Bisphosphonates: Update from the ProFiDys Study - Dr. Roland Chapurlat

Bisphosphonates: Update from the ProFiDys Study - Dr. Roland Chapurlat at the 2023 FD/MAS Community Conference and ICFDMAS meeting
Dr. Chapurlat discusses a trial aimed at assessing the impact on bone pain and radiological improvement over three years. The primary endpoint focused on radiological improvement, and the study included 32 patients. The results indicated that risedronate did not show a significant difference in radiological improvement compared to a placebo. The study was underpowered, but no substantial difference was observed. Some patients on risedronate experienced negative changes in lesions. While the primary endpoint was not met, the study provided insights into the feasibility of using CT scans as an outcome measure for future trials. The discussion also highlighted challenges in conducting placebo-controlled trials for rare diseases like fibrous dysplasia.
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.