Outcomes With Bone Marrow Transplantation for Sickle Cell Disease

The majority of patients with sickle cell disease (SCD) who received bone marrow transplantation (BMT) in a study of US Medicaid claims data were free of vaso-occlusive crises (VOCs) after 2 years, but they showed high rates of other complications. Results were reported in the European Journal of Haematology.

In this analysis, Medicaid claims from 2000 through 2013 for patients with SCD were evaluated for outcomes following BMT. For the purposes of this study, SCD case definition was confined to sickle cell hemoglobin form. The primary study outcome involved VOC events that led to hospitalization or a visit to an emergency department.

Data for 204 patients were identified in this analysis. The mean age of patients was 10.6 years (standard deviation, 7.3), and patients had an average of 3.1 VOC events at baseline. Slightly more than half of patients (52.9%) were men, and approximately two-thirds (67.6%) of patients were African American.

Median study follow-up occurred at 2.1 years (interquartile range [IQR], 0.8-4.3). The rate of VOC events over this time was 0.99 per person-year (95% CI, 0.91-1.07) after BMT. The majority of patients (67.6%) were free of VOCs throughout the follow-up period. In patients who had VOC events after BMT, the first such event occurred at a median of 226.5 days following BMT (IQR, 45-632).

A total of 11 patients died throughout the follow-up period, with mortality occurring at a rate of 1.7 (95% CI, 0.9-3.1) per 100 person-years. Complications associated with transplantation occurred in 55.4% of patients, and the incidence rate of these was 38.2 (95% CI, 31.7-45.9) per 100 person-years. Nearly a quarter (23%) of patients experienced graft-vs-host disease, which showed an incidence rate of 8.0 (95% CI, 6.0-10.7) per 100 person-years.

“In this cohort of 204 patients with SCD undergoing BMT, we observed that more than two thirds of the transplant recipients remained VOC event free over a median follow-up of two years. Moreover, our study also demonstrated a high survival rate (94.6%) over the study follow-up period,” the study authors wrote in their report.

However, the authors also concluded that there is a continuing unmet need for curative treatment options for patients with SCD because of the high rate of complications.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.