Beta-thalassemia and Microparticles

NCT01284738 | Completed

• 2 other identifiers: 2010-A00198-312009-18
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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

Conditions

Thalassemia Major (TM); Thalassemia Intermedia (TI); Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes

Keywords

TM; TI; MP

Outcome Measures

Primary Outcomes (1)

• Relationship between TM and TI

  * 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.

  36 months

Secondary Outcomes (1)

• Investigate the mechanisms of the elevated production of MP in thalassemias

  36 months

Study Arms (2)

TM patients

ACTIVE COMPARATOR

thalassemia major (TM) Need transfusion for survive

Other: Physiopathology

TI patients

ACTIVE COMPARATOR

thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM

Other: Physiopathology

Interventions

Physiopathology OTHER

Three sequential biological evaluations will be performed for each patient and will consist in : * the dosages of MP carried out by the UMR 608 in Marseille, * the evaluation of the oxidative stress markers and of iron performed in the UMR 773 in Paris-Bichat. In vitro production of MP of transfused red blood cells origin will also be evaluated in erythrocytes concentrates during the storage of the units.

TM patients

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Assistance Publique Hopitaux De Marseille: lead

Study Sites (1)

APHM

Marseille, 13, France

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Isabelle Thuret, Doctor

  APHM

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2011
• First Posted: January 27, 2011
• Study Start: March 1, 2010
• Primary Completion: February 1, 2014
• Last Updated: August 29, 2014

Record last verified: 2014-08

Locations

FR (1)