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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stem Cell Transplantation | Experimental |
| |
| Transfusion program | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Stem cell transplantation | Procedure | Stem cell transplantation |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Cerebral vasculopathy | velocity in the artery with highest velocity | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| ischemic lesions on magnetic resonance imaging (MRI) | percentage of patients with ischemic lesions on magnetic resonance imaging (MRI) | 1 year |
| stenoses on magnetic resonance imaging (MRI) | percentage of patients with stenoses on magnetic resonance imaging (MRI) |
| Measure | Description | Time Frame |
|---|---|---|
| cognitive dysfunction | 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 | 12 months |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Françoise Bernaudin, MD | Assistance Publique - Hôpitaux de Paris | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHIC | Créteil | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30667500 | Derived | Bernaudin F, Verlhac S, Peffault de Latour R, Dalle JH, Brousse V, Petras E, Thuret I, Paillard C, Neven B, Galambrun C, Divialle-Doumdo L, Pondarre C, Guitton C, Missud F, Runel C, Jubert C, Elana G, Ducros-Miralles E, Drain E, Taieb O, Arnaud C, Kamdem A, Malric A, Elmaleh-Berges M, Vasile M, Leveille E, Socie G, Chevret S; DREPAGREFFE Trial Investigators. Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia. JAMA. 2019 Jan 22;321(3):266-276. doi: 10.1001/jama.2018.20059. | |
| 28821470 |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D020521 | Stroke |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D033581 | Stem Cell Transplantation |
| ID | Term |
|---|---|
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
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| Transfusion program |
| Procedure |
Transfusion program |
|
| 1 year |
| normalisation of arterial velocities | percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(< 170 cm/sec) | 12 months |
| Derived |
| Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarre 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, Freard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socie G, Bernaudin F. 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). Contemp Clin Trials. 2017 Nov;62:91-104. doi: 10.1016/j.cct.2017.08.008. Epub 2017 Aug 15. |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D002561 | Cerebrovascular Disorders |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D014180 |
| Transplantation |
| D013514 | Surgical Procedures, Operative |