The FD/MAS Patient Registry has been gathering data for almost a full year. In July, Fibrous Dysplasia Foundation (FDF) received the exciting news that a team of researchers and patient advocates will rely on registry data to design and develop a research proposal. Together, FDF President Catherine Fairchild, President Emeritus Amanda Konradi, Phd, Andrea Burke DMD, MD, and FDF Program Manager Tovah Burstein have received a PCORI award to pursue a study comparing the experiences of craniofacial FD patients who have surgery to those who do not.
This PCORI project is especially exciting for the FD/MAS Patient Registry because it marks the first study that will develop questions and analyze data from the information that patients have provided on this platform. So far 517 patients have signed up for the registry and 230 have shared some of their medical data by taking one or more registry surveys. The registry is an important step in fighting FD/MAS because it gathers a variety of data on a patient’s life and medical history. The surveys are designed to gather that information in a scientific method that will offer researchers endless opportunities to study, analyze, and form new ideas about FD/MAS. “The registry has been a collaboration between patients, caregivers, and researchers from the beginning.” said Dr. Konradi. “PCORI has helped us develop a tool that prioritizes patients’ concerns and turns them into quantifiable data to be studied.”
While the data that patients have already provided will support the PCORI project as well as other future study projects, there is still much work to be done. “It is of utmost importance that we get as many patients as possible to fill out all of the registry surveys.” explained Burke. “Currently, clinical indications for craniofacial surgery are inconsistent and not evidence-based. By using patient data to retroactively analyze treatments, we can help patients make more informed decisions. Patients will really benefit by researchers compiling a broad spectrum of survey data to determine what treatments have been most effective.” The more data that scientists have, the better they will be able to understand this disease.
Part of the reason Fairchild, Konradi, Burke, and Burstein decided to pursue this project is because so many medical professionals lack accurate and well-informed information on effective treatments for FD/MAS. “Patients are often frustrated by the number of questions about their daily care that remain unanswered.” Said Catherine Fairchild. “As a parent of a patient, I want to do everything I can to get answers, and the registry represents an easy and effective way to make progress.” The team will continue to reference deidentified data from the registry as they develop their research into a proposal and, hopefully, receive funding to pursue a better understanding of craniofacial FD and the treatment options available to those patients.