Materiały źródłowe

• #1 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  If your child has rhabdomyosarcoma (RMS), you may have questions about the prognosis (outcome). A prognosis is the doctor’s best estimate of how cancer will affect someone and how it will respond to treatment. Prognosis and survival depend on many things. Only a doctor familiar with a child’s medical history, the type, stage and characteristics of the cancer, the treatments chosen and the response to treatment can put all of this information together with survival statistics to arrive at a prognosis. […] A prognostic factor is an aspect of the cancer or a characteristic of the child that the doctor will consider when making a prognosis. A predictive factor influences how a cancer will respond to a certain treatment. Prognostic and predictive factors are often discussed together. They both play a part in deciding on a treatment plan and a prognosis.

• #2 Rhabdomyosarcoma: Current Therapy, Challenges, and Future Approaches to Treatment Strategies

  https://www.mdpi.com/2072-6694/15/21/5269

  Rhabdomyosarcoma (RMS) is a rare pediatric sarcoma affecting skeletal muscle in children and young adults. It is responsible for 3% of all childhood malignant tumors and is the third most prevalent pediatric extracranial solid tumor. Despite advances in diagnostic and treatment methods and clinical trials to improve pediatric RMS survival rates, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The overall 5-year survival rates for RMS in children have exceeded 70%. However, despite advances in diagnostic and treatment methods over the past few decades, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The cure rate remains low and the current RMS therapies continue to pose potential life-threatening toxicities, which can lead to lifelong morbidity.

• #3 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] The survival rate is the percentage of people who live at least a certain amount of time (usually 5 years) after being diagnosed with cancer. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #4 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  Continual improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2017, the 5-year relative survival rate for patients with rhabdomyosarcoma increased from 53% to 71% for children younger than 15 years and from 30% to 52% for adolescents aged 15 to 19 years. In more recent years, improvements in outcome have plateaued. […] Rhabdomyosarcoma is usually curable in children with localized disease who receive combined-modality therapy, with more than 70% of patients surviving 5 years after diagnosis. Relapses are uncommon in patients who were alive and event free at 5 years, with a 10-year late-event rate of 9%. Relapses are more common in patients who have unresectable disease, tumor in an unfavorable site at diagnosis, or metastatic disease at diagnosis.

• #5 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] The survival rate is the percentage of people who live at least a certain amount of time (usually 5 years) after being diagnosed with cancer. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #6 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] The survival rate is the percentage of people who live at least a certain amount of time (usually 5 years) after being diagnosed with cancer. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #7 Survival Rates for Rhabdomyosarcoma by Risk Group | American Cancer Society

  https://www.cancer.org/cancer/types/rhabdomyosarcoma/detection-diagnosis-staging/staging-survival-rates.html

  Survival rates can give you an idea of what percentage of people in a similar situation (such as with the same type and stage of cancer) are still alive a certain amount of time after they were diagnosed. […] The survival rate is the percentage of people who live at least a certain amount of time (usually 5 years) after being diagnosed with cancer. […] For a person with RMS, the risk group is important in estimating their outlook. […] The overall 5-year survival for children with RMS is about 70%, while survival in adults is lower. […] Overall, the survival rate for children in the low-risk group ranges from about 70% to over 90%. […] For those in the intermediate-risk group, the survival rates range from about 50% to about 70%. […] The survival rate in this group is generally around 20% to 30%. […] Even when taking risk groups and other factors into account, survival rates are at best rough estimates.

• #8 Rhabdomyosarcoma: Current Therapy, Challenges, and Future Approaches to Treatment Strategies

  https://www.mdpi.com/2072-6694/15/21/5269

  Rhabdomyosarcoma (RMS) is a rare pediatric sarcoma affecting skeletal muscle in children and young adults. It is responsible for 3% of all childhood malignant tumors and is the third most prevalent pediatric extracranial solid tumor. Despite advances in diagnostic and treatment methods and clinical trials to improve pediatric RMS survival rates, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The overall 5-year survival rates for RMS in children have exceeded 70%. However, despite advances in diagnostic and treatment methods over the past few decades, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The cure rate remains low and the current RMS therapies continue to pose potential life-threatening toxicities, which can lead to lifelong morbidity.

• #9 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Rhabdomyosarcoma is usually curable in children with localized disease who receive combined-modality therapy, with more than 70% of patients surviving 5 years after diagnosis. Relapses are uncommon in patients who were alive and event free at 5 years, with a 10-year late-event rate of 9%. Relapses are more common in patients who have unresectable disease, tumor in an unfavorable site at diagnosis, or metastatic disease at diagnosis. […] The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including the following: Age, Site of origin, Tumor size, Resectability, Histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, Metastases at diagnosis, Lymph node involvement at diagnosis, Bone marrow involvement, Biological characteristics, Response to therapy, Circulating tumor DNA and RNA.

• #10 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children between the ages of 1 and 9 years tend to have a better prognosis than children 10 years of age and older or younger than 1 year. […] Where the tumour started in the body can affect the prognosis. […] There is a better prognosis with tumours that begin in: the area around the eye (called the orbit), areas of the head and neck not near the tissue surrounding the brain and spinal cord (called non-parameningeal areas), organs of the genitourinary system (except the kidney, bladder and prostate), the gallbladder or the bile ducts. […] Tumours in any other location have a poorer prognosis. This includes tumours that begin in: the bladder, prostate, arm, leg, hand or foot, areas of the head and neck near the meninges (called parameningeal areas). […] Tumours that start in the arm or leg tend to spread to other areas earlier than tumours that start in other parts of the body, so they have a poorer prognosis.

• #11 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] The extent of disease after the primary surgical procedure is correlated with outcome. […] The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] The prognostic significance of metastatic disease is modified by the following: Tumor histology, Age at diagnosis, The site of primary disease, The number of metastatic sites.

• #12 Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis From Four International Cooperative Groups

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5002346/

  Extremity rhabdomyosarcomas do not always show satisfactory outcomes. We analyzed data from 643 patients treated in 14 studies conducted by European and North American groups between 1983 and 2004 to identify factors predictive of outcome. […] 5-year OS were 67% (se 1.8). Multivariate analysis showed that lower OS correlated with age 3 years, T2 and N1 stage, incomplete initial surgery, treatment before 1995, and European cooperative group treatment. […] This meta-analysis confirms the role of many established prognostic factors but identifies for the first time that chemotherapy duration may have an impact on outcome in patients with grossly resected tumors. […] The 10-year OS rate of 62% confirms the relatively less favorable prognosis of extremity RMS as compared to other sites. […] Age is a prognostic factor and patients 10 years old or more had a poorer survival than younger patients, consistent with data from other studies.

• #13 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  Nonetheless, intensive local therapy should be always considered for those at high risk for local recurrence, including intermediate-risk disease, advanced IRS stage, large tumors or narrow surgical margins. […] The 5y and 10y-OS rates were 52.7% and 42.7%, respectively, with a median survival time of 74 months (range, 1266 months). […] Low-risk patients had better 5y-OS and PFS rates (77% and 73%) than intermediate-risk patients (35% and 40% respectively, p0.001). […] Risk of disease progression increased by 1.32 for every unit (year) increase in age. […] The survival benefit of chemotherapy was more evident in PFS compared to OS results. […] On multivariate analysis, the chemotherapy effect on OS was lost, but remained significant in PFS analysis. […] Use of anthracycline-only based chemotherapy (no cyclophosphamide/ifosfamide) was associated with a significant high risk of disease progression.

• #14 Rhabdomyosarcoma: Current Therapy, Challenges, and Future Approaches to Treatment Strategies

  https://www.mdpi.com/2072-6694/15/21/5269

  The treatment strategies for RMS include multi-agent chemotherapies after surgical resection with or without radiotherapy. […] Prognostic stratification is significant because 15–20% of children have diffused metastatic disease at the time of diagnosis. […] Adults with RMS also experience poor outcomes, with 5-year survival rates ranging from 26.6 to 61%. […] Over the last three decades, there have been several national and international clinical trials which have resulted in refined treatment regimens based on the tumor stage and clinical group, leading to improved pediatric RMS survival rates. […] However, despite these developments, the cure rate for pediatric patients with metastatic or recurrent disease remains low and current RMS therapies continue to pose potential life-threatening toxicities, which can lead to lifelong morbidity.

• #15 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children between the ages of 1 and 9 years tend to have a better prognosis than children 10 years of age and older or younger than 1 year. […] Where the tumour started in the body can affect the prognosis. […] There is a better prognosis with tumours that begin in: the area around the eye (called the orbit), areas of the head and neck not near the tissue surrounding the brain and spinal cord (called non-parameningeal areas), organs of the genitourinary system (except the kidney, bladder and prostate), the gallbladder or the bile ducts. […] Tumours in any other location have a poorer prognosis. This includes tumours that begin in: the bladder, prostate, arm, leg, hand or foot, areas of the head and neck near the meninges (called parameningeal areas). […] Tumours that start in the arm or leg tend to spread to other areas earlier than tumours that start in other parts of the body, so they have a poorer prognosis.

• #16 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children between the ages of 1 and 9 years tend to have a better prognosis than children 10 years of age and older or younger than 1 year. […] Where the tumour started in the body can affect the prognosis. […] There is a better prognosis with tumours that begin in: the area around the eye (called the orbit), areas of the head and neck not near the tissue surrounding the brain and spinal cord (called non-parameningeal areas), organs of the genitourinary system (except the kidney, bladder and prostate), the gallbladder or the bile ducts. […] Tumours in any other location have a poorer prognosis. This includes tumours that begin in: the bladder, prostate, arm, leg, hand or foot, areas of the head and neck near the meninges (called parameningeal areas). […] Tumours that start in the arm or leg tend to spread to other areas earlier than tumours that start in other parts of the body, so they have a poorer prognosis.

• #17 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children between the ages of 1 and 9 years tend to have a better prognosis than children 10 years of age and older or younger than 1 year. […] Where the tumour started in the body can affect the prognosis. […] There is a better prognosis with tumours that begin in: the area around the eye (called the orbit), areas of the head and neck not near the tissue surrounding the brain and spinal cord (called non-parameningeal areas), organs of the genitourinary system (except the kidney, bladder and prostate), the gallbladder or the bile ducts. […] Tumours in any other location have a poorer prognosis. This includes tumours that begin in: the bladder, prostate, arm, leg, hand or foot, areas of the head and neck near the meninges (called parameningeal areas). […] Tumours that start in the arm or leg tend to spread to other areas earlier than tumours that start in other parts of the body, so they have a poorer prognosis.

• #18 Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis From Four International Cooperative Groups

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5002346/

  Extremity rhabdomyosarcomas do not always show satisfactory outcomes. We analyzed data from 643 patients treated in 14 studies conducted by European and North American groups between 1983 and 2004 to identify factors predictive of outcome. […] 5-year OS were 67% (se 1.8). Multivariate analysis showed that lower OS correlated with age 3 years, T2 and N1 stage, incomplete initial surgery, treatment before 1995, and European cooperative group treatment. […] This meta-analysis confirms the role of many established prognostic factors but identifies for the first time that chemotherapy duration may have an impact on outcome in patients with grossly resected tumors. […] The 10-year OS rate of 62% confirms the relatively less favorable prognosis of extremity RMS as compared to other sites. […] Age is a prognostic factor and patients 10 years old or more had a poorer survival than younger patients, consistent with data from other studies.

• #19 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #20 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The prognosis for children or adolescents with rhabdomyosarcoma is related to many clinical and biological factors, including age, site of origin, tumor size, resectability, histopathological subtype, PAX3::FOXO1 or PAX7::FOXO1 gene fusion status, metastases at diagnosis, lymph node involvement at diagnosis, biological characteristics, response to therapy, circulating tumor DNA and RNA. […] Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Children with tumors 5 cm or smaller have improved survival, compared with children with tumors larger than 5 cm. […] The extent of disease after the primary surgical procedure is correlated with outcome.

• #21 Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis From Four International Cooperative Groups

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5002346/

  Tumor invasiveness (T2 stage), tumor size (5 cm), and locoregional nodal involvement, findings similar to those reported for RMS regardless of tumor site. […] Overall, patients treated in North American studies fared better than patients treated in Europe, even after adjustment for other prognostic factors. […] In this subset of patients, multivariate analysis showed that tumor invasiveness and location in hands/feet were prognostically important in addition to cooperative group. […] The impact of treatment duration has not been previously reported as prognostic in studies of RMS, but the value of prolonged additional treatment with maintenance chemotherapy is now under exploration in current European studies.

• #22 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The COG analyzed ctDNA in 124 patients with newly diagnosed, intermediate-risk rhabdomyosarcoma. Patients with fusion-negative rhabdomyosarcoma with detectable ctDNA at diagnosis had significantly worse outcomes than patients without detectable ctDNA. […] The estimated 4-year EFS rate was 53% for patients with confirmed FOXO1 fusion-positive rhabdomyosarcoma. Multivariate analysis identified older age and larger tumor size as independent, adverse prognostic factors for EFS within this population.

• #23 Outcome and prognostic variables in childhood rhabdomyosarcoma (RMS) with emphasis on impact of FOXO1 Fusions in non-metastatic RMS: Experience from a tertiary

  https://ecancer.org/en/journal/article/1539-outcome-and-prognostic-variables-in-childhood-rhabdomyosarcoma-rms-with-emphasis-on-impact-of-foxo1-fusions-in-non-metastatic-rms-experience-from-a-tertiary-cancer-centre-in-india

  While factors influencing outcomes of rhabdomyosarcoma (RMS) in developed countries have evolved from clinical characteristics to molecular profiles, similar data from developing countries are scarce. […] The 5-year-event-free survival (EFS)/overall survival (OS) of Cohorts 1, 2 and 3 was 48.5%/55.5%, 54.6%/62.6% and 55.1%/63.7%, respectively. […] Amongst the localised RMS, presence of nodal metastases and primary tumour size 10 cms were adverse prognostic factors (p0.05). […] While FOXO1-fusions have superior prognostic utility compared to histology alone in localised, favourable-site RMS, traditional prognostic factors (tumour size and nodal metastases) impacted outcome the most in this subset. […] The 5-year EFS and OS in our cohort is inferior to comparable cohorts from western studies.

• #24 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #25 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] The extent of disease after the primary surgical procedure is correlated with outcome. […] The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] The prognostic significance of metastatic disease is modified by the following: Tumor histology, Age at diagnosis, The site of primary disease, The number of metastatic sites.

• #26 An adult spindle cell rhabdomyosarcoma in the head and neck region with long-term survival: a case report | Journal of Medical Case Reports | Full Text

  https://jmedicalcasereports.biomedcentral.com/articles/10.1186/1752-1947-8-208

  Spindle cell rhabdomyosarcoma of the head and neck is a very rare tumor in adults. […] In contrast to childhood tumors, spindle cell rhabdomyosarcoma in adulthood is often associated with a poor prognosis. […] The key factors for predicting overall survival are more likely metastatic disease at presentation, a poor response to chemotherapy, tumor size and a negative margin status after primary resection. […] The poorer prognosis of adult sc-rms is in striking contrast to the prognosis of childhood sc-rms, which could be because older patients are less tolerant of intensive chemotherapy and that their tumors are less chemosensitive. […] The radical surgical treatment performed in the presented case could have helped to prolong the patients overall survival until today. […] Of particular interest in this case is the patients overall survival of 82 months since his primary diagnosis in 2007, which is, to our knowledge, the longest overall survival of an adult patient with an sc-rms in the head and neck reported so far. […] Despite aggressive surgical resection with free margins, local recurrence is common.

• #27 An adult spindle cell rhabdomyosarcoma in the head and neck region with long-term survival: a case report | Journal of Medical Case Reports | Full Text

  https://jmedicalcasereports.biomedcentral.com/articles/10.1186/1752-1947-8-208

  Spindle cell rhabdomyosarcoma of the head and neck is a very rare tumor in adults. […] In contrast to childhood tumors, spindle cell rhabdomyosarcoma in adulthood is often associated with a poor prognosis. […] The key factors for predicting overall survival are more likely metastatic disease at presentation, a poor response to chemotherapy, tumor size and a negative margin status after primary resection. […] The poorer prognosis of adult sc-rms is in striking contrast to the prognosis of childhood sc-rms, which could be because older patients are less tolerant of intensive chemotherapy and that their tumors are less chemosensitive. […] The radical surgical treatment performed in the presented case could have helped to prolong the patients overall survival until today. […] Of particular interest in this case is the patients overall survival of 82 months since his primary diagnosis in 2007, which is, to our knowledge, the longest overall survival of an adult patient with an sc-rms in the head and neck reported so far. […] Despite aggressive surgical resection with free margins, local recurrence is common.

• #28 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  This is the second-largest retrospective analysis addressing the controversy of whether adult rhabdomyosarcoma (RMS) should be treated with chemotherapy regimens adopted from pediatric RMS protocols or adult soft-tissue sarcoma protocols. […] Increasing age, intermediate-risk disease, no chemotherapy use, anthacycline-based and poor chemotherapy response were significant predictors of poor overall and progression-free survival. […] Intermediate-risk disease was a significant predictor of poor chemotherapy response. […] Overall survival of clinical group-III patients was significantly improved if they underwent delayed complete resection. […] This study highlights the superiority of chemotherapy regimens adapted from pediatric protocols- compared to anthracycline-based regimens. […] There is lack of data to support the routine use of adjuvant radiotherapy for non-parameningeal group-I patients.

• #29 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #30 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features and is generally associated with a worse outcome than in similar patients with embryonal rhabdomyosarcoma. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] Lymph node involvement at diagnosis is seen in about 23% of patients with rhabdomyosarcoma and is associated with an inferior prognosis. […] It is unlikely that response to induction chemotherapy or best tumor response during therapy correlates with the likelihood of survival in patients with rhabdomyosarcoma. […] A retrospective study of 99 children with rhabdomyosarcoma used reverse transcription-polymerase chain reaction to analyze an 11-gene panel in peripheral blood and bone marrow samples at the time of initial diagnosis. The 5-year EFS rate was 35.5% for the 33 patients who were RNA positive, compared with 88.0% for the 66 patients who were RNA negative.

• #31 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] The extent of disease after the primary surgical procedure is correlated with outcome. […] The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] The prognostic significance of metastatic disease is modified by the following: Tumor histology, Age at diagnosis, The site of primary disease, The number of metastatic sites.

• #32 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features and is generally associated with a worse outcome than in similar patients with embryonal rhabdomyosarcoma. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] Lymph node involvement at diagnosis is seen in about 23% of patients with rhabdomyosarcoma and is associated with an inferior prognosis. […] It is unlikely that response to induction chemotherapy or best tumor response during therapy correlates with the likelihood of survival in patients with rhabdomyosarcoma. […] A retrospective study of 99 children with rhabdomyosarcoma used reverse transcription-polymerase chain reaction to analyze an 11-gene panel in peripheral blood and bone marrow samples at the time of initial diagnosis. The 5-year EFS rate was 35.5% for the 33 patients who were RNA positive, compared with 88.0% for the 66 patients who were RNA negative.

• #33 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Lymph node involvement at diagnosis is seen in about 23% of patients with rhabdomyosarcoma and is associated with an inferior prognosis. […] The COG reviewed the correlation between anaplastic histology and germline TP53 pathogenic variants in patients with rhabdomyosarcoma. […] The COG performed a retrospective analysis of 269 patients with confirmed FOXO1 fusion-positive rhabdomyosarcoma who were enrolled in three completed clinical trials for localized rhabdomyosarcoma. […] The estimated 4-year EFS rate was 53%, and the OS rate was 69%. […] The COG performed a retrospective analysis of patients with rhabdomyosarcoma who had bone marrow metastasis at initial presentation and were treated in COG protocols. […] The 3-year EFS rate was 15.4%, and the 5-year EFS rate was 14.5%. […] The 3-year OS rate was 30.4%, and the 5-year OS rate was 18.0%.

• #34 Outcome and prognostic variables in childhood rhabdomyosarcoma (RMS) with emphasis on impact of FOXO1 Fusions in non-metastatic RMS: Experience from a tertiary

  https://ecancer.org/en/journal/article/1539-outcome-and-prognostic-variables-in-childhood-rhabdomyosarcoma-rms-with-emphasis-on-impact-of-foxo1-fusions-in-non-metastatic-rms-experience-from-a-tertiary-cancer-centre-in-india

  In our cohort, the associations were strongest for traditional clinical variables like tumour size, nodal status and site of the tumour, with tumour size retaining its significance in multivariate analysis. […] FOXO1-positive tumours did have an inferior survival but this association was not significant on univariate analysis. […] In the subset of favourable tumours however, presence of FOXO1 fusions portended an inferior survival trending towards statistical significance (p = 0.063). […] All patients who were re-categorised as LR (FOXO1 negative) had a 5-year OS of 91%. […] Streamlining risk-stratification using FOXO1 fusions has teased out a smaller yet highly favourable subset within the LR (A) and LR (B) RMS, who could be candidates for de-escalation of therapy.

• #35 Outcome and prognostic variables in childhood rhabdomyosarcoma (RMS) with emphasis on impact of FOXO1 Fusions in non-metastatic RMS: Experience from a tertiary

  https://ecancer.org/en/journal/article/1539-outcome-and-prognostic-variables-in-childhood-rhabdomyosarcoma-rms-with-emphasis-on-impact-of-foxo1-fusions-in-non-metastatic-rms-experience-from-a-tertiary-cancer-centre-in-india

  In our cohort, the associations were strongest for traditional clinical variables like tumour size, nodal status and site of the tumour, with tumour size retaining its significance in multivariate analysis. […] FOXO1-positive tumours did have an inferior survival but this association was not significant on univariate analysis. […] In the subset of favourable tumours however, presence of FOXO1 fusions portended an inferior survival trending towards statistical significance (p = 0.063). […] All patients who were re-categorised as LR (FOXO1 negative) had a 5-year OS of 91%. […] Streamlining risk-stratification using FOXO1 fusions has teased out a smaller yet highly favourable subset within the LR (A) and LR (B) RMS, who could be candidates for de-escalation of therapy.

• #36 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using Somatic Molecular Biomarkers | medRxiv

  https://www.medrxiv.org/content/10.1101/2024.09.04.24313032v1.full-text

  By predicting 5-year EFS for individual patients in Table 4, we demonstrated that expected survival can change dramatically in ways that may suggest alternative therapies when molecular markers are included. […] This study provides supportive evidence to reinforce the importance of TP53 and MYOD1 mutations as molecular markers of poor prognosis. […] In a large cohort of pediatric patients with RMS, we identified several promising prognostic biomarkers that may improve risk-adapted treatment assignment. Mutations in NF1 are associated with a substantially decreased risk of treatment failure, and mutations in CDNK2A, MET, and MYCN are associated with an increased risk.

• #37 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using… | Mark Zobeck, MD, MPH

  https://www.linkedin.com/posts/markzobeck_improving-individualized-rhabdomyosarcoma-activity-7293674069013508097-1-gN

  We have a paper out in JCO Precision Oncology describing new biomarkers for pediatric rhabdomyosarcoma! Prediction modeling revealed gene mutations that clearly predict survival outcomes: – CDKN2A, MET, MYCN mutations associated with decreased survival. – NF1 mutations associated with increased survival. […] We’re hoping these biomarkers help make progress in RMS treatment, which has been very difficult over the last few decades.

• #38 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #39 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] The extent of disease after the primary surgical procedure is correlated with outcome. […] The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] The prognostic significance of metastatic disease is modified by the following: Tumor histology, Age at diagnosis, The site of primary disease, The number of metastatic sites.

• #40 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Children aged 1 to 9 years have the best prognosis, while those younger than 1 year and older than 10 years fare less well. […] The prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] Prognosis for childhood rhabdomyosarcoma varies according to the primary tumor site. […] The extent of disease after the primary surgical procedure is correlated with outcome. […] The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] The prognostic significance of metastatic disease is modified by the following: Tumor histology, Age at diagnosis, The site of primary disease, The number of metastatic sites.

• #41 Rhabdomyosarcoma: Current Therapy, Challenges, and Future Approaches to Treatment Strategies

  https://www.mdpi.com/2072-6694/15/21/5269

  The treatment strategies for RMS include multi-agent chemotherapies after surgical resection with or without radiotherapy. […] Prognostic stratification is significant because 15–20% of children have diffused metastatic disease at the time of diagnosis. […] Adults with RMS also experience poor outcomes, with 5-year survival rates ranging from 26.6 to 61%. […] Over the last three decades, there have been several national and international clinical trials which have resulted in refined treatment regimens based on the tumor stage and clinical group, leading to improved pediatric RMS survival rates. […] However, despite these developments, the cure rate for pediatric patients with metastatic or recurrent disease remains low and current RMS therapies continue to pose potential life-threatening toxicities, which can lead to lifelong morbidity.

• #42 An emerging evidence base for PET-CT in the management of childhood rhabdomyosarcoma: systematic review | BMJ Open

  https://bmjopen.bmj.com/content/5/1/e006030

  Also predictive of survival were PET-CT detection of nodal involvement (p=0.016), PET-CT detection of metastases (p=0.002) and a composite outcome (PET group; p=0.002). […] PET-CT at initial staging may have predictive value for OS and EFS. […] The ultimate impact of FI with PET-CT on treatment outcomes could not be addressed and it remains unclear whether and how increasing accuracy at initial staging might alter patient management and survival. […] This review highlights potential from PET-CT in imaging of children and adolescents with RMS but there is a high level of uncertainty in these data and their relevance to clinical practice.

• #43 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #44 Childhood Rhabdomyosarcoma Treatment (PDQ®) – NCI

  https://www.cancer.gov/types/soft-tissue-sarcoma/hp/rhabdomyosarcoma-treatment-pdq

  Lymph node involvement at diagnosis is seen in about 23% of patients with rhabdomyosarcoma and is associated with an inferior prognosis. […] The COG reviewed the correlation between anaplastic histology and germline TP53 pathogenic variants in patients with rhabdomyosarcoma. […] The COG performed a retrospective analysis of 269 patients with confirmed FOXO1 fusion-positive rhabdomyosarcoma who were enrolled in three completed clinical trials for localized rhabdomyosarcoma. […] The estimated 4-year EFS rate was 53%, and the OS rate was 69%. […] The COG performed a retrospective analysis of patients with rhabdomyosarcoma who had bone marrow metastasis at initial presentation and were treated in COG protocols. […] The 3-year EFS rate was 15.4%, and the 5-year EFS rate was 14.5%. […] The 3-year OS rate was 30.4%, and the 5-year OS rate was 18.0%.

• #45 Prognosis and survival for rhabdomyosarcoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/rhabdomyosarcoma/prognosis-and-survival

  Children with tumours that are 5 cm or less in diameter have a better prognosis than children with larger tumours. […] Children with cancer that has already spread, or metastasized, to distant parts of the body when it is diagnosed have a poorer prognosis. Children with cancer that has spread to regional lymph nodes have a poorer prognosis than children with cancer that hasn’t spread to any lymph nodes. Children with cancer in the brain and spinal cord (called the central nervous system, or CNS) have a poor prognosis. […] How much cancer is left behind after surgery is also an important prognostic factor. Children with tumours that can be completely removed usually have a better prognosis. […] Alveolar tumours tend to be more aggressive and have a poorer prognosis than embryonal tumours.

• #46 An emerging evidence base for PET-CT in the management of childhood rhabdomyosarcoma: systematic review | BMJ Open

  https://bmjopen.bmj.com/content/5/1/e006030

  Also predictive of survival were PET-CT detection of nodal involvement (p=0.016), PET-CT detection of metastases (p=0.002) and a composite outcome (PET group; p=0.002). […] PET-CT at initial staging may have predictive value for OS and EFS. […] The ultimate impact of FI with PET-CT on treatment outcomes could not be addressed and it remains unclear whether and how increasing accuracy at initial staging might alter patient management and survival. […] This review highlights potential from PET-CT in imaging of children and adolescents with RMS but there is a high level of uncertainty in these data and their relevance to clinical practice.

• #47 Outcome and prognostic variables in childhood rhabdomyosarcoma (RMS) with emphasis on impact of FOXO1 Fusions in non-metastatic RMS: Experience from a tertiary

  https://ecancer.org/en/journal/article/1539-outcome-and-prognostic-variables-in-childhood-rhabdomyosarcoma-rms-with-emphasis-on-impact-of-foxo1-fusions-in-non-metastatic-rms-experience-from-a-tertiary-cancer-centre-in-india

  In our cohort, the associations were strongest for traditional clinical variables like tumour size, nodal status and site of the tumour, with tumour size retaining its significance in multivariate analysis. […] FOXO1-positive tumours did have an inferior survival but this association was not significant on univariate analysis. […] In the subset of favourable tumours however, presence of FOXO1 fusions portended an inferior survival trending towards statistical significance (p = 0.063). […] All patients who were re-categorised as LR (FOXO1 negative) had a 5-year OS of 91%. […] Streamlining risk-stratification using FOXO1 fusions has teased out a smaller yet highly favourable subset within the LR (A) and LR (B) RMS, who could be candidates for de-escalation of therapy.

• #48 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  In contrast, patients treated with cyclophosphamide-based, cyclophosphamide+anthracycline-based, or cyclophosphamide+ifosfamide+anthracycline-based combinations fared significantly better in terms of 5y-PFS (64%, 74%, 80% vs. 47%; p=0.091, 0.016, 0.037 respectively) and 5y-OS (56%, 78% and 48% vs. 36%; p=0.022,0.001, 0.039 respectively). […] Belonging to the intermediate-risk group retained prognostic significance after including chemotherapy response in the multivariate models of OS (HR 2.69; 95%CI 1.774.095; C-index: 72%). […] The prognosis of intermediate-risk group patients still improved if their disease responded to chemotherapy (p0.001). […] The highest risk of local recurrence was seen in patients with IRS stage-II (unfavorable site), intermediate-risk disease, residual tumor (Group II-III), anthracycline-based regimen and PR to initial CT.

• #49 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  This is the second-largest retrospective analysis addressing the controversy of whether adult rhabdomyosarcoma (RMS) should be treated with chemotherapy regimens adopted from pediatric RMS protocols or adult soft-tissue sarcoma protocols. […] Increasing age, intermediate-risk disease, no chemotherapy use, anthacycline-based and poor chemotherapy response were significant predictors of poor overall and progression-free survival. […] Intermediate-risk disease was a significant predictor of poor chemotherapy response. […] Overall survival of clinical group-III patients was significantly improved if they underwent delayed complete resection. […] This study highlights the superiority of chemotherapy regimens adapted from pediatric protocols- compared to anthracycline-based regimens. […] There is lack of data to support the routine use of adjuvant radiotherapy for non-parameningeal group-I patients.

• #50 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The alveolar subtype of childhood rhabdomyosarcoma is more prevalent among patients with less favorable clinical features and is generally associated with a worse outcome than in similar patients with embryonal rhabdomyosarcoma. […] Approximately 80% of rhabdomyosarcoma cases morphologically defined as alveolar rhabdomyosarcoma express a FOXO1 fusion. […] Children with metastatic disease at diagnosis have the worst prognosis. […] Lymph node involvement at diagnosis is seen in about 23% of patients with rhabdomyosarcoma and is associated with an inferior prognosis. […] It is unlikely that response to induction chemotherapy or best tumor response during therapy correlates with the likelihood of survival in patients with rhabdomyosarcoma. […] A retrospective study of 99 children with rhabdomyosarcoma used reverse transcription-polymerase chain reaction to analyze an 11-gene panel in peripheral blood and bone marrow samples at the time of initial diagnosis. The 5-year EFS rate was 35.5% for the 33 patients who were RNA positive, compared with 88.0% for the 66 patients who were RNA negative.

• #51 The Value of Early Tumor Size Response to Chemotherapy in Pediatric Rhabdomyosarcoma

  https://www.mdpi.com/2072-6694/13/3/510

  The results of this systematic review, including data from large international study groups, clearly illustrate the discordant results in the existing literature on the prognostic value of early tumor size response in pediatric RMS. There is no evidence that the degree of response at early response evaluation, except for patients with progressive disease, is a prognostic marker for survival. As the majority of patients do not have progressive disease, the use of early tumor size response should be reconsidered for individual treatment adjustments or to evaluate new treatment strategies. However, the results do illustrate that the early progression of disease impacts survival. […] The results of the included studies are summarized in Table 4. Different response criteria were used to assess tumor size response by conventional imaging; three studies used two-dimensional measurements according to WHO criteria, two studies used three-dimensional measurements.

• #52 The Value of Early Tumor Size Response to Chemotherapy in Pediatric Rhabdomyosarcoma

  https://www.mdpi.com/2072-6694/13/3/510

  The current literature shows no evidence for early tumor size response as an early surrogate marker of survival in children with non-progressive RMS. Therefore, tumor size response should not be used to adapt subsequent treatment, except for patients showing progression of disease at early assessment.

• #53 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  This is the second-largest retrospective analysis addressing the controversy of whether adult rhabdomyosarcoma (RMS) should be treated with chemotherapy regimens adopted from pediatric RMS protocols or adult soft-tissue sarcoma protocols. […] Increasing age, intermediate-risk disease, no chemotherapy use, anthacycline-based and poor chemotherapy response were significant predictors of poor overall and progression-free survival. […] Intermediate-risk disease was a significant predictor of poor chemotherapy response. […] Overall survival of clinical group-III patients was significantly improved if they underwent delayed complete resection. […] This study highlights the superiority of chemotherapy regimens adapted from pediatric protocols- compared to anthracycline-based regimens. […] There is lack of data to support the routine use of adjuvant radiotherapy for non-parameningeal group-I patients.

• #54 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  In contrast, patients treated with cyclophosphamide-based, cyclophosphamide+anthracycline-based, or cyclophosphamide+ifosfamide+anthracycline-based combinations fared significantly better in terms of 5y-PFS (64%, 74%, 80% vs. 47%; p=0.091, 0.016, 0.037 respectively) and 5y-OS (56%, 78% and 48% vs. 36%; p=0.022,0.001, 0.039 respectively). […] Belonging to the intermediate-risk group retained prognostic significance after including chemotherapy response in the multivariate models of OS (HR 2.69; 95%CI 1.774.095; C-index: 72%). […] The prognosis of intermediate-risk group patients still improved if their disease responded to chemotherapy (p0.001). […] The highest risk of local recurrence was seen in patients with IRS stage-II (unfavorable site), intermediate-risk disease, residual tumor (Group II-III), anthracycline-based regimen and PR to initial CT.

• #55 Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis From Four International Cooperative Groups

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5002346/

  Extremity rhabdomyosarcomas do not always show satisfactory outcomes. We analyzed data from 643 patients treated in 14 studies conducted by European and North American groups between 1983 and 2004 to identify factors predictive of outcome. […] 5-year OS were 67% (se 1.8). Multivariate analysis showed that lower OS correlated with age 3 years, T2 and N1 stage, incomplete initial surgery, treatment before 1995, and European cooperative group treatment. […] This meta-analysis confirms the role of many established prognostic factors but identifies for the first time that chemotherapy duration may have an impact on outcome in patients with grossly resected tumors. […] The 10-year OS rate of 62% confirms the relatively less favorable prognosis of extremity RMS as compared to other sites. […] Age is a prognostic factor and patients 10 years old or more had a poorer survival than younger patients, consistent with data from other studies.

• #56 Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis From Four International Cooperative Groups

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5002346/

  Tumor invasiveness (T2 stage), tumor size (5 cm), and locoregional nodal involvement, findings similar to those reported for RMS regardless of tumor site. […] Overall, patients treated in North American studies fared better than patients treated in Europe, even after adjustment for other prognostic factors. […] In this subset of patients, multivariate analysis showed that tumor invasiveness and location in hands/feet were prognostically important in addition to cooperative group. […] The impact of treatment duration has not been previously reported as prognostic in studies of RMS, but the value of prolonged additional treatment with maintenance chemotherapy is now under exploration in current European studies.

• #57 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using Somatic Molecular Biomarkers | medRxiv

  https://www.medrxiv.org/content/10.1101/2024.09.04.24313032v1.full-text

  Molecular markers, such as FOXO1 fusion genes and TP53 and MYOD1 mutations, increasingly influence risk-stratified treatment selection for pediatric rhabdomyosarcoma (RMS). This study aims to integrate molecular and clinical data to produce individualized prognosis predictions that can further improve treatment selection. […] The GE6 model demonstrated superior predictive performance, offering 39% more predictive information than the BC model (LR p0.001) and 15% more than the GE2 model (LR p0.001). […] Incorporating molecular markers into RMS prognosis models improves prognosis predictions. Individualized prognosis predictions may suggest alternative treatment regimens compared to traditional risk-classification schemas. […] Our results demonstrate that known and new molecular markers can improve prognosis predictions for RMS.

• #58 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using Somatic Molecular Biomarkers | medRxiv

  https://www.medrxiv.org/content/10.1101/2024.09.04.24313032v1.full-text

  Molecular markers, such as FOXO1 fusion genes and TP53 and MYOD1 mutations, increasingly influence risk-stratified treatment selection for pediatric rhabdomyosarcoma (RMS). This study aims to integrate molecular and clinical data to produce individualized prognosis predictions that can further improve treatment selection. […] The GE6 model demonstrated superior predictive performance, offering 39% more predictive information than the BC model (LR p0.001) and 15% more than the GE2 model (LR p0.001). […] Incorporating molecular markers into RMS prognosis models improves prognosis predictions. Individualized prognosis predictions may suggest alternative treatment regimens compared to traditional risk-classification schemas. […] Our results demonstrate that known and new molecular markers can improve prognosis predictions for RMS.

• #59 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  The COG analyzed ctDNA in 124 patients with newly diagnosed, intermediate-risk rhabdomyosarcoma. Patients with fusion-negative rhabdomyosarcoma with detectable ctDNA at diagnosis had significantly worse outcomes than patients without detectable ctDNA. […] The estimated 4-year EFS rate was 53% for patients with confirmed FOXO1 fusion-positive rhabdomyosarcoma. Multivariate analysis identified older age and larger tumor size as independent, adverse prognostic factors for EFS within this population.

• #60 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  The objective of this study was to estimate overall survival in children with extremity rhabdomyosarcoma (RMS). […] Multivariable analysis identified age group, N classification, M classification, and treatment combinations as independent predictive factors for patient overall survival. […] For overall survival, the bootstrap-adjusted c-index was 0.76 (95% CI, 0.730.80) for the nomogram. […] We performed a population-based analysis of data from the SEER registry in an effort to identify prognostic factors and develop a nomogram in children with extremity RMS. […] The nomogram appears to be suitable for the survival stratification of children with RMS and will help clinicians identify patients who may be at a reduced probability of survival and assist them in making treatment and surveillance decisions.

• #61 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  The objective of this study was to estimate overall survival in children with extremity rhabdomyosarcoma (RMS). […] Multivariable analysis identified age group, N classification, M classification, and treatment combinations as independent predictive factors for patient overall survival. […] For overall survival, the bootstrap-adjusted c-index was 0.76 (95% CI, 0.730.80) for the nomogram. […] We performed a population-based analysis of data from the SEER registry in an effort to identify prognostic factors and develop a nomogram in children with extremity RMS. […] The nomogram appears to be suitable for the survival stratification of children with RMS and will help clinicians identify patients who may be at a reduced probability of survival and assist them in making treatment and surveillance decisions.

• #62 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  The overall 3-year failure-free survival and overall survival rates in children with nonmetastatic RMS were 77% and 86%, respectively. […] The new survival stratification model was developed from a median 46.0 months of follow-up in the SEER-based cohort. […] The calibration plots demonstrated good concordance between the predicted and actual survival at 2, 3, and 5 years. […] Ultimately, the tree was pruned to generate 3 prognostic subgroups using the endpoint of overall survival. […] In the entire cohort, the 5-year overall survival rates of patients with low risk, intermediate risk, and high risk were 75.8% (95% CI, 0.720.80), 36.7% (95% CI, 0.290.45), and 0%, respectively. […] We concluded that patients who underwent surgery alone had worse survival than patients who underwent surgery combined with radiotherapy.

• #63 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  The overall 3-year failure-free survival and overall survival rates in children with nonmetastatic RMS were 77% and 86%, respectively. […] The new survival stratification model was developed from a median 46.0 months of follow-up in the SEER-based cohort. […] The calibration plots demonstrated good concordance between the predicted and actual survival at 2, 3, and 5 years. […] Ultimately, the tree was pruned to generate 3 prognostic subgroups using the endpoint of overall survival. […] In the entire cohort, the 5-year overall survival rates of patients with low risk, intermediate risk, and high risk were 75.8% (95% CI, 0.720.80), 36.7% (95% CI, 0.290.45), and 0%, respectively. […] We concluded that patients who underwent surgery alone had worse survival than patients who underwent surgery combined with radiotherapy.

• #64 Rhabdomyosarcoma: Current Therapy, Challenges, and Future Approaches to Treatment Strategies

  https://www.mdpi.com/2072-6694/15/21/5269

  Rhabdomyosarcoma (RMS) is a rare pediatric sarcoma affecting skeletal muscle in children and young adults. It is responsible for 3% of all childhood malignant tumors and is the third most prevalent pediatric extracranial solid tumor. Despite advances in diagnostic and treatment methods and clinical trials to improve pediatric RMS survival rates, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The overall 5-year survival rates for RMS in children have exceeded 70%. However, despite advances in diagnostic and treatment methods over the past few decades, children with high-risk RMS and recurrent disease have 5-year survival rates of less than 30% and 17%, respectively. […] The cure rate remains low and the current RMS therapies continue to pose potential life-threatening toxicities, which can lead to lifelong morbidity.

• #65 Survival stratification in childhood rhabdomyosarcoma of the extremities: a derivation and validation study | Scientific Reports

  https://www.nature.com/articles/s41598-020-62656-x

  Further, we also concluded that the survival advantage in patients who received surgery and radiation disappeared in patients with high risk. […] Taken together, we can justify the use of surgery and radiotherapy in patients who have a higher probability of 5-year survival and do not recommend more aggressive surgery combined with radiotherapy in patients who have a lower probability of 5-year survival (high risk). […] The outcome for children with extremity RMS remains suboptimal compared with that of children with RMS in more favorable sites. […] The nomogram appears to be suitable for survival stratification in children with RMS and will help clinicians identify patients who may have a reduced probability of overall survival and assist them in making treatment and surveillance decisions.

• #66 Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment – Health Professional Information [NCI] – Health Information Library | PeaceHealth

  https://www.peacehealth.org/medical-topics/id/ncicdr0000062792

  Continual improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2017, the 5-year relative survival rate for patients with rhabdomyosarcoma increased from 53% to 71% for children younger than 15 years and from 30% to 52% for adolescents aged 15 to 19 years. In more recent years, improvements in outcome have plateaued. […] Rhabdomyosarcoma is usually curable in children with localized disease who receive combined-modality therapy, with more than 70% of patients surviving 5 years after diagnosis. Relapses are uncommon in patients who were alive and event free at 5 years, with a 10-year late-event rate of 9%. Relapses are more common in patients who have unresectable disease, tumor in an unfavorable site at diagnosis, or metastatic disease at diagnosis.

• #67 Management of Low and Intermediate Risk Adult Rhabdomyosarcoma: A Pooled Survival Analysis of 553 Patients | Scientific Reports

  https://www.nature.com/articles/s41598-018-27556-1

  Clinical group-III patients were compared based on whether they underwent delayed primary excisions (DPE) or not. […] The only significant difference between controlled and recurrent tumors among non-PM group-I patients was in their risk group distribution. […] Our findings highlight the local, distant and overall PFS benefit with the use of chemotherapy regimens, adapted from the pediatric RMS protocols, in adults with non-metastatic RMS. […] Development and assessment of novel chemotherapeutic agents is critical, especially for patients with intermediate-risk disease who could not benefit from conventional chemotherapeutic regimens.

• #68 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using Somatic Molecular Biomarkers | medRxiv

  https://www.medrxiv.org/content/10.1101/2024.09.04.24313032v1.full-text

  By predicting 5-year EFS for individual patients in Table 4, we demonstrated that expected survival can change dramatically in ways that may suggest alternative therapies when molecular markers are included. […] This study provides supportive evidence to reinforce the importance of TP53 and MYOD1 mutations as molecular markers of poor prognosis. […] In a large cohort of pediatric patients with RMS, we identified several promising prognostic biomarkers that may improve risk-adapted treatment assignment. Mutations in NF1 are associated with a substantially decreased risk of treatment failure, and mutations in CDNK2A, MET, and MYCN are associated with an increased risk.

• #69 Improving Individualized Rhabdomyosarcoma Prognosis Predictions Using… | Mark Zobeck, MD, MPH

  https://www.linkedin.com/posts/markzobeck_improving-individualized-rhabdomyosarcoma-activity-7293674069013508097-1-gN

  We have a paper out in JCO Precision Oncology describing new biomarkers for pediatric rhabdomyosarcoma! Prediction modeling revealed gene mutations that clearly predict survival outcomes: – CDKN2A, MET, MYCN mutations associated with decreased survival. – NF1 mutations associated with increased survival. […] We’re hoping these biomarkers help make progress in RMS treatment, which has been very difficult over the last few decades.

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