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, Comprehensive Sickle Cell Centers (CSCC) Clinical Trial Consortium (CTC), Lillian McMahon, Asif Qureshi, Shafat Quadri, Matthew Heeney, Tiffany Kang, Amber Smith, Maureen Okam, Ainsley Ross, Carole Tremonti, Lennette Benjamin, Gwendolyn Swinson, Arlette Paul, Catherine Driscoll, Kim Smith-Whitley, Henrietta Enninful-Eghan, Tannoa Jackson, Karen Kalinyak, Tammy Nordheim, Marlene Eaton, Laura DeCastro, Michael Armstrong, Jude Jonassaint, Christle Cameron, Amanda Mandy, Kenneth Ataga, Ruth Baldwin, Perrior Anderson, Carlton Dampier, Camille Coleman, Samir Ballas, April Deoria, Carol Wexler, Ashok Raj, Salvatore Bertolone, Claudia Grandinetti, Elliott Vichinsky, Christine Hoehner, Susan Paulukonis, Ofelia Alvarez, Patricia Williams, Mary Donovan, James Huang, Laura Quill, Jonah Todd-Geddes, Winfred Wang, Karen Winton, Lynn Wynn, Zora Rogers, Cynthia Rutherford, Leah Adix, Nancy Lee, Joan Parkhurst Cain, Christina Gonzalez, Annette Johnson, David Bessman, Lisa Hernandez Garcia, Phyllis Crawford, Carlton Dampier, Susan Lieff, Petra LeBeau, Seungshin Rhee, Marsha McMurray, Zora Rogers, Kim Smith-Whitley, Winfred Wang, Comprehensive Sickle Cell Centers (CSCC) Clinical Trial Consortium (CTC), Lillian McMahon, Asif Qureshi, Shafat Quadri, Matthew Heeney, Tiffany Kang, Amber Smith, Maureen Okam, Ainsley Ross, Carole Tremonti, Lennette Benjamin, Gwendolyn Swinson, Arlette Paul, Catherine Driscoll, Kim Smith-Whitley, Henrietta Enninful-Eghan, Tannoa Jackson, Karen Kalinyak, Tammy Nordheim, Marlene Eaton, Laura DeCastro, Michael Armstrong, Jude Jonassaint, Christle Cameron, Amanda Mandy, Kenneth Ataga, Ruth Baldwin, Perrior Anderson, Carlton Dampier, Camille Coleman, Samir Ballas, April Deoria, Carol Wexler, Ashok Raj, Salvatore Bertolone, Claudia Grandinetti, Elliott Vichinsky, Christine Hoehner, Susan Paulukonis, Ofelia Alvarez, Patricia Williams, Mary Donovan, James Huang, Laura Quill, Jonah Todd-Geddes, Winfred Wang, Karen Winton, Lynn Wynn, Zora Rogers, Cynthia Rutherford, Leah Adix, Nancy Lee, Joan Parkhurst Cain, Christina Gonzalez, Annette Johnson, David Bessman, Lisa Hernandez Garcia, Phyllis Crawford
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.
2010 Wiley-Liss, Inc.
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Source: PubMed