PedsQL Neurofibromatosis Type 1 Module for children, adolescents and young adults: feasibility, reliability, and validity
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The objective of the present study was to report on the measurement properties of the Pediatric Quality of Life Inventory (PedsQL) Neurofibromatosis Type 1 Module for pediatric patients ages 5–25 from the perspectives of patients and parents. The 104-item PedsQL NF1 Module and 23-item PedsQL Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents (343 families). Patients were diagnosed with NF1 using the National Institutes of Health diagnostic criteria. In addition to a Total Scale Score, 18 unidimensional scales were derived measuring skin itch bother, skin sensations, pain, pain impact, pain management, cognitive functioning, speech, fine motor, balance, vision, perceived physical appearance, communication, worry, treatment anxiety, medicines, stomach discomfort, constipation, and diarrhea. The PedsQL NF1 Module Scales evidenced excellent feasibility, excellent reliability for the Total Scale Scores (patient self-report α = 0.98; parent proxy-report α = 0.98), and good to excellent reliability for the 18 individual scales (patient self-report α = 0.71–0.96; parent proxy-report α = 0.73–0.98). Intercorrelations with the Generic Core Scales supported construct validity. Factor analysis supported the unidimensionality of the 18 individual scales. The PedsQL NF1 Module Scales demonstrated acceptable to excellent measurement properties, and may be utilized as standardized metrics to assess NF1-specific symptoms and problems in clinical research and practice in children, adolescents, and young adults.
KeywordsNeurofibromatosis Symptoms Pediatrics Patient-reported outcomes Health-related quality of life PedsQL
Pediatric Quality of Life Inventory
Health-related quality of life
Neurofibromatosis type 1
This study was fully funded by the Neurofibromatosis Therapeutic Acceleration Program (NTAP) at Johns Hopkins University School of Medicine, Baltimore, MD. We would like to thank Ms. Pamela Knight and Ms. Rebecca Harris at the Children’s Tumor Foundation, Dr. Jaishri Blakeley, Ms. Marigo Stathis and Dr. Sharad Verma at the NTAP, Dr. Cynthia Hingtgen and Ms. Rosemary Anderson at the NF Michigan, Ms. Ellynore Florendo and Dr. Linda Randolph at the Children’s Hospital of Los Angeles, Ms. Diana Haberkamp at the NF Midwest, and Ms. Kelly Walsh-Curtis at the NF Network for their help in reaching out to potential participants.
This study was fully funded by the Neurofibromatosis Therapeutic Acceleration Program (NTAP) at Johns Hopkins University School of Medicine, Baltimore, MD.
Compliance with ethical standards
Conflict of interest
Dr. Varni holds the copyright and the trademark for the PedsQL and receives financial compensation from the Mapi Research Trust, which is a nonprofit research institute that charges distribution fees to for-profit companies that use the Pediatric Quality of Life Inventory. All other authors have no competing interests.
The Institutional Review Board at Indiana University, Indianapolis, approved this study (Protocol # 1403632840).
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