Observational study on Langerhans Cell Histiocytosis in adults

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

This study is collecting and analyzing health information from adults with Langerhans Cell Histiocytosis to see how well first-line treatments work and to look at certain gene mutations.

Quick facts

• Study type: Observational
• Enrollment: 186 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto (other)
• Locations: 8 sites (Cagliari and 7 other locations)
• Trial ID: NCT04627090 on ClinicalTrials.gov

What this trial studies

This multicenter observational study aims to collect and analyze clinical data from adult patients diagnosed with Langerhans Cell Histiocytosis (LCH) from January 2001 onwards. The study includes both retrospective data from previously diagnosed patients and prospective data collected after patient enrollment, with each patient followed for at least one year. The goal is to evaluate the efficacy of first-line therapies through routine clinical evaluations and laboratory investigations. Additionally, a sub-cohort will assess biological data related to specific gene mutations.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of LCH.

Not a fit: Patients with presumptive diagnoses of LCH or other forms of histiocytosis will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of LCH treatment efficacy and improve patient outcomes.

How similar studies have performed: Other studies on LCH have shown varying degrees of success, but this specific observational approach is relatively novel.

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.).

Where this trial is running

Cagliari and 7 other locations

• Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo — Cagliari, Italy (RECRUITING)
• Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania — Catania, Italy (RECRUITING)
• Fondazione Irccs "Istituto Nazionale Tumori" - Milano - Sc Ematologia — Milano, Italy (RECRUITING)
• Ao Regionale S. Carlo - Potenza - Sic Ematologia — Potenza, Italy (RECRUITING)
• Fondazione Policlinico Universitario Agostino Gemelli Irccs — Roma, Italy (RECRUITING)
• Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia — Roma, Italy (RECRUITING)
• Aou "San Giovanni Di Dio E Ruggi D'Aragona" - Salerno - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche — Salerno, Italy (RECRUITING)
• Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia — Sassari, Italy (RECRUITING)

Study contacts

• Study coordinator: Paola Fazi

Conditions: Langerhans Cell Histiocytosis