Histiocytic Disorder Follow-up Study

NCT05915208 | Recruiting

• 1 other identifier: IRB-300008744
• Study Type: observational
• Target: 6,000
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Histiocytosis; Langerhans Cell Histiocytosis; Erdheim-Chester Disease; Rosai Dorfman Disease; Xanthogranuloma; Malignant Histiocytoses

Keywords

survivorship; long-term; targeted therapy; second cancer

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (1)

• Factors associated with new-onset morbidity

  From diagnosis to age at completion of survey, assessed up to 200 months

Eligibility Criteria

• Age: 0 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals with diagnosis of histiocytic disorder are eligible to participate, including family members (next of kin) of deceased individuals.

You may qualify if:

• 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

You may not qualify if:

• None

Sponsors & Collaborators

• University of Alabama at Birmingham: lead

Study Sites (1)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

MeSH Terms

Conditions

Histiocytosis; Histiocytosis, Langerhans-Cell; Erdheim-Chester Disease; Histiocytosis, Sinus; Histiocytic Sarcoma; Neoplasms, Second Primary

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytic Disorders, Malignant; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Gaurav Goyal, MD

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gaurav Goyal, MD

CONTACT

1-866-438-1640; histio@uabmc.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 25, 2023
• First Posted: June 22, 2023
• Study Start: September 1, 2022
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: June 11, 2025

Record last verified: 2025-06

Locations

US (1)