A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune‐suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium
Year: 2020
Type: article
Study design: Clinical Trial
Abstract: BACKGROUND: Recent data show survival after matched unrelated donor (MUD) bone marrow transplantation (BMT) is similar to matched sibling procedures for young patients with severe aplastic anemia (SAA). Donor delays, risk of transplant-related mortality (TRM), and concern about chronic graft versus host disease raise questions about whether MUD BMT or immune suppression therapy (IST) should be preferred initial therapy for young patients lacking matched sibling donors. PROCEDURE: We performed a pilot trial to assess the feasibility of randomizing patients under age 26 with newly diagnosed SAA to receive IST versus MUD BMT. Primary aims assessed the acceptability of randomization and timing of BMT. Secondary aims measured toxicities, response, and survival. RESULTS: Sixty-seven patients with possible SAA were screened at nine centers. Of 57 with confirmed SAA, 23 underwent randomization and received therapy with a median follow-up of 18 months. Of 12 randomized to BMT, 10 started BMT as initial therapy at a median of 36 days after randomization. One BMT recipient experienced secondary graft failure, requiring a second procedure. Six of 11 randomized to IST responded, whereas five with refractory disease underwent successful salvage BMT. One patient achieving complete response relapsed after discontinuation of immune suppression and died of infection after salvage BMT. CONCLUSIONS: This feasibility study showed that a high percentage of patients underwent randomization and received up-front MUD BMT. Our study lays the groundwork for a larger randomized trial that will define best initial therapy for young patients with SAA who have an available MUD.
Source: Pediatric Blood & Cancer
DOI: 10.1002/pbc.28444
Authors: Michael A. Pulsipher, Leslie E. Lehmann, Alison A. Bertuch, Ghadir Sasa, Timothy Olson, Taizo Nakano, Alfred Gilio, Lauri M. Burroughs, Jeffrey M. Lipton, James N. Huang, Kathryn E. Dickerson, Alice Bertaina, Cindy Zhuang, Maggie Malsch, Mark Fleming, Edie Weller, Akiko Shimamura, David A. Williams
Institutions: University of Southern California, Children's Hospital of Los Angeles, Boston Children's Hospital, Harvard University, Baylor College of Medicine, Texas Children's Hospital, Children's Hospital of Philadelphia, Children's Hospital Colorado, University of Washington, Fred Hutch Cancer Center, Cohen Children's Medical Center, University of California, San Francisco, UCSF Benioff Children's Hospital, The University of Texas Southwestern Medical Center, Stanford Medicine, Stanford University
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: green
Awards: G1332931553, G225285241, G2774526201, Unrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant Consortium, Translational Studies of Inherited Marrow Failure and Myelodysplastic Syndromes, A Vital tool for the Study of DBA: The Diamond Blackfan Anemia Registry, G7436788619, G8445828852, G9000064834, G9000132497, G9000248292
OpenAlex ID: W3048868992