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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

2020; Wiley; Volume: 67; Issue: 10 Linguagem: Inglês

10.1002/pbc.28444

1545-5017

Michael A. Pulsipher, Leslie E. Lehmann, Alison A. Bertuch, Ghadir Sasa, Timothy S. Olson, Taizo A. Nakano, Alfred Gilio, Lauri M. Burroughs, Jeffrey M. Lipton, James N. Huang, Kathryn E. Dickerson, Alice Bertaina, Cindy Zhuang, Maggie Malsch, Mark D. Fleming, Edie Weller, Akiko Shimamura, David A. Williams,

Childhood Cancer Survivors' Quality of Life

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.