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Introduction: Bone marrow transplantation (BMT) is curative for hemoglobinopathies but its utilization in sickle cell disease (SCD) is sparse. Though there were 100,000 affected with SCD in the USA, BMT has been utilized in less than 1000 individuals. As SCD has variable clinical course, confusion prevails regarding indication, timing of BMT, benefits and risks for individuals due to morbidity/mortality of the procedure.
Methods: We used the Pediatric Health Information System (PHIS), an electronic database of children's hospitals in the US. Patients under the age of 21 who underwent BMT at one of the 26 PHIS hospitals from 2000-2011 were analyzed. We abstracted data on demographics, hospitalizations, BMT and related complications.
Results: From 2000 to 2011, there were 1365 unique pediatric patients with SCD were identified. Among these 228(17%) children underwent BMT and their demographic and baseline characteristics are shown in Figure 1A. Overall, the number of new patients with SCD getting hospitalized has decreased consistently whereas children undergoing BMT has increased (Figure 1B). There was a significant decrease in hospitalizations after BMT compared to pre-BMT state (median hospitalization 2 vs. 4 respectively, n=173, p=<0.0001). After BMT, 6%(13/228) of patients developed stroke, 14%(31/228) had CMV infection, 9%(21/228) developed invasive fungal infections, 2.2%(5/228) had VOD, and 23%(24/102) developed GVHD. Seventeen patients (7.4%) died related to BMT with more deaths occurred when BMT performed at age ³11 years compared to age ²10 years (13/108 vs. 4/120 respectively, p=0.029).
Conclusions: We show an increasing trend of BMT utilization in children with SCD. In this largest BMT cohort of SCD, transplant related mortality is very low especially if BMT is performed before 10 years of age. BMT significantly decreases the overall hospitalizations in these patients. Studies addressing the barriers of BMT and hydroxyurea utilization are needed to decrease the health burden in this chronic disease.
