Materiały źródłowe

• #1 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. […] Current management of DSRCT includes a combination of chemotherapy, radiation and aggressive cytoreductive surgery plus intra-peritoneal hyperthermic chemotherapy (HIPEC). […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. […] The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors was evaluated by small trials. […] Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases.

• #2 Management of Desmoplastic Small Round Cell Tumor

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5614508/

  Desmoplastic small round cell tumor (DSRCT) is a soft tissue sarcoma of mesenchymal cell origin that typically presents with multiple intra-abdominal tumors and exhibits a multi-phenotypic pattern of immunohistochemical staining. […] With this challenging disease, multidisciplinary therapy, including aggressive surgery, is warranted. This review will address DSRCT biology and treatment options and discuss outcomes. […] Since its description in 1989 by Gerald and Rosai at Memorial Sloan Kettering Cancer Center, multimodality chemotherapy has been used for DSRCT. Ewings type chemotherapy, aggressive surgery, tumor debulking, total abdominal radiation therapy, and high-dose chemotherapy followed by autologous stem cell rescue have all been used in the treatment of DSRCT, with little improvement in survival.

• #3 Desmoplastic small-round-cell tumor – Wikipedia

  https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor

  DSRCT is frequently misdiagnosed. Adult patients should always be referred to a sarcoma specialist. This is an aggressive, rare, fast spreading tumor and both pediatric and adult patients should be treated at a sarcoma center. […] There is no standard protocol for the disease. However, recent research has reported that some patients respond to high-dose (P6 Protocol) chemotherapy, maintenance chemotherapy, debulking operation, cytoreductive surgery, and radiation therapy. […] Other treatment options include: hematopoietic stem cell transplantation, intensity-modulated radiation therapy, radiofrequency ablation, stereotactic body radiation therapy, intraperitoneal hyperthermic chemoperfusion, and clinical trials. […] A multi-modality approach of high-dose chemotherapy, aggressive surgical resection, radiation, and stem cell rescue improves survival for some patients. Reports have indicated that patients will initially respond to first line chemotherapy and treatment but that relapse is common. […] Some patients in remission or with inoperable tumor seem to benefit from long term low dose chemotherapy, turning DSRCT into a chronic disease.

• #4 SSA – POMS: DI 23022.146 – Desmoplastic Small Round Cell Tumors – 09/11/2020

  https://secure.ssa.gov/poms.nsf/%20lnx/0423022146

  TREATMENT […] Treatment of DSRCT may include chemotherapy, hyperthermic intraperitoneal chemotherapy (HIPEC), radiation therapy, surgery, and stem cell transplantation. DSRCT is often resistant to treatment and frequently recurs. Despite aggressive therapy, 3-year overall survival has been estimated at 44% and the 5-year survival rate remains around 15%.

• #5 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases. As most of the cases present as intra-peritoneal, aggressive tumors, the main guidelines recommend initiating treatment with systemic chemotherapy. Although DSRCT is not specifically addressed by ESMO or NCCN guideline, this recommendation is stated by the Chicago Consensus on Peritoneal Malignancies and by the MDAnderson Cancer Center nomogram. […] The role of surgery in the management of DSRCT is well established in the literature. In a review of 12 patients treated at Mayo Clinic, Hassan et al. showed the median survival of patients treated with surgical resection was 34 months, whereas the median survival of those who underwent biopsy alone was 14 months. In the report of Wong et al., the median survival for patients who had a resection for their abdominal or pelvic tumors was 47 months, compared to 16 months for those who did not. Complete cytoreductive surgery is associated with improved survival and should be considered a cornerstone of treatment together with chemotherapy.

• #6 Management of Desmoplastic Small Round Cell Tumor

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5614508/

  Desmoplastic small round cell tumor (DSRCT) is a soft tissue sarcoma of mesenchymal cell origin that typically presents with multiple intra-abdominal tumors and exhibits a multi-phenotypic pattern of immunohistochemical staining. […] With this challenging disease, multidisciplinary therapy, including aggressive surgery, is warranted. This review will address DSRCT biology and treatment options and discuss outcomes. […] Since its description in 1989 by Gerald and Rosai at Memorial Sloan Kettering Cancer Center, multimodality chemotherapy has been used for DSRCT. Ewings type chemotherapy, aggressive surgery, tumor debulking, total abdominal radiation therapy, and high-dose chemotherapy followed by autologous stem cell rescue have all been used in the treatment of DSRCT, with little improvement in survival.

• #7

  http://waocp.com/journal/index.php/apjcc/article/view/1022

  The optimal treatment approach for DSRCT involves a multidisciplinary team. The standard chemotherapy regimen used is typically the Ewing’s sarcoma protocol of multiagent chemotherapy. Surgical interventions, such as cytoreduction and intraperitoneal hyperthermic chemotherapy, are the preferred procedures, although their feasibility is limited. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. For advanced disease, symptom palliation is paramount. […] Although complete cytoreductive surgery (CRS) is the standard therapy, the microscopic residual disease is often present. Hence, Hyperthermic Intraperitoneal Chemotherapy(HIPEC) has been examined in some studies as an adjunct strategy but with conflicting results. […] Proper consensus about treatment has not yet been established. DSRCT still remains a clinical challenge for oncologists because of the lack of standard guidelines.

• #8 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  The most effective chemotherapeutic regimen with curative intent is still debated, but most are based on those used to treat other small round cell sarcomas, with a combination of an anthracycline, alkylating agent and vinca alkaloid. DSRCT is somehow sensitive to chemotherapy, although a transient response followed by disease progression is common. Farhat et al. reported four patients with the intra-abdominal disease who experienced disease stabilization lasting 4–9 months after chemotherapy including cyclophosphamide, etoposide, doxorubicin and cisplatin. Kushner et al. reported 12 patients with a median survival of 19 months with the P6-protocol, which has seven courses of chemotherapy consisting of cyclophosphamide, doxorubicin, vincristine (HD-CAV), etoposide and ifosfamide. This was followed by surgery, radiotherapy, and myeloablative chemotherapy using thiotepa and carboplatin with stem cell rescue in some cases. Bertuzzi et al. published a trial that included 7 patients with DSRCT treated with induction chemotherapy consisting of ifosfamide, epirubicin and vincristine, and those who responded were then treated with high-dose chemotherapy and autologous bone marrow transplantation in conjunction with local therapy (surgery and/or radiotherapy). The authors concluded that high-dose chemotherapy probably has no role in the treatment of DSRCT. More recently, Scheer et al. found that patients treated with the VAIA regimen (ifosfamide, vincristine, doxorubicin, actinomycin D) presented longer event-free survival (29.4 months) compared to other protocols, including the P6 protocol. The interval-compressed regimen of vincristine, irinotecan, temozolamide (VIT) was evaluated in 6 pediatric patients and showed a tolerable profile with an objective response rate of 50% to the first 2 cycles of VIT.

• #9 A Case of Intra-Abdominal Desmoplastic Small Round Cell Tumor in a Young Girl Patient

  https://www.clinmedjournals.org/articles/ogcr/obstetrics-and-gynaecology-cases-reviews-ogcr-5-128.php?jid=ogcr

  The main aim of this case report was to present the method of diagnosis, management, and prognosis of a young adolescent diagnosed with a desmoplastic small round cell tumor (DSRCT). […] Due to our limited knowledge of the pathologic and clinical nature of this disease, there is no clear consensus regarding the optimal therapeutic procedures for treating this neoplasm and a multimodal treatment with surgery, chemotherapy and radiotherapy is used for these rare cases. […] A multidisciplinary approach is required. It combines: […] Total resection of the mass and postoperative multiagent chemotherapy and radiation therapy as the best therapeutic options. Total resection is not often feasible because of the numerous peritoneal implants. […] Preoperative chemotherapy, with the P6 protocol including cyclophosphamide, doxorubicin, vincristine, ifosfamide and etoposide, as a way of shrinking the widespread mass and reducing its vascularity.

• #10 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  The most effective chemotherapeutic regimen with curative intent is still debated, but most are based on those used to treat other small round cell sarcomas, with a combination of an anthracycline, alkylating agent and vinca alkaloid. DSRCT is somehow sensitive to chemotherapy, although a transient response followed by disease progression is common. Farhat et al. reported four patients with the intra-abdominal disease who experienced disease stabilization lasting 4–9 months after chemotherapy including cyclophosphamide, etoposide, doxorubicin and cisplatin. Kushner et al. reported 12 patients with a median survival of 19 months with the P6-protocol, which has seven courses of chemotherapy consisting of cyclophosphamide, doxorubicin, vincristine (HD-CAV), etoposide and ifosfamide. This was followed by surgery, radiotherapy, and myeloablative chemotherapy using thiotepa and carboplatin with stem cell rescue in some cases. Bertuzzi et al. published a trial that included 7 patients with DSRCT treated with induction chemotherapy consisting of ifosfamide, epirubicin and vincristine, and those who responded were then treated with high-dose chemotherapy and autologous bone marrow transplantation in conjunction with local therapy (surgery and/or radiotherapy). The authors concluded that high-dose chemotherapy probably has no role in the treatment of DSRCT. More recently, Scheer et al. found that patients treated with the VAIA regimen (ifosfamide, vincristine, doxorubicin, actinomycin D) presented longer event-free survival (29.4 months) compared to other protocols, including the P6 protocol. The interval-compressed regimen of vincristine, irinotecan, temozolamide (VIT) was evaluated in 6 pediatric patients and showed a tolerable profile with an objective response rate of 50% to the first 2 cycles of VIT.

• #11 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  The most effective chemotherapeutic regimen with curative intent is still debated, but most are based on those used to treat other small round cell sarcomas, with a combination of an anthracycline, alkylating agent and vinca alkaloid. DSRCT is somehow sensitive to chemotherapy, although a transient response followed by disease progression is common. Farhat et al. reported four patients with the intra-abdominal disease who experienced disease stabilization lasting 4–9 months after chemotherapy including cyclophosphamide, etoposide, doxorubicin and cisplatin. Kushner et al. reported 12 patients with a median survival of 19 months with the P6-protocol, which has seven courses of chemotherapy consisting of cyclophosphamide, doxorubicin, vincristine (HD-CAV), etoposide and ifosfamide. This was followed by surgery, radiotherapy, and myeloablative chemotherapy using thiotepa and carboplatin with stem cell rescue in some cases. Bertuzzi et al. published a trial that included 7 patients with DSRCT treated with induction chemotherapy consisting of ifosfamide, epirubicin and vincristine, and those who responded were then treated with high-dose chemotherapy and autologous bone marrow transplantation in conjunction with local therapy (surgery and/or radiotherapy). The authors concluded that high-dose chemotherapy probably has no role in the treatment of DSRCT. More recently, Scheer et al. found that patients treated with the VAIA regimen (ifosfamide, vincristine, doxorubicin, actinomycin D) presented longer event-free survival (29.4 months) compared to other protocols, including the P6 protocol. The interval-compressed regimen of vincristine, irinotecan, temozolamide (VIT) was evaluated in 6 pediatric patients and showed a tolerable profile with an objective response rate of 50% to the first 2 cycles of VIT.

• #12 Desmoplastic small round cell tumors – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/dsrct/diagnosis-treatment/drc-20449484

  Treatment for desmoplastic small round cell tumor depends on your situation. Your healthcare team considers your cancer’s location and whether it has spread to other parts of the body. Most people with this type of cancer receive a combination of treatments. […] The goal of surgery is to remove all of the cancer. It might not be possible if the cancer has grown into nearby organs. If that happens, your healthcare professional may recommend chemotherapy with powerful drugs to shrink the cancer first. […] Chemotherapy uses powerful drugs to kill cancer cells. Chemotherapy may be used before surgery to shrink the cancer. This makes it easier to remove with surgery. Chemotherapy may also be used after surgery to kill any cells that might remain after the operation. […] Radiation therapy uses powerful beams of energy to kill cancer cells. The energy can come from sources such as X-rays and protons. […] Targeted drug treatments attack specific chemicals present within cancer cells. By blocking these chemicals, targeted drug treatments can cause cancer cells to die.

• #13 Desmoplastic small round cell tumors

  https://johnsonmemorial.org/jmh-health/disease-conditions/con-20248516

  Treatment for desmoplastic small round cell tumors typically involves a combination of treatments. Options might include surgery, chemotherapy and radiation therapy. […] Treatment for desmoplastic small round cell tumor depends on your situation. Your health care team considers your cancer’s location and whether it has spread to other parts of the body. Most people with this type of cancer receive a combination of treatments. […] The goal of surgery is to remove all of the cancer. It might not be possible if the cancer has grown into nearby organs. If that happens, your health care provider may recommend chemotherapy with powerful drugs to shrink the cancer first. […] Chemotherapy uses powerful drugs to kill cancer cells. Chemotherapy may be used before surgery to shrink the cancer. This makes it easier to remove with surgery. Chemotherapy may also be used after surgery to kill any cells that might remain after the operation.

• #14 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. Second-line treatment is ineffective in most cases. In our cohort, out of 19 patients treated with first-line chemotherapy, 13 received the second-line and the progression-free survival was only 3.9 months. This short survival time highlights the aggressiveness of this disease and the challenge in developing new therapeutic strategies to treat these young patients. Despite the development of new regimens for ES and other soft tissue and bone sarcoma in recent years, DSRCT is underrepresented or were not included in the trials that lead to the drug approval. As a result, the evidence to use second-line therapy is very limited. It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT.

• #15 Desmoplastic Small Round Cell Tumor Treatment | St. Jude Care & Treatment

  https://www.stjude.org/care-treatment/treatment/childhood-cancer/solid-tumors/desmoplastic-small-round-cell-tumor.html

  Because this tumor is so rare, there is no standard way to treat it. Treatments for DSRCT may include: […] Surgery is used to remove as much of the cancer as possible. Often, DSRCT has spread too far for complete removal. […] Chemotherapy (chemo) uses powerful medicines to kill cancer cells or stop them from growing (dividing) and making more cancer cells. Typically, 5-7 different types of chemotherapies are used to treat DSRCT. […] Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or stop them from growing. […] Hyperthermic intraperitoneal chemotherapy (HIPEC) may be given during surgery. HIPEC kills cancer cells that cannot be removed surgically. It is done by washing the inside of the abdomen with a warm chemo solution. This procedure lasts up to 2 hours.

• #16 9 Innovative Desmoplastic Small Round Cell Tumor Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors/desmoplastic-small-round-cell-tumors-treatment.html

  MD Anderson’s Children’s Cancer Hospital is among the few cancer centers in the nation with extensive experience treating desmoplastic small round cell tumors, a rare and aggressive form of pediatric cancer. […] Using the latest research, and backed by the most modern technology and techniques, Children’s Cancer Hospital physicians customize your child’s comprehensive course of treatment to address specific problems. Our goal is to offer the best chances for effective treatment with the least impact on your child’s body. […] Surgery is usually the first line of treatment for DSRCT. […] HIPEC, or hyperthermic peritoneal perfusion with chemotherapy, an innovative surgical procedure pioneered at Children’s Cancer Hospital, has shown to be safe and effective for many children with DSRCT.

• #17 Management of Desmoplastic Small Round Cell Tumor

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5614508/

  Control of DSRCT with chemotherapy is most effective in children treated with Ewings type chemotherapy, which has become the standard after efficacy with this regimen was demonstrated by Kushner et al. […] This chemotherapy regimen was used in combination with aggressive surgical complete excision and post-operative whole abdominal radiation, providing improved survival. […] Complete surgical resection, including cytoreduction and hyperthermic intraperitoneal chemotherapy (HIPEC), is standard therapy for appendiceal carcinoma pseudomyxoma peritonei, and other diseases, and multimodal therapy consisting of complete cytoreduction and HIPEC has been found to improve survival in many studies of carcinomatosis. […] In a report on the impact of complete surgical resection of DSRCT, LaQuaglia and colleagues found a 3-year overall survival of 58% with complete resection and 0% when resection was not done and the patients were treated with chemotherapy and radiotherapy alone.

• #18 Desmoplastic Small Round Cell Tumor Treatments for Your Child | MSK Kids | Memorial Sloan Kettering Cancer Center

  https://www.mskcc.org/pediatrics/cancer-care/types/desmoplastic-small-round-cell-tumors-dsrcts/treatment

  Chemotherapy and surgery are common treatments for desmoplastic small round cell tumor (DSRCT) in children. MSK Kids offers all treatments for DSRCT. […] We often give chemotherapy (chemo) to first shrink the tumor and make it easier to remove with surgery. These drugs also lower the chances the cancer will come back. […] MSK Kids cancer surgeons in New York City have improved DSRCT treatment. They have developed new ways to remove these tumors. […] Desmoplastic small round cell tumors can be very hard to remove all the way. They often are wrapped around blood vessels and other organs. That’s why we often give chemotherapy first to shrink the tumor, which makes surgery easier. […] The operation to remove DSRCT is very long, sometimes lasting 12 hours. Experience matters with this type of surgery. MSK Kids surgeons were the first to show it’s best to remove as much of the tumor as possible. This is called debulking surgery. Taking out the tumor and nearby tissues helps children live longer.

• #19 New Study Demonstrates Effectiveness of Cytoreductive Surgery for Desmoplastic Small Round Cell Tumor

  https://consultqd.clevelandclinic.org/new-study-demonstrates-effectiveness-of-cytoreductive-surgery-for-desmoplastic-small-round-cell-tumor

  Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive form of sarcoma with the greatest incidence in children, adolescents and young adults. […] The major challenge is getting patients to as low of a disease burden as possible using chemotherapy, at which point surgery can significantly contribute to the cure. […] In our study, patients with DSRCT had significantly longer median overall survival (OS) after cytoreductive surgery compared with patients with other sarcomas, (44.3 vs. 12.5 months, P = 0.0013), Dr. Anderson notes, adding that recurrence-free survival (RFS) was also significantly longer for patients with DSRCT compared with patients with other tumors (14.9 vs. 4.5 months, P = 0.0012). […] Dr. Anderson says this trial has shown that not only is cytoreductive surgery technically feasible, but we also know what dose of cis-platinum to use.

• #20 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Even after chemotherapeutic cytoreduction and surgical resection of gross, visible disease, the microscopic residual disease is often present. Hence, hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. In a recent phase 2 trial, 14 DSRCT patients were treated with neoadjuvant chemotherapy, followed by cytoreductive surgery (CRS), which was complete (CR0) or near-complete (CR1 ≤ 2.5 cm of tumor remaining) in all patients, with closed technique HIPEC using 100 mg/m² of cisplatin for 90 min at 41 degrees Celsius, then followed by WAP-RT. The 3-year overall survival from time of diagnosis for DSRCT patients was 79%, and the estimated median recurrence-free survival (RFS) was 14.0 months. In 100% of patients without hepatic or portal metastasis, there was no peritoneal disease recurrence after CRS-HIPEC. They concluded that CRS, HIPEC and WART are effective local control therapy in DSRCT patients.

• #21 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #22 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Even after chemotherapeutic cytoreduction and surgical resection of gross, visible disease, the microscopic residual disease is often present. Hence, hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. In a recent phase 2 trial, 14 DSRCT patients were treated with neoadjuvant chemotherapy, followed by cytoreductive surgery (CRS), which was complete (CR0) or near-complete (CR1 ≤ 2.5 cm of tumor remaining) in all patients, with closed technique HIPEC using 100 mg/m² of cisplatin for 90 min at 41 degrees Celsius, then followed by WAP-RT. The 3-year overall survival from time of diagnosis for DSRCT patients was 79%, and the estimated median recurrence-free survival (RFS) was 14.0 months. In 100% of patients without hepatic or portal metastasis, there was no peritoneal disease recurrence after CRS-HIPEC. They concluded that CRS, HIPEC and WART are effective local control therapy in DSRCT patients.

• #23 Desmoplastic Small Round Cell Tumor Treatment | St. Jude Care & Treatment

  https://www.stjude.org/care-treatment/treatment/childhood-cancer/solid-tumors/desmoplastic-small-round-cell-tumor.html

  Because this tumor is so rare, there is no standard way to treat it. Treatments for DSRCT may include: […] Surgery is used to remove as much of the cancer as possible. Often, DSRCT has spread too far for complete removal. […] Chemotherapy (chemo) uses powerful medicines to kill cancer cells or stop them from growing (dividing) and making more cancer cells. Typically, 5-7 different types of chemotherapies are used to treat DSRCT. […] Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or stop them from growing. […] Hyperthermic intraperitoneal chemotherapy (HIPEC) may be given during surgery. HIPEC kills cancer cells that cannot be removed surgically. It is done by washing the inside of the abdomen with a warm chemo solution. This procedure lasts up to 2 hours.

• #24 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Even after chemotherapeutic cytoreduction and surgical resection of gross, visible disease, the microscopic residual disease is often present. Hence, hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. In a recent phase 2 trial, 14 DSRCT patients were treated with neoadjuvant chemotherapy, followed by cytoreductive surgery (CRS), which was complete (CR0) or near-complete (CR1 ≤ 2.5 cm of tumor remaining) in all patients, with closed technique HIPEC using 100 mg/m² of cisplatin for 90 min at 41 degrees Celsius, then followed by WAP-RT. The 3-year overall survival from time of diagnosis for DSRCT patients was 79%, and the estimated median recurrence-free survival (RFS) was 14.0 months. In 100% of patients without hepatic or portal metastasis, there was no peritoneal disease recurrence after CRS-HIPEC. They concluded that CRS, HIPEC and WART are effective local control therapy in DSRCT patients.

• #25 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Even after chemotherapeutic cytoreduction and surgical resection of gross, visible disease, the microscopic residual disease is often present. Hence, hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. In a recent phase 2 trial, 14 DSRCT patients were treated with neoadjuvant chemotherapy, followed by cytoreductive surgery (CRS), which was complete (CR0) or near-complete (CR1 ≤ 2.5 cm of tumor remaining) in all patients, with closed technique HIPEC using 100 mg/m² of cisplatin for 90 min at 41 degrees Celsius, then followed by WAP-RT. The 3-year overall survival from time of diagnosis for DSRCT patients was 79%, and the estimated median recurrence-free survival (RFS) was 14.0 months. In 100% of patients without hepatic or portal metastasis, there was no peritoneal disease recurrence after CRS-HIPEC. They concluded that CRS, HIPEC and WART are effective local control therapy in DSRCT patients.

• #26 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Given the propensity for morbid intra-peritoneal progression, consolidative whole abdominopelvic radiotherapy (WAP-RT) as part of multimodal treatment (chemotherapy, surgical debulking and WAP-RT) was first reported using the P6 protocol, in an attempt to improve local control. Honoré et al. reported in a series of 38 patients with a median follow-up of almost 5 years, that multimodal treatment combining systemic chemotherapy, complete macroscopic resection, and postoperative WAP-RT could prolong survival in patients without the extra-peritoneal disease (EPM)—median survival of 37.7 months (range 7.9–42.9 months). The factors predictive of 3-year overall survival were the absence of EPM, complete surgical resection, postoperative WAP-RT and postoperative chemotherapy. Atallah et al. studied the prognostic role of WAP-RT on oncologic outcomes as part of multimodal treatment in 103 patients with abdominal DSRCT treated at eight French centers from 1991 to 2014. They concluded that RT seems to improve survival after cytoreductive surgery, with better PPFS, PFS and OS for the patients treated in a multimodal approach, with the limitations of a retrospective study, lack of statistical power (due to the small number of patients), and the need of randomized prospective studies to confirm these results.

• #27 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Given the propensity for morbid intra-peritoneal progression, consolidative whole abdominopelvic radiotherapy (WAP-RT) as part of multimodal treatment (chemotherapy, surgical debulking and WAP-RT) was first reported using the P6 protocol, in an attempt to improve local control. Honoré et al. reported in a series of 38 patients with a median follow-up of almost 5 years, that multimodal treatment combining systemic chemotherapy, complete macroscopic resection, and postoperative WAP-RT could prolong survival in patients without the extra-peritoneal disease (EPM)—median survival of 37.7 months (range 7.9–42.9 months). The factors predictive of 3-year overall survival were the absence of EPM, complete surgical resection, postoperative WAP-RT and postoperative chemotherapy. Atallah et al. studied the prognostic role of WAP-RT on oncologic outcomes as part of multimodal treatment in 103 patients with abdominal DSRCT treated at eight French centers from 1991 to 2014. They concluded that RT seems to improve survival after cytoreductive surgery, with better PPFS, PFS and OS for the patients treated in a multimodal approach, with the limitations of a retrospective study, lack of statistical power (due to the small number of patients), and the need of randomized prospective studies to confirm these results.

• #28 Treatment of Abdominal Desmoplastic Small Round Cell Tumor | OTT

  https://www.dovepress.com/treatment-of-abdominal-desmoplastic-small-round-cell-tumor-induces-acu-peer-reviewed-fulltext-article-OTT

  Radiotherapy is commonly used to relieve local symptoms and is a supplementary treatment to surgery and chemotherapy. Whole-abdominal radiation therapy (WART) can be used as a consolidated chemotherapy method for small abdominal lesions. […] Although the combination of chemotherapy, surgery, and radiotherapy significantly improves the survival rate of patients with DSRCT, the overall prognosis remains poor because the majority of patients experience disease recurrence and die within three years. […] Tyrosine kinase inhibitors (TKI) targeting VEGF, VEGFR, and other proteins involved in tumor vascular proliferation have also been used in clinical practice. […] The patient we reported took oral anlotinib for maintenance treatment after radiotherapy, and the disease remained stable with mild diarrhea for more than half a year. […] This case highlights the importance of individualized multimodal treatment strategies for rare cancers. Additionally, it underscores the need to closely monitor the risk of a secondary tumor induced by high-intensity radiotherapy and chemotherapy.

• #29 Desmoplastic Small Round Cell Tumor Treatments for Your Child | MSK Kids | Memorial Sloan Kettering Cancer Center

  https://www.mskcc.org/pediatrics/cancer-care/types/desmoplastic-small-round-cell-tumors-dsrcts/treatment

  Some children have radiation therapy, a treatment that directs beams of high-energy rays to kill cancer cells. Your doctor will let you know if this is an option for your child. […] Intensity-modulated radiation therapy (IMRT) is a very precise form of radiation therapy used for some children with DSRCT. […] Proton therapy is a treatment choice for some kids with DSRCT in the abdomen (belly). It uses charged particles called protons to aim high doses of radiation right at tumors. […] An anesthesiologist uses anesthesia (medicine to make your child sleep) during surgery. MSK Kids anesthesiologists are all pediatric anesthesiologists, with special training in the care of children. As part of your child’s care team, anesthesiologists help with pain or discomfort related to DSRCT treatments. They will help control your child’s pain.

• #30 Desmoplastic small-round-cell tumor – Wikipedia

  https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor

  DSRCT is frequently misdiagnosed. Adult patients should always be referred to a sarcoma specialist. This is an aggressive, rare, fast spreading tumor and both pediatric and adult patients should be treated at a sarcoma center. […] There is no standard protocol for the disease. However, recent research has reported that some patients respond to high-dose (P6 Protocol) chemotherapy, maintenance chemotherapy, debulking operation, cytoreductive surgery, and radiation therapy. […] Other treatment options include: hematopoietic stem cell transplantation, intensity-modulated radiation therapy, radiofrequency ablation, stereotactic body radiation therapy, intraperitoneal hyperthermic chemoperfusion, and clinical trials. […] A multi-modality approach of high-dose chemotherapy, aggressive surgical resection, radiation, and stem cell rescue improves survival for some patients. Reports have indicated that patients will initially respond to first line chemotherapy and treatment but that relapse is common. […] Some patients in remission or with inoperable tumor seem to benefit from long term low dose chemotherapy, turning DSRCT into a chronic disease.

• #31 Desmoplastic Small Round Cell Tumor Treatments for Your Child | MSK Kids | Memorial Sloan Kettering Cancer Center

  https://www.mskcc.org/pediatrics/cancer-care/types/desmoplastic-small-round-cell-tumors-dsrcts/treatment

  Some children have radiation therapy, a treatment that directs beams of high-energy rays to kill cancer cells. Your doctor will let you know if this is an option for your child. […] Intensity-modulated radiation therapy (IMRT) is a very precise form of radiation therapy used for some children with DSRCT. […] Proton therapy is a treatment choice for some kids with DSRCT in the abdomen (belly). It uses charged particles called protons to aim high doses of radiation right at tumors. […] An anesthesiologist uses anesthesia (medicine to make your child sleep) during surgery. MSK Kids anesthesiologists are all pediatric anesthesiologists, with special training in the care of children. As part of your child’s care team, anesthesiologists help with pain or discomfort related to DSRCT treatments. They will help control your child’s pain.

• #32 Desmoplastic Small Round Cell Tumors | Symptoms, Diagnosis & Treatment

  https://www.cincinnatichildrens.org/health/d/desmoplastic-small-round-cell-tumors

  Desmoplastic small round cell tumors (DSRCT) are a type of rare, soft tissue sarcoma. This type of cancer usually begins in the abdomen or pelvis, and does not respond well to treatment. […] DSRCT is a very rare cancer. Therefore, patients who have it should be treated at a comprehensive sarcoma center with experts in the field of radiation oncology, surgery and oncology who have experience and expertise in diagnosing and treating this disease. […] The doctor may recommend surgery to remove as much of the tumor(s) as possible. Because these tumors grow in and around other organs, it usually is not possible to remove the entire tumor this way. Most patients need to have other therapies after their surgery. The medical team may offer some or all of the following treatments: Traditional radiation therapy, Proton therapy, which the doctor may recommend in special cases. Proton therapy is a type of radiation therapy that targets the tumor while avoiding organs and healthy tissue. This means fewer short-term and long-term side effects from radiation. Cincinnati Childrens is one of only a few pediatric hospitals in the country that offer proton therapy, High doses of chemotherapy to shrink the tumor(s) and prevent new ones from forming. This therapy may require the use of rescuing stem cells, which serve as an internal repair system for the body. Stem cell rescue involves removing stem cells from the blood between chemotherapy cycles and freezing them. After very high dose chemotherapy, the doctor thaws and injects the stem cells back into the body, where they begin forming new, healthy blood cells, A two-step surgical procedure called hyperthermic intra-peritoneal chemotherapy (HIPEC). This involves removing as much of the tumor as possible, then delivering highly concentrated, heated chemotherapy drugs directly to the abdominal area. This therapy is for patients whose cancer is located in the abdominal cavity, Maintenance therapy, which may be an option after an initial round of treatment. It can involve giving chemotherapy or molecularly targeted therapy, which uses drugs or other substances to interfere with specific molecules that allow cancer to grow and spread, One of several targeted tumor ablation techniques, which kill tumors.

• #33 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Given the propensity for morbid intra-peritoneal progression, consolidative whole abdominopelvic radiotherapy (WAP-RT) as part of multimodal treatment (chemotherapy, surgical debulking and WAP-RT) was first reported using the P6 protocol, in an attempt to improve local control. Honoré et al. reported in a series of 38 patients with a median follow-up of almost 5 years, that multimodal treatment combining systemic chemotherapy, complete macroscopic resection, and postoperative WAP-RT could prolong survival in patients without the extra-peritoneal disease (EPM)—median survival of 37.7 months (range 7.9–42.9 months). The factors predictive of 3-year overall survival were the absence of EPM, complete surgical resection, postoperative WAP-RT and postoperative chemotherapy. Atallah et al. studied the prognostic role of WAP-RT on oncologic outcomes as part of multimodal treatment in 103 patients with abdominal DSRCT treated at eight French centers from 1991 to 2014. They concluded that RT seems to improve survival after cytoreductive surgery, with better PPFS, PFS and OS for the patients treated in a multimodal approach, with the limitations of a retrospective study, lack of statistical power (due to the small number of patients), and the need of randomized prospective studies to confirm these results.

• #34 Complete response of giant desmoplastic small round cell tumor treated with chemoradiotherapy: A case report

  https://www.spandidos-publications.com/10.3892/ol.2015.4024

  Desmoplastic small round cell tumor (DSRCT) is a rare tumor that mainly affects adolescents, and typically involves the abdominal and pelvic peritoneum. […] Following laparotomy, the patient was treated with external beam radiotherapy to the whole abdomen and pelvis to a dose of 40 Gy plus a 20 Gy boost to the residual disease. […] In conclusion, DSRCT is a rare malignancy requiring multidisciplinary treatment, including surgery, chemotherapy and radiotherapy. […] The results of the present study confirm that radiotherapy has a significant role in the treatment of advanced abdominal DSRCT and may contribute to durable remission. […] The standard treatment protocol for DSRCT has not been well established. […] Potential therapeutic strategies include, debulking surgery, radiotherapy and chemotherapy.

• #35 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #36 9 Innovative Desmoplastic Small Round Cell Tumor Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors/desmoplastic-small-round-cell-tumors-treatment.html

  The specialists at Children’s Cancer Hospital are researching new ways to treat DSRCT, including targeted therapies to help the body fight cancer on a cellular level. […] After carefully evaluating your child’s case, our team of experts will discuss a recommended course of treatment for DSRCT. Since DSRCT is complex and seldom seen, no standard treatment exists. […] Surgery is almost always part of treatment for DSRCT. […] Some DSRCT patients respond to chemotherapy, but most relapse. Long-term, low-dose chemotherapy may help patients in remission or with a tumor that cannot be surgically removed. […] Radiation therapy (also called radiotherapy) uses high-energy beams to destroy cancer cells. […] Children’s Cancer Hospital is leading into the future of DSRCT treatment by developing innovative targeted therapies.

• #37 Desmoplastic small round cell tumors

  https://johnsonmemorial.org/jmh-health/disease-conditions/con-20248516

  Radiation therapy uses powerful beams of energy to kill cancer cells. The energy can come from sources such as X-rays and protons. During radiation therapy, you lie very still on a table and a machine moves around you. The machine directs radiation to precise points on your body. […] Targeted drug treatments attack specific chemicals present within cancer cells. By blocking these chemicals, targeted drug treatments can cause cancer cells to die.

• #38 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. […] Current management of DSRCT includes a combination of chemotherapy, radiation and aggressive cytoreductive surgery plus intra-peritoneal hyperthermic chemotherapy (HIPEC). […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. […] The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors was evaluated by small trials. […] Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases.

• #39 Treatment of Abdominal Desmoplastic Small Round Cell Tumor | OTT

  https://www.dovepress.com/treatment-of-abdominal-desmoplastic-small-round-cell-tumor-induces-acu-peer-reviewed-fulltext-article-OTT

  Radiotherapy is commonly used to relieve local symptoms and is a supplementary treatment to surgery and chemotherapy. Whole-abdominal radiation therapy (WART) can be used as a consolidated chemotherapy method for small abdominal lesions. […] Although the combination of chemotherapy, surgery, and radiotherapy significantly improves the survival rate of patients with DSRCT, the overall prognosis remains poor because the majority of patients experience disease recurrence and die within three years. […] Tyrosine kinase inhibitors (TKI) targeting VEGF, VEGFR, and other proteins involved in tumor vascular proliferation have also been used in clinical practice. […] The patient we reported took oral anlotinib for maintenance treatment after radiotherapy, and the disease remained stable with mild diarrhea for more than half a year. […] This case highlights the importance of individualized multimodal treatment strategies for rare cancers. Additionally, it underscores the need to closely monitor the risk of a secondary tumor induced by high-intensity radiotherapy and chemotherapy.

• #40 Desmoplastic Small Round Cell Tumor of the Pleura Successfully Treated with a Lower Dose of Pazopanib

  https://www.jstage.jst.go.jp/article/internalmedicine/55/17/55_55.6395/_article

  Desmoplastic small round cell tumor (DSRCT) is an aggressive mesenchymal tumor which primarily affects the abdomen. […] Initial therapy, which included chemotherapy, surgery and radiotherapy, achieved a partial response for only two months. […] Although salvage chemotherapies had no effect, pazopanib treatment shrank the tumors and was well-tolerated on an outpatient basis. […] From the viewpoint of quality of life, pazopanib may therefore be a good therapeutic option for this aggressive disease.

• #41 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. […] Current management of DSRCT includes a combination of chemotherapy, radiation and aggressive cytoreductive surgery plus intra-peritoneal hyperthermic chemotherapy (HIPEC). […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. […] The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors was evaluated by small trials. […] Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases.

• #42 Multi-site desmoplastic small round cell tumors are genetically related and immune-cold | npj Precision Oncology

  https://www.nature.com/articles/s41698-022-00257-9

  Desmoplastic small round cell tumor (DSRCT) is a highly aggressive soft tissue sarcoma that is characterized by the EWSR1-WT1 fusion protein. […] Treatment of JN-DSRCT cells with the PI3K inhibitor alpelisib and mTOR inhibitor temsirolimus reduced cell proliferation. […] Treatments directed at the EWSR1-WT1 fusion protein or downstream signaling pathways are not currently available. […] Given the low prevalence of gene-level mutations, we interrogated whether pathways were recurrently altered by mapping all the mutated genes to pathways. […] Therefore, we tested the effect of the PI3K inhibitor alpelisib and the mTOR inhibitor temsirolimus on the proliferation of the JN-DSRCT cells. […] The results support the prediction of our integrative network analysis that indicates the PI3K/mTOR pathway would be therapeutic targets in DSRCT.

• #43 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #44 Trabectedin for desmoplastic small round cell tumours: a possible treatment option? | Clinical Sarcoma Research | Full Text

  https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-4-3

  Desmoplastic small round cell tumour (DSRCT) is a rare sarcoma typically affecting young males and usually widely metastatic at presentation. Despite multimodal treatment approaches, the prognosis for DSRCT is extremely poor. Alkylator- and anthracyclines- based regimens are widely used as therapy and an initial response is common. Durable responses are exceptionally rare so further systemic treatment options for these patients represent an unmet medical need. We report two cases of metastatic, pretreated DSRCT patients achieving disease stabilisation with Trabectedin. […] This report supports Trabectedin to be active and safe in pre-treated DSRCT patients. Further prospective and collaborative efforts are desirable to better define its role in the management of this disease. […] Trabectedin is a marine-derived antineoplastic agent, initially isolated from the tunicate Ecteinascidia turbinata and now produced synthetically, which is currently approved in Europe for the treatment of advanced soft tissue sarcoma after prior treatment with an anthracycline.

• #45 Third-Line Trabectedin for a Metastatic Desmoplastic Small Round Cell Tumour Treated with Multimodal Therapy | Anticancer Research

  https://ar.iiarjournals.org/content/34/7/3683

  Trabectedin (Ecteinascidin-743; ET-743; Yondelis, Pharma Mar S.A., Madrid, Spain) is novel tetrahydroisoquinoline alkaloid originally extracted from the Caribbean sea squirt Ecteinascidia turbinata, and now produced synthetically. […] The response to trabectedin in our patient is notable. Following disease progression despite two lines of intensive chemotherapy, HIPEC, and three rounds of aggressive surgery, he achieved a partial response after 12 weeks of trabectedin at 1.5 mg/m2 every three weeks. […] Our case report suggests that trabectedin may be considered as a treatment option in multimodality therapy for the management of DSRCT. Further research is warranted to explore the impact of trabectedin on the treatment of this disease.

• #46

  https://link.springer.com/article/10.1186/s12885-018-4135-x

  Desmoplastic small round cell tumor (DSRCT) is a rare malignant sarcoma with poor prognosis due to lack of effective treatments. Apatinib is a new potent oral small-molecule tyrosine kinase inhibitor, and targets the intracellular domain of vascular endothelial growth factor receptor 2 (VEGFR-2). In this study, we presented a case of intra-abdominal DSRCT which was effectively treated by apatinib. […] Apatinib revealed outstanding effect on reducing mass size and ascites during 2-month treatment. Apatinib therapy continued for additional 2 months, and the patient was in good condition. […] It is the first report that apatinib is effective on DSRCT. This report may provide an additional option for the treatment of metastatic DSRCT. […] The treatment of DSRCT remains challenging. No standard treatment regimen is available nowadays. Current therapies mainly include surgical therapy, chemotherapy, and radiotherapy.

• #47 Effective treatment of apatinib in desmoplastic small round cell tumor: a case report and literature review | BMC Cancer | Full Text

  https://bmccancer.biomedcentral.com/articles/10.1186/s12885-018-4135-x

  Desmoplastic small round cell tumor (DSRCT) is a rare malignant sarcoma with poor prognosis due to lack of effective treatments. Apatinib is a new potent oral small-molecule tyrosine kinase inhibitor, and targets the intracellular domain of vascular endothelial growth factor receptor 2 (VEGFR-2). […] Apatinib was then recommended. Apatinib revealed outstanding effect on reducing mass size and ascites during 2-month treatment. Apatinib therapy continued for additional 2 months, and the patient was in good condition. […] It is the first report that apatinib is effective on DSRCT. This report may provide an additional option for the treatment of metastatic DSRCT. […] The treatment of DSRCT remains challenging. No standard treatment regimen is available nowadays. Current therapies mainly include surgical therapy, chemotherapy, and radiotherapy.

• #48

  https://journals.lww.com/cancerjournal/fulltext/2020/16050/chemotherapy_combined_with_apatinib_for_the.34.aspx

  We chose apatinib combined with chemotherapy considering the high degree of malignancy of DSRCT, the advantages of combination therapy, and the availability of the drugs. The patient was disease free for 5 months after applying this combination therapy with apatinib and it may continue to be effective. […] To our knowledge, this is the first valid case report of the use of apatinib combined with chemotherapy for the treatment of DSRCT, where it was determined that CA125 and NSE may be indicators of the degree of disease. More prospective studies are required in the future to verify the safety and efficacy of apatinib combined with chemotherapy, and more effective tumor markers need to be discovered.

• #49

  https://journals.lww.com/cancerjournal/fulltext/2020/16050/chemotherapy_combined_with_apatinib_for_the.34.aspx

  We chose apatinib combined with chemotherapy considering the high degree of malignancy of DSRCT, the advantages of combination therapy, and the availability of the drugs. The patient was disease free for 5 months after applying this combination therapy with apatinib and it may continue to be effective. […] To our knowledge, this is the first valid case report of the use of apatinib combined with chemotherapy for the treatment of DSRCT, where it was determined that CA125 and NSE may be indicators of the degree of disease. More prospective studies are required in the future to verify the safety and efficacy of apatinib combined with chemotherapy, and more effective tumor markers need to be discovered.

• #50 Desmoplastic small round cell tumour | Sarcoma UK

  https://sarcoma.org.uk/about-sarcoma/what-is-sarcoma/types-of-sarcoma/desmoplastic-small-round-cell-tumour/

  Researchers have been learning more about treatments known as targeted therapies and immunotherapies. Targeted therapy is a treatment that stops cancer cells from growing and spreading. It targets specific characteristics within the cells. Immunotherapy is a treatment that helps your immune system kill cancer cells. Clinical trials have found that some targeted and immunotherapies work well for desmoplastic small round cell tumour. But, more research and trials are needed before these treatments are approved.

• #51 Desmoplastic small round cell tumor: an update of current management practices | Journal of the Egyptian National Cancer Institute | Full Text

  https://jenci.springeropen.com/articles/10.1186/s43046-025-00276-0

  Immunotherapy represents an exciting frontier in cancer treatment, and potential targets for DSRCT have been identified. […] Recent studies have also shown genomic alterations beyond the known EWSR1 rearrangement. […] HER2 antibody-drug conjugates, such as trastuzumab deruxtecan and trastuzumab emtansine, have also demonstrated efficacy against HER2-positive DSRCT. […] Anlotinib, a multitarget tyrosine-kinase inhibitor showed promising results when combined with chemotherapy for the treatment of pediatric DSRCT.

• #52 Desmoplastic small round cell tumor: an update of current management practices | Journal of the Egyptian National Cancer Institute | Full Text

  https://jenci.springeropen.com/articles/10.1186/s43046-025-00276-0

  Immunotherapy represents an exciting frontier in cancer treatment, and potential targets for DSRCT have been identified. […] Recent studies have also shown genomic alterations beyond the known EWSR1 rearrangement. […] HER2 antibody-drug conjugates, such as trastuzumab deruxtecan and trastuzumab emtansine, have also demonstrated efficacy against HER2-positive DSRCT. […] Anlotinib, a multitarget tyrosine-kinase inhibitor showed promising results when combined with chemotherapy for the treatment of pediatric DSRCT.

• #53

  https://link.springer.com/article/10.1007/s00432-025-06102-3

  Since 1996, the mainstay of treatment has been aggressive chemotherapy followed by surgical resection where possible and radiotherapy to high-risk sites. […] In our case, the patient underwent chemotherapy regimens for urothelial carcinoma before trying a DSRCT-targeted regimen. […] Finally, the patients most sustained response was on enfortumab-vedotin, a novel anti-nectin-4 targeted antibody-drug conjugate highlighting a potential crossover between therapies for urothelial carcinoma and DSRCT. […] DSRCT is an ultra-rare sarcoma with limited treatment options and poor prognosis. Such a case of long sustained response and clinical benefit with EV is encouraging and merits exploration. Nectin-4 expression should be examined in DSRCT and other sarcomas as a potential prognostic and predictive biomarker.

• #54

  https://link.springer.com/article/10.1007/s00432-025-06102-3

  Desmoplastic small round cell tumor is a rare sarcoma type with poor prognosis for which there is no standard of care. It primarily affects adolescents and young adults and treatment is both multimodal and challenging due to the aggressive nature of the disease. […] After failing multiple lines of chemotherapy and also pembrolizumab, the patient was started on enfortumab-vedotin (EV), an antibody-drug conjugate targeting Nectin-4. Patient sustained partial response and documented disease control with clinical benefit for 23 months. […] This case report is the first documented usage of EV in the management of a desmoplastic small round cell tumor case and gives light to a new potential therapeutic strategy for this rare and aggressive tumor. […] The standard of care for DSRCT is constantly evolving as we explore new lines of therapy for an aggressive tumor with a generally poor prognosis; overall survival at five years is roughly 10-25%.

• #55 Multi-site desmoplastic small round cell tumors are genetically related and immune-cold | npj Precision Oncology

  https://www.nature.com/articles/s41698-022-00257-9

  Therefore, it is advantageous to combine bioinformatic predictions with patient profiling and in vitro data to identify viable therapeutic options for DSRCT patients. […] Similar to other types of fusion-driven sarcomas such as synovial sarcoma, our DSRCT samples had lower overall immune infiltrate than other tumors types, especially those against which ICI are effective. […] Therefore, as with other types of immunologically cold tumors, the immune response may require stimulation with vaccine, CAR T-cell, or NK cell-based therapies to elicit an immune response. […] The EWSR1-WT1 fusion is almost certainly a major driver in the early development and dispersion of DSRCT tumors.

• #56 Enzalutamide induces cytotoxicity in desmoplastic small round cell tumor independent of the androgen receptor | Communications Biology

  https://www.nature.com/articles/s42003-024-06003-0

  Desmoplastic Small Round Cell Tumor (DSRCT) is a rare, pediatric cancer caused by the EWSR1::WT1 fusion protein. DSRCT survival remains poor with a 5-year survival rate of 15-25% necessitating the development of novel therapeutic strategies. […] One hypothesis for the DSRCT male prevalence is that elevated testosterone levels in males activate the androgen receptor (AR) pathway and drive tumor growth. […] Based on these promising findings, Fine et al. treated six patients with combined androgen blockage including the first-generation AR blocker bicalutamide followed by the gonadotropin-releasing hormone Lupron. […] More recently, Lamhamedi-Cherradi et al. found that 75% of DSRCT patients from a set of 60 tumors stain positive for AR localized to the nucleus. […] We sought to use this novel model to enable the establishment of DSRCT xenografts with a lower number of cells.

• #57 Enzalutamide induces cytotoxicity in desmoplastic small round cell tumor independent of the androgen receptor | Communications Biology

  https://www.nature.com/articles/s42003-024-06003-0

  We expand on previous work by demonstrating that AR is expressed in the three most used DSRCT cell lines and that androgen treatment leads to AR nuclear localization. […] These findings suggest sustained AR dependence is not the reason for male predominance in DSRCT and that AR-targeted therapies may provide therapeutic benefits to patients in an AR-independent manner. […] The finding that DSRCT cells can proliferate with near complete knockdown of AR suggests enzalutamide and flutamide may reduce DSRCT growth via a mechanism independent of AR. […] Together these findings suggest enzalutamide cytotoxicity in DSRCT is independent of AR and may involve downregulation of the EWSR1::WT1 oncogene.

• #58 Desmoplastic small round cell tumor: an update of current management practices | Journal of the Egyptian National Cancer Institute | Full Text

  https://jenci.springeropen.com/articles/10.1186/s43046-025-00276-0

  Systemic chemotherapy, notably the P6 regimen, is the cornerstone of DSRCT treatment, while other options, including CDK4/6 inhibitors, antibody-drug conjugates, and anlotinib present varying efficacy. […] A novel chemotherapeutic agent, ONC-201, exhibits promise, inhibiting tumor growth in preclinical models. […] Surgical management, encompassing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (HIPEC), reveals improved survival rates, particularly when combined with chemotherapy. […] Whole abdomen radiotherapy (WART) emerges as a post-chemotherapy treatment option, despite potential complications. […] Immunotherapy, targeting receptors such as B7-H3, GD2, EGFR, HER2, tyrosine kinase inhibitors, and androgen receptors is a promising avenue, especially in younger populations, given its favorable long-term effects.

• #59 New Study Demonstrates Effectiveness of Cytoreductive Surgery for Desmoplastic Small Round Cell Tumor

  https://consultqd.clevelandclinic.org/new-study-demonstrates-effectiveness-of-cytoreductive-surgery-for-desmoplastic-small-round-cell-tumor

  In terms of treatment expectations, Dr. Anderson believes the study has set a minimum for what should be expected by a DSRCT patient to get to a low disease burden. […] We recently treated six patients with a drug called ONC201, which Dr. Hayes-Jordan previously tested in her lab. […] I think that this drug has promise in the adjuvant setting and the potential for use in relapse prevention, he says. […] According to Dr. Anderson, getting a consultation from an expert who is familiar with the principles of therapy for DSRCT is one of the most important steps in treating a very rare tumor such as DSRCT. […] I provide a summary, articles, clinicaltrials.gov information, and often PowerPoint images, so they have a much better idea of systemic and local control options. […] Also, we talk about how to improve intestinal health to make chemotherapy, surgery and radiotherapy less toxic and more effective for DSRCT, he says.

• #60 Desmoplastic small round cell tumor: an update of current management practices | Journal of the Egyptian National Cancer Institute | Full Text

  https://jenci.springeropen.com/articles/10.1186/s43046-025-00276-0

  Systemic chemotherapy, notably the P6 regimen, is the cornerstone of DSRCT treatment, while other options, including CDK4/6 inhibitors, antibody-drug conjugates, and anlotinib present varying efficacy. […] A novel chemotherapeutic agent, ONC-201, exhibits promise, inhibiting tumor growth in preclinical models. […] Surgical management, encompassing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (HIPEC), reveals improved survival rates, particularly when combined with chemotherapy. […] Whole abdomen radiotherapy (WART) emerges as a post-chemotherapy treatment option, despite potential complications. […] Immunotherapy, targeting receptors such as B7-H3, GD2, EGFR, HER2, tyrosine kinase inhibitors, and androgen receptors is a promising avenue, especially in younger populations, given its favorable long-term effects.

• #61 Desmoplastic small round cell tumor: an update of current management practices | Journal of the Egyptian National Cancer Institute | Full Text

  https://jenci.springeropen.com/articles/10.1186/s43046-025-00276-0

  Immunotherapy represents an exciting frontier in cancer treatment, and potential targets for DSRCT have been identified. […] Recent studies have also shown genomic alterations beyond the known EWSR1 rearrangement. […] HER2 antibody-drug conjugates, such as trastuzumab deruxtecan and trastuzumab emtansine, have also demonstrated efficacy against HER2-positive DSRCT. […] Anlotinib, a multitarget tyrosine-kinase inhibitor showed promising results when combined with chemotherapy for the treatment of pediatric DSRCT.

• #62 Pre-Targeted Radioimmunotherapy for Desmoplastic Small Round Cell Tumors | Alex’s Lemonade Stand Foundation for Childhood Cancer

  https://www.alexslemonade.org/project/pre-targeted-radioimmunotherapy-desmoplastic-small-round-cell-tumors

  Desmoplastic small round cell tumor is a rare cancer that affects children and young adults. Most patients have very advanced disease already when they are diagnosed, and the long-term survival rate is low. Current treatments can’t cure DSRCT and are very toxic. Patients who do survive are often left with debilitating side effects that last the rest of their lives. We have developed a new type of radiation therapy that targets the tumor cells specifically without harming normal tissue. In mice, this type of therapy can cure tumors without causing toxicity. […] The goal of our project is to test our new therapy in cells in mice to determine if it can destroy tumors and to make sure it doesn’t cause toxicity to normal organs. If these experiments are successful, doctors could start clinical trials using our therapy based on the evidence we generate during this project. Our long-term goal is to develop therapy that can cure DSRCT without leaving patients with lifelong adverse effects.

• #63 CXCR4-Directed Imaging and Endoradiotherapy in Desmoplastic Small Round Cell Tumors | Journal of Nuclear Medicine

  https://jnm.snmjournals.org/content/64/9/1424

  Four patients with end-stage progressive disease underwent CXCR4-based endoradiotherapy. We report the safety, response by RECIST 1.1, and survival after endoradiotherapy. […] We validated CXCR4 as a diagnostic biomarker and a promising target for endoradiotherapy in DSRCT, demonstrated its feasibility, and provided the first evidence of its clinical efficacy. […] CXCR4-directed endoradiotherapy in DSRCT is feasible and might prove a new option for patients with otherwise limited treatment alternatives.

• #64 Desmoplastic Small Round Cell Tumour Successfully Treated with Caffeine-assisted Chemotherapy: A Case Report and Review of the Literature | Anticancer Research

  https://ar.iiarjournals.org/content/30/9/3769

  Desmoplastic small round cell tumour (DSRCT) is a rare tumour, usually arising in the abdominal cavity. DSRCT remains an aggressive malignancy, with a poor prognosis despite multi-modality treatments. […] This report describes a case of DSRCT arising from the brachial plexus and successfully treated with caffeine-assisted chemotherapy. […] Therefore, it is concluded that caffeine-assisted chemotherapy should be one of the treatment options for DSRCT. […] This study presents the first report of DSRCT occurring in the brachial plexus and being successfully treated with caffeine-assisted chemotherapy. […] After five courses of chemotherapy were performed using cisplatin (120 mg/m2 for 2 h), doxorubicin (30 mg/m2/day for 2 days) and caffeine (1.5 g/m2/day for 3 days), the size of the tumour markedly decreased on MRI, and the accumulation of 201Tl and 99mTc-MIBI disappeared.

• #65 Desmoplastic Small Round Cell Tumour Successfully Treated with Caffeine-assisted Chemotherapy: A Case Report and Review of the Literature | Anticancer Research

  https://ar.iiarjournals.org/content/30/9/3769

  Three courses of additional chemotherapy were performed using ifosfamide (3 g/m2/day for 3 days), etoposide (60 mg/m2/day for 3 days) and caffeine (1.5 g/m2/day for 3 days), and radiation therapy with 60 Gy in total. […] In the present case, caffeine-assisted chemotherapy was performed. […] The present case showed good response to caffeine-assisted chemotherapy. […] Although the current treatments for DSRCT are not curative, caffeine-assisted chemotherapy combined with radiotherapy was effective for DSRCT in the present case.

• #66 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases. As most of the cases present as intra-peritoneal, aggressive tumors, the main guidelines recommend initiating treatment with systemic chemotherapy. Although DSRCT is not specifically addressed by ESMO or NCCN guideline, this recommendation is stated by the Chicago Consensus on Peritoneal Malignancies and by the MDAnderson Cancer Center nomogram. […] The role of surgery in the management of DSRCT is well established in the literature. In a review of 12 patients treated at Mayo Clinic, Hassan et al. showed the median survival of patients treated with surgical resection was 34 months, whereas the median survival of those who underwent biopsy alone was 14 months. In the report of Wong et al., the median survival for patients who had a resection for their abdominal or pelvic tumors was 47 months, compared to 16 months for those who did not. Complete cytoreductive surgery is associated with improved survival and should be considered a cornerstone of treatment together with chemotherapy.

• #67 Intra-Abdominal Desmoplastic Small Round Cell Tumor (DSRCT) and the Role of Hyperthermic Intraperitoneal Chemotherapy (HIPEC): A Review

  https://www.mdpi.com/1718-7729/30/4/299

  Given the chemosensitivity of DSRCT and the high proportion of metastatic disease, initial treatment is usually systemic chemotherapy. The regimens employed include those for soft tissue sarcoma which incorporate ifosfamide and doxorubicin or those established for Ewing sarcoma which are most commonly the P6 regimen (cyclophosphamide, doxorubicin, vincristine, ifosfamide, and etoposide) or VAIA (vincristine, dactinomycin, ifosfamide, and doxorubicin). […] There are no clear guidelines on patient selection for surgery in DSRCT. Almost all patients receive systemic chemotherapy prior to surgery, where progression on chemotherapy indicates poor tumor biology and precludes resection. […] The ultimate goal of surgery is complete cytoreduction but the potential risks of morbidity should be considered since this could prevent or delay postoperative treatments such as chemotherapy and/or radiotherapy. If the expected surgical morbidity and mortality from optimal cytoreduction are excessive, the surgeon may consider debulking surgery as an alternative.

• #68 Intra-Abdominal Desmoplastic Small Round Cell Tumor (DSRCT) and the Role of Hyperthermic Intraperitoneal Chemotherapy (HIPEC): A Review

  https://www.mdpi.com/1718-7729/30/4/299

  Given the chemosensitivity of DSRCT and the high proportion of metastatic disease, initial treatment is usually systemic chemotherapy. The regimens employed include those for soft tissue sarcoma which incorporate ifosfamide and doxorubicin or those established for Ewing sarcoma which are most commonly the P6 regimen (cyclophosphamide, doxorubicin, vincristine, ifosfamide, and etoposide) or VAIA (vincristine, dactinomycin, ifosfamide, and doxorubicin). […] There are no clear guidelines on patient selection for surgery in DSRCT. Almost all patients receive systemic chemotherapy prior to surgery, where progression on chemotherapy indicates poor tumor biology and precludes resection. […] The ultimate goal of surgery is complete cytoreduction but the potential risks of morbidity should be considered since this could prevent or delay postoperative treatments such as chemotherapy and/or radiotherapy. If the expected surgical morbidity and mortality from optimal cytoreduction are excessive, the surgeon may consider debulking surgery as an alternative.

• #69 Intra-Abdominal Desmoplastic Small Round Cell Tumor (DSRCT) and the Role of Hyperthermic Intraperitoneal Chemotherapy (HIPEC): A Review

  https://www.mdpi.com/1718-7729/30/4/299

  In the setting of disseminated abdominal disease, surgical resection alone may not provide durable control, as microscopic residual disease appears almost inevitable. The theoretical benefit of adding HIPEC immediately after CRS is to eradicate potential microscopic disease. […] At present, there is no clear evidence on the benefit of performing HIPEC after CRS for soft tissue sarcoma with peritoneal metastases. […] Considering the high risk of relapse, postoperative treatment is critical. After surgery, further consolidative regimens with radiation therapy and systemic therapy have been employed to target microscopic disease. […] Induction chemotherapy and complete cytoreduction are essential cornerstones in the treatment of DSRCT. Although CRS and even debulking surgery have demonstrated survival benefits, they should be restricted to patients who respond to chemotherapy and those without extra-peritoneal metastases.

• #70 Intra-Abdominal Desmoplastic Small Round Cell Tumor (DSRCT) and the Role of Hyperthermic Intraperitoneal Chemotherapy (HIPEC): A Review

  https://www.mdpi.com/1718-7729/30/4/299

  In the setting of disseminated abdominal disease, surgical resection alone may not provide durable control, as microscopic residual disease appears almost inevitable. The theoretical benefit of adding HIPEC immediately after CRS is to eradicate potential microscopic disease. […] At present, there is no clear evidence on the benefit of performing HIPEC after CRS for soft tissue sarcoma with peritoneal metastases. […] Considering the high risk of relapse, postoperative treatment is critical. After surgery, further consolidative regimens with radiation therapy and systemic therapy have been employed to target microscopic disease. […] Induction chemotherapy and complete cytoreduction are essential cornerstones in the treatment of DSRCT. Although CRS and even debulking surgery have demonstrated survival benefits, they should be restricted to patients who respond to chemotherapy and those without extra-peritoneal metastases.

• #71 Desmoplastic Small Round Cell Tumors | Symptoms, Diagnosis & Treatment

  https://www.cincinnatichildrens.org/health/d/desmoplastic-small-round-cell-tumors

  Desmoplastic small round cell tumors (DSRCT) are a type of rare, soft tissue sarcoma. This type of cancer usually begins in the abdomen or pelvis, and does not respond well to treatment. […] DSRCT is a very rare cancer. Therefore, patients who have it should be treated at a comprehensive sarcoma center with experts in the field of radiation oncology, surgery and oncology who have experience and expertise in diagnosing and treating this disease. […] The doctor may recommend surgery to remove as much of the tumor(s) as possible. Because these tumors grow in and around other organs, it usually is not possible to remove the entire tumor this way. Most patients need to have other therapies after their surgery. The medical team may offer some or all of the following treatments: Traditional radiation therapy, Proton therapy, which the doctor may recommend in special cases. Proton therapy is a type of radiation therapy that targets the tumor while avoiding organs and healthy tissue. This means fewer short-term and long-term side effects from radiation. Cincinnati Childrens is one of only a few pediatric hospitals in the country that offer proton therapy, High doses of chemotherapy to shrink the tumor(s) and prevent new ones from forming. This therapy may require the use of rescuing stem cells, which serve as an internal repair system for the body. Stem cell rescue involves removing stem cells from the blood between chemotherapy cycles and freezing them. After very high dose chemotherapy, the doctor thaws and injects the stem cells back into the body, where they begin forming new, healthy blood cells, A two-step surgical procedure called hyperthermic intra-peritoneal chemotherapy (HIPEC). This involves removing as much of the tumor as possible, then delivering highly concentrated, heated chemotherapy drugs directly to the abdominal area. This therapy is for patients whose cancer is located in the abdominal cavity, Maintenance therapy, which may be an option after an initial round of treatment. It can involve giving chemotherapy or molecularly targeted therapy, which uses drugs or other substances to interfere with specific molecules that allow cancer to grow and spread, One of several targeted tumor ablation techniques, which kill tumors.

• #72 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  Given the propensity for morbid intra-peritoneal progression, consolidative whole abdominopelvic radiotherapy (WAP-RT) as part of multimodal treatment (chemotherapy, surgical debulking and WAP-RT) was first reported using the P6 protocol, in an attempt to improve local control. Honoré et al. reported in a series of 38 patients with a median follow-up of almost 5 years, that multimodal treatment combining systemic chemotherapy, complete macroscopic resection, and postoperative WAP-RT could prolong survival in patients without the extra-peritoneal disease (EPM)—median survival of 37.7 months (range 7.9–42.9 months). The factors predictive of 3-year overall survival were the absence of EPM, complete surgical resection, postoperative WAP-RT and postoperative chemotherapy. Atallah et al. studied the prognostic role of WAP-RT on oncologic outcomes as part of multimodal treatment in 103 patients with abdominal DSRCT treated at eight French centers from 1991 to 2014. They concluded that RT seems to improve survival after cytoreductive surgery, with better PPFS, PFS and OS for the patients treated in a multimodal approach, with the limitations of a retrospective study, lack of statistical power (due to the small number of patients), and the need of randomized prospective studies to confirm these results.

• #73 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. Second-line treatment is ineffective in most cases. In our cohort, out of 19 patients treated with first-line chemotherapy, 13 received the second-line and the progression-free survival was only 3.9 months. This short survival time highlights the aggressiveness of this disease and the challenge in developing new therapeutic strategies to treat these young patients. Despite the development of new regimens for ES and other soft tissue and bone sarcoma in recent years, DSRCT is underrepresented or were not included in the trials that lead to the drug approval. As a result, the evidence to use second-line therapy is very limited. It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT.

• #74

  https://journals.lww.com/cancerjournal/fulltext/2020/16050/chemotherapy_combined_with_apatinib_for_the.34.aspx

  This patient has no chance of surgery, so he was treated with vincristine, epirubicin, and cyclophosphamide, which is classic and economical. The chemotherapy regimen of gemcitabine combined with docetaxel also showed a therapeutic effect on the disease. A chemotherapy of gemcitabine combined with nedaplatin showed partial reactivity after two cycles, which indicated a possible partial efficacy of the regimen. […] Apatinib is an oral TKI with antiangiogenic properties targeting vascular endothelial growth factor receptor (VEGFR)-2. Furthermore, there are encouraging clinical data that confirm the effectiveness of apatinib for gastric cancer and other solid tumors. When combined with chemotherapy, apatinib has been proven to be safe and to prolong survival in the treatment of solid tumors and soft tissue tumors.

• #75 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. […] Current management of DSRCT includes a combination of chemotherapy, radiation and aggressive cytoreductive surgery plus intra-peritoneal hyperthermic chemotherapy (HIPEC). […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. […] The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors was evaluated by small trials. […] Multimodal therapy combining multi-agent intensive chemotherapy, aggressive debulking surgery and adjuvant radiotherapy is considered the standard of care for patients presenting without extra-abdominal metastases.

• #76 Treatment of Abdominal Desmoplastic Small Round Cell Tumor | OTT

  https://www.dovepress.com/treatment-of-abdominal-desmoplastic-small-round-cell-tumor-induces-acu-peer-reviewed-fulltext-article-OTT

  Radiotherapy is commonly used to relieve local symptoms and is a supplementary treatment to surgery and chemotherapy. Whole-abdominal radiation therapy (WART) can be used as a consolidated chemotherapy method for small abdominal lesions. […] Although the combination of chemotherapy, surgery, and radiotherapy significantly improves the survival rate of patients with DSRCT, the overall prognosis remains poor because the majority of patients experience disease recurrence and die within three years. […] Tyrosine kinase inhibitors (TKI) targeting VEGF, VEGFR, and other proteins involved in tumor vascular proliferation have also been used in clinical practice. […] The patient we reported took oral anlotinib for maintenance treatment after radiotherapy, and the disease remained stable with mild diarrhea for more than half a year. […] This case highlights the importance of individualized multimodal treatment strategies for rare cancers. Additionally, it underscores the need to closely monitor the risk of a secondary tumor induced by high-intensity radiotherapy and chemotherapy.

• #77 Desmoplastic small-round-cell tumor – Wikipedia

  https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor

  DSRCT is frequently misdiagnosed. Adult patients should always be referred to a sarcoma specialist. This is an aggressive, rare, fast spreading tumor and both pediatric and adult patients should be treated at a sarcoma center. […] There is no standard protocol for the disease. However, recent research has reported that some patients respond to high-dose (P6 Protocol) chemotherapy, maintenance chemotherapy, debulking operation, cytoreductive surgery, and radiation therapy. […] Other treatment options include: hematopoietic stem cell transplantation, intensity-modulated radiation therapy, radiofrequency ablation, stereotactic body radiation therapy, intraperitoneal hyperthermic chemoperfusion, and clinical trials. […] A multi-modality approach of high-dose chemotherapy, aggressive surgical resection, radiation, and stem cell rescue improves survival for some patients. Reports have indicated that patients will initially respond to first line chemotherapy and treatment but that relapse is common. […] Some patients in remission or with inoperable tumor seem to benefit from long term low dose chemotherapy, turning DSRCT into a chronic disease.

• #78 9 Innovative Desmoplastic Small Round Cell Tumor Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors/desmoplastic-small-round-cell-tumors-treatment.html

  The specialists at Children’s Cancer Hospital are researching new ways to treat DSRCT, including targeted therapies to help the body fight cancer on a cellular level. […] After carefully evaluating your child’s case, our team of experts will discuss a recommended course of treatment for DSRCT. Since DSRCT is complex and seldom seen, no standard treatment exists. […] Surgery is almost always part of treatment for DSRCT. […] Some DSRCT patients respond to chemotherapy, but most relapse. Long-term, low-dose chemotherapy may help patients in remission or with a tumor that cannot be surgically removed. […] Radiation therapy (also called radiotherapy) uses high-energy beams to destroy cancer cells. […] Children’s Cancer Hospital is leading into the future of DSRCT treatment by developing innovative targeted therapies.

• #79 9 Innovative Desmoplastic Small Round Cell Tumor Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors/desmoplastic-small-round-cell-tumors-treatment.html

  In this minimally invasive surgery, doctors inject material to stop blood flow to an area. By starving the tumor of blood, its growth can be slowed or stopped. […] This procedure is similar to standard embolization, except chemotherapy agents are injected. […] DSRCT is treated in our Children’s Cancer Hospital. […] MD Anderson patients have access to clinical trials offering promising new treatments that cannot be found anywhere else.

• #80 9 Innovative Desmoplastic Small Round Cell Tumor Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors/desmoplastic-small-round-cell-tumors-treatment.html

  In this minimally invasive surgery, doctors inject material to stop blood flow to an area. By starving the tumor of blood, its growth can be slowed or stopped. […] This procedure is similar to standard embolization, except chemotherapy agents are injected. […] DSRCT is treated in our Children’s Cancer Hospital. […] MD Anderson patients have access to clinical trials offering promising new treatments that cannot be found anywhere else.

• #81 What are Desmoplastic Small Round Cell Tumors (DSRCT)? Symptoms, Risk Factors & Treatments | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/desmoplastic-small-round-cell-tumors.html

  Desmoplastic small round cell tumors (DSRCT) are a type of soft-tissue sarcoma. DSRCT is a dangerous type of cancer that often is resistant to treatment and frequently comes back after treatment. A team of highly focused renowned physicians, including surgeons, oncologists and radiologists, as well as a specialized support staff of nurses, physicians assistants and others follow your child from diagnosis through treatment. DSRCT often is difficult to diagnose. The Childrens Cancer Hospital utilizes the most advanced technology and techniques to pinpoint the cancer. We are at the forefront of discovering new therapies to help manage DSRCT, including: […] HIPEC (hyperthermic peritoneal perfusion with chemotherapy) which was pioneered at Childrens Cancer hospital, combines surgery with heated chemotherapy for a safe and often effective treatment. It has been shown to extend survival time in many patients. […] Targeted therapies that help fight DSRCT at the cellular level. DSRCT is treated in our Children’s Cancer Hospital. MD Anderson patients have access to clinical trials offering promising new treatments that cannot be found anywhere else.

• #82 Treatment of Abdominal Desmoplastic Small Round Cell Tumor | OTT

  https://www.dovepress.com/treatment-of-abdominal-desmoplastic-small-round-cell-tumor-induces-acu-peer-reviewed-fulltext-article-OTT

  Radiotherapy is commonly used to relieve local symptoms and is a supplementary treatment to surgery and chemotherapy. Whole-abdominal radiation therapy (WART) can be used as a consolidated chemotherapy method for small abdominal lesions. […] Although the combination of chemotherapy, surgery, and radiotherapy significantly improves the survival rate of patients with DSRCT, the overall prognosis remains poor because the majority of patients experience disease recurrence and die within three years. […] Tyrosine kinase inhibitors (TKI) targeting VEGF, VEGFR, and other proteins involved in tumor vascular proliferation have also been used in clinical practice. […] The patient we reported took oral anlotinib for maintenance treatment after radiotherapy, and the disease remained stable with mild diarrhea for more than half a year. […] This case highlights the importance of individualized multimodal treatment strategies for rare cancers. Additionally, it underscores the need to closely monitor the risk of a secondary tumor induced by high-intensity radiotherapy and chemotherapy.

• #83 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #84 Desmoplastic small round cell tumor: the report of two cases and literature analysis review of the radiological findings – Chen – Quantitative Imaging in Medicine and Surgery

  https://qims.amegroups.org/article/view/114164/html

  At present, there is no standard treatment protocol for DSRCT, and complete surgical resection of DSRCT is crucial when technically feasible. […] For most patients in advanced stages, effective systemic chemotherapy helps in systemic control. […] Other studies have shown that hyperthermic intraperitoneal chemotherapy (HIPEC) after cytoreductive surgery (CRS) is an effective local control method and can improve the survival rate of patients with DSRCT. […] Despite advances in multimodal therapy, patients with DSRCT have a poor prognosis, with about 60-70% dying from the disease within 2-3 years.

• #85 Development of Combination Therapies for Desmoplastic Small Round Cell Tumor (DSRCT) | Alex’s Lemonade Stand Foundation for Childhood Cancer

  https://www.alexslemonade.org/project/development-combination-therapies-desmoplastic-small-round-cell-tumor-dsrct

  Development of Combination Therapies for Desmoplastic Small Round Cell Tumor (DSRCT) […] Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive malignancy that is most commonly diagnosed in adolescent and young adult males. The patients are frequently metastatic at diagnosis and the overall prognosis is poor. There is currently no accepted standard treatment for this disease and despite aggressive chemotherapy, surgery, radiation therapy, and stem cell transplant, the 5-year survival is less than 15%. Therefore, there is a need for new therapies for this disease. […] Our lab has recently shown that DSRCT depends on the activity of the EWS-WT1 transcription factor for cell survival. Blocking the activity of this protein leads to tumor cell death. We have identified a compound, lurbinectedin, which inhibits EWS-WT1 leading to cell death in vitro and a decrease in tumor size in xenograft mouse models. The goal of this study is to identify compounds that work with lurbinectedin to further reduce cell viability.

• #86

  https://link.springer.com/article/10.1007/s00432-025-06102-3

  Since 1996, the mainstay of treatment has been aggressive chemotherapy followed by surgical resection where possible and radiotherapy to high-risk sites. […] In our case, the patient underwent chemotherapy regimens for urothelial carcinoma before trying a DSRCT-targeted regimen. […] Finally, the patients most sustained response was on enfortumab-vedotin, a novel anti-nectin-4 targeted antibody-drug conjugate highlighting a potential crossover between therapies for urothelial carcinoma and DSRCT. […] DSRCT is an ultra-rare sarcoma with limited treatment options and poor prognosis. Such a case of long sustained response and clinical benefit with EV is encouraging and merits exploration. Nectin-4 expression should be examined in DSRCT and other sarcomas as a potential prognostic and predictive biomarker.

• #87 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #88 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #89 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7865637/

  Hyperthermic intra-peritoneal chemotherapy (HIPEC) has been examined as an adjunctive intraoperative strategy. […] Despite aggressive multidisciplinary approaches, patients have a poor prognosis. […] DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. […] It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT. […] The use of preclinical models is an important step in the development of new therapies for tumors in general and is crucial for rare tumors such as DSRCT. […] The combination of trabectedin and PARP inhibitor is under investigation in clinical trials. […] The oncogenic fusion product EWSR1-WT1 in DSRCT was reported to activate the IGF-1R gene promoter, providing the basis to test the activity of anti-IGF-1R antibodies in the metastatic setting.

• #90 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

  https://www.mdpi.com/2072-6694/13/3/498

  DSRCT is characterized by poor response to conventional chemotherapy and early relapse after radical surgery. Second-line treatment is ineffective in most cases. In our cohort, out of 19 patients treated with first-line chemotherapy, 13 received the second-line and the progression-free survival was only 3.9 months. This short survival time highlights the aggressiveness of this disease and the challenge in developing new therapeutic strategies to treat these young patients. Despite the development of new regimens for ES and other soft tissue and bone sarcoma in recent years, DSRCT is underrepresented or were not included in the trials that lead to the drug approval. As a result, the evidence to use second-line therapy is very limited. It is paramount to develop active cooperative groups to quickly collect data and propose new strategies for the treatment of DSRCT.

• #91 The Role of Immunotherapy in Treating Desmoplastic Small Round Cell Tumors – Site Title

  https://carevitahealth.tech.blog/2025/02/20/the-role-of-immunotherapy-in-treating-desmoplastic-small-round-cell-tumors/

  The Desmoplastic Small Round Cell Tumors Drugs Market is witnessing a surge in research and clinical trials focused on targeted therapies, immunotherapy, and novel chemotherapeutic agents. Targeted therapies, such as tyrosine kinase inhibitors and immune checkpoint inhibitors, are gaining attention for their potential to offer more effective and less toxic treatment options. Additionally, the exploration of personalized medicine based on the genetic profile of individual tumors holds promise for improving treatment efficacy. […] As clinical trials progress and new drugs are approved, the Desmoplastic Small Round Cell Tumors Therapeutics Market is expected to expand, providing hope for better management of this rare and challenging disease.

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