Presented by Dr. Gaurav Goyal, University of Alabama at Birmingham In partnership with the Erdheim-Chester Disease Global Alliance (ECDGA) This presentation explores the importance of survivorship research in Erdheim-Chester Disease (ECD) and the critical role patients play in shaping that research. Dr. Gaurav Goyal, adult hematologist-oncologist at the University of Alabama at Birmingham, discusses how survivorship extends beyond medical treatment—it includes the emotional, psychological, and social experiences of living with a rare disease.
Through the Histiocytic Disorder Follow-Up Study, developed in collaboration with the ECD Global Alliance, researchers are gathering first-hand insights directly from patients and caregivers to better understand what life looks like after an ECD diagnosis. The study focuses on long-term outcomes such as fatigue, pain, cognitive changes, anxiety, and quality-of-life factors that are often overlooked in traditional clinical data.
Dr. Goyal explains why this type of research is essential for rare diseases like ECD, where survivorship data remain limited. By listening to the patient voice and collecting information on physical health, mental well-being, and daily living challenges, the study aims to create a more holistic understanding of the ECD experience. These findings will help guide evidence-based recommendations, improve patient care, and influence future advocacy and policy efforts.
Key topics covered in this presentation include:
- Why patient-reported outcomes are vital for improving care
- Early findings from the Histiocytic Disorder Follow-Up Study
- How to participate in ongoing research
- The value of large-scale community involvement in rare disease studies
By sharing their experiences, patients help shape the future of ECD care—ensuring that treatment progress goes hand-in-hand with improved quality of life.