Desmoplastic small round cell tumors: Multimodality treatment and new risk factors

Scheer, Monika; Vokuhl, Christian; Blank, Bernd; Hallmen, Erika; Kalle, Thekla von; Münter, Marc W.; Wessalowski, Rüdiger; Hartwig, Maite; Sparber‐Sauer, Monika; Schlegel, Paul‐Gerhardt; Kramm, Christof M.; Kontny, Udo; Spriewald, Bernd; Kegel, Thomas; Bauer, Sebastian; Kazanowska, Bernarda; Niggli, Felix; Ladenstein, Ruth; Ljungman, Gustaf; Jahnukainen, Kirsi; Fuchs, Jörg; Bielack, Stefan; Klingebiel, Thomas; Kościelniak, Ewa; Studiengruppe, Cooperative Weichteilsarkom

doi:10.1002/cam4.1940

Cited by 46 publications

(64 citation statements)

References 63 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Last, in patients with desmoplastic small round-cell tumors, MC was associated with more frequent complete remission and longer event-free survival. 27…”

Section: Discussionmentioning

confidence: 99%

Metronomic Chemotherapy for Children in Low- and Middle-Income Countries: Survey of Current Practices and Opinions of Pediatric Oncologists

Revon‐Rivière

Banavali

Heississen

et al. 2019

JGO

View full text Add to dashboard Cite

PURPOSE Low- and middle-income countries (LMICs) experience the burden of 80% of new childhood cancer cases worldwide, with cure rates as low as 10% in some countries. Metronomics combines frequent administrations of low-dose chemotherapy with drug repurposing, which consists of using already-approved drugs for new medical applications. With wide availability, limited costs, and little infrastructure needs, metronomics can be part of constraint-adapted regimens in these resource-limited settings—with the understanding that metronomics shall not be a substitute for standard treatments when available and doable. Our study aims to describe the experience, practices, opinions, and needs in metronomics of physicians working in LMICs. METHODS An online questionnaire was sent to more than 1,200 physicians in pediatric oncology networks in LMICs. Items included the type of center, physician’s demographics, experience in pediatric oncology, and experience with current knowledge of metronomics. Opinions and perspectives were explored using multiple-answer and open questions. RESULTS Of physicians, 17% responded. Of respondents, 54.9% declared that they had already used a metronomic regimen. The most frequently cited repositioned drugs were celecoxib (44%) followed by propranolol and valproic acid (17%). Respondents highlighted the advantages of outpatient use (20%) and expected low toxicity (24%). In considering the drawbacks of metronomics, 47% of responses highlighted the lack of scientific evidence or guidelines, 33% the availability or affordability of drugs, and 18% the problem of acceptance or compliance. Of physicians, 79% believed that use of metronomics will spread in LMICs in the near future and 98% of them were willing to participate in international metronomic protocols or registries. CONCLUSION Metronomics is already used in LMICs and is a potential answer to unmet needs in pediatric oncology. There is room for improvement in the availability of drugs and a necessity to develop collaborative protocols and research to generate level A evidence.

show abstract

“…Last, in patients with desmoplastic small round-cell tumors, MC was associated with more frequent complete remission and longer event-free survival. 27…”

Section: Discussionmentioning

confidence: 99%

Metronomic Chemotherapy for Children in Low- and Middle-Income Countries: Survey of Current Practices and Opinions of Pediatric Oncologists

Revon‐Rivière

Banavali

Heississen

et al. 2019

JGO

View full text Add to dashboard Cite

show abstract

“…Recientes estudios comparativos plantean un esquema menos tóxico y tan beneficioso como el anterior: VAIA (ifosfamida, vincristina, adriamicina D, carboplatino y actinomicina D). 19 Un esquema de citorreducción más quimioterapia intraperitoneal hipertérmica (CRS/HIPEC) se vislumbra como una opción terapéutica con morbilidad periope ratoria aceptable. Sin embargo, el impacto a largo plazo sigue siendo mal definido.…”

Section: Discussionunclassified

Tumor abdominal desmoplásico de células pequeñas y redondas

Chavarría-Caldas

Terán-Vásquez

Páucar-Pérez

et al. 2019

spmi

View full text Add to dashboard Cite

show abstract

“…The authors concluded that high-dose chemotherapy probably has no role in the treatment of DSRCT [ 51 ]. More recently, Scheer et al [ 52 ] found that patients treated with the VAIA regimen (ifosfamide, vincristine, doxorubicin, actinomycin D) presented longer event-free survival (29.4 months) compared to other protocols, including the P6 protocol. The interval-compressed regimen of vincristine, irinotecan, temozolamide (VIT) was evaluated in 6 pediatric patients and showed a tolerable profile with an objective response rate of 50% to the first 2 cycles of VIT [ 53 ].…”

Section: Treatmentmentioning

confidence: 99%

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

Mello

Campos

Santos

et al. 2021

Cancers

View full text Add to dashboard Cite

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 EWSR1–WT1 gene fusion. This translocation up-regulates the expression of PDGFRα, 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. The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors was evaluated by small trials. Recent studies using comprehensive molecular profiling of DSRCT identified potential therapeutic targets. In this review, we aim to describe the current studies conducted to better understand DSRCT biology and to explore the new therapeutic strategies under investigation in preclinical models and in early phase clinical trials.

show abstract

Desmoplastic small round cell tumors: Multimodality treatment and new risk factors

Cited by 46 publications

References 63 publications

Metronomic Chemotherapy for Children in Low- and Middle-Income Countries: Survey of Current Practices and Opinions of Pediatric Oncologists

Metronomic Chemotherapy for Children in Low- and Middle-Income Countries: Survey of Current Practices and Opinions of Pediatric Oncologists

Tumor abdominal desmoplásico de células pequeñas y redondas

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

Contact Info

Product

Resources

About