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

Recruiting
Conditions
Langerhans Cell Histiocytosis
Interventions
Other: Data collection
Registration Number
NCT04627090
Lead Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto
Brief Summary

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

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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
186
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 & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
LCH PatientsData collectionAdult patients with LCH, diagnosed starting from January 2001
Primary Outcome Measures
NameTimeMethod
Evaluation of efficacy of first-line therapiesup to 3 months

Number of patients who achive complete/intermediate response

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (8)

Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo

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Cagliari, Italy

Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania

🇮🇹

Catania, Italy

Fondazione Irccs "Istituto Nazionale Tumori" - Milano - Sc Ematologia

🇮🇹

Milano, Italy

Ao Regionale S. Carlo - Potenza - Sic Ematologia

🇮🇹

Potenza, Italy

Fondazione Policlinico Universitario Agostino Gemelli Irccs

🇮🇹

Roma, Italy

Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia

🇮🇹

Roma, Italy

Aou "San Giovanni Di Dio E Ruggi D'Aragona" - Salerno - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche

🇮🇹

Salerno, Italy

Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia

🇮🇹

Sassari, Italy

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