Sheppard D, Bredeson C, Huebsch L, Allan D, Tay J
Blood and Marrow Transplant Program, Division of Hematology, Department of Medicine, The Ottawa Hospital, The Ottawa Hospital Research Institute, The University of Ottawa, Ottawa, Canada.
Bone Marrow Transplant. 2014 Jun;49(6):751-5. doi: 10.1038/bmt.2014.33. Epub 2014 Mar 10.
Plerixafor effectively mobilizes hematopoietic stem cells (HSCs). However, most patients' cells are successfully collected using traditional strategies and there is limited cost-effectiveness data. The objectives of this study were to: (1) summarize the published reports of mobilization using a plerixafor-based strategy during compassionate access programs and (2) describe the Canadian experience with plerixafor during its availability by Health Canada's Special Access Program. A literature search identified reports of plerixafor-based mobilization during compassionate access programs. Overall, successful collection of at least 2 × 10(6) CD34+ cells/kg was achieved in ~75% of patients, and about two-thirds of patients went on to HSCT. A greater proportion of patients had successful collections when plerixafor was used in the upfront or preemptive settings. Plerixafor was made available by Health Canada's SAP from September 2008 to December 2010. In 96 of 132 (73%) patients, there was successful collection of at least 2 × 10(6) CD34+ cells/kg. Ninety-nine (75%) patients went on to receive an autologous transplant. Plerixafor-based mobilization is effective in perceived poor mobilizers. The optimal way to incorporate plerixafor into a mobilization strategy, however, remains to be determined. Centre-specific analysis of resource utilization may help to identify the most cost-effective way to implement various plerixafor-based mobilization strategies.
普乐沙福能有效动员造血干细胞(HSCs)。然而,大多数患者的细胞是通过传统策略成功采集的,且成本效益数据有限。本研究的目的是:(1)总结在同情用药项目期间使用基于普乐沙福的策略进行动员的已发表报告,以及(2)描述加拿大在加拿大卫生部特殊准入计划下可获得普乐沙福期间的使用经验。文献检索确定了同情用药项目期间基于普乐沙福进行动员的报告。总体而言,约75%的患者成功采集到至少2×10⁶个CD34⁺细胞/kg,约三分之二的患者随后接受了造血干细胞移植(HSCT)。当普乐沙福用于初始或抢先设置时,成功采集的患者比例更高。加拿大卫生部的特殊准入计划在2008年9月至2010年12月期间提供了普乐沙福。在132例患者中的96例(73%)成功采集到至少2×10⁶个CD34⁺细胞/kg。99例(75%)患者随后接受了自体移植。基于普乐沙福的动员对认为动员效果不佳的患者有效。然而,将普乐沙福纳入动员策略的最佳方式仍有待确定。对资源利用进行中心特异性分析可能有助于确定实施各种基于普乐沙福的动员策略的最具成本效益的方式。