Journal
PEDIATRIC TRANSPLANTATION
Volume 19, Issue 6, Pages E157-E159Publisher
WILEY
DOI: 10.1111/petr.12547
Keywords
hematopoietic stem cell transplant; matched-unrelated donor; reduced-intensity conditioning; cirrhosis; Diamond-Blackfan anemia; hereditary bone marrow failure; hemochromatosis
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For patients with DBA who are transfusion dependent, HSCT is the only cure. Chronic transfusions can lead to cirrhosis secondary to iron overload, making them poor candidates for myeloablative HSCT. RIC regimens are associated with lower morbidity and mortality compared to myeloablative regimens, but use of RIC in DBA has been limited. Here we present a 14-yr-old girl with DBA and multiple comorbidities including liver cirrhosis, who underwent MUD HSCT utilizing a RIC regimen that is novel to this condition. She tolerated the regimen well, and at 21months, she remains transfusion independent with chimerisms at 99%.
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