Assessing quality of life in pediatric fibrous dysplasia and McCune Albright syndrome: PEDS-QL and HADS data from the Fibrous Dysplasia Foundation Patient Registry

PURPOSE: The International FD/MAS Consortium recently encouraged using the Pediatric Quality of Life Inventory (PEDS-QL) and the Hospital Anxiety and Depression scales (HADS) in clinical care. This study examines scores on these measures among pediatric fibrous dysplasia and McCune Albright (FD/MAS) patients to initiate consideration of their use in clinical treatment. METHODS: This is a retrospective analysis of pediatric data from 39 minors, ages 2–17, entered in the Fibrous Dysplasia Foundation Patient Registry from July 2016 to December 2018. Sample means and score distributions are compared to general population and chronic disease benchmarks. Associations with medical and demographic variables are also explored. RESULTS: Mean PEDS-QL scores for children 2–7 were inconclusive in determining at risk status for impaired quality of life (QOL). Individual score distributions suggested up to half experienced extensive physical or social impairment. Means and individual score distributions for the physical and psychosocial components of the PEDS-QL for children 8–17 suggested many were at risk of impaired QOL. Over half of 13–17 year-olds met the clinical benchmark for anxiety. Older males scored better than females on the PEDS-QL and HADS. Pain frequency was associated with physical function for older children. CONCLUSIONS: Older children with FD/MAS may be more compromised in terms of psychosocial QOL than previously reported. Clinicians should be attentive to the influence of gender on QOL in older children. Online patient registries associated with rare diseases have the potential to serve as efficient and cost-effective mechanisms to jumpstart examination of new measures in consideration for clinical use.