Identification and characterization of hematopoietic stem cell transplant candidates in a sickle cell disease cohort

Abstract Sickle cell disease (SCD) is associated with significant morbidity and allogeneic hematopoietic stem cell transplantation (HSCT) remains the primary curative treatment. Recently, the Brazilian Ministry of Health released a regulation to require the publically funded healthcare system pay for HSCT for SCD patients with defined indications. We used an existing 2794-member SCD cohort established during the period 2013-2015 to characterize candidates for HSCT and estimate number of possible donors. Of 2064 patients with SC anemia (SCA), 152 of 974 children (16%) and 279 of 1090 adults (26%) had at least 1 HSCT indication. The most common indication for transplant was stroke (n=239) followed by avascular necrosis (n=96), priapism (n=82), cerebrovascular disease (n=55), >2 vasocclusive episodes (n=38), alloantibodies and chronic transfusion therapy (n=18) and >2 acute chest syndrome episodes (n=11). Increasing age, number of transfusions, abnormal transcranial Doppler, retinopathy, dactilytis and use of hydroxyurea were more frequent in the 152 children with an indication for HSCT compared to 822 without (p