Idiopathic Chronic Thrombocytopenia of Undetermined Significance : Pathogenesis and Biomarker

NCT04003220 | Unknown

• 1 other identifier: CHUBX 2018/47
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 4

Brief Summary

It is a translational research study with mechanistical objectives and including biological samples of patients with thrombocytopenia

Conditions

Thrombocytopenia; Purpura, Thrombocytopenic; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Measure of clonality in patients with ICTUS

  identification of mutations in genes known to be involved in clonal expansion

  Day 1

Study Arms (4)

MyeloDysplastic Syndrome

platelets \<150 G/l and diagnosis of myelodysplasia according to the WHO classification (abnormal bone marrow features).

Procedure: blood sample

Idiopathic Chronic Thrombocytopenia of Unknown Significance

Acquired thrombocytopenia lasting\>6 months, without feature of dysimmunity (antiplatelet Ab, or anti nuclear Ab, anti ENA Ab, ANCA, anti-CCP Ab, cryoglobulinemia), normal bone marrow examination, absence of other thrombocytopenia in the family. All ICTUS patients included in the study will thus have a bone marrow aspiration.

Procedure: blood sample; Procedure: Bone marrow sample

Immune Thrombocytopenic Purpura

Diagnosis of Immune Thrombocytopenic Purpura (ITP) is made according to the international ITP consensus (diagnostic criteria of Rodeghiero){Provan, 2010 #18}, knowing that a presumptive diagnosis of ITP is made when the history, physical examination, complete blood count, and examination of the peripheral blood smears do not suggest other etiologies for the thrombocytopenia

Procedure: blood sample

healthy volunteers undergoing cardiac surgery

patients with normal blood counts undergoing cardiovascular surgery for valve replacement, or healthy bone-marrow donors

Procedure: Bone marrow sample

Interventions

blood sample PROCEDURE

The extra samples will be taken Blood sampling

Idiopathic Chronic Thrombocytopenia of Unknown Significance; Immune Thrombocytopenic Purpura; MyeloDysplastic Syndrome

Bone marrow sample PROCEDURE

This bone marrow sampling will be performed for 10 patients with ICTUS, and 10 controls

Idiopathic Chronic Thrombocytopenia of Unknown Significance; healthy volunteers undergoing cardiac surgery

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

In this project we will study 30 patients with ICTUS, 20 ITP with immunological features, 20 MDS patients and 10 Healthy volunteers (normal bone marrow donors, obtained during a sternotomy performed in the cardiac surgery unit).

You may qualify if:

• Age: 18-80
• Nosological criteria for thrombocytopenic patients:
• ICTUS: acquired thrombocytopenia lasting\>6 months, without feature of dysimmunity (antiplatelet Ab, or anti nuclear Ab, anti ENA Ab, ANCA, anti-CCP Ab, cryoglobulinemia), normal bone marrow examination, absence of other thrombocytopenia in the family. All ICTUS patients included in the study will thus have a bone marrow aspiration.
• ITP: Diagnosis of ITP is made according to the international ITP consensus (diagnostic criteria of Rodeghiero){Provan, 2010 #18}, knowing that a presumptive diagnosis of ITP is made when the history, physical examination, complete blood count, and examination of the peripheral blood smears do not suggest other etiologies for the thrombocytopenia. There is no "gold standard" test that can reliably establish the diagnosis. We are concerned that this group of patients may include ICTUS patients.
• ITP with immunological features: ITP with detectable anti-platelet antibodies (Ab) and/or autoimmunity (anti nuclear Ab, anti ENA Ab, ANCA, anti-CCP Ab, cryoglobulinemia), and/or previously responsive to immunomodulatory ITP-directed therapies (steroids, intravenous Immunoglobulin, rituximab, splenectomy). We will use these patients as non-ICTUS, ITP positive controls.
• Thrombocytopenic myelodysplasia: platelets \<150 G/l and diagnosis of myelodysplasia according to the WHO classification (abnormal bone marrow features).
• Healthy controls: patients with normal blood counts undergoing cardiovascular surgery for valve replacement, or healthy bone-marrow donors.

You may not qualify if:

• Age under 18 or over 80
• Persons placed under judicial protection,
• Persons in emergency situation,
• Pregnant or nursing women,
• Women of childbearing age who do not benefit from effective contraception (HAS criteria),
• Brain death or deceased persons,
• Person who doesn't speak French, doesn't read it, doesn't write it, and doesn't have a schooling in France.

Sponsors & Collaborators

• University Hospital, Bordeaux: lead
• Institut National de la Santé Et de la Recherche Médicale, France: collaborator

Study Sites (4)

Immunopathologie Clinique - Hôpital Saint Louis - AP HP

Paris, 75010, France

RECRUITING

Chirurgie cardiaque - Hôpital Haut Lévêque - CHU de Bordeaux

Pessac, 33600, France

RECRUITING

Service des maladies du sang - Hôpital Haut-Lévêque - avenue de Magellan

Pessac, 33600, France

RECRUITING

service de médecine interne et maladies infectieuses - Groupe hospitalier Sud

Pessac, France

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

the samples for research will be taken in the department where the patient is: * Blood sampling: 5 ACD-A tubes (Acid Citrate Dextrose) and 2 EDTA tubes (for 30 ICTUS patients, 20 ITP patients, and 20 patients with MDS) * One supplementary tube of bone marrow (for 10 patients with ICTUS, and 10 Healthy controls) * 25 Hair bulbs (for 20 ICTUS patients)

MeSH Terms

Conditions

Thrombocytopenia; Purpura, Thrombocytopenic; Myelodysplastic Syndromes

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• JAMES, MD, PhD

  University Hospital, Bordeaux

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chloé JAMES, MD,PhD

CONTACT

+335 57 65 64 78; chloe.james@chu-bordeaux.fr

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2019
• First Posted: July 1, 2019
• Study Start: September 7, 2020
• Primary Completion: September 7, 2023
• Study Completion: September 7, 2023
• Last Updated: November 28, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4)