Significant improvement of child physical and emotional functioning after familial haploidentical stem cell...

Area of Science:

• Hematology
• Pediatric Oncology
• Transplantation Immunology

Background:

• Allogeneic stem cell transplantation (AlloSCT) is the only cure for sickle cell disease (SCD).
• Limited matched related donors and suboptimal outcomes with unrelated donors necessitate alternative donor investigations.
• Familial haploidentical SCT is explored as a viable alternative for high-risk pediatric SCD patients.

Purpose of the Study:

• To evaluate the health-related quality of life (HRQoL) impact in children with high-risk SCD.
• To assess HRQoL changes over two years following familial haploidentical SCT.
• To compare parent and child perspectives on HRQoL trajectories.

Main Methods:

• A prospective study involving 19 children with high-risk SCD and their parents.
• Health-related quality of life (HRQoL) assessed using Child Health Ratings Inventories and an SCT-specific module.
• Repeated measures models analyzed HRQoL changes over time and by rater.

Main Results:

• No significant differences in 2-year physical or emotional functioning (EF) trajectories between parent and child raters.
• Initial decrease in physical functioning and EF at day +45 post-transplant, with recovery by day +180.
• Significant improvement in EF observed at 2 years compared to baseline (p=0.03).

Conclusions:

• Familial haploidentical SCT leads to significant recovery or improvement in patient-reported HRQoL within two years.
• Despite initial treatment intensity, children experience sustained HRQoL gains post-transplant.
• This approach offers a promising therapeutic strategy for high-risk pediatric SCD, restoring quality of life.