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Histiocytic Disorder Follow-up Study

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ClinicalTrials.gov Identifier: NCT05915208
Recruitment Status : Recruiting
First Posted : June 22, 2023
Last Update Posted : June 22, 2023
Sponsor:
Information provided by (Responsible Party):
Gaurav Goyal, University of Alabama at Birmingham

Study Description
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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

Condition or disease
Histiocytosis Langerhans Cell Histiocytosis Erdheim-Chester Disease Rosai Dorfman Disease Xanthogranuloma Malignant Histiocytoses

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 6000 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Histiocytic Disorder Follow-up Study
Actual Study Start Date : September 1, 2022
Estimated Primary Completion Date : July 2027
Estimated Study Completion Date : December 2028

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
    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.

  2. All-cause and cause-specific mortality [ Time Frame: 2 years preceding death ]
    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.


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 ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 89 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05915208


Contacts
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Contact: Gaurav Goyal, MD 1-866-438-1640 histio@uabmc.edu

Locations
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United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Gaurav Goyal, MD    866-438-1640    histio@uabmc.edu   
Sponsors and Collaborators
University of Alabama at Birmingham
Investigators
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Principal Investigator: Gaurav Goyal, MD University of Alabama at Birmingham
More Information
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Responsible Party: Gaurav Goyal, Principal Investigator, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT05915208    
Other Study ID Numbers: IRB-300008744
First Posted: June 22, 2023    Key Record Dates
Last Update Posted: June 22, 2023
Last Verified: June 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Gaurav Goyal, University of Alabama at Birmingham:
survivorship
long-term
targeted therapy
second cancer
Additional relevant MeSH terms:
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Histiocytic Sarcoma
Histiocytosis, Langerhans-Cell
Histiocytosis
Erdheim-Chester Disease
Histiocytosis, Sinus
Lymphatic Diseases
Lung Diseases, Interstitial
 Lung Diseases
Respiratory Tract Diseases
Histiocytosis, Non-Langerhans-Cell
Histiocytic Disorders, Malignant
Neoplasms by Histologic Type
Neoplasms