Beta-thalassemia and Microparticles
Recruitment status was Recruiting
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
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
| Condition | Intervention |
|---|---|
|
Thalassemia Major (TM) Thalassemia Intermedia (TI) Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes |
Other: Physiopathology |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: Open Label |
| Official Title: | Beta-thalassemia and Microparticles |
- Relationship between TM and TI [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- 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.
- Investigate the mechanisms of the elevated production of MP in thalassemias [ Time Frame: 36 months ] [ Designated as safety issue: No ]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.
| Estimated Enrollment: | 55 |
| Study Start Date: | March 2010 |
| Estimated Primary Completion Date: | March 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Active Comparator: TM patients
thalassemia major (TM) Need transfusion for survive
|
Other: Physiopathology
Three sequential biological evaluations will be performed for each patient and will consist in :
In vitro production of MP of transfused red blood cells origin will also be evaluated in erythrocytes concentrates during the storage of the units. |
|
Active Comparator: TI patients
thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM
|
Other: Physiopathology
Three sequential biological evaluations will be performed for each patient and will consist in :
|
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.
Eligibility| Ages Eligible for Study: | 15 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| Contact: Isabelle Thuret, Doctor | +33491386370 | isabelle.thuret@ap-hm.fr |
| France | |
| APHM | Recruiting |
| Marseille, France, 13 | |
| Contact: Isabelle Thuret, Doctor +33491386370 isabelle.thuret@ap-hm.fr | |
| Study Director: | Isabelle Thuret, Doctor | APHM |
More Information
No publications provided
| Responsible Party: | APHM, Direction de la recherche |
| ClinicalTrials.gov Identifier: | NCT01284738 History of Changes |
| Other Study ID Numbers: | 2010-A00198-31, 2009-18 |
| Study First Received: | January 26, 2011 |
| Last Updated: | January 26, 2011 |
| Health Authority: | France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) |
Keywords provided by Assistance Publique Hopitaux De Marseille:
|
TM TI MP |
Additional relevant MeSH terms:
|
Beta-Thalassemia Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 23, 2013