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

Assistance Publique - Hôpitaux de Paris

Status

Completed

Conditions

Cerebrovascular Accident

Sickle Cell Anemia

Treatments

Procedure: Stem cell transplantation

Procedure: Transfusion program

Study type

Interventional

Funder types

Other

Identifiers

NCT01340404

P071247

Details and patient eligibility

About

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.

Full 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

Enrollment

63 patients

Sex

All

Ages

Under 15 years old

Volunteers

No Healthy Volunteers

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