摘要

Background Little is known regarding the health-related quality of life (HRQL) trajectory of children with sickle cell disease or thalassemia (hemoglobinopathies) following hematopoietic stem cell transplantation (HSCT). Procedures We serially evaluated the HRQL of 13 children with hemoglobinopathies who received HSCT during two prospective multi-center studies using the Child Health Ratings Inventories (CHRIs). The HRQL scores among children with hemoglobinopathies, as reported separately by the children and their parents were compared using repeated measures models to scores of a comparison group of children receiving HSCT for malignancies or severe aplastic anemia. Results The sample included 13 children with hemoglobinopathies (median age: 8 years, range 518) and 268 children in the comparison group (median age: 11 years, range 518). There were similar rates of early infection, chronic graft versus host disease, and all-cause mortality between the two groups. There was no significant difference in recovery to baseline scores for physical, emotional, and role functioning by 3 months for either group. Children with hemoglobinopathies had higher HRQL scores for physical (beta?=?12, se?=?5.5, P?=?0.01) and baseline emotional functioning (beta?=?11.6, se?=?5.5, P?=?0.03) than the comparison group. For all domains for both groups, parent reports demonstrated a nadir at 45 days with recovery to baseline by 3 months following transplant. Children's ratings were higher than those of their parents in both diagnostic groups. Conclusions Children with hemoglobinopathies had higher physical and emotional functioning scores prior to HSCT and experienced a similar pattern of recovery to their baseline functioning by 3 months post-HSCT when compared to children receiving HSCT for acquired conditions. Pediatr Blood Cancer 2012;59:725731.

  • 出版日期2012-10