title: Health-related quality of life in children with sickle cell disease: a report from the Comprehensive Sickle Cell Centers Clinical Trial Consortium.
abstract: 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 Sbeta(0) thalassemia occurred in 68% and 32% had SC or Sbeta(+) 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.
authors.affiliation: Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia 30322, USA. cdampie@emory.edu
authors.forename: Carlton
authors.initials: C
authors.lastname: Dampier
authors.forename: Susan
authors.initials: S
authors.lastname: Lieff
authors.forename: Petra
authors.initials: P
authors.lastname: LeBeau
authors.forename: Seungshin
authors.initials: S
authors.lastname: Rhee
authors.forename: Marsha
authors.initials: M
authors.lastname: McMurray
authors.forename: Zora
authors.initials: Z
authors.lastname: Rogers
authors.forename: Kim
authors.initials: K
authors.lastname: Smith-Whitley
authors.forename: Winfred
authors.initials: W
authors.lastname: Wang
mesh_terms: D000203:Activities of Daily Living; D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications; D001249:Asthma / Q000150:complications; D002648:Child; D002675:Child, Preschool; D005221:Fatigue / Q000150:complications; D005260:Female; D006760:Hospitalization; D006801:Humans; D008297:Male; D010146:Pain; D010290:Parents / Q000523:psychology; D011788:Quality of Life; D011795:Surveys and Questionnaires