Research Archive

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

An international group of thought leaders concerned with the care of patients with Fibrous Dysplasia/McCune Albright Syndrome (FD/MAS) recently encouraged treating physicians to use two measures to assess children’s health-related quality of life (QOL), the Pediatric Quality of Life Inventory (PEDS-QL) and the Hospital Anxiety and Depression scales (HADS). To date, no one has applied these measures to the FD/MAS population. This study was conducted to initiate consideration of their use in clinical management. The study is a retrospective analysis of data from 39 children, ages 2 to 17, who are participating in the Fibrous Dysplasia Foundation Patient Registry (FDFPR). This is an online resource that enables patients to report on demographics, symptoms, surgical and medical treatments received; to complete validated psychological and social measures (including the PEDS-QL and HADS), and to answer questions about access to and satisfaction with treatment.

The scores of children with FD/MAS were compared to published scores from the general population and from children with chronic diseases. Results showed that on average, young children, 2–7, had significantly worse physical health than the general population, but similar psychological and social health. However, the PEDS-QL scores of 30–50% of these young children suggested they were at risk in some aspect of QOL. PEDS-QL score averages for older children, 8–17, and the distribution of individual scores in relation to published benchmarks, suggested half were at risk of impaired QOL. More than half the 13–17 year-olds also scored above 7 on the HADS, the published value for clinical levels of anxiety. Older males scored better than females on the PEDS-QL and the HADS. Pain frequency was associated with physical function for older children.

In sum, older children with FD/MAS may be more compromised in terms of psychosocial QOL than previously reported. Clinicians should be attentive to the potential influence of gender on QOL in older children. Online patient registries associated with rare diseases, such as the FDFPR, have the potential to serve as efficient and cost-effective mechanisms to jumpstart examination of new measures in consideration for clinical use.