Objective: To investigate health-related quality of life (HR-QOL) in children with neurofibromatosis type 1 (NF1) with parental reports and children's self-reports, and to investigate the potential contribution of demographic factors, disease-specific factors, and problems in school performance or behavior. Study design: In a prospective observational study, parents of 58 children with NF1 (32 boys, 26 girls, age 12.2 ± 2.5 years) visiting a university clinic, and their 43 children 10 years or older were assessed with the Child Health Questionnaire (CHQ). Potential determinants of domain scores were assessed in 3 explorative regression models. Results: Parents reported a significant impact of NF1 on 9/13 CHQ scales, with moderate effect sizes on 8 (general health perceptions, physical functioning, general behavior, mental health, self esteem, family activities, role functioning emotional/behavioral, and parent emotional impact). Children report an impact on bodily pain, and an above average general behavior. Multiple CHQ scales were sensitive to demographic factors and behavioral problems, and 1 to NF1 severity. NF1 visibility and school problems did not influence HR-QOL. Conclusions: Parents, but not the children with NF1, report a profound impact of NF1 on physical, social, behavioral, and emotional aspects of HR-QOL. Multiple HR-QOL domains were most sensitive to behavioral problems, which points to an exciting potential opportunity to improve HR-QOL in children with NF1 by addressing these behavioral problems.

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Persistent URL dx.doi.org/10.1016/j.jpeds.2008.08.045, hdl.handle.net/1765/24438
Citation
Krab, L.C, Oostenbrink, R, de Goede-Bolder, A, Aarsen, F.K, Elgersma, Y, & Moll, H.A. (2009). Health-Related Quality of Life in Children with Neurofibromatosis Type 1: Contribution of Demographic Factors, Disease-Related Factors, and Behavior. Journal of Pediatrics, 154(3). doi:10.1016/j.jpeds.2008.08.045