The University of Alabama at Birmingham
Project Term: July 1, 2023 - June 30, 2025
Advances in the treatment of Erdheim-Chester Disease (ECD) have led to a growing survivor population; however, there is a lack of information on the burden of chronic health problems, symptomatology, psychological dysfunction, and mortality experienced by this group of individuals. We propose a multi-institutional international study in collaboration with the ECD Global Alliance to answer these critical questions. Results from our study will help in counseling patients in the clinic about what to expect in the future and develop interventions to reduce the risk of health issues and disease or treatment-related symptoms.
The prognosis of ECD has improved tremendously due to the approval of targeted therapies. However, these treatments are administered for prolonged duration, and can lead to side effects and complications. These factors have led to a growing survivor population that is at an increased risk of disease/treatment-related symptoms, chronic health conditions, psychological problems, pain, and poor health-related quality of life. There is a lack of understanding about these health effects among people with ECD. We propose a multi-institutional international study in collaboration with the ECD Global Alliance to answer these critical questions. The study will aim to address the long-term health problems experienced by people with ECD and evaluate the symptom burden and evolution after starting treatments. The proposed study will involve completion of survey questionnaires by individuals at participating institutions and those registered with the ECD Global Alliance. The results from the proposed study will help define the healthcare needs of people with ECD and the symptoms experienced by them while on treatment. Ultimately, the study results will be used to counsel patients in the clinic about long-term effects and devise follow-up guidelines. These results will also lead to targeted interventions aimed at improving the symptoms and other health problems among the ECD population.