Improved stenosis outcome in stroke-free sickle cell anemia children after transplantation compared to chronic transfusion.

British Journal of Haematology
Suzanne Verlhacon behaf of the Drepagreffe Investigators

Abstract

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 = 0·006). No patient developed stenosis after SCT, while two on standard-care did, indicating better stenosis prevention and improved outcome after SCT.

References

Dec 31, 2005·The New England Journal of Medicine·Robert J AdamsUNKNOWN Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators
Dec 9, 2008·Haematologica·Brigitte Bader-MeunierMalika Benkerrou
Jun 9, 2009·British Journal of Haematology·Janet L KwiatkowskiKwaku Ohene-Frempong
Feb 19, 2013·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Mari H DallasWing Leung
May 7, 2014·Advances in Medical Sciences·Michal ArkuszewskiElias R Melhem
Mar 13, 2016·Pediatric Neurology·Michael J RivkinCatherine Amlie-Lefond
Jan 17, 2017·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Nancy S GreenAdam M Brickman
Jan 23, 2019·JAMA : the Journal of the American Medical Association·Françoise BernaudinUNKNOWN DREPAGREFFE Trial Investigators

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