LCH in Adults: a Collaborative, Prospective-retrospective, Observational Study

Study Overview
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Brief Summary
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This is a multicenter, retrospective and prospective, observational, no profit study including adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient. Each patient will be followed up to one year after the last enrolled patient. Each patient will be followed up to one year after the last enrolled patient.This study plans to collect clinical information at the time of diagnosis and at various follow-ups to evaluate the efficacy of first-line therapies. Diagnostic and therapeutic data will be collected from routine clinical evaluations and laboratory and instrumental investigations carried out during clinical practice

Detailed Description
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This is a multicenter, retrospective and prospective, observational, no profit study including adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient. Data from patients, diagnosed from January 2001 to the date of patient enrolment, is considered retrospective, while data collected after patient enrolment, and up to two years after the first enrolled patient, is considered prospective. At least one year of follow-up is expected for each patient. Each patient will be followed up to one year after the last enrolled patient. This study plans to collect clinical information at the time of diagnosis and at various follow-ups to evaluate the efficacy of first-line therapies. Diagnostic and therapeutic data will be collected from routine clinical evaluations and laboratory and instrumental investigations carried out during clinical practice. A sub-cohort of patients with available biological data on somatic mutations of the RAF-MEK-ERK genes will also be evaluated.

Official Title
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Langerhans Cell Histiocytosis in Adults: a Collaborative, Prospective-retrospective, Observational GIMEMA Study

Conditions
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Langerhans Cell Histiocytosis

Intervention / Treatment
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* Other: Data collection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed histological and immunohistochemical diagnosis of LCH (CD1a+, S-100+, CD207+), from January 2001 up to two years after the first enrolled patient. Patients with isolated vertebra plana, not related to a malignancy, and without a soft tissue component, are included without a histological and immunohistochemical diagnosis; Age ≥18 years at the time of definitive diagnosis; Signed, written informed consent, according to ICH/EU/GCP, and national, local laws. Exclusion Criteria: Age ≥18 years and presumptive diagnosis of LCH, or definitive diagnosis of non-Langerhans Histiocytosis (Juvenile xantogranuloma, Rosai-Dorfman disease, Erdheim-Chester disease, etc.).

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| LCH Patients<br>Adult patients with LCH, diagnosed starting from January 2001 | Other: Data collection<br>* Diagnostic and therapeutic data of adult patients with LCH, collected from clinical and laboratory evaluations, carried out during clinical practice.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of efficacy of first-line therapies | Number of patients who achive complete/intermediate response | up to 3 months |