Gaurav Goyal (University of Alabama at Birmingham, Birmingham, AL US)
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 the 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.
Amount: 200,000 USD in partnership with the Leukemia & Lymphoma Society
Interim Report
The survival of people with ECD has improved with the introduction of targeted therapies, resulting in a growing population of people living with ECD as a chronic illness. There is a knowledge gap with respect to the burden of chronic medical conditions, mortality, symptoms, and health-related quality of life in ECD. Our study aimed to address these questions through the Histiocytic Disorder Follow-Up Study, which involves the completion of a detailed survey pertaining to health problems experienced by individuals with ECD through the ECDGA registry, including nearly 450 individuals with ECD from the US. We also aim to evaluate the physical and mental health symptoms that encompass the health-related quality of life through a survey administered in the clinic. As of December 2023, we have completed survey data from 75 individuals with ECD from the ECDGA registry, with a goal of approximately 300 completed surveys over the next 2-3 years. We have also received completed health-related quality-of-life surveys from 39 individuals with ECD. These surveys have suggested that individuals with ECD may experience worse physical health and fatigue compared with the general population without ECD. We anticipate that the completion of this study will lead to the development of follow-up guidelines to care for people with ECD in the clinic and strategies to improve long-term outcomes and symptoms.