期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 193, 期 1, 页码 188-193出版社
WILEY
DOI: 10.1111/bjh.17178
关键词
sickle cell anaemia; cerebral arterial stenosis; cerebral magnetic resonance imaging; magnetic resonance angiography; haematopoietic stem cell transplantation; transfusions
类别
资金
- Assistance Publique-Hopitaux de Paris from the French Ministry of Health
- Cordons de Vie
- BlueBirdBio
In stroke-free children with sickle cell anaemia, stem cell transplantation significantly improved stenosis outcomes over 3 years compared to standard-care, indicating better prevention and improved outcome.
We report here the 3-year stenosis outcome in 60 stroke-free children with sickle cell anaemia (SCA) and an abnormal transcranial Doppler history, enrolled in the DREPAGREFFE trial, which compared stem cell transplantation (SCT) with standard-care (chronic transfusion for 1-year minimum). Twenty-eight patients with matched sibling donors were transplanted, while 32 remained on standard-care. Stenosis scores were calculated after performing cerebral/cervical 3D time-of-flight magnetic resonance angiography. Fourteen patients had stenosis at enrollment, but only five SCT versus 10 standard-care patients still had stenosis at 3 years. Stenosis scores remained stable on standard-care, but significantly improved after SCT (P = 0 center dot 006). No patient developed stenosis after SCT, while two on standard-care did, indicating better stenosis prevention and improved outcome after SCT.
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