Updates on the NIH trials Denosumab/Burosumab – Dr. Alison Boyce at the 2023 FD/MAS Community Conference and ICFDMAS meeting

Dr. Boyce shares updates on NIH clinical trials for Fibrous Dysplasia/McCune-Albright Syndrome (FD/MS), focusing on denosumab and burosumab.

Denosumab Study:

Denosumab shows promise in reducing bone turnover markers and impacting FD lesion metabolic activity. Challenges include bone turnover rebound after discontinuation, with severe hypercalcemia observed in some cases. Considerations for optimal dosing, treatment duration, and patient selection are discussed. A phase two study in children aims to prevent FD lesion progression.

Burosumab (Anti-FGF23) Study:

Burosumab is studied for treating hypophosphatemia in FD patients by maintaining high-normal phosphorus levels. Safety endpoints include monitoring FD lesion activity, with bone biopsies providing insights.

Patient Testimonial:

A patient on denosumab shares positive effects on FD lesions. Challenges, such as bone turnover rebound, are acknowledged, emphasizing the need for individualized treatment.

Insurance Coverage Concerns:

Concerns about insurance coverage for expensive medications are addressed. Advocacy for high-normal phosphorus levels as a therapeutic goal is highlighted.

In conclusion, ongoing studies aim to provide insights into the efficacy, safety, and optimal use of denosumab and burosumab in FD patients, emphasizing individualized treatment approaches and addressing challenges.

The FD/MAS Alliance Community Conference combined patient-centered science and real-world strategies for patients and families living with fibrous dysplasia, McCune-Albright syndrome (FD/MAS).

The FD/MAS Alliance (formerly Fibrous Dysplasia Foundation) is a community-driven 501c3 nonprofit that fosters the development of evidence-based treatments for Fibrous Dysplasia and McCune-Albright syndrome (FD/MAS). We advance research, provide education and channel the voices of individuals and caregivers with FD/MAS. To support our work visit www.fdmasalliance.org/donate