Guzy drobne, okrągłe z desmoplastyczną stroma – Rokowania, prognozy i postęp choroby

Guzy drobne, okrągłe z desmoplastyczną stroma
Rokowania, prognozy i postęp choroby

Desmoplastic Small Round Cell Tumor (DSRCT) to rzadki, wysoce agresywny mięsak, charakteryzujący się translokacją t(11;22)(p13;q12) i fuzją genów EWSR1-WT1, najczęściej lokalizujący się w otrzewnej jamy brzusznej młodych mężczyzn. Rokowanie jest niekorzystne, z 5-letnim wskaźnikiem przeżycia około 15%, medianą całkowitego przeżycia od 16 do 42 miesięcy oraz medianą przeżycia wolnego od choroby 10-15,5 miesiąca. Czynniki prognostyczne obejmują wiek (gorsze rokowanie powyżej 70 lat, HRadj 29,4), lokalizację guza (lepsze rokowanie w przypadku lokalizacji pozabrzusznej) oraz obecność przerzutów pozaotrzewnowych. Leczenie wielomodalne, obejmujące chirurgię, chemioterapię i radioterapię, poprawia przeżycie, zwłaszcza całkowita resekcja chirurgiczna (mediana przeżycia 47 miesięcy vs 16 miesięcy bez operacji, P=0,0235) oraz pooperacyjna radioterapia całej jamy brzusznej (WAP-RT), która jest czynnikiem predykcyjnym 3-letniego przeżycia całkowitego i przeżycia wolnego od nawrotu.

Wprowadzenie do Guzów drobnych, okrągłych z desmoplastyczną stroma
Statystyki przeżycia pacjentów z DSRCT
Czynniki prognostyczne w DSRCT

Czynniki demograficzne
Lokalizacja i zaawansowanie choroby
Czynniki molekularne

Wpływ metod terapeutycznych na rokowanie

Chirurgia i chemioterapia
Radioterapia i terapia skojarzona
HIPEC i EPIC

Charakterystyka nawrotu choroby
Nowe kierunki w leczeniu i rokowaniu DSRCT
Strategia leczenia w kontekście rokowania
Wnioski prognostyczne

Kolejne rozdziały

Wprowadzenie do Guzów drobnych, okrągłych z desmoplastyczną stroma

Guzy drobne, okrągłe z desmoplastyczną stroma (ang. Desmoplastic Small Round Cell Tumor, DSRCT) to rzadki, wysoce agresywny nowotwór, który dotyka głównie młodych mężczyzn.¹² Jest to złośliwy mięsak charakteryzujący się obecnością translokacji chromosomowej t(11;22)(p13;q12), która prowadzi do fuzji genów EWSR1 i WT1.³ Nowotwór ten zazwyczaj zajmuje otrzewną jamy brzusznej, choć może występować również w innych lokalizacjach.⁴ DSRCT wykazuje się wyjątkowo agresywnym przebiegiem klinicznym, co przekłada się na niekorzystne rokowanie, pomimo stosowania wielomodalnego podejścia terapeutycznego.⁵

Statystyki przeżycia pacjentów z DSRCT

Analiza dostępnych danych pokazuje, że odsetek przeżyć wśród pacjentów z DSRCT wynosi: 81% po 12 miesiącach, 39,9% po 36 miesiącach i jedynie 23,4% po 60 miesiącach.⁶ Pięcioletni wskaźnik przeżycia szacuje się na około 15%.⁷ Mediana całkowitego przeżycia w różnych badaniach waha się od 16 do 42 miesięcy, co wskazuje na duże zróżnicowanie wyników w zależności od badanej populacji i zastosowanych metod terapeutycznych.⁸⁹¹⁰

W jednym z największych badań populacyjnych mediana przeżycia dla wszystkich przypadków DSRCT wynosiła 27 miesięcy.¹¹ W innych analizach mediana przeżycia wahała się od 17 do 25 miesięcy.¹²¹³ Mediana przeżycia wolnego od choroby jest znacznie krótsza i wynosi około 10-15,5 miesięcy.¹⁴ Te dane podkreślają agresywny charakter DSRCT, w którym nawet po intensywnym leczeniu dochodzi często do nawrotu choroby.

Czynniki prognostyczne w DSRCT

Czynniki demograficzne

Analizy przeżycia pacjentów z DSRCT wykazały, że niektóre czynniki demograficzne mogą wpływać na rokowanie:

Wiek: Pacjenci w wieku powyżej 70 lat mają istotnie gorsze rokowanie w porównaniu z osobami poniżej 60 roku życia (skorygowany współczynnik ryzyka [HRadj] 29,4, 95% CI 7,02-123,1, P<0,001).¹⁵
Płeć: Nie wykazano istotnych różnic w przeżyciu pomiędzy kobietami a mężczyznami (HRadj 0,85, 95% CI 0,52-1,4, P=0,5).¹⁶ Mediana przeżycia dla mężczyzn wynosiła 27 miesięcy, a dla kobiet 25 miesięcy.¹⁷
Rasa: Mediana przeżycia dla rasy białej wynosiła 25 miesięcy, w porównaniu do 31 miesięcy dla rasy czarnej, jednak po uwzględnieniu innych czynników różnice te nie były statystycznie istotne.¹⁸

Lokalizacja i zaawansowanie choroby

Lokalizacja nowotworu ma istotny wpływ na rokowanie pacjentów z DSRCT:

Choroba wewnątrzbrzuszna vs pozabrzuszna: Pacjenci z chorobą pozabrzuszną mają lepsze rokowanie w porównaniu do pacjentów z guzami zlokalizowanymi w jamie brzusznej.¹⁹
Obecność przerzutów pozaotrzewnowych: Brak przerzutów pozaotrzewnowych jest czynnikiem predykcyjnym korzystniejszego 3-letniego przeżycia całkowitego.²⁰

Czynniki molekularne

Mimo że wszystkie przypadki DSRCT charakteryzują się obecnością fuzji EWSR1-WT1, obserwuje się znaczną heterogenność w odpowiedzi na leczenie i wynikach przeżycia wśród pacjentów. Ta heterogenność może wynikać z drugorzędowych zmian genomowych, które są różnie rozpowszechnione w różnych lokalizacjach tkankowych.²¹ Analiza mutacji w wielu lokalizacjach wykazała, że większość tych drugorzędowych mutacji w genach nowotworowych ma charakter subklonalny (tzw. mutacje gałęziowe). Kilka z nich to mutacje pniowe, ale żadna nie była powtarzalnie zmutowana w całej kohorcie badanej. Wyniki te potwierdzają pogląd, że fuzja EWSR1-WT1 jest prawdopodobnym czynnikiem sprawczym rozwoju guza, co często ma miejsce w przypadku innych nowotworów napędzanych fuzjami genowymi.²²

Wpływ metod terapeutycznych na rokowanie

Chirurgia i chemioterapia

Leczenie chirurgiczne odgrywa kluczową rolę w poprawie rokowania pacjentów z DSRCT:

Zabieg chirurgiczny w chorobie zlokalizowanej: Pacjenci z nierozsiany DSRCT wewnątrzbrzusznym, którzy przeszli zabieg chirurgiczny, mieli medianę przeżycia 47 miesięcy, w porównaniu do 16 miesięcy u pacjentów, którzy nie zostali poddani operacji (P=0,0235).²³
Całkowita cytoredukcja: Całkowita resekcja chirurgiczna jest jednym z czynników predykcyjnych 3-letniego przeżycia całkowitego.²⁴
Chemioterapia po zabiegu: W porównaniu do zabiegu chirurgicznego z chemioterapią, sama operacja nie wpływała na zmianę przeżycia (HRadj 0,37, 95% CI 0,14-1,01, P=0,06).²⁵
Sama chemioterapia: Chemioterapia bez zabiegu chirurgicznego w porównaniu do operacji z chemioterapią wiązała się z 79% zwiększonym ryzykiem zgonu (HRadj 1,79, 95% CI 1,10-2,9, P<0,01).²⁶

Radioterapia i terapia skojarzona

Włączenie radioterapii do schematu leczenia może znacząco poprawić rokowanie:

Chemioradioterapia: W porównaniu do zabiegu chirurgicznego z chemioterapią, zabieg z chemioradioterapią wiązał się z 53% niższym ryzykiem zgonu (HRadj 0,47, 95% CI 0,25-0,90, P<0,01).²⁷²⁸
Mediana przeżycia w terapii skojarzonej: Pacjenci poddani zabiegowi chirurgicznemu z chemioradioterapią mieli medianę przeżycia 45 miesięcy, podczas gdy pacjenci poddani zabiegowi chirurgicznemu z chemioterapią mieli przeżycie 31 miesięcy.²⁹
Paliatywna radioterapia: W przypadku przerzutowego wewnątrzbrzusznego DSRCT, radioterapia w celu kontroli miejscowej wiązała się z dłuższym przeżyciem (mediana przeżycia 47 vs 14 miesięcy; P=0,0147).³⁰³¹
Napromienianie całej jamy brzusznej: Pooperacyjna radioterapia całej jamy brzusznej (WAP-RT) jest czynnikiem predykcyjnym 3-letniego przeżycia całkowitego.³² W analizie wieloczynnikowej była ona jedynym czynnikiem istotnie związanym z przeżyciem wolnym od nawrotu otrzewnowego i przeżyciem wolnym od choroby.³³

HIPEC i EPIC

Rola dootrzewnowej chemioterapii w hipertermii (HIPEC) lub wczesnej pooperacyjnej chemioterapii dootrzewnowej (EPIC) po całkowitej cytoredukcji chirurgicznej pozostaje niejasna:

Brak jednoznacznych korzyści: W badaniu retrospektywnym pacjenci, którzy otrzymali HIPEC/EPIC, mieli istotnie wyższy wskaźnik raka otrzewnej (PCI), co sugeruje bardziej zaawansowaną chorobę. Nie wykazano jednak poprawy przeżycia związanej z tymi metodami.³⁴³⁵
Potrzeba prospektywnych badań: Korzyści z HIPEC powinny zostać ocenione w badaniu prospektywnym.³⁶

Charakterystyka nawrotu choroby

Pomimo stosowania agresywnych metod leczenia, DSRCT charakteryzuje się wysokim odsetkiem nawrotów:

Częstość nawrotów otrzewnowych: Około 69% pacjentów doświadcza nawrotu otrzewnowego po medianie czasu 13 miesięcy.³⁷
Przerzuty odległe: Mimo że wielomodalny schemat leczenia łączący chemioterapię, zabieg cytoredukcyjny, HIPEC i WAP-RT może zapewnić miejscową kontrolę choroby, pacjenci nadal będą prezentować przerzuty odległe podczas obserwacji.³⁸
Odpowiedź na pierwszą linię leczenia: Doniesienia wskazują, że pacjenci początkowo odpowiadają na chemioterapię pierwszej linii i leczenie, ale nawrót jest powszechny.³⁹

Nowe kierunki w leczeniu i rokowaniu DSRCT

Biorąc pod uwagę niekorzystne rokowanie w DSRCT, istnieje pilna potrzeba opracowania nowych podejść terapeutycznych:

Terapie celowane: Translokacja EWSR1-WT1 prowadzi do nadekspresji PDGFRα, VEGF i innych białek związanych z proliferacją komórek nowotworowych i naczyniowych, co może stanowić potencjalne cele terapeutyczne.⁴⁰
Przewlekła chemioterapia: Niektórzy pacjenci w remisji lub z nieresekcyjnym guzem wydają się odnosić korzyści z długotrwałej chemioterapii w małych dawkach, przekształcając DSRCT w chorobę przewlekłą.⁴¹
Wielomodalny intensywny schemat leczenia: Podejście multimodalne obejmujące wysokodawkową chemioterapię, agresywną resekcję chirurgiczną, radioterapię i ratunkowe przeszczepienie komórek macierzystych poprawia przeżycie u niektórych pacjentów.⁴²
Badania kliniczne: Ze względu na rzadkość choroby nie opracowano jednoznacznego protokołu leczenia. Obecne dane i protokoły leczenia nie wykazały poprawy wyników, co podkreśla potrzebę przyszłych badań klinicznych.⁴³

Strategia leczenia w kontekście rokowania

Optymalne podejście terapeutyczne dla DSRCT wymaga zespołu wielodyscyplinarnego i powinno uwzględniać następujące aspekty:⁴⁴

Wczesne wykrycie: Wykrycie choroby we wczesnym stadium i całkowita resekcyjność mogą zapewnić istotne korzyści prognostyczne.⁴⁵
Skojarzenie różnych modalności: Połączenie całkowitej resekcji i chemioterapii wydaje się być obecnie najbardziej preferowaną strategią.⁴⁶
Znaczenie postawienia właściwej diagnozy: DSRCT powinien być uwzględniany w diagnostyce różnicowej guzów drobnokomórkowych nerki u pacjentów pediatrycznych. Jest to istotne, ponieważ każdy z tych guzów ma różne zachowanie kliniczne, rokowanie i implikacje terapeutyczne.⁴⁷

Wnioski prognostyczne

Podsumowując dostępne dane na temat rokowania w DSRCT, można wyciągnąć następujące wnioski:

Rokowanie ogólne: DSRCT pozostaje chorobą złożoną o niekorzystnym rokowaniu, nawet przy zastosowaniu agresywnych wielomodalnych terapii miejscowych.⁴⁸
Czynniki korzystnie wpływające na rokowanie: Brak przerzutów pozaotrzewnowych, całkowita resekcja chirurgiczna i pooperacyjna radioterapia całej jamy brzusznej są czynnikami predykcyjnymi lepszego przeżycia 3-letniego.⁴⁹
Strategia terapeutyczna: Do czasu opracowania związków bezpośrednio hamujących fuzję EWSR1-WT1, najlepszą krótkoterminową strategią zwalczania tego agresywnego mięsaka pediatrycznego jest pośrednie ukierunkowanie na szlaki, od których fuzja EWSR1-WT1 zależy w celu mediacji wzrostu i przeżycia guza.⁵⁰

Mimo postępów w terapii wielomodalnej, wyniki pozostają niezadowalające, ponieważ większość pacjentów doświadcza nawrotu choroby i umiera w ciągu trzech lat. Ta niekorzystna statystyka przeżycia sprawia, że DSRCT jest chorobą sierocą, z pilną potrzebą opracowania nowych leków.⁵¹

Kolejne rozdziały

Zapraszamy do dalszego czytania naszego leksykonu.

Wybierz kolejny rozdział z menu poniżej, aby otworzyć nową podstronę kompedium wiedzy i uzyskać szczegółowe informację o leku, substancji lub chorobie.

Materiały źródłowe

#1
http://waocp.com/journal/index.php/apjcc/article/view/1022
Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive malignancy that predominantly affects males. […] DSRCT is a tumor associated with a poor prognosis. The development of novel treatment approaches is essential to enhance survival rates for patients with this disease. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. For advanced disease, symptom palliation is paramount. The reported Median Survival of DSRCT ranges from 17 to 25 months. […] A definite treatment protocol has not been devised owing to the uncommonness of the disease. The current data and treatment protocols have not shown improved outcomes, thereby invoking the need for future clinical trials. Newer treatment approaches are the need of the hour in this disease with a bleak prognosis.
#2 Desmoplastic Small Round Cell Tumors: A Case Series of a Rare Tumor and Literature Review
http://waocp.com/journal/index.php/apjcc/article/view/1022/2355
Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive malignancy that predominantly affects males. […] The optimal treatment approach for DSRCT involves a multidisciplinary team. […] DSRCT is a tumor associated with a poor prognosis. […] The development of novel treatment approaches is essential to enhance survival rates for patients with this disease. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. […] The reported Median Survival of DSRCT ranges from 17 to 25 months. […] Two retrospective studies have shown improvement in survival with maximal surgical debulking. […] A definite treatment protocol has not been devised owing to the uncommonness of the disease. […] The current data and treatment protocols have not shown improved outcomes, thereby invoking the need for future clinical trials. […] Newer treatment approaches are the need of the hour in this disease with a bleak prognosis.
#3
https://journals.lww.com/cancerjournal/fulltext/2015/11040/desmoplastic_small_round_cell_tumor__diagnostic.107.aspx
Desmoplastic small round cell tumor (DSRCT) is rare and highly malignant neoplasm. […] Prognosis is uncertain in such an aggressive neoplasm as chemotherapy (CT) or radiotherapy (RT) shows various outcomes. […] Median survival is less than 3 year. […] Role of chemotherapy (CT) or radiotherapy (RT) is unpredictable. […] The biologic behavior of DSRCT is aggressive. […] A study by Wong et al., in 2013 found DSRCT as a fatal disease and intra-abdominal tumor with a poorer prognosis. […] They also found surgery for local disease and RT in the metastatic setting improved survival. […] DSRCT is associated with a unique chromosomal translocation t (11:22) (p 13; q 12) that involves the EWSR1 and WT1 genes.
#4 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. Generally, it originates from the serosal surface of the abdominal cavity. The hallmark characteristic of DSRCT is the EWSR1âWT1 gene fusion. This translocation up-regulates the expression of PDGFRÎ±, VEGF and other proteins related to tumor and vascular cell proliferation. 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 dismal survival makes DSRCT an orphan disease with an urgent need for new drugs.
#5 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 is a rare neoplasm with extremely aggressive behavior. Despite the multimodal treatment for newly diagnosed patients with chemotherapy, cytoreductive surgery and radiation, the cure rate is still low. […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. The dismal survival makes DSRCT an orphan disease with an urgent need for new drugs. […] The median overall survival varies between 28-60 months, with a median disease-free survival between 10-15.5 months. […] One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. […] Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60-70% of patients die due to disease progression usually within 3 years after diagnosis.
#6 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The incidence and prognosis of desmoplastic small round cell tumor (DSRCT) is inadequately understood. […] Survival analysis for DSRCT has not been investigated in a population-based study. […] The observed survival at 12, 36, and 60 months was 81%, 39.9%, and 23.4%, respectively. […] Those 70 years had a poorer survival than those 60 years (P0.001). […] Compared to surgery with chemotherapy, surgery with chemoradiotherapy was linked to a 53% lower risk of mortality (P0.001). […] We conclude that the DSRCT incidence has been increasing since 2000 with a white male predominance. […] Gender doesn’t affect survival in DSRCT, and surgery combined with chemoradiotherapy improves survival compared to surgical management with chemotherapy alone. […] The median survival time for all cases was 27 months.
#7 Desmoplastic small-round-cell tumor – Wikipedia
https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor
Prognosis Five-year survival rate 15% […] The prognosis for DSRCT remains poor. […] Prognosis depends upon the stage of the cancer. […] Because the disease can be misdiagnosed or remain undetected, tumors frequently grow large within the abdomen and metastasize or seed to other parts of the body. […] 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.
#8 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The incidence and prognosis of desmoplastic small round cell tumor (DSRCT) is inadequately understood. […] Survival analysis for DSRCT has not been investigated in a population-based study. […] The observed survival at 12, 36, and 60 months was 81%, 39.9%, and 23.4%, respectively. […] Those 70 years had a poorer survival than those 60 years (P0.001). […] Compared to surgery with chemotherapy, surgery with chemoradiotherapy was linked to a 53% lower risk of mortality (P0.001). […] We conclude that the DSRCT incidence has been increasing since 2000 with a white male predominance. […] Gender doesn’t affect survival in DSRCT, and surgery combined with chemoradiotherapy improves survival compared to surgical management with chemotherapy alone. […] The median survival time for all cases was 27 months.
#9 Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature | Clinical Sarcoma Research | Full Text
https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-3-14
Desmoplastic small round cell tumour (DSRCT) is a rare but frequently fatal sarcoma, and many of its characteristics still require further clarification. […] The overall median survival (MS) was 16 months. […] Patients with extra-abdominal disease survived longer compared to those with tumours in the abdomen (all still alive vs MS of 15 months; P= 0.0246). […] Patients with non-metastatic intra-abdominal disease who underwent surgery had an MS of 47 months (16 months for those who did not have surgery; P=0.0235). […] Radiotherapy for locoregional control in patients with metastatic intra-abdominal DSRCT was associated with longer survival (MS of 47 vs 14 months; P=0.0147). […] DSRCT is a rare but often fatal disease that mainly affects younger male patients. Those with intra-abdominal DSRCT have a poorer prognosis, although surgical resection for localised disease and radiotherapy in the metastatic setting are associated with improved survival.
#10 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare disease affecting predominantly children and young adults and for which the benefit of hyperthermic intraperitoneal chemotherapy (HIPEC) after complete cytoreductive surgery (CCRS) remains unknown. […] The median overall survival (OS) of the entire cohort was 42 months. The 2-y and 5-y OS were 72% and 19%. The 2-y and 5-y disease-free survival (DFS) were 30% and 12%. […] The benefit of HIPEC is still unknown and should be evaluated in a prospective trial. The value of postoperative WAP-RT seems to be confirmed. […] This multicentre retrospective study shows that DSRCT remains a complex disease with a dismal prognosis even when using aggressive multimodal locoregional treatments. After complete CRS, the median OS in patients with DSRCT without EPM was 42 months. No prognostic factor was identified. Moreover, 69% of patients had a peritoneal recurrence after a median time of 13 months. The only factor significantly associated with peritoneal recurrence-free survival and DFS was WAP-RT. Intraperitoneal chemotherapy (HIPEC or EPIC) did not improve survival, although patients who received HIPEC/EPIC had a significantly higher PCI.
#11 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The incidence and prognosis of desmoplastic small round cell tumor (DSRCT) is inadequately understood. […] Survival analysis for DSRCT has not been investigated in a population-based study. […] The observed survival at 12, 36, and 60 months was 81%, 39.9%, and 23.4%, respectively. […] Those 70 years had a poorer survival than those 60 years (P0.001). […] Compared to surgery with chemotherapy, surgery with chemoradiotherapy was linked to a 53% lower risk of mortality (P0.001). […] We conclude that the DSRCT incidence has been increasing since 2000 with a white male predominance. […] Gender doesn’t affect survival in DSRCT, and surgery combined with chemoradiotherapy improves survival compared to surgical management with chemotherapy alone. […] The median survival time for all cases was 27 months.
#12
http://waocp.com/journal/index.php/apjcc/article/view/1022
Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive malignancy that predominantly affects males. […] DSRCT is a tumor associated with a poor prognosis. The development of novel treatment approaches is essential to enhance survival rates for patients with this disease. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. For advanced disease, symptom palliation is paramount. The reported Median Survival of DSRCT ranges from 17 to 25 months. […] A definite treatment protocol has not been devised owing to the uncommonness of the disease. The current data and treatment protocols have not shown improved outcomes, thereby invoking the need for future clinical trials. Newer treatment approaches are the need of the hour in this disease with a bleak prognosis.
#13 A Case of Desmoplastic Small Round Cell Tumor Diagnosed in a Young Female Patient
https://www.e-crt.org/journal/view.php?number=187
Desmoplastic small round cell tumor is a very rare malignancy. […] Most patients are diagnosed in the advanced stages and have a poor prognosis. […] The prognosis of patients with DSRCT is generally poor. […] Kretschmar et al. reported summarized results on the prognosis of patients with DSRCT from previous reports: the median overall survival of patients was 17 months (range, 3~72). […] Our patient also had a poor prognosis. […] Given these results, chemotherapy may be beneficial in patients with DSRCT and further comparative studies with larger population are necessary. […] Our patient was treated with combination chemotherapy consisting of VAC/IE as first line palliative chemotherapy and subsequently treated with VIP second line chemotherapy. The maximum response in this case was stable disease (14% decreases) to first line chemotherapy and the time to progression after initial chemotherapy was 5 months. […] In conclusion, we report on the case of a young female patient who was diagnosed with DSRCT and treated with combination chemotherapy. Chemotherapy could be an option in the treatment of patients with DSRCT.
#14 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60â70% of patients die due to disease progression usually within 3 years after diagnosis. The median overall survival varies between 28â60 months, with a median disease-free survival between 10â15.5 months. One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. The multimodality treatment combining chemotherapy, cytoreductive surgery, HIPEC and WAP-RT can warrant local control of the disease, but patients will still present distant metastasis during follow-up, meaning that more effective chemotherapy is necessary to improve long-term outcomes.
#15 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#16 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#17 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#18 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#19 Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature | Clinical Sarcoma Research | Full Text
https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-3-14
Desmoplastic small round cell tumour (DSRCT) is a rare but frequently fatal sarcoma, and many of its characteristics still require further clarification. […] The overall median survival (MS) was 16 months. […] Patients with extra-abdominal disease survived longer compared to those with tumours in the abdomen (all still alive vs MS of 15 months; P= 0.0246). […] Patients with non-metastatic intra-abdominal disease who underwent surgery had an MS of 47 months (16 months for those who did not have surgery; P=0.0235). […] Radiotherapy for locoregional control in patients with metastatic intra-abdominal DSRCT was associated with longer survival (MS of 47 vs 14 months; P=0.0147). […] DSRCT is a rare but often fatal disease that mainly affects younger male patients. Those with intra-abdominal DSRCT have a poorer prognosis, although surgical resection for localised disease and radiotherapy in the metastatic setting are associated with improved survival.
#20 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60â70% of patients die due to disease progression usually within 3 years after diagnosis. The median overall survival varies between 28â60 months, with a median disease-free survival between 10â15.5 months. One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. The multimodality treatment combining chemotherapy, cytoreductive surgery, HIPEC and WAP-RT can warrant local control of the disease, but patients will still present distant metastasis during follow-up, meaning that more effective chemotherapy is necessary to improve long-term outcomes.
#21 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. Patients present with hundreds of tumor implants in their abdominal cavity at various sites. […] Although patients all harbor the EWSR1-WT1 driver mutation, there is substantial heterogeneity in treatment response and survival outcomes among DSRCT patients. Such heterogeneity may be due to secondary genomic alterations that are distributed differently across tissue sites. […] Our multiple-site mutation analysis indicated that most of these secondary mutations in cancer genes were subclonal (i.e., branch mutations). Several were trunk mutations, but none were recurrently mutated in the entire cohort. These results support the view that the EWSR1-WT1 fusion is the likely perpetrator of tumor development, which is often the case for other fusion-driven cancers.
#22 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. Patients present with hundreds of tumor implants in their abdominal cavity at various sites. […] Although patients all harbor the EWSR1-WT1 driver mutation, there is substantial heterogeneity in treatment response and survival outcomes among DSRCT patients. Such heterogeneity may be due to secondary genomic alterations that are distributed differently across tissue sites. […] Our multiple-site mutation analysis indicated that most of these secondary mutations in cancer genes were subclonal (i.e., branch mutations). Several were trunk mutations, but none were recurrently mutated in the entire cohort. These results support the view that the EWSR1-WT1 fusion is the likely perpetrator of tumor development, which is often the case for other fusion-driven cancers.
#23 Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature | Clinical Sarcoma Research | Full Text
https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-3-14
Desmoplastic small round cell tumour (DSRCT) is a rare but frequently fatal sarcoma, and many of its characteristics still require further clarification. […] The overall median survival (MS) was 16 months. […] Patients with extra-abdominal disease survived longer compared to those with tumours in the abdomen (all still alive vs MS of 15 months; P= 0.0246). […] Patients with non-metastatic intra-abdominal disease who underwent surgery had an MS of 47 months (16 months for those who did not have surgery; P=0.0235). […] Radiotherapy for locoregional control in patients with metastatic intra-abdominal DSRCT was associated with longer survival (MS of 47 vs 14 months; P=0.0147). […] DSRCT is a rare but often fatal disease that mainly affects younger male patients. Those with intra-abdominal DSRCT have a poorer prognosis, although surgical resection for localised disease and radiotherapy in the metastatic setting are associated with improved survival.
#24 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60â70% of patients die due to disease progression usually within 3 years after diagnosis. The median overall survival varies between 28â60 months, with a median disease-free survival between 10â15.5 months. One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. The multimodality treatment combining chemotherapy, cytoreductive surgery, HIPEC and WAP-RT can warrant local control of the disease, but patients will still present distant metastasis during follow-up, meaning that more effective chemotherapy is necessary to improve long-term outcomes.
#25 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#26 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
Chemotherapy alone compared to surgery with chemotherapy was linked to a 79% increased risk of mortality (HRadj 1.79, 95% CI 1.102.9, P0.01). […] Our survival analysis revealed that surgery combined with chemoradiotherapy was superior in prolonging survival compared to surgery with chemotherapy alone (HRadj 0.47, 95% CI 0.250.90, P0.01).
#27 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#28 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
Chemotherapy alone compared to surgery with chemotherapy was linked to a 79% increased risk of mortality (HRadj 1.79, 95% CI 1.102.9, P0.01). […] Our survival analysis revealed that surgery combined with chemoradiotherapy was superior in prolonging survival compared to surgery with chemotherapy alone (HRadj 0.47, 95% CI 0.250.90, P0.01).
#29 Changing incidence and survival of desmoplastic small round cell tumor in the USA
https://pmc.ncbi.nlm.nih.gov/articles/PMC9196647/
The median survival time for men was 27 months, while it was 25 months for women. […] The median survival time for the white race was 25 months vs. 31 months for the black race. […] Those who underwent surgery with chemoradiotherapy had a median survival time of 45 months, while patients who underwent surgery with chemotherapy had survival of 31 months. […] After adjusting for age, gender, and race, there was no difference in survival in women compared to men (adjusted hazard ratio [HRadj] 0.85, 95% CI 0.521.4, P=0.5). […] Those 70 years had poor survival compared to those 60 years (HRadj 29.4, 95% CI 7.02123.1, P0.001). […] Compared to surgery with chemotherapy, surgery alone had no change in survival (HRadj 0.37, 95% CI 0.141.01, P=0.06); however, surgery combined with chemoradiotherapy was linked to a 53% lower risk of mortality (HRadj 0.47, 95% CI 0.250.90, P0.01).
#30 Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature | Clinical Sarcoma Research | Full Text
https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-3-14
Desmoplastic small round cell tumour (DSRCT) is a rare but frequently fatal sarcoma, and many of its characteristics still require further clarification. […] The overall median survival (MS) was 16 months. […] Patients with extra-abdominal disease survived longer compared to those with tumours in the abdomen (all still alive vs MS of 15 months; P= 0.0246). […] Patients with non-metastatic intra-abdominal disease who underwent surgery had an MS of 47 months (16 months for those who did not have surgery; P=0.0235). […] Radiotherapy for locoregional control in patients with metastatic intra-abdominal DSRCT was associated with longer survival (MS of 47 vs 14 months; P=0.0147). […] DSRCT is a rare but often fatal disease that mainly affects younger male patients. Those with intra-abdominal DSRCT have a poorer prognosis, although surgical resection for localised disease and radiotherapy in the metastatic setting are associated with improved survival.
#31 Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature | Clinical Sarcoma Research | Full Text
https://clinicalsarcomaresearch.biomedcentral.com/articles/10.1186/2045-3329-3-14
The MS of patients diagnosed with DSRCT was 16 months in this study, which is slightly lower than those reported previously. […] In our study, the MS observed for patients who had resection for their abdominal or pelvic tumours was 47 months, compared to 16 months for those who did not. […] For patients with metastatic intra-abdominal DSRCT, palliative radiotherapy for locoregional disease control appeared to confer a survival advantage (MS of 47 vs 14 months in those who did not have radiotherapy). […] Advanced DSRCT is a rare, aggressive disease with invariably poor outcome that generally occurs in young men. It has a propensity to metastasise and at present, surgery, combination cytotoxic chemotherapy and radiotherapy remain the only standard therapeutic options. In our study, we found that patients with intra-abdominal DSRCT have a poorer prognosis, although surgical resection for localised disease and radiotherapy, even in the metastatic setting for locoregional control, are associated with improved survival.
#32 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60â70% of patients die due to disease progression usually within 3 years after diagnosis. The median overall survival varies between 28â60 months, with a median disease-free survival between 10â15.5 months. One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. The multimodality treatment combining chemotherapy, cytoreductive surgery, HIPEC and WAP-RT can warrant local control of the disease, but patients will still present distant metastasis during follow-up, meaning that more effective chemotherapy is necessary to improve long-term outcomes.
#33 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare disease affecting predominantly children and young adults and for which the benefit of hyperthermic intraperitoneal chemotherapy (HIPEC) after complete cytoreductive surgery (CCRS) remains unknown. […] The median overall survival (OS) of the entire cohort was 42 months. The 2-y and 5-y OS were 72% and 19%. The 2-y and 5-y disease-free survival (DFS) were 30% and 12%. […] The benefit of HIPEC is still unknown and should be evaluated in a prospective trial. The value of postoperative WAP-RT seems to be confirmed. […] This multicentre retrospective study shows that DSRCT remains a complex disease with a dismal prognosis even when using aggressive multimodal locoregional treatments. After complete CRS, the median OS in patients with DSRCT without EPM was 42 months. No prognostic factor was identified. Moreover, 69% of patients had a peritoneal recurrence after a median time of 13 months. The only factor significantly associated with peritoneal recurrence-free survival and DFS was WAP-RT. Intraperitoneal chemotherapy (HIPEC or EPIC) did not improve survival, although patients who received HIPEC/EPIC had a significantly higher PCI.
#34 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare disease affecting predominantly children and young adults and for which the benefit of hyperthermic intraperitoneal chemotherapy (HIPEC) after complete cytoreductive surgery (CCRS) remains unknown. […] The median overall survival (OS) of the entire cohort was 42 months. The 2-y and 5-y OS were 72% and 19%. The 2-y and 5-y disease-free survival (DFS) were 30% and 12%. […] The benefit of HIPEC is still unknown and should be evaluated in a prospective trial. The value of postoperative WAP-RT seems to be confirmed. […] This multicentre retrospective study shows that DSRCT remains a complex disease with a dismal prognosis even when using aggressive multimodal locoregional treatments. After complete CRS, the median OS in patients with DSRCT without EPM was 42 months. No prognostic factor was identified. Moreover, 69% of patients had a peritoneal recurrence after a median time of 13 months. The only factor significantly associated with peritoneal recurrence-free survival and DFS was WAP-RT. Intraperitoneal chemotherapy (HIPEC or EPIC) did not improve survival, although patients who received HIPEC/EPIC had a significantly higher PCI.
#35 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
The benefit of HIPEC or EPIC after complete CRS in patients with a DSRCT without EPM is still unknown. Patients who underwent HIPEC or EPIC in this study likely had more advanced disease with a significantly higher PCI. Conversely, Data suggest a confirmed benefit of postoperative WAP-RT. The benefit of HIPEC should be evaluated in a prospective trial.
#36 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
The benefit of HIPEC or EPIC after complete CRS in patients with a DSRCT without EPM is still unknown. Patients who underwent HIPEC or EPIC in this study likely had more advanced disease with a significantly higher PCI. Conversely, Data suggest a confirmed benefit of postoperative WAP-RT. The benefit of HIPEC should be evaluated in a prospective trial.
#37 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare disease affecting predominantly children and young adults and for which the benefit of hyperthermic intraperitoneal chemotherapy (HIPEC) after complete cytoreductive surgery (CCRS) remains unknown. […] The median overall survival (OS) of the entire cohort was 42 months. The 2-y and 5-y OS were 72% and 19%. The 2-y and 5-y disease-free survival (DFS) were 30% and 12%. […] The benefit of HIPEC is still unknown and should be evaluated in a prospective trial. The value of postoperative WAP-RT seems to be confirmed. […] This multicentre retrospective study shows that DSRCT remains a complex disease with a dismal prognosis even when using aggressive multimodal locoregional treatments. After complete CRS, the median OS in patients with DSRCT without EPM was 42 months. No prognostic factor was identified. Moreover, 69% of patients had a peritoneal recurrence after a median time of 13 months. The only factor significantly associated with peritoneal recurrence-free survival and DFS was WAP-RT. Intraperitoneal chemotherapy (HIPEC or EPIC) did not improve survival, although patients who received HIPEC/EPIC had a significantly higher PCI.
#38 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60â70% of patients die due to disease progression usually within 3 years after diagnosis. The median overall survival varies between 28â60 months, with a median disease-free survival between 10â15.5 months. One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. The multimodality treatment combining chemotherapy, cytoreductive surgery, HIPEC and WAP-RT can warrant local control of the disease, but patients will still present distant metastasis during follow-up, meaning that more effective chemotherapy is necessary to improve long-term outcomes.
#39 Desmoplastic small-round-cell tumor – Wikipedia
https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor
Prognosis Five-year survival rate 15% […] The prognosis for DSRCT remains poor. […] Prognosis depends upon the stage of the cancer. […] Because the disease can be misdiagnosed or remain undetected, tumors frequently grow large within the abdomen and metastasize or seed to other parts of the body. […] 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.
#40 Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy
https://www.mdpi.com/2072-6694/13/3/498
Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. Generally, it originates from the serosal surface of the abdominal cavity. The hallmark characteristic of DSRCT is the EWSR1âWT1 gene fusion. This translocation up-regulates the expression of PDGFRÎ±, VEGF and other proteins related to tumor and vascular cell proliferation. 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 dismal survival makes DSRCT an orphan disease with an urgent need for new drugs.
#41 Desmoplastic small-round-cell tumor – Wikipedia
https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor
Prognosis Five-year survival rate 15% […] The prognosis for DSRCT remains poor. […] Prognosis depends upon the stage of the cancer. […] Because the disease can be misdiagnosed or remain undetected, tumors frequently grow large within the abdomen and metastasize or seed to other parts of the body. […] 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.
#42 Desmoplastic small-round-cell tumor – Wikipedia
https://en.wikipedia.org/wiki/Desmoplastic_small-round-cell_tumor
Prognosis Five-year survival rate 15% […] The prognosis for DSRCT remains poor. […] Prognosis depends upon the stage of the cancer. […] Because the disease can be misdiagnosed or remain undetected, tumors frequently grow large within the abdomen and metastasize or seed to other parts of the body. […] 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.
#43 Desmoplastic Small Round Cell Tumors: A Case Series of a Rare Tumor and Literature Review
http://waocp.com/journal/index.php/apjcc/article/view/1022/2355
Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive malignancy that predominantly affects males. […] The optimal treatment approach for DSRCT involves a multidisciplinary team. […] DSRCT is a tumor associated with a poor prognosis. […] The development of novel treatment approaches is essential to enhance survival rates for patients with this disease. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. […] The reported Median Survival of DSRCT ranges from 17 to 25 months. […] Two retrospective studies have shown improvement in survival with maximal surgical debulking. […] A definite treatment protocol has not been devised owing to the uncommonness of the disease. […] The current data and treatment protocols have not shown improved outcomes, thereby invoking the need for future clinical trials. […] Newer treatment approaches are the need of the hour in this disease with a bleak prognosis.
#44 Desmoplastic Small Round Cell Tumors: A Case Series of a Rare Tumor and Literature Review
http://waocp.com/journal/index.php/apjcc/article/view/1022/2355
Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive malignancy that predominantly affects males. […] The optimal treatment approach for DSRCT involves a multidisciplinary team. […] DSRCT is a tumor associated with a poor prognosis. […] The development of novel treatment approaches is essential to enhance survival rates for patients with this disease. […] DSRCT is known to be at least somewhat chemosensitive and radiosensitive. […] The reported Median Survival of DSRCT ranges from 17 to 25 months. […] Two retrospective studies have shown improvement in survival with maximal surgical debulking. […] A definite treatment protocol has not been devised owing to the uncommonness of the disease. […] The current data and treatment protocols have not shown improved outcomes, thereby invoking the need for future clinical trials. […] Newer treatment approaches are the need of the hour in this disease with a bleak prognosis.
#45 Desmoplastic small round cell tumor of the kidney: a case report | Diagnostic Pathology | Full Text
https://diagnosticpathology.biomedcentral.com/articles/10.1186/s13000-020-01015-w
Desmoplastic small round cell tumor (DSRCT) is known to have a poor prognosis. Our patient who presented with multiple distant metastases at the initial diagnosis died at the 30th month despite radical operation and intensive chemotherapy. […] However, the detection of the disease at early stage and complete resectability may provide significant prognostic benefit as previously reported: 6 out of 11 renal DSRCTs were stated alive without disease, keeping in mind that the follow-up durations are too short to drive a reliable conclusion. The best therapeutic modality has yet to be explored for renal DSRCT. A combination of total resection and chemotherapy seems to be the most preferred strategy at the moment. […] As a conclusion, DSRCT is a rare disease, but should be considered in the differential diagnosis of small round cell tumors of the kidney in pediatric patients. This is important as each one of those tumors has different clinical behavior, prognosis, and treatment implications. Immunohistochemical and molecular studies have particular guidance for the right analytic approach, and documentation of EWSR1-WT1 fusion is the gold standard for the diagnosis of DSRCT as it appears exceedingly characteristic for this disease.
#46 Desmoplastic small round cell tumor of the kidney: a case report | Diagnostic Pathology | Full Text
https://diagnosticpathology.biomedcentral.com/articles/10.1186/s13000-020-01015-w
Desmoplastic small round cell tumor (DSRCT) is known to have a poor prognosis. Our patient who presented with multiple distant metastases at the initial diagnosis died at the 30th month despite radical operation and intensive chemotherapy. […] However, the detection of the disease at early stage and complete resectability may provide significant prognostic benefit as previously reported: 6 out of 11 renal DSRCTs were stated alive without disease, keeping in mind that the follow-up durations are too short to drive a reliable conclusion. The best therapeutic modality has yet to be explored for renal DSRCT. A combination of total resection and chemotherapy seems to be the most preferred strategy at the moment. […] As a conclusion, DSRCT is a rare disease, but should be considered in the differential diagnosis of small round cell tumors of the kidney in pediatric patients. This is important as each one of those tumors has different clinical behavior, prognosis, and treatment implications. Immunohistochemical and molecular studies have particular guidance for the right analytic approach, and documentation of EWSR1-WT1 fusion is the gold standard for the diagnosis of DSRCT as it appears exceedingly characteristic for this disease.
#47 Desmoplastic small round cell tumor of the kidney: a case report | Diagnostic Pathology | Full Text
https://diagnosticpathology.biomedcentral.com/articles/10.1186/s13000-020-01015-w
Desmoplastic small round cell tumor (DSRCT) is known to have a poor prognosis. Our patient who presented with multiple distant metastases at the initial diagnosis died at the 30th month despite radical operation and intensive chemotherapy. […] However, the detection of the disease at early stage and complete resectability may provide significant prognostic benefit as previously reported: 6 out of 11 renal DSRCTs were stated alive without disease, keeping in mind that the follow-up durations are too short to drive a reliable conclusion. The best therapeutic modality has yet to be explored for renal DSRCT. A combination of total resection and chemotherapy seems to be the most preferred strategy at the moment. […] As a conclusion, DSRCT is a rare disease, but should be considered in the differential diagnosis of small round cell tumors of the kidney in pediatric patients. This is important as each one of those tumors has different clinical behavior, prognosis, and treatment implications. Immunohistochemical and molecular studies have particular guidance for the right analytic approach, and documentation of EWSR1-WT1 fusion is the gold standard for the diagnosis of DSRCT as it appears exceedingly characteristic for this disease.
#48 Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: Is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? | PLOS One
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0171639
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare disease affecting predominantly children and young adults and for which the benefit of hyperthermic intraperitoneal chemotherapy (HIPEC) after complete cytoreductive surgery (CCRS) remains unknown. […] The median overall survival (OS) of the entire cohort was 42 months. The 2-y and 5-y OS were 72% and 19%. The 2-y and 5-y disease-free survival (DFS) were 30% and 12%. […] The benefit of HIPEC is still unknown and should be evaluated in a prospective trial. The value of postoperative WAP-RT seems to be confirmed. […] This multicentre retrospective study shows that DSRCT remains a complex disease with a dismal prognosis even when using aggressive multimodal locoregional treatments. After complete CRS, the median OS in patients with DSRCT without EPM was 42 months. No prognostic factor was identified. Moreover, 69% of patients had a peritoneal recurrence after a median time of 13 months. The only factor significantly associated with peritoneal recurrence-free survival and DFS was WAP-RT. Intraperitoneal chemotherapy (HIPEC or EPIC) did not improve survival, although patients who received HIPEC/EPIC had a significantly higher PCI.
#49 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 is a rare neoplasm with extremely aggressive behavior. Despite the multimodal treatment for newly diagnosed patients with chemotherapy, cytoreductive surgery and radiation, the cure rate is still low. […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. The dismal survival makes DSRCT an orphan disease with an urgent need for new drugs. […] The median overall survival varies between 28-60 months, with a median disease-free survival between 10-15.5 months. […] One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. […] Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60-70% of patients die due to disease progression usually within 3 years after diagnosis.
#50 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
DSRCT is often diagnosed at a late stage, after tumors have already become large and spread to other parts of the body, mainly in the abdomen. To clarify whether multiple masses arose independently or have a shared origin, we constructed mutation and SCNA phylogenetic analysis for four patients for whom multi-site sampling were available. We observed that all samples had identical EWSR1-WT1 fusion breakpoints and that most mutations and SCNAs occurred within the trunks of the phylogenetic trees. […] Our findings demonstrate that multi-site tumors from individual DSRCT patients have a shared origin and are closely related. The EWSR1-WT1 fusion is almost certainly a major driver in the early development and dispersion of DSRCT tumors. Therefore, until compounds that directly inhibit the EWSR1-WT1 fusion are developed, the best short-term strategy to eradicating this aggressive pediatric sarcoma is indirect targeting of pathways that the EWSR1-WT1 fusion relies on to mediate tumor growth and survival.
#51 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 is a rare neoplasm with extremely aggressive behavior. Despite the multimodal treatment for newly diagnosed patients with chemotherapy, cytoreductive surgery and radiation, the cure rate is still low. […] Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within three years. The dismal survival makes DSRCT an orphan disease with an urgent need for new drugs. […] The median overall survival varies between 28-60 months, with a median disease-free survival between 10-15.5 months. […] One study proposed that the absence of extra-peritoneal metastasis, complete surgical resection and postoperative WAP-RT are factors predictive of 3-year overall survival. […] Despite multimodal treatment, DSRCT has a poor prognosis, and approximately 60-70% of patients die due to disease progression usually within 3 years after diagnosis.