Multimodality Treatment of Desmoplastic Small Round Cell Tumor: Chemotherapy and Complete Cytoreductive Surgery Improve Patient Survival

Abstract

Desmoplastic small round cell tumor (DSRCT), which harbors EWSR1-WT1 t(11;22)(p13:q12) chromosomal translocation, is an aggressive malignancy that typically presents as intra-abdominal sarcomatosis in young males. Given its rarity, optimal treatment has not been defined. We conducted a retrospective study of 187 patients with DSRCT treated at MD Anderson Cancer Center over 2 decades. Univariate and multivariate regression analyses were performed. We determined whether chemotherapy, complete cytoreductive surgery (CCS), hyperthermic intraperitoneal cisplatin (HIPEC), and/or whole abdominal radiation (WART) improve overall survival (OS) in patients with DSRCT. Critically, because our institutional practice limits HIPEC and WART to patients with less extensive, potentially resectable disease that had benefited from neoadjuvant chemotherapy, a time-variant analysis was performed to evaluate those adjunct treatment modalities. The pre-2003 5-year OS rate of 5% has substantially improved to 25% with the advent of newer chemotherapies and better surgical and radiotherapy techniques (HR, 0.47; 95% CI, 0.29-0.75). Chemotherapy response (log rank = 0.004) and CCS (log rank < 0.0001) were associated with improved survival. Although WART and HIPEC lacked statistical significance, our study was not powered to detect their potential impact upon OS. Improved 3- and 5-year OS were observed following multidisciplinary treatment that includes Ewing sarcoma (ES)-based chemotherapy and complete tumor cytoreductive surgery, but few if any patients are cured. Prospective randomized studies will be required to prove whether HIPEC or WART are important. In the meantime, chemotherapy and CCS remain the cornerstone of treatment and provide a solid foundation to evaluate new biologically targeted therapies. .

Olaparib and temozolomide in desmoplastic small round cell tumors: a promising combination in vitro and in vivo

“…These treatments can again induce favorable, however short-lived, responses (Chen and Feng 2019;Italiano et al 2013;Menegaz et al 2018;Tarek et al 2018;Thijs et al 2010). Overall, treatment results in a 5-year overall survival (OS) rate of 15-25%, which shows the high unmet need for novel treatments in DSRCTs (Bent et al 2016;Subbiah et al 2018).…”

Cytoreductive surgery and hyperthermic intraperitoneal chemotherapy in children

“…представили собственный опыт комплексного лечения пациентов с ДМКО. С 1990 по 2016 год в MDACC получили лечение 187 пациентов в возрасте 22,6 ± 10,6 года (от 0,5 до 52,9 года) [42]. При статистическом анализе данных авторы выделили три фактора, определяющие прогноз заболевания:…”

“…Так, группа из Франции использует комбинированные схемы препаратов и более высокие дозы при меньшем времени химиоперфузии по сравнению с авторами из США и Германии. Большинство авторов использует цисплатин в дозе 100 мг/м 2 (перфузия в течение 60 мин) для проведения HIPEC [38,39,42,43,50]. Немецкие исследователи применяют доксорубицин из расчета 15 мг/м 2 [48].…”

A case of submandibular desmoplastic small round cell tumor: Diagnostic and management approaches to an atypical presentation of a rare tumor

“…Typically affecting pediatric and young adult patients, the overwhelming majority of cases originate in the abdomen and pelvis with coincident distant metastases . Outcomes have traditionally been dismal in this population, with five‐year overall survival rates on the order of only 18% to 25% even with aggressive multimodal treatment regimens . To some extent, this is driven by difficulty achieving microscopically negative local control due to the frequently widespread disease state at presentation .…”

“…2 Outcomes have traditionally been dismal in this population, with five-year overall survival rates on the order of only 18% to 25% even with aggressive multimodal treatment regimens. 3,4 To some extent, this is driven by difficulty achieving microscopically negative local control due to the frequently widespread…”