Histiocytic Disorder Follow-up Study
Purpose
The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders
Conditions
- Histiocytosis
- Langerhans Cell Histiocytosis
- Erdheim-Chester Disease
- Rosai Dorfman Disease
- Xanthogranuloma
- Malignant Histiocytoses
Eligibility
- Eligible Ages
- Between 0 Years and 89 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- diagnosis of histiocytic disorder at any age 1. Langerhans cell histiocytosis, 2. Erdheim-Chester disease, 3. Rosai-Dorfman disease, 4. Xanthogranuloma, 5. Mixed histiocytosis 6. Malignant histiocytosis (Histiocytic sarcoma, langerhans cell sarcoma, interdigitating cell sarcoma) 7. Hemophagocytic lymphohistiocytosis
Exclusion Criteria
- None
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Retrospective
Recruiting Locations
Birmingham, Alabama 35233
More Details
- Status
- Recruiting
- Sponsor
- University of Alabama at Birmingham
Detailed Description
Background Over the last decade, major advances have occurred in histiocytic disorders with the discovery of MAPK-ERK pathway mutations leading to targeted therapeutics using BRAF- and MEK-inhibitors. However, there is a lack of large studies informing the burden of morbidity and mortality among people with histiocytosis. Institutional studies in pediatric LCH suggest that survivors suffer from long-term impairment of health related quality of life, cognitive dysfunction, pituitary dysfunction, and hearing difficulties in 20-50% cases. Studies from the investigative team and others have also shown a high incidence of acute myeloid leukemia and other second primary malignancies in pediatric and adult LCH. Chronic medical conditions may arise as a function of the disease biology or due to cancer therapy, as seen in other hematologic malignancies. Design Retrospective cohort study aimed at determining the risk of chronic health conditions and cause-specific mortality in pediatric and adult patients with histiocytic disorders. The study will include patients from the Histiocytosis Association registry and other institutions including UAB (n~6000). The participants will complete a validated questionnaire capturing details of chronic health conditions, health related quality of life, cognitive/psychological function, and healthcare utilization. Future directions The results from our study will be instrumental in formulating follow-up guidelines for histiocytic disorders and developing targeted survivorship programs to improve overall outcomes.