Clinical trial NCT05915208: Histiocytic Disorder Follow-up Study
Official title: Histiocytic Disorder Follow-up Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Histiocytosis
Langerhans Cell Histiocytosis
Erdheim-Chester Disease
Rosai Dorfman Disease
Xanthogranuloma
Malignant Histiocytoses
Keywords: survivorship
long-term
targeted therapy
second cancer
Lead sponsor: University of Alabama at Birmingham
Sponsor class: OTHER
First posted: 2023-06-22
Enrollment: 6000
Sex: ALL
Minimum age (years): 0
Maximum age (years): 89
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

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

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.

Eligibility 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

Primary outcomes:
measure: Chronic health conditions diagnosed after LCH diagnosis graded using the Common Terminology Criteria for Adverse Events (CTCAE), v5.0 (https://ctep.cancer.gov)
timeFrame: From diagnosis of histiocytic disorder to age at completion of survey, assessed up to 200 months
measure: All-cause and cause-specific mortality
timeFrame: 2 years preceding death
Secondary outcomes:
measure: Factors associated with new-onset morbidity
timeFrame: From diagnosis to age at completion of survey, assessed up to 200 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
Study officials:
affiliation: University of Alabama at Birmingham
name: Gaurav Goyal, MD
role: PRINCIPAL_INVESTIGATOR
References: