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

Completed

Phase N/A Results N/A

Trial 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.

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

Conditions

Interventions

  • Stem cell transplantation Procedure
    Intervention Desc: Stem cell transplantation
    ARM 1: Kind: Experimental
    Label: Stem Cell Transplantation
  • Transfusion program Procedure
    Intervention Desc: Transfusion program
    ARM 1: Kind: Experimental
    Label: Transfusion program

Trial Design

  • Allocation: Non-Randomized
  • Masking: Open Label
  • Purpose: Treatment
  • Endpoint: Efficacy Study
  • Intervention: Parallel Assignment

Outcomes

Type Measure Time Frame Safety Issue
Primary Cerebral vasculopathy 1 year No
Secondary ischemic lesions on magnetic resonance imaging (MRI) 1 year No
Secondary stenoses on magnetic resonance imaging (MRI) 1 year No

Sponsors