Denosumab: Summary of the LUMC studies and The DeFiD trial – Dr. Natasha Appelman-Dijkstra at the 2023 FD/MAS Community Conference and ICFDMAS meeting

Dr. Natasha Appelman-Dijkstra discusses LUMC on Fibrous Dysplasia/McCune-Albright Syndrome (FD/MS), focusing on denosumab and burosumab. Differences between the care at LUMC and NIH are highlighted, emphasizing a mix of monostotic and polyostotic cases at LUMC.

Denosumab study: Positive effects observed, including decreased lesion size on sodium fluoride PET scans.

Burosumab study: Investigating hypophosphatemia treatment in FD patients. Safety endpoints include monitoring FD lesion activity and bone biopsies.

Patient testimonial: Positive effects of denosumab shared, emphasizing individualized treatment approaches.

Insurance coverage concerns: Addressing challenges related to expensive medications and advocating for high-normal phosphorus levels as a therapeutic goal.

Dr. Appelman-Dijkstra also emphasized the need to move beyond presenting case reports in the literature and focus on larger studies involving more patients.

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