Health-related quality of life in children with sickle cell disease: A report from the comprehensive sickle cell centers clinical trial consortium

Carlton Dampier, Susan Lieff, Petra LeBeau, Seungshin Rhee, Marsha McMurray, Zora Rogers, Kim Smith-Whitley, Winfred Wang

Research output: Contribution to journalArticlepeer-review

131 Scopus citations


Background. Pediatric health-related quality of life (HRQOL) questionnaires have been validated in children with sickle cell disease (SCD), but small sample sizes in these studies have limited clinical comparisons. We used the baseline clinical data from the Collaborative Data (C-Data) Project of the Comprehensive Sickle Cell Centers (CSCC) Clinical Trial Consortium to perform a detailed, descriptive study of HRQOL using the PedsQL™ version 4.0 generic core and fatigue scales. Methods. Retrospective clinical data were obtained via medical record abstraction. Staff-administered health history, psychosocial, and health behavior interviews were completed by a parent or guardian. PedsQL™ forms were completed separately by the child and a parent/guardian. Results. The study enrolled 1,772 subjects (53% boys) with a mean age of 9.6 years (SD 4.7). SS or Sβ0 thalassemia occurred in 68% and 32% had SC or Sβ+ thalassemia. The occurrences of pain, priapism, avascular necrosis of hips/shoulders (AVN), or asthma were the most common complications/conditions reported. Multiple regression models controlling for hemoglobinopathies, gender, and age suggested that parent reports of physical functioning and sleep/rest fatigue declined in response to pain or AVN, while school functioning scales declined in response to pain or asthma. Sickle pain, and to a lesser extent asthma, negatively influenced child reports on almost all functioning and fatigue scales. Conclusions. While longitudinal studies will be necessary to determine sensitivity to change, the current study suggests the potential utility of several PedsQL™ HRQOL scales as patient-reported outcome measures for observational or interventional treatment studies of children and adolescents with SCD.

Original languageEnglish (US)
Pages (from-to)485-494
Number of pages10
JournalPediatric Blood and Cancer
Issue number3
StatePublished - Sep 2010


  • Peds QL
  • Sickle cell disease

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology


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