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.