Objectives: XLHR in children with a Rickets Severity Score ≥2 can now be treated with the new antibody drug, Burosumab, which prevents bone deformities and increases gait endurance. This study illustrates the extent of complications in the traditional orthopedic treatment of XLHR. The impact of surgery and severity of complications in this patient population has not been systematically assessed before. Methods: The search strategy resulted in 215 studies and data were collected from 19 eligible studies and complications were categorized. Four medical charts of patients with XLHR at Aalborg University Hospital were assessed. Results: One complication occurred on average per surgical procedure for XLHR in the published literature. The 168 reported complications were categorized as follows: Type I (n=79): Complications with minimal intervention required and treatment goal still achieved, Type II (n=41): Complications with substantial change in treatment plan and treatment goal still achieved, Type IIIA (n=23): Complications with failure to achieve treatment goal and no new pathology or permanent sequelae, Type IIIB (n=25): Complications with failure to achieve treatment goal and/or new pathology or permanent sequelae. Conclusions: In average, one complication occurred per surgery and the severity of complications were substantial. The treatment goal was not achieved in 28% of surgeries whereof half of them resulted in permanent sequalae or new pathology. Our findings support the use of Burosumab for treatment of the skeletal changes in XLHR as the reported side-effects in Burosumab treatment appear negligible compared to the impact of surgeries and related complications (Imel EA, Glorieux FH, Whyte MP, Munns CF, Ward LM, Nilsson O, et al. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Lancet 2019;393:2416-27). However, orthopedic surgery might still be needed for correcting deformities restricting activities of daily living in XLHR patients.
|Journal||Journal of Pediatric Endocrinology and Metabolism|
|Number of pages||7|
|Publication status||Published - 26 Aug 2022|
Bibliographical note© 2022 Walter de Gruyter GmbH, Berlin/Boston.
- X-linked hypophosphatemic rickets
- complication reporting