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

Not Applicable

Completed

• Conditions: Sickle Cell Anemia; Cerebrovascular Accident
• Interventions: Procedure: Stem cell transplantation; Procedure: Transfusion program

• Registration Number: NCT01340404
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

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

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

Study Timeline

• Study Start: 2010-12
• Study First Submitted: 2011-04-21
• Study First Submitted QC: 2011-04-21
• Study First Posted: 2011-04-22
• Primary Completion: 2013-04
• Study Completion: 2015-10
• Status Verified: 2016-11
• Last Update Submitted: 2016-12-02
• Last Update Posted: 2016-12-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 63

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Stem Cell Transplantation	Stem cell transplantation	-
Transfusion program	Transfusion program	-

Primary Outcome Measures

Name	Time	Method
Cerebral vasculopathy	1 year	velocity in the artery with highest velocity

Secondary Outcome Measures

Name	Time	Method
ischemic lesions on magnetic resonance imaging (MRI)	1 year	percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)
stenoses on magnetic resonance imaging (MRI)	1 year	percentage of patients with stenoses on magnetic resonance imaging (MRI)
normalisation of arterial velocities	12 months	percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(\< 170 cm/sec)

Trial Locations

Locations (1)

CHIC; 🇫🇷 Créteil, France