Work

Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor

Year: 2020

Type: article

Abstract: Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixaforHematopoietic stem cell (HSC) gene therapy is potentially curative for sickle cell disease (SCD); 1 however, options for HSC collection are limited in this population, [2][3][4] and investigation of the collection, efficiency, and safety of peripheral blood (PB) mobilization with plerixafor from start to finish is needed.Here we describe consistent, safe, and sufficient PB HSC collection and processing after plerixafor mobilization from the greatest number of participants reported to date and the first twoinstitutional study.Our data suggest plerixafor mobilized HSC in SCD are enriched for long-term engrafting HSC, which is not true of HSC from SCD bone marrow (BM), 5 supporting a paradigm shift in the optimal HSC source for patients with SCD.This open-label phase I study was sponsored by the National Heart, Lung and Blood Institute at the National Institutes of Health (NIH) and was conducted at the NIH Clinical Center and St. Jude Children's Research Hospital (SJCRH) (clinicaltrials.govidentifier: NCT03226691).All participants provided written informed consent for a protocol approved by each institution's Institutional Review Board.Hydroxyurea (HU) was stopped at least two weeks prior to mobilization, and all participants received red blood cell exchange the day prior to mobilization and collection to target <30% sickle hemoglobin (HbS).Participants received a single subcutaneous dose (240 mg/kg) of plerixafor (Mozobil TM , Sanofi, Bridgewater, NJ, USA) 4 hours (h) before leukapheresis.Participants in the NIH cohort also received 325 mg aspirin.If the minimum target CD34 + cell dose of 1.5x10 6 cells/kg (goal tar-

Source: Haematologica

DOI: 10.3324/haematol.2019.236182