Changes of the lower limb deformity in children with FGF23-related hypophosphatemic rickets treated with Burosumab: a single-center prospective study.

Abstract

Fibroblast growth factor 23 (FGF23)-related hypophosphatemic rickets (HPR) are characterized by excess circulating FGF23 and low concentrations of serum phosphorus, leading to skeletal manifestations of rickets, including lower limb deformities in children. The objective of this study was to prospectively evaluate whether treatment with burosumab, a monoclonal antibody neutralizing FGF23, changes lower limb deformities in HPR. Patients who were 15 years of age or younger with a documented clinical diagnosis of HPR, receiving burosumab treatment, and had a minimum follow-up period of one year were included in the study. Various radiological parameters were measured from anteroposterior and lateral radiographs of the bilateral lower limbs taken before administration of burosumab and at 3, 6, 9, and 12 months after treatment for evaluation of lower limb alignment. Outcome was classified as 'improvement', 'no change', or 'deterioration' after 12 months treatment. Five patients (10 limbs), with a mean age of 7.2 years were included in this study. The outcome was 'improvement' in six limbs and 'no change' in four limbs. There were no limbs of 'deterioration'. The improvement in deformities after treatment was more significant in younger patients who originally showed severe lower limb deformities. Older patients with milder deformities, on the other hand, showed less improvement. Burosumab therapy favorably changed lower-limb malalignment in children with FGF23-related HPR.