X-linked hypophosphatemia and burosumab: Practical clinical points from the French experience - 26/09/21
pages | 7 |
Iconographies | 0 |
Vidéos | 0 |
Autres | 0 |
Highlights |
• | X-linked hypophosphatemia is the most common cause of inherited hypophosphatemia. |
• | Burosumab: a novel monoclonal antibody that specifically targets FGF23 restores the phosphate renal reabsorption and the endogenous calcitriol synthesis panel discussion on the management of young XLH patients treated with burosumab. |
• | Practical clinical points based on the French experience with burosumab. |
Abstract |
Hereditary hypophosphatemia with increased FGF23 levels are rare inherited metabolic diseases characterized by low serum phosphate because of impaired renal tubular phosphate reabsorption. The most common form is X-linked hypophosphatemia (XLH), secondary to a mutation in the PHEX gene. In children, XLH is often manifested by rickets, delayed development of gait, lower limb deformities, growth retardation, craniosynostosis, and spontaneous dental abscesses. In adults, patients present diffuse musculoskeletal pain (bone and joints), early osteoarthritis, entesopathies, pseudo-fractures, muscular weakness, and severe dental damage. Conventional medical management is based on the combined administration of oral phosphate supplementation with active vitamin D analogs. Treatment with the recently approved anti-FGF23 burosumab is an alternative, especially in severe forms. Burosumab restores phosphate reabsorption in the proximal tubule and stimulates the endogenous synthesis of calcitriol. In Europe, burosumab has been approved for the treatment of XLH with radiographic evidence of bone disease in pediatric patients from one year of age and in adults. This manuscript will discuss the specific management of burosumab in children and adolescents in daily practice.
Le texte complet de cet article est disponible en PDF.Keywords : X-linked hypophosphatemia (XLH), Burosumab, Osteomalacia, Phosphate, Vitamin D, FGF23, PHEX, Rickets
Plan
Vol 88 - N° 5
Article 105208- octobre 2021 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’achat d’article à l’unité est indisponible à l’heure actuelle.
Déjà abonné à cette revue ?