Effect of Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts From Patients Requiring Craniosynostosis Surgery for Idiopathic Reason or Due to Hypophosphatemic Rickets

What this Trial Is About

Fibroblast Growth Factor 23 (FGF23) plays a key role in managing phosphate, calcium, and vitamin D metabolism, mainly produced by bone cells called osteocytes. Its direct effect on bone cells, especially osteoblasts that form bone, has not been clearly established. This research focuses on patients with hypophosphatemic rickets (HR), a rare genetic condition marked by excessive FGF23 levels, often leading to bone deformities and growth issues. The study aims to understand how treatments like burosumab, a new antibody therapy that blocks FGF23, and active vitamin D impact bone cell biology in these patients compared to controls. The study involves observing human osteoblast cells from patients undergoing surgery for craniosynostosis, a condition affecting skull bone development either idiopathically or due to HR. Researchers will examine the effects of burosumab and 1-25 (OH) vitamin D on these cells in the lab to understand how these treatments influence bone formation and mineralization. Two groups are involved: patients with idiopathic craniosynostosis and those with craniosynostosis linked to HR. Participants will be children aged between 4 months and 18 years who need craniosynostosis surgery. The study includes collecting osteoblast cells during surgery and analyzing their growth and differentiation. The primary measure is the number of osteoblast cells obtained after differentiation. This observational study does not involve administering treatments but rather studies cells in vitro to assess the biological effects. Participation lasts through the surgical period and laboratory analysis, with ongoing monitoring of cell behavior and treatment impact.

Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts