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

Assistance Publique - Hôpitaux de Paris63 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Anemia Cerebrovascular Accident

Interventions

Stem cell transplantationPROCEDURE

Stem cell transplantation

Transfusion programPROCEDURE

Transfusion program

Eligibility

Sex: ALLMax age: 15 Years

Medical Language ↔ Plain English

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

Locations (1)

CHIC

Créteil, France

Outcomes

Primary Outcomes

Cerebral vasculopathy

velocity in the artery with highest velocity

Time frame: 1 year

Secondary Outcomes

ischemic lesions on magnetic resonance imaging (MRI)

percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)

Time frame: 1 year

stenoses on magnetic resonance imaging (MRI)

percentage of patients with stenoses on magnetic resonance imaging (MRI)

Time frame: 1 year

normalisation of arterial velocities

percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(\< 170 cm/sec)

Time frame: 12 months

Data from ClinicalTrials.gov

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