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Beta-thalassemia and Microparticles

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ClinicalTrials.gov Identifier: NCT01284738
Recruitment Status : Completed
First Posted : January 27, 2011
Last Update Posted : August 29, 2014
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

Study Description
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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

Condition or disease Intervention/treatment Phase
Thalassemia Major (TM) Thalassemia Intermedia (TI) Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes Other: Physiopathology Not Applicable

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Official Title: Beta-thalassemia and Microparticles
Study Start Date : March 2010
Actual Primary Completion Date : February 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
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 :

  • 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.
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 :

  • 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.


Outcome Measures
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Primary Outcome Measures :
  1. Relationship between TM and TI [ Time Frame: 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 :
  1. Investigate the mechanisms of the elevated production of MP in thalassemias [ Time Frame: 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.


Eligibility Criteria
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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01284738


Locations
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France
APHM
Marseille, France, 13
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
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Study Director: Isabelle Thuret, Doctor APHM
More Information
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Responsible Party: Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier: NCT01284738    
Other Study ID Numbers: 2010-A00198-31
2009-18
First Posted: January 27, 2011    Key Record Dates
Last Update Posted: August 29, 2014
Last Verified: August 2014
Keywords provided by Assistance Publique Hopitaux De Marseille:
TM
TI
MP
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn