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

Conditions

Cerebrovascular Accident, Sickle Cell Anemia

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

Françoise Bernaudin, Suzanne Verlhac, Elisabeth Ducros-Miralles, Cécile Arnaud, I. Genty et al. (31 authors)

Blood2024-11-25

10.1182/blood-2024-212967

Stenosis Outcome at 1 and 3 Years after Transplantation Vs Standard-Care in Children with Sickle-Cell Anemia and Abnormal Transcranial Doppler with Stroke or No-Stroke History

Suzanne Verlhac, Catherine Paillard, Flaviu Gabor, Charlotte Jubert, Jean‐François Chateil et al. (24 authors)

Blood2019-11-13

10.1182/blood-2019-122699

Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia

Françoise Bernaudin, Suzanne Verlhac, Régis Peffault de Latour, Jean‐Hugues Dalle, Valentine Brousse et al. (29 authors)

JAMA2019-01-2274 citations

10.1001/jama.2018.20059

Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404).

Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarré C, Thuret I, Guitton C, Lesprit E, Etienne-Julan M, Elana G, Vannier JP, Lutz P, Neven B, Galambrun C, Paillard C, Runel C, Jubert C, Arnaud C, Kamdem A, Brousse V, Missud F, Petras M, Doumdo-Divialle L, Berger C, Fréard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socié G, Bernaudin F.

2017-08-1510 citations

10.1016/j.cct.2017.08.008

Interventions

PROCEDUREStem cell transplantation

Stem cell transplantation

PROCEDURETransfusion program

Transfusion program

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 15 Years

Healthy volunteers

No

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

Design outcomes

Primary

Measure	Time frame	Description
Cerebral vasculopathy	1 year	velocity in the artery with highest velocity

Secondary

Measure	Time frame	Description
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)

Other

Measure	Time frame	Description
cognitive dysfunction	12 months	the scale of cognitive dyfunction depends on the child's age : definition of dysfunction uses the WISC-4, WPPSI-4 or WAIS-3 according to age

Countries

France

Outcome results

None listed