Targeted busulfan-based conditioning unrelated donor bone marrow
transplantation for Diamond-Blackfan anemia
Abstract
Hematopoietic cell transplantation corrects a hematological phenotype of
Diamond-Blackfan anemia (DBA). Reduced conditioning has been recommended
for patients with the cancer predisposition, but appropriate intensity
is required to prevent graft failure. We conducted unrelated-donor bone
marrow transplantations in six consecutive transfusion-dependent
patients (median age, 5.4 years; range, 1.8–23) after targeted-busulfan
(60–75 mg/L×h of cumulative AUC) and fludarabine regimen without
irradiation. All obtained chronic graft-versus-host-disease-free
complete donor chimerism, including one unrelated cord blood
transplantation rescue. The rescued 7-year-old girl had the longest
transfusion-dependency (>40 months) and highest ferritin
level (>2,000 ng/mL) pretransplant. Intermediate-intensity
targeted busulfan-based conditioning may cure DBA.