Idiopathic Chronic Thrombocytopenia of Undetermined Significance : Pathogenesis and Biomarker (ICTUS)

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

Study Overview

• Status: Recruiting
• Conditions: Myelodysplastic Syndromes; Purpura, Thrombocytopenic; Thrombocytopenia
• Intervention / Treatment: Procedure: blood sample; Procedure: Bone marrow sample

Detailed Description

This study is a prospective study to collect blood, bone marrow and hair bulb from patients with thrombocytopenia, to better understand the mechanism of idiopathic chronic thrombocytopenia of undetermined significance (ICTUS)

• Study Type: Observational
• Enrollment (Anticipated): 80

Contacts and Locations

• Study Contact: Name: Chloé JAMES, MD,PhD; Phone Number: +335 57 65 64 78; Email: chloe.james@chu-bordeaux.fr

Study Locations

• France
  • Paris, France, 75010
    • Recruiting
    • Immunopathologie Clinique - Hôpital Saint Louis - AP HP
    • Contact: Lionel GALICIER, MD; Phone Number: +331.42.49.96.90; Email: lionel.galicier@aphp.fr
    • Principal Investigator: Lionel GALICIER, MD
  • Pessac, France, 33600
    • Recruiting
    • Chirurgie cardiaque - Hôpital Haut Lévêque - CHU de Bordeaux
    • Contact: Mathieu PERNOT, MD; Phone Number: +335.57.65.69.69; Email: mathieu.pernot@chu-bordeaux.fr
    • Principal Investigator: Mathieu PERNOT, MD
  • Pessac, France, 33600
    • Recruiting
    • Service des maladies du sang - Hôpital Haut-Lévêque - avenue de magellan
    • Contact: Noel MILPIED; Email: noel.milpied@chu-bordeaux.fr
    • Sub-Investigator: Krimo BOUABDALLAH
    • Sub-Investigator: Marie-Sarah DILHUYDY
    • Principal Investigator: Noel MILPIED
  • Pessac, France
    • Recruiting
    • service de médecine interne et maladies infectieuses - Groupe hospitalier Sud

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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).

Description

Inclusion Criteria:

• 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.
• Affiliated person or beneficiary of a social security scheme. Free, informed and written consent signed by the participant and the investigator (at the latest on the day of inclusion and before any examination required by the research).

Exclusion Criteria:

• Age under 18 or over 80
• Persons placed under judicial protection,
• Persons participating in another research including a period of exclusion still in course
• 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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Control
• Time Perspectives: Prospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
MyeloDysplastic Syndrome; platelets <150 G/l and diagnosis of myelodysplasia according to the WHO classification (abnormal bone marrow features).	Procedure: blood sample; The extra samples will be taken Blood sampling
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; The extra samples will be taken Blood sampling; Procedure: Bone marrow sample; This bone marrow sampling will be performed for 10 patients with ICTUS, and 10 controls
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; The extra samples will be taken Blood sampling
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; This bone marrow sampling will be performed for 10 patients with ICTUS, and 10 controls

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measure of clonality in patients with ICTUS	identification of mutations in genes known to be involved in clonal expansion	Day 1

Collaborators and Investigators

• Sponsor: University Hospital, Bordeaux
• Collaborators: Institut National de la Santé Et de la Recherche Médicale, France
• Investigators: Principal Investigator: JAMES, MD, PhD, University Hospital, Bordeaux

Study record dates

Study Major Dates

• Study Start (Actual): September 7, 2020
• Primary Completion (Anticipated): September 7, 2023
• Study Completion (Anticipated): September 7, 2023

Study Registration Dates

• First Submitted: June 26, 2019
• First Submitted That Met QC Criteria: June 28, 2019
• First Posted (Actual): July 1, 2019

Study Record Updates

• Last Update Posted (Actual): November 28, 2022
• Last Update Submitted That Met QC Criteria: November 25, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Bone Marrow Diseases; Hematologic Diseases; Hemorrhage; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Myelodysplastic Syndromes; Purpura; Purpura, Thrombocytopenic; Thrombocytopenia
• Other Study ID Numbers: CHUBX 2018/47

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

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