Adult Pulmonary Langerhans Cell Histiocytosis: a National Registry-based Prospective Cohort Study

Enquête épidémiologique Sur la prévalence et le Pronostic de l'Histiocytose Langerhansienne Pulmonaire de l'Adulte en France

The long-term outcomes of adult patients with pulmonary Langerhans cell histiocytosis (PLCH), particularly survival, is largely unknown. This is the first prospective study in the field evaluating the long-term outcomes of PLCH patients. This french countrywide registry-based study included a large cohort of PLCH patients followed for a sufficiently long period to address risk factors of long-term outcomes of PLCH patients.

Study Overview

• Status: Recruiting
• Conditions: Histiocytosis; Histiocytosis, Langerhans-Cell; Histiocytosis Pulmonary
• Intervention / Treatment: Other: Standard of care

• Study Type: Observational
• Enrollment (Anticipated): 500

Contacts and Locations

• Study Contact: Name: Matthieu RESCHE-RIGON, MD PhD; Phone Number: 33 142499742; Email: matthieu.resche-rigon@u-paris.fr
• Study Contact Backup: Name: Abdellatif TAZI, MD PhD; Phone Number: 33 142499198; Email: abdellatif.tazi@aphp.fr

Study Locations

• France
  • Paris, France, 75010
    • Recruiting
    • French national reference centre for Histiocytoses
    • Contact: Matthieu RESCHE-RIGON, MD PhD; Phone Number: 33 142499742; Email: matthieu.resche-rigon@u-paris.fr
    • Contact: Abdellatif TAZI, MD PhD; Phone Number: 33 142499198; Email: abdellatif.tazi@aphp.fr

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Probability Sample

Study Population

All patients with newly diagnosed PLCH at adulthood (i.e. 18 years of age or older) referred to the French national reference centre for Histiocytoses

Description

Inclusion Criteria:

• Age >= 18 years
• Diagnosis of PLCH was either histologically confirmed on a biopsy of an involved tissue, or based on the combination of the following criteria: 1) an appropriate clinical picture; 2) a typical nodulo-cystic pattern on lung high-resolution computed tomography (HRCT) and 3) exclusion of alternative diagnoses

Exclusion Criteria:

-

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pulmonary Langerhans cell histiocytosis (PLCH); All patients with newly diagnosed PLCH at adulthood (i.e. 18 years of age or older) referred to the French national reference centre for Histiocytoses	Other: Standard of care; Standard of car of pulmonary Langerhans cell histiocytosis (PLCH)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	Overall survival defined as the time from inclusion to death from any cause.	20 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
CRF	Cumulative incidence of chronic respiratory failure (CRF) from inclusion	20 years
PH	Cumulative incidence of pulmonary hypertension (PH) from inclusion	20 years
Malignant diseases	Cumulative incidence of Malignant diseases from inclusion	20 years
Extra-pulmonary involvement in isolated PLCH	Cumulative incidence of extra pulmonary localisations from inclusion	20 years
Annual prevalence	Number of people with the disease at any time during a year	20 years

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris

Publications and helpful links

General Publications

• Benattia A, Bugnet E, Walter-Petrich A, de Margerie-Mellon C, Meignin V, Seguin-Givelet A, Lorillon G, Chevret S, Tazi A. Long-term outcomes of adult pulmonary Langerhans cell histiocytosis: a prospective cohort. Eur Respir J. 2022 May 26;59(5):2101017. doi: 10.1183/13993003.01017-2021. Print 2022 May.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2004
• Primary Completion (Anticipated): December 31, 2034
• Study Completion (Anticipated): December 31, 2035

Study Registration Dates

• First Submitted: November 30, 2020
• First Submitted That Met QC Criteria: December 7, 2020
• First Posted (Actual): December 11, 2020

Study Record Updates

• Last Update Posted (Actual): December 17, 2020
• Last Update Submitted That Met QC Criteria: December 15, 2020
• Last Verified: November 1, 2020

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: 2014_TAZI

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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