Clinical trial NCT05969821: Clonal Hematopoiesis of Immunological Significance
Official title: National Cohort of Patients With Dysimmune Manifestations With or Without Clonal Hematopoiesis
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Immune System Diseases
Autoimmune Diseases
Inflammation
Autoinflammatory Diseases
Vexas Syndrome
Hematopoiesis Clonal
Clonal Hematopoiesis of Indeterminate Potential
Hematologic Diseases
Myelodysplastic-Myeloproliferative Diseases
Leukemia Myelomonocytic Chronic
Myelodysplastic Syndromes
Myeloproliferative Disorders
Lymphoproliferative Disorders
Lymphoma
Leukemia
Monoclonal Gammopathy of Undetermined Significance
Keywords: Inflammation
Autoinflammatory Diseases
Vexas syndrome
Autoimmune Diseases
Clonal Hematopoiesis of Indeterminate Potential
Myelodysplastic-Myeloproliferative Diseases
Lymphoproliferative Disorders
Monoclonal Gammopathy of Undetermined Significance
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
Collaborators: name: Sorbonne University
name: Institut National de la Santé Et de la Recherche Médicale, France
name: Club MINHEMON (MEDECINE INTERNE, HEMATO ET ONCO)
First posted: 2023-08-01
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The research concerns :

* patients whose inflammatory disease without or with haemopathy is already known when the cohort is set up, and for whom data will be collected retrospectively and then prospectively
* incident cases identified after the cohort was set up.

Brief summary: Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome.

Detailed description: The clinical spectrum of dysimmune manifestations associated with blood diseases is wide. The pathophysiology of these manifestations is not well understood and their management is poorly codified. This observational cohort aims to list the different clinical pictures, the therapeutic management and the prognosis of patients according to the type of dysimmune manifestations and the type of hemopathy. We wish to have an inventory of the demographic, genetic, clinical and evolutionary data of patients with an inflammatory manifestation associated or not with a myeloid or lymphoid hemopathy. This will make it possible to establish quantitative data on the morbidity and mortality of these rare diseases and to propose therapeutic trials for the most serious patients.

Eligibility criteria: Inclusion Criteria:

* Age \>=18 years old
* Confirmed dysimmune manifestations: clinical or biological abnormality or systemic disease
* Presence or absence of myeloid or lymphoid blood disease according to World Health Organization (WHO) classification

Exclusion Criteria:

* Persons benefiting from special protection: adults under guardianship and curatorship; people hospitalized without their consent and not protected by law; persons deprived of liberty.
* Persons not affiliated to the social security system

Primary outcomes:
measure: Incidence of dysimmune manifestations associated with hematological disorders
timeFrame: Baseline
Secondary outcomes:
measure: VEXAS syndrome
timeFrame: 10 years
measure: Dysimmune manifestations other than VEXAS syndrome
timeFrame: 10 years
measure: Myeloid hemopathy
timeFrame: 10 years
measure: Lymphoid hemopathy
timeFrame: 10 years
measure: Clonal hematopoiesis of undeterminate potential
timeFrame: 10 years
measure: Skin involvement
timeFrame: 10 years
measure: Musculoskeletal involvement
timeFrame: 10 years
measure: Ocular involvement
timeFrame: 10 years
measure: Vascular involvement
timeFrame: 10 years
measure: Neurological involvement
timeFrame: 10 years
measure: Digestive system involvement
timeFrame: 10 years
measure: Cardiac involvement
timeFrame: 10 years
measure: Pulmonary involvement
timeFrame: 10 years
measure: Renal involvement
timeFrame: 10 years
measure: Therapeutic interventions received
timeFrame: 10 years
measure: Progression to acute myeloid leukemia
timeFrame: 10 years
measure: Overall mortality
timeFrame: 10 years
Other outcomes:
Locations:
Study officials:
affiliation: Service de médecine interne, Hôpital Saint Antoine, APHP, Paris
name: Arsene MEKINIAN, MD PhD
role: PRINCIPAL_INVESTIGATOR
References: