HCT Significantly Improves Quality of Life in Children with Sickle Cell Disease

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

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