Clinical trial NCT04627090: LCH in Adults: a Collaborative, Prospective-retrospective, Observational Study
Official title: Langerhans Cell Histiocytosis in Adults: a Collaborative, Prospective-retrospective, Observational GIMEMA Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Langerhans Cell Histiocytosis
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2020-11-13
Enrollment: 186
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient.

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.

Eligibility criteria: 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.).

Primary outcomes:
measure: Evaluation of efficacy of first-line therapies
timeFrame: up to 3 months
Secondary outcomes:
Other outcomes:
Locations:
city: Cagliari
country: Italy
facility: Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
city: Catania
country: Italy
facility: Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania
city: Milano
country: Italy
facility: Fondazione Irccs "Istituto Nazionale Tumori" - Milano - Sc Ematologia
city: Potenza
country: Italy
facility: Ao Regionale S. Carlo - Potenza - Sic Ematologia
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli Irccs
city: Roma
country: Italy
facility: Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia
city: Salerno
country: Italy
facility: Aou "San Giovanni Di Dio E Ruggi D'Aragona" - Salerno - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche
city: Sassari
country: Italy
facility: Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia
Study officials:
References: