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Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial.

作者信息

Tisdale John F, Pierciey Francis J, Bonner Melissa, Thompson Alexis A, Krishnamurti Lakshmanan, Mapara Markus Y, Kwiatkowski Janet L, Shestopalov Ilya, Ribeil Jean-Antoine, Huang Wenmei, Asmal Mohammed, Kanter Julie, Walters Mark C

机构信息

Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland, USA.

bluebird bio, Cambridge, Massachusetts, USA.

出版信息

Am J Hematol. 2020 Sep;95(9):E239-E242. doi: 10.1002/ajh.25867. Epub 2020 Jun 3.

DOI:10.1002/ajh.25867
PMID:32401372
Abstract
摘要

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1
Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial.在多中心HGB - 206试验中，对于镰状细胞病患者，通过普乐沙福动员后进行单采术采集造血祖干细胞与骨髓采集相比的安全性和可行性。
Am J Hematol. 2020 Sep;95(9):E239-E242. doi: 10.1002/ajh.25867. Epub 2020 Jun 3.
2
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.单用培洛昔康成功动员和采集镰状细胞病患者的造血干细胞。
Blood Adv. 2018 Oct 9;2(19):2505-2512. doi: 10.1182/bloodadvances.2018016725.
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Effect of voxelotor on murine bone marrow and peripheral blood with hematopoietic progenitor cell mobilization for gene therapy of sickle cell disease.羟基脲对镰状细胞病基因治疗中用于造血祖细胞动员的小鼠骨髓和外周血的影响。
Blood Cells Mol Dis. 2024 Mar;105:102824. doi: 10.1016/j.bcmd.2024.102824. Epub 2024 Jan 19.
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Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.普乐沙福可使镰状细胞病患者在输血交换后安全、快速、有效地动员造血干细胞。
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Evidence for continued dose escalation of plerixafor for hematopoietic progenitor cell collections in sickle cell disease.在镰状细胞病中，继续增加培利昔洛韦剂量以采集造血祖细胞的证据。
Blood Cells Mol Dis. 2021 Sep;90:102588. doi: 10.1016/j.bcmd.2021.102588. Epub 2021 Jun 15.
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Safety and efficacy of plerixafor dose escalation for the mobilization of CD34 hematopoietic progenitor cells in patients with sickle cell disease: interim results.培瑞克昔福剂量递增用于镰状细胞病患者造血祖细胞动员的安全性和有效性：中期结果。
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A Pilot, Exploratory, Randomized, Phase II Safety Study Evaluating Tumor Cell Mobilization and Apheresis Product Contamination in Patients Treated with Granulocyte Colony-Stimulating Factor Alone or Plus Plerixafor.一项评估单用粒细胞集落刺激因子或加用普乐沙福治疗的患者肿瘤细胞动员和单采产品污染的探索性、随机、Ⅱ期安全性研究
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No evidence for cell activation or brain vaso-occlusion with plerixafor mobilization in sickle cell mice.在镰状细胞小鼠中，没有证据表明普乐沙福动员会导致细胞活化或脑血管闭塞。
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Evaluation of mobilization efficacy with an extended interval following plerixafor administration.普乐沙福给药后延长间隔时间对动员效果的评估。
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引用本文的文献

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Gene Therapies for Sickle Cell Disease.镰状细胞病的基因疗法
J Pharm Technol. 2024 Oct;40(5):236-247. doi: 10.1177/87551225241268742. Epub 2024 Aug 15.
2
Cxcl10 and Cxcr3 regulate self-renewal and differentiation of hematopoietic stem cells.趋化因子Cxcl10和趋化因子受体Cxcr3调节造血干细胞的自我更新和分化。
Stem Cell Res Ther. 2024 Aug 7;15(1):248. doi: 10.1186/s13287-024-03861-7.
3
Hematopoietic stem cell collection for sickle cell disease gene therapy.镰状细胞病基因治疗的造血干细胞采集
Curr Opin Hematol. 2024 May 1;31(3):104-114. doi: 10.1097/MOH.0000000000000807. Epub 2024 Feb 9.
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Effect of voxelotor on murine bone marrow and peripheral blood with hematopoietic progenitor cell mobilization for gene therapy of sickle cell disease.羟基脲对镰状细胞病基因治疗中用于造血祖细胞动员的小鼠骨髓和外周血的影响。
Blood Cells Mol Dis. 2024 Mar;105:102824. doi: 10.1016/j.bcmd.2024.102824. Epub 2024 Jan 19.
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Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.制定改善镰状细胞病结局的全球战略：柳叶刀血液学委员会。
Lancet Haematol. 2023 Aug;10(8):e633-e686. doi: 10.1016/S2352-3026(23)00096-0. Epub 2023 Jul 11.
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Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.动员后和骨髓来源的人类造血干细胞在基因治疗后的造血重建动力学。
Nat Commun. 2023 May 27;14(1):3068. doi: 10.1038/s41467-023-38448-y.
7
Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.同种异体移植与基因治疗：走向治愈之路。
Hematol Oncol Clin North Am. 2022 Dec;36(6):1313-1335. doi: 10.1016/j.hoc.2022.06.007.
8
Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.洛沃细胞基因治疗镰状细胞病：HGB-206 研究初始组的治疗过程演变和结果。
Am J Hematol. 2023 Jan;98(1):11-22. doi: 10.1002/ajh.26741. Epub 2022 Oct 10.
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Peripheral blood stem and progenitor cell collection in pediatric candidates for gene therapy: a 10-year series.基因治疗儿科候选患者的外周血干细胞和祖细胞采集：一项为期10年的研究系列
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