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Autografting

Clinical outcomes of patients with desmoplastic small round cell tumor of the peritoneum undergoing autologous HCT: a CIBMTR retrospective analysis

Abstract

Desmoplastic small round cell tumor of the peritoneum (DSRCTP) is a rare, frequently fatal tumor. This retrospective study, based on CIBMTR registry data, describes the largest reported cohort of DSRCTP patients who have undergone Auto-SCT. The probabilities of disease-free survival (DFS) at 1 year for patients in CR and not in CR were 75% (95% confidence interval: 48–94%) and 35% (15–59%), respectively. The probability of OS at 3 years was 57% (29–83%) and 28% (9–51%) for patients in CR and not in CR, respectively. Median survival for the entire cohort was 31 months (36 months and 21 months for those in CR and not in CR, respectively). Engraftment at 42 days was 97% (88–100%). Treatment-related mortality was low, with only one death in the first 100 days. Auto-SCT is a tolerable approach in patients with DSRCTP, with the greatest benefit seen in those patients who obtain CR. For those not in CR, the median OS in this series is greater than previously reported (21 months vs 17 months), suggesting Auto-SCT is useful in prolonging DFS and OS, even in patients with residual or persistent disease pre-transplant.

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Acknowledgements

CIBMTR is supported by the Public Health Service Grant/Cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U01HL069294 from NHLBI and NCI; a contract HHSH234200637015C with the Health Resources and Services Administration; two Grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research; and Grants from Allos, Inc.; Amgen, Inc.; Angioblast; anonymous donation to the Medical College of Wisconsin; Ariad; Be the Match Foundation; Blue Cross and Blue Shield Association; Buchanan Family Foundation; CaridianBCT; Celgene Corporation; CellGenix, GmbH; Children's Leukemia Research Association; Fresenius-Biotech North America, Inc.; Gamida Cell Teva Joint Venture Ltd.; Genentech, Inc.; Genzyme Corporation; GlaxoSmithKline; Kiadis Pharma; the Leukemia & Lymphoma Society; the Medical College of Wisconsin; Millennium Pharmaceuticals, Inc.; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Seattle Genetics; Sigma-Tau Pharmaceuticals; Soligenix, Inc.; Swedish Orphan Biovitrum; THERAKOS, Inc.; and Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the NIH, the Department of the Navy, the Department of Defense or any other agency of the US government.

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Correspondence to R J Cook.

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Cook, R., Wang, Z., Arora, M. et al. Clinical outcomes of patients with desmoplastic small round cell tumor of the peritoneum undergoing autologous HCT: a CIBMTR retrospective analysis. Bone Marrow Transplant 47, 1455–1458 (2012). https://doi.org/10.1038/bmt.2012.57

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