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

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

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

Study Overview

• Status: Recruiting
• Conditions: Langerhans Cell Histiocytosis
• Intervention / Treatment: Other: Data collection

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 186

Contacts and Locations

• Study Contact: Name: Paola Fazi; Phone Number: 06 70390528; Email: p.fazi@gimema.it
• Study Contact Backup: Name: Enrico Crea; Phone Number: 06 70390515; Email: e.crea@gimema.it

Study Locations

• Italy
  • Cagliari, Italy
    • Recruiting
    • Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
    • Contact: Simula
  • Catania, Italy
    • Recruiting
    • Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania
    • Contact: Amalia Figuera
  • Milano, Italy
    • Recruiting
    • Fondazione Irccs "Istituto Nazionale Tumori" - Milano - Sc Ematologia
    • Contact: Chiaravalli
  • Potenza, Italy
    • Recruiting
    • Ao Regionale S. Carlo - Potenza - Sic Ematologia
    • Contact: Angela Amendola; Email: angela.amendola@ospedalesancarlo.it
  • Roma, Italy
    • Recruiting
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
    • Contact: Livio Pagano
  • Roma, Italy
    • Recruiting
    • Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia
    • Contact: Fiorina Giona; Email: giona@bce.uniroma1.it
  • Salerno, Italy
    • Recruiting
    • Aou "San Giovanni Di Dio E Ruggi D'Aragona" - Salerno - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche
    • Contact: Carmine Selleri
  • Sassari, Italy
    • Recruiting
    • Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia
    • Contact: Claudio Fozza; Email: cfozza@uniss.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient.

Description

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.).

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
LCH Patients; Adult patients with LCH, diagnosed starting from January 2001	Other: Data collection; Diagnostic and therapeutic data of adult patients with LCH, collected from clinical and laboratory evaluations, carried out during clinical practice.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2021
• Primary Completion (Estimated): March 1, 2024
• Study Completion (Estimated): March 1, 2024

Study Registration Dates

• First Submitted: November 2, 2020
• First Submitted That Met QC Criteria: November 6, 2020
• First Posted (Actual): November 13, 2020

Study Record Updates

• Last Update Posted (Estimated): February 7, 2024
• Last Update Submitted That Met QC Criteria: February 6, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lymphatic Diseases; Lung Diseases; Lung Diseases, Interstitial; Histiocytosis, Langerhans-Cell; Histiocytosis
• Other Study ID Numbers: RD0120

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No