Rhabdomyosarcoma (RMS), the most common soft-tissue sarcoma in children, is cured with conventional therapy in 70%. However, the 5-year survival for those who relapse is about 30%, and drops to about 15% for those with unfavorable histologies (alveolar/undifferentiated subtypes). We describe outcomes of 62 subjects receiving autologous blood/bone marrow (BM) transplants for RMS between 1989 and 2003, and reported to the Center for International Blood and Marrow Transplantation Research (CIBMTR). Histologic subtype was confirmed by reviewing pathology reports. Treatment-related mortality (TRM), progression-free survival (PFS), and overall survival (OS) were evaluated. Overall, 73% of subjects were <20 years; 39% had cancer bulk >5 cm, 63% had metastasis at diagnosis, 55% had unfavorable histologies, 92% had cancer responsive to chemotherapy pretransplant, and 67% were in first remission. The 1-year TRM was 5% (95% confidence interval [CI], 1%-12%) and the 5-year PFS and OS were 29% (95% CI, 18%-41%) and 32% (95% CI, 21%-44%), respectively. There was only a 4% relapse rate after the first year. There were no differences in 5-year PFS or survival based on histological subtype, transplant in first remission versus relapse (36% versus 29%; P = .5), or transplantation for poor-risk histologies in first remission versus relapse (34% versus 33%; P = .9). Our data indicate that autotransplants for RMS disease are typically done in patients with disease responsive to chemotherapy pretransplant, with approximately one-third long-term survivors. Despite high-risk factors, we also found a low TRM, perhaps reflecting the migration from marrow to blood stem cells as the graft source. Even when performed after relapse for alveolar/undifferentiated histologies, long-term survivals were seen seemingly better than results with conventional therapies.
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Financial disclosure: The CIBMTR is supported by 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 Health Resources and Services Administration (HRSA/DHHS); 2 Grants N00014-06-1-0704 and N00014-08-1-0058 from the Office of Naval Research ; and grants from AABB ; Aetna; American Society for Blood and Marrow Transplantation ; Amgen, Inc. ; anonymous donation to the Medical College of Wisconsin ; Astellas Pharma US, Inc. ; Baxter International, Inc. ; Bayer HealthCare Pharmaceuticals ; Be the Match Foundation ; Biogen IDEC ; BioMarin Pharmaceutical, Inc. ; Biovitrum AB ; BloodCenter of Wisconsin ; Blue Cross and Blue Shield Association ; Bone Marrow Foundation ; Canadian Blood and Marrow Transplant Group ; CaridianBCT ; Celgene Corporation ; CellGenix , GmbH ; Centers for Disease Control and Prevention ; Children's Leukemia Research Association ; ClinImmune Labs ; CTI Clinical Trial and Consulting Services ; Cubist Pharmaceuticals ; Cylex Inc. ; CytoTherm ; DOR BioPharma, Inc. ; Dynal Biotech , an Invitrogen Company ; Eisai, Inc. ; Enzon Pharmaceuticals, Inc. ; European Group for Blood and Marrow Transplantation ; Gamida Cell, Ltd. ; GE Healthcare; Genentech, Inc. ; Genzyme Corporation ; Histogenetics, Inc. ; HKS Medical Information Systems ; Hospira, Inc. ; Infectious Diseases Society of America ; Kiadis Pharma ; Kirin Brewery Co., Ltd. ; The Leukemia & Lymphoma Society ; Merck & Company ; The Medical College of Wisconsin ; MGI Pharma, Inc. ; Michigan Community Blood Centers ; Millennium Pharmaceuticals, Inc. ; Miller Pharmacal Group ; Milliman USA, Inc. ; Miltenyi Biotec, Inc. ; National Marrow Donor Program ; Nature Publishing Group ; New York Blood Center ; Novartis Oncology ; Oncology Nursing Society ; Osiris Therapeutics, Inc. ; Otsuka America Pharmaceutical, Inc. ; Pall Life Sciences ; Pfizer Inc ; Saladax Biomedical, Inc. ; Schering Corporation ; Society for Healthcare Epidemiology of America ; StemCyte, Inc. ; StemSoft Software, Inc. ; Sysmex America, Inc. ; Teva Pharmaceutical Industries ; THERAKOS, Inc. ; Thermogenesis Corporation ; Vidacare Corporation ; Vion Pharmaceuticals, Inc. ; ViraCor Laboratories ; ViroPharma, Inc. ; and Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institute of Health, the Department of the Navy, the Department of Defense, or any other agency of the U.S. Government.