Can we cure patients with abdominal Desmoplastic Small Round Cell Tumor? Results of a retrospective multicentric study on 100 patients

Honoré, Charles; Delhorme, Jean-Baptiste; Nassif, Elise F.; Faron, Matthieu; Ferron, Gwénaël; Bompas, Emmanuelle; Gléhen, Olivier; Italiano, Antoîne; Bertucci, François; Orbach, Daniel; Pocard, Marc; Quénet, François; Blay, Jean‐Yves; Carrère, Sébastien; Chevreau, Christine; Mir, Olivier; Cesne, Axel Le

doi:10.1016/j.suronc.2019.04.002

Cited by 58 publications

(89 citation statements)

References 19 publications

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“…Given the small number of patients, it is difficult to determine whether WART or total radiation dose impacted disease outcomes. For patients with DSRCT, relapses most frequently occur in the first 2 years after diagnosis 2,5,6,10 . In our case series, two patients who completed 20 cycles of planned chemotherapy are disease‐free at 46.7 and 60.3 months.…”

Section: Discussionmentioning

confidence: 74%

“…Patients often present with a primary abdominal mass with diffuse intraabdominal dissemination. Reported long‐term outcomes are exceedingly poor with 5‐year overall survival (OS) rates of approximately 20% 2‐11 . Current multimodal therapeutic approaches consist of intensive multiagent chemotherapy and local control measures including surgery and whole abdomen radiotherapy (WART) with or without hyperthermic intraperitoneal chemotherapy (HIPEC).…”

Section: Introductionmentioning

confidence: 99%

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The use of interval‐compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor

Liu

Collins

Greenzang

et al. 2020

Pediatric Blood & Cancer

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Background Desmoplastic small round cell tumor (DSRCT) is a rare aggressive sarcoma that affects children and young adults, and portends poor outcomes despite intensive multimodal treatment approaches. We report toxicity, response, and outcomes of patients with DSRCT treated with the addition of vincristine, irinotecan, and temozolomide (VIT) to interval‐compressed chemotherapy as per Children's Oncology Group ARST08P1. Methods All newly diagnosed pediatric patients with DSRCT treated at Dana‐Farber Cancer Institute and Boston Children's Hospital between 2014 and 2019 as per ARST08P1, Arm P2 with replacement of VAC cycles with VIT, were identified. Medical records were reviewed for clinical and disease characteristics, and treatment response and outcomes. Results Six patients were treated as per the above regimen. Median age at diagnosis was 15.1 years (range 3.2‐16.4) and five patients were male. Five patients had abdominal primary tumors, of which one had exclusively intraabdominal and four had extraabdominal metastases. Two initial cycles of VIT were well tolerated with nausea, vomiting, diarrhea, and constipation as the most common adverse events. Overall response rate defined as partial or complete response after two initial cycles of VIT was 50%. For local control, all patients had surgical resection followed by radiotherapy, and two patients received hyperthermic intraperitoneal chemotherapy at the time of surgery. Of the four patients who have completed therapy to date, three remain disease‐free with median follow‐up time of 46.7 months. Conclusions The addition of VIT to interval‐compressed chemotherapy is tolerable and active in DSRCT, with activity warranting additional investigation.

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Section: Discussionmentioning

confidence: 74%

Section: Introductionmentioning

confidence: 99%

The use of interval‐compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor

Liu

Collins

Greenzang

et al. 2020

Pediatric Blood & Cancer

View full text Add to dashboard Cite

show abstract

“…The median time to progression on first-line treatment is short: in a UK study median time to progression was between 3.9 and 14.9 months depending on type of chemotherapy protocol used (2) and in a French study the progression-free survival was 11 months (14). The occasional long-term survivors are those with localized disease at presentation (15) and those who have complete surgical resection (14). The 5-year overall survival is dismal at 5-18% (14,15) highlighting the urgent need for better systemic therapies for this highly aggressive tumor.…”

Section: Introduction Clinical Presentation and Current Treatmentmentioning

confidence: 99%

“…Despite initial response to chemotherapy, DSRCT often progresses while patients are on treatment (6). The median time to progression on first-line treatment is short: in a UK study median time to progression was between 3.9 and 14.9 months depending on type of chemotherapy protocol used (2) and in a French study the progression-free survival was 11 months (14). The occasional long-term survivors are those with localized disease at presentation (15) and those who have complete surgical resection (14).…”

Section: Introduction Clinical Presentation and Current Treatmentmentioning

confidence: 99%

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Mini-Review on Targeted Treatment of Desmoplastic Small Round Cell Tumor

2020

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Desmoplastic small round cell tumor (DSRCT) is a devastating disease which most commonly affects adolescents, with a male predominance. Despite the best multimodality treatment efforts, most patients will ultimately not survive more than 3-5 years after diagnosis. Some research trials in soft-tissue sarcoma and Ewing sarcoma include DSRCT patients but few studies have been tailored to the specific clinical needs and underlying cytogenetic abnormalities characterizing this disease such as the typical EWSR1-WT1 gene fusion. Downstream activation of EWSR1-WT1 gene fusion includes signaling pathways of platelet-derived growth factor (PDGF), vascular endothelial growth factor (VEGF), and insulin growth factor (IGF)-1. Other biological pathways that are activated and expressed in DSRCT cells include endothelial growth factor receptor (EGFR), androgen receptor pathway, c-KIT, MET, and transforming growth factor (TGF) beta. Investigation of somatic mutations, copy number alterations (CNA), and chromosomes in DSRCT samples suggests that deregulation of mesenchymal-epithelial reverse transition (MErT)/epithelial-mesenchymal transition (EMT) and DNA damage repair (DDR) may be important in DSRCT. This mini review looks at known druggable targets in DSRCT and existing clinical evidence for targeted treatments, particularly multityrosine kinase inhibitors such as pazopanib, imatinib, and sorafenib alone or in combination with other agents such as mTOR (mammalian target of rapamycin) inhibitors. The aim is to increase shared knowledge about current available treatments and identify gaps in research to further efforts toward clinical development of targeted agents.

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Long‐term survivors with desmoplastic small round cell tumor (DSRCT): Results from a retrospective single‐institution case series analysis

et al. 2023

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Objective To report on a retrospective study of primary DSRCT aiming at characterizing long‐term survivors (LTS). Methods All consecutive patients treated at our institution for a primary DSRCT between 2000 and 2021 were retrospectively identified. Patients received multiagent chemotherapy ± surgery ± hyperthermic intraperitoneal chemotherapy (HIPEC) ± whole abdomino‐pelvic radiotherapy (WAP‐RT) ± high‐dose chemotherapy ± maintenance chemotherapy (MC). Event‐free survival (EFS) and overall survival (OS) were estimated by Kaplan–Meier method. Patients alive, without evidence of disease at ≥36 months from diagnosis, were defined as LTS. Results Thirty‐eight patients were identified. All received multiagent chemotherapy; 27/38 (71%) surgery (7/27 [26%] plus HIPEC), 9/38 (24%) WAP‐RT, 12/38 (32%) MC. At a median‐follow‐up of 37 months (IQR 18–63), overall median‐EFS and median‐OS were 15 and 37 months, respectively. All events occurred within 35 months. In patients who underwent surgery, median‐EFS and median‐OS were 19 and 37 months (23 and 43 months after R0/R1, and 10 and 19 months after R2 resection), respectively. LTS were 5/38 (13%), alive at 37, 39, 53, 64, 209 months. None had liver or extra‐abdominal metastasis at diagnosis, they all received R0/R1 resection, 3/5 had WAP‐RT, 2/5 MC, 1/5 received high‐dose chemotherapy, none HIPEC. Conclusions In our series cure was likely achieved in 13% of DSRCT. LTS had no liver/extra‐abdominal disease, were treated with complete surgery, and possibly WAP‐RT/MC.

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Can we cure patients with abdominal Desmoplastic Small Round Cell Tumor? Results of a retrospective multicentric study on 100 patients

Cited by 58 publications

References 19 publications

The use of interval‐compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor

The use of interval‐compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor

Mini-Review on Targeted Treatment of Desmoplastic Small Round Cell Tumor

Long‐term survivors with desmoplastic small round cell tumor (DSRCT): Results from a retrospective single‐institution case series analysis

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