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Trial Title:
Histiocytic Disorder Follow-up Study
NCT ID:
NCT05915208
Condition:
Histiocytosis
Langerhans Cell Histiocytosis
Erdheim-Chester Disease
Rosai Dorfman Disease
Xanthogranuloma
Malignant Histiocytoses
Conditions: Official terms:
Histiocytic Sarcoma
Histiocytosis, Langerhans-Cell
Histiocytosis
Erdheim-Chester Disease
Histiocytosis, Sinus
Conditions: Keywords:
survivorship
long-term
targeted therapy
second cancer
Study type:
Observational
Overall status:
Recruiting
Study design:
Time perspective:
Retrospective
Summary:
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.
Criteria for eligibility:
Study pop:
Individuals with diagnosis of histiocytic disorder are eligible to participate, including
family members (next of kin) of deceased individuals.
Sampling method:
Non-Probability Sample
Criteria:
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
Gender:
All
Minimum age:
0 Years
Maximum age:
89 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
University of Alabama at Birmingham
Address:
City:
Birmingham
Zip:
35233
Country:
United States
Status:
Recruiting
Contact:
Last name:
Gaurav Goyal, MD
Phone:
866-438-1640
Email:
histio@uabmc.edu
Start date:
September 1, 2022
Completion date:
December 2028
Lead sponsor:
Agency:
University of Alabama at Birmingham
Agency class:
Other
Source:
University of Alabama at Birmingham
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05915208
