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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
Author(s) -
Pulsipher Michael A.,
Lehmann Leslie E.,
Bertuch Alison A.,
Sasa Ghadir,
Olson Timothy,
Nakano Taizo,
Gilio Alfred,
Burroughs Lauri M.,
Lipton Jeffrey M.,
Huang James N.,
Dickerson Kathryn E.,
Bertaina Alice,
Zhuang Cindy,
Malsch Maggie,
Fleming Mark,
Weller Edie,
Shimamura Akiko,
Williams David A.
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28444
Subject(s) - medicine , randomization , randomized controlled trial , surgery , transplantation , salvage therapy , aplastic anemia , sibling , bone marrow failure , bone marrow , pediatrics , chemotherapy , haematopoiesis , stem cell , psychology , developmental psychology , biology , genetics
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.