Children with sickle cell disease (SCD) have significantly better physical health and school functioning after transplantation than do those receiving chronic transfusion, according to results of a donor/no donor study of 67 patients with SCD.
Researchers assessed outcomes and the health-related quality of life (HRQL) in children <15 years who were regularly transfused for a history of abnormal cerebral velocities and who were enrolled in the DREPAGREFFE trial, a French prospective trial with 2 groups, transfusions or transplantation.
Children in the transplant arm (n=32) received grafts from HLA-identical siblings. In the 35 children without sibling donors assigned to the transfusion arm, sickle hemoglobin was maintained below 30% after transfusion.
After one year, all children in both arms were alive. No strokes were observed in either arm during the 12 months of follow-up. All transplanted patients successfully engrafted.
Researchers collected HRQL using the PedsQL 4.0 Generic Core Scale, a generic HRQL questionnaire containing 23 items assessing physical (8), emotional (5), social (5) and school functioning (5). Four composite scores were calculated, ranging from 0 to 100, with higher scores indicating higher HRQL.
At 1 year, the child self-report showed significantly better scores in the transplant arm than the transfused arm for physical health (p=0.048), indicating less “hurting” and more energy, and school functioning (p=0.031) when children had fewer absences.
The parent proxy-report showed significantly better scores in transplanted children for all scales: physical (p<0.001), emotional (p<0.001), social (p<0.001), and school functioning (p<0.001).
In contrast to previous studies, results of this trial showed that parents of transplanted children reported a better HRQL than their child, which the researchers noted “is probably related to the better HRQL observed in their child and to anticipation of a better future.”
Bernaudin F, et al. Abstract oral presentation, 2017 ASH annual meeting