James W Varni1, Kavitha Nutakki2,3, Nancy L Swigonski2,4. 1. Department of Pediatrics, College of Medicine, Department of Landscape Architecture and Urban Planning, College of Architecture, Texas A&M University, 3137 TAMU, College Station, TX, 77843-3137, USA. jvarni@tamu.edu. 2. Children's Health Services Research, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA. 3. Brigham and Women's Hospital, Boston, MA, USA. 4. Fairbanks School of Public Health, Indiana University, Indianapolis, IN, USA.
Abstract
OBJECTIVES: The aim was to investigate pain, skin sensations symptoms and patient self-reported, and parent proxy-reported cognitive functioning as predictors of generic health-related quality of life (HRQOL) in pediatric patients with Neurofibromatosis Type 1 (NF1) from the perspectives of patients and parents. METHODS: The Pain, Skin Itch Bother, Skin Sensations, and Cognitive Functioning Scales from the PedsQL™ Neurofibromatosis Type 1 Module and the PedsQL™ Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents. Patients were 5-25 years of age. Pain and skin symptoms and cognitive functioning were tested for bivariate and multivariate linear associations with generic HRQOL. RESULTS: Pain, skin itch bother, skin sensations, and cognitive functioning were associated with decreased HRQOL in bivariate analyses (Ps < 0.001). In predictive analytics models, utilizing hierarchical multiple regression analyses controlling for demographic covariates, pain, skin itch bother, skin sensations, and cognitive functioning as a group accounted for 61 percent of the variance in patient-reported generic HRQOL (P < 0.001), reflecting a large effect size. For parent proxy-report, the predictor variables as a group accounted for 53% of the variance in generic HRQOL. CONCLUSIONS: Pain, skin symptoms, and patient self-reported and parent proxy-reported cognitive functioning are key predictors of generic HRQOL in pediatric patients with NF1. Delineating NF1-specific symptoms and cognitive functioning as high-priority predictors from the patient and parents perspective enhances a family-centered approach in clinical research, clinical trials, and clinical practice intended to improve the global generic HRQOL of pediatric patients with NF1.
OBJECTIVES: The aim was to investigate pain, skin sensations symptoms and patient self-reported, and parent proxy-reported cognitive functioning as predictors of generic health-related quality of life (HRQOL) in pediatric patients with Neurofibromatosis Type 1 (NF1) from the perspectives of patients and parents. METHODS: The Pain, Skin Itch Bother, Skin Sensations, and Cognitive Functioning Scales from the PedsQL™ Neurofibromatosis Type 1 Module and the PedsQL™ Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents. Patients were 5-25 years of age. Pain and skin symptoms and cognitive functioning were tested for bivariate and multivariate linear associations with generic HRQOL. RESULTS:Pain, skin itch bother, skin sensations, and cognitive functioning were associated with decreased HRQOL in bivariate analyses (Ps < 0.001). In predictive analytics models, utilizing hierarchical multiple regression analyses controlling for demographic covariates, pain, skin itch bother, skin sensations, and cognitive functioning as a group accounted for 61 percent of the variance in patient-reported generic HRQOL (P < 0.001), reflecting a large effect size. For parent proxy-report, the predictor variables as a group accounted for 53% of the variance in generic HRQOL. CONCLUSIONS:Pain, skin symptoms, and patient self-reported and parent proxy-reported cognitive functioning are key predictors of generic HRQOL in pediatric patients with NF1. Delineating NF1-specific symptoms and cognitive functioning as high-priority predictors from the patient and parents perspective enhances a family-centered approach in clinical research, clinical trials, and clinical practice intended to improve the global generic HRQOL of pediatric patients with NF1.
Authors: Kiarash Khosrotehrani; Sylvie Bastuji-Garin; Vincent M Riccardi; Patricia Birch; Jan M Friedman; Pierre Wolkenstein Journal: Am J Med Genet A Date: 2005-01-01 Impact factor: 2.802
Authors: Rajesh Khanna; Aubin Moutal; Katherine A White; Aude Chefdeville; Pedro Negrao de Assis; Song Cai; Vicki J Swier; Shreya S Bellampalli; Marissa D Giunta; Benjamin W Darbro; Dawn E Quelle; Jessica C Sieren; Margaret R Wallace; Christopher S Rogers; David K Meyerholz; Jill M Weimer Journal: Pain Date: 2019-11 Impact factor: 7.926