Histiocytic Disorder Follow-up Study

Langerhans Cell Histiocytosis Clinical Trial

Official title:

Histiocytic Disorder Follow-up Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05915208
• Other study ID #: IRB-300008744
• Status: Recruiting
• Start date: September 1, 2022
• Est. completion date: December 2028

Study information

• Verified date: June 2024
• Source: University of Alabama at Birmingham
• Contact: Gaurav Goyal, MD
• Phone: 1-866-438-1640
• Email: histio[@]uabmc.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 6000
• Est. completion date: December 2028
• Est. primary completion date: July 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 89 Years

Eligibility

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

Related Conditions & MeSH terms

• Erdheim-Chester Disease
• Histiocytic Sarcoma
• Histiocytosis
• Histiocytosis, Langerhans-Cell
• Langerhans Cell Histiocytosis
• Rosai Dorfman Disease

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama

Sponsors (1)

Lead Sponsor	Collaborator
University of Alabama at Birmingham

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov)	The CTCAE criteria for CHCs distinguishes each acute and chronic condition (including SPMs) in persons with cancer from grades 1 through 4 with unique clinical descriptions of severity (grade 1, mild; grade 2, moderate; grade 3, severe; grade 4, life-threatening/disabling). The main outcome will be the number of distinct CTCAE grade 3-4 conditions among LCH cases compared with unaffected (non-cancer) controls.	From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months
Primary	All-cause and cause-specific mortality	Causes of death will be grouped into primary cancer (LCH)-related mortality (PCRM) and non-primary cancer-related mortality (NPCRM - death from all causes other than the index LCH diagnosis) by two independent investigators.	2 years preceding death
Secondary	Factors associated with new-onset morbidity	We will build a prediction model that has a good discrimination for predicting CHC, SPMs, and psychological outcomes separately using data from the LCH (case) cohort.	From diagnosis to age at completion of survey, assessed up to 200 months