Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy

Study Overview
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Brief Summary
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The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients.

Detailed Description
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The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD)are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients. Sixty-three patients will be enrolled, namely 21 sickle-cell patients in the SCT group and 42 in the Transfusion Program (TP) group

Official Title
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A Multicenter Study Comparing the Results of Allogeneic Stem Cell Genoidentical in Children With Sickle Cell Anemia and Cerebral Vascular Disease Detected by Transcranial Doppler

Conditions
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Sickle Cell Anemia, Cerebrovascular Accident

Intervention / Treatment
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* Procedure: Stem cell transplantation
* Procedure: Transfusion program


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Sickle cell anemia patients (SS/Sb0) < 15 years old History of abnormal transcranial doppler (TCD) (≥ 200 cm/sec) Siblings from the same parental couple Parents amenable to Human Leucocyte Antigen (HLA) typing, SCT if an HLA-identical sibling is available or to long-term transfusion program Exclusion Criteria: Sickle cell patients older than 15 years no history of abnormal transcranial doppler (TCD) (≥ 200 cm/sec) no sibling from the same parental couple parents averse to HLA typing, SCT if an HLA-identical sibling is available or to long-term transfusion program

Ages Eligible for Study
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Maximum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stem Cell Transplantation<br> | Procedure: Stem cell transplantation<br>* Stem cell transplantation<br>|
| Active Comparator: Transfusion program<br> | Procedure: Transfusion program<br>* Transfusion program<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cerebral vasculopathy | velocity in the artery with highest velocity | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ischemic lesions on magnetic resonance imaging (MRI) | percentage of patients with ischemic lesions on magnetic resonance imaging (MRI) | 1 year | 
| stenoses on magnetic resonance imaging (MRI) | percentage of patients with stenoses on magnetic resonance imaging (MRI) | 1 year | 
| normalisation of arterial velocities | percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec) | 12 months |