Burosumab prevents further height deficit in toddlers affected by XLH
Most patients with X-linked hypophosphatemic rickets (XLH) present with short stature. This is the first large study on XLH children younger than 5 years treated with burosumab since a mean age of 2.1 years. We analyzed growth over a period of 2 years of therapy. Our data show that, in XLH toddlers, burosumab therapy prevents the further loss of height and the weight gain observed in patients treated with oral phosphate and active vitamin D therapy at the same age. As burosumab leads to better long-term outcomes in these children, we hope that our work will encourage the early use of burosumab and motivate further research in this field.
Authors
- Marco Pitea
- Anne-Sophie Lambert (ORCID: https://orcid.org/0000-0002-4232-3882)
- Diana‐Alexandra Ertl (ORCID: https://orcid.org/0000-0001-9404-0975)
- Stefano Mora (ORCID: https://orcid.org/0000-0002-4781-6715)
- Barbara Girerd
- Agnès Linglart (ORCID: https://orcid.org/0000-0003-3455-002X)
- Jugurtha Berkenou
- Christelle Audrain
- Anya Rothenbühler (ORCID: https://orcid.org/0000-0001-9246-5160)
- Elisa Sala (ORCID: https://orcid.org/0009-0003-0294-8591)
Institutions
- Vita-Salute San Raffaele University (IT)
- Inserm (FR)
- Université Paris-Saclay (FR)
- San Raffaele University of Rome (IT)
- Bicêtre Hospital (FR)
Publication Details
- Journal
- Endocrine Connections
- Published
- 2025-09-30
- DOI
- https://doi.org/10.1530/ec-25-0435
- Citations
- 1
- Primary Topic
- Pharmaceutical studies and practices
- Type
- article
- Field-Weighted Citation Impact
- 3.49