This multi-center phase II trial conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) analyzed outcomes of 29 children (age range 6-19 years) with sickle cell disease (SCD) who underwent reduced-intensity conditioning unrelated donor hematopoietic cell transplantation (HCT) between 2008 and 2014. One- and two-year overall survival was 86% and 79% respectively. Graft rejection among recipients was 10%.
Health-related quality of life scores for “change in health” improved significantly by one year post-transplant compared to pre-transplant scores. The report indicated that children felt better and experienced physical health improvements following HCT.
The day-100 incidence of grade II-IV acute graft-versus-host disease (GVHD) was 28%. One-year incidence of chronic GVHD was higher than expected at 62%, with 38% classified as extensive. There were 7 deaths, primarily in older adolescents. Researchers attributed higher GVHD rates to race, potential HLA mismatches and timing of GVHD prophylaxis.
The researchers suggest that “future trials on unrelated donor transplantation for SCD should focus on strategies that minimize risks of GVHD and include stopping rules for chronic GVHD.”