Clinical trial NCT02855957: Evaluation of the Free α-hemoglobin Pool in the Red Blood Cells : Prognostic Marker and Severity Index in Thalassemic Syndromes
Official title: Evaluation of the Free α-hemoglobin Pool in the Red Blood Cells : Prognostic Marker and Severity Index in Thalassemic Syndromes
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Thalassemic Syndromes
Keywords: Alpha-Hemoglobin Stabilizing Protein
alpha-Hb pool
thalassemias
diagnostic method
biomarker
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
Collaborators: name: Institut National de la Santé Et de la Recherche Médicale, France
name: Paris 12 Val de Marne University
First posted: 2016-08-04
Enrollment: 135
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: Our project aims to assess and validate an innovative prognostic and diagnostic test in order to i) define a severity index of the β-thalassemia according to the free α-Hb pool for a better management of the patient; ii) differentiate the α-thalassemia and β-thalassemia and iii) follow the evolution of this pool in response to treatment of patients. In the future, this prognostic test based on the measurement of free α-Hb pool may be suitable for routine laboratory practice. To carry out this project, cohort of 85 thalassemic patients and a group of 50 healthy volunteers have been recruited.


Eligibility criteria: Inclusion Criteria Patient

* Age ≥ 18 years
* Written informed consent
* Affiliation to social security
* Known Hb Phenotypes
* Biological phenotype corresponding to a classical picture of thalassemia

Healthy volunteers

* Age ≥ 18 years
* Written informed consent
* Affiliation to social security

Exclusion Criteria Patient

* Transfusion for less than 3 months
* Chronic active viral disease: hepatitis B, C, HIV
* Ongoing infections or known inflammatory diseases
* Hyper or Hypothyroidism known or subject treated by Levothyrox
* Active pathology or tumor remission for less than 5 years
* Oral corticosteroid
* Hemoglobinopathy other than thalassemia for patients
* Treatment by Hydroxyurea for more than 3 months
* Treatment by stimulating agent erythropoiesis for longer than 3 months

Healthy volunteers

* Transfusion for less than 3 months
* Chronic active viral disease: hepatitis B, C, HIV
* Ongoing infections or known inflammatory diseases
* Hyper or Hypothyroidism known or subject treated by Levothyrox
* Active pathology or tumor remission for less than 5 years
* Oral corticosteroid
* Genetically related to person with hemoglobinopathy

Primary outcomes:
measure: Correlation between the pool of free α-Hb and genotype α and β -globin of the patient and if necessary with the genotype AHSP gene
timeFrame: Day 1
Secondary outcomes:
measure: Correlation between the pool of free α-Hb and Hb value
timeFrame: Day 1
measure: Correlation between the pool of free α-Hb and Mean Corpuscular Hb
timeFrame: Day 1
measure: Correlation between the pool of free α-Hb and Mean Cell Volume
timeFrame: Day 1
measure: Correlation between the pool of free α-Hb and percent of reticulocytes
timeFrame: Day 1
measure: Correlation between the pool of free α-Hb and fetal Hb
timeFrame: Day 1
measure: Correlation between the pool of free α-Hb and erythrocyte enzymes
timeFrame: Day 1
Other outcomes:
Locations:
city: Creteil
country: France
facility: Henri Mondor Hospital
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Frédéric Galacteros, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Vasseur C, Pissard S, Domingues-Hamdi E, Marden MC, Galacteros F, Baudin-Creuza V. Evaluation of the free alpha-hemoglobin pool in red blood cells: a new test providing a scale of beta-thalassemia severity. Am J Hematol. 2011 Feb;86(2):199-202. doi: 10.1002/ajh.21918.