Beta-thalassemia and Microparticles

Not Applicable

Completed

• Conditions: Thalassemia Major (TM); Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes; Thalassemia Intermedia (TI)
• Interventions: Other: Physiopathology

• Registration Number: NCT01284738
• Lead Sponsor: Assistance Publique Hopitaux De Marseille

Brief Summary

The results will allow us to evaluate the role of MP in the thrombo-embolic risk observed in thalassemic patients and to underline a possible difference between TM and TI. The in vitro and in vivo study of MP in erythrocytes concentrates is a new approach to explore the consequence of transfusion in polytransfused patients. Finally, the identification of a possible relationship between the oxidative stress and the production of MP may lead to the development of specific therapeutical approaches

Detailed Description

Microparticles (MP) are intact vesicles derived from cell membranes which arise mainly through cell membrane activation processes and from apoptosis. MP originating from platelets, endothelial cells and monocytes have been most extensively studied, though similar particles can arise from red cells and granulocytes. The ability to form microparticles is an essential part of physiological coagulation.However, MP may play an important procoagulant role in several diseases including sickle cell disease, and paroxysmal nocturnal haemoglobinuria (PNH).

Several studies reported the presence of MP in TI and their potential role in the hypercoagulable state. The investigators propose in this study to investigate the presence and origin of MP in TM patients.

Study Timeline

• Study Start: 2010-03
• Study First Submitted: 2011-01-26
• Study First Submitted QC: 2011-01-26
• Study First Posted: 2011-01-27
• Primary Completion: 2014-02
• Status Verified: 2014-08
• Last Update Submitted: 2014-08-28
• Last Update Posted: 2014-08-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

• Patient recorded in the national register of the patients attained by beta-thalassemia (TI) or (TM)
• Patient monitoring in one of 5 recruiters centers
• Patient more than 15 years
• Patient consented and informed

Exclusion Criteria

• Blood transfusion dating from less than 3 months for TI
• Composite Heterozygotes HbE /beta-thalassemia
• pregnant women
• other disease

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
TM patients	Physiopathology	thalassemia major (TM) Need transfusion for survive
TI patients	Physiopathology	thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM

Primary Outcome Measures

Name	Time	Method
Relationship between TM and TI	36 months	* In TM, to quantify the elevation of MP as well as their procoagulant activity, to describe their production kinetic, to determine the transfusional or endogenous origin of erythrocytic MP and finally to compare their characteristics with those found in TI patients.; * To study, in TM and TI patients, the relationship between the number, the procoagulant activity of MP and the clinical (thromboembolic episodes,splenectomy, presence of pulmonary hypertension) biological and plasmatic data reflecting the patient's prothrombotic state.

Secondary Outcome Measures

Name	Time	Method
Investigate the mechanisms of the elevated production of MP in thalassemias	36 months	Studying the correlation between the number, the activity of erythrocytes and platelets derived-MP and the hemolysis, the dyserythropoiesis, the oxidative stress and iron overload markers.

Trial Locations

Locations (1)

APHM; 🇫🇷 Marseille, France