Histiocytic Disorder Follow-up Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Histiocytosis, Langerhans Cell Histiocytosis, Erdheim-Chester Disease, Rosai Dorfman Disease. Basic parameters: 0 years — 89 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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
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.
Primary outcome measures
- Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov) [Time frame: From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months]
- All-cause and cause-specific mortality [Time frame: 2 years preceding death]
Secondary outcome measures (1)
- Factors associated with new-onset morbidity [Time frame: From diagnosis to age at completion of survey, assessed up to 200 months]
Eligibility criteria
Inclusion criteria
- diagnosis of histiocytic disorder at any age
- Langerhans cell histiocytosis,
- Erdheim-Chester disease,
- Rosai-Dorfman disease,
- Xanthogranuloma,
- Mixed histiocytosis
- Malignant histiocytosis (Histiocytic sarcoma, langerhans cell sarcoma, interdigitating cell sarcoma)
- Hemophagocytic lymphohistiocytosis
Exclusion criteria
- None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- University of Alabama at Birmingham — Birmingham
Identifiers
NCT: NCT05915208 · IRB-300008744 · UAB