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评估普乐沙福在干细胞动员中的应用——PHANTASTIC试验的经济学分析。

Evaluating the use of plerixafor in stem cell mobilisation - an economic analysis of the PHANTASTIC trial.

作者信息

Martin Antony P, Richards Sarah, Haycox Alan, Houten Rachel, McLeod Claire, Braithwaite Barbara, Clark Jack O, Bell Joanne, Clark Richard E

机构信息

Liverpool Health Economics, Department of Economics, University of Liverpool Management School, Liverpool, United Kingdom.

Haematology Department, Royal Liverpool University Hospital, Liverpool, United Kingdom.

出版信息

J Clin Apher. 2016 Oct;31(5):434-42. doi: 10.1002/jca.21433. Epub 2015 Sep 29.

DOI:10.1002/jca.21433
PMID:26415895
Abstract

Plerixafor is an effective haematopoietic stem cell mobilising agent in candidates for autologous transplantation, including patients with myeloma and lymphoma. Here we compare 98 plerixafor recipients in the PHANTASTIC trial with 151 historic controls mobilised by conventional chemotherapy (each with granulocyte colony-stimulating factor, G-CSF). Seventy (71.4%) plerixafor-mobilised patients achieved the composite primary endpoint of ≥4 × 10(6) CD34+ cells kg(-1) in ≤2 aphereses and no clinically significant neutropenia, compared to 48 (31.8%) historic controls (P < 0.001), and this significant advantage was maintained in scenario analyses testing components of this composite endpoint. A patient-level cost analysis was undertaken for 249 patients, which included the cost of remobilising patients where initial mobilisation had failed. Combined mean treatment cost for plerixafor mobilised patients was £12,679 compared with £11,694 for historical controls. However, plerixafor produces an average saving of £3,828 per lymphoma patient but average cost increase by £5,245 per myeloma patient. The present data demonstrate cost-effectiveness for plerixafor as a first line mobilisation agent, certainly for lymphoma patients, where substantial resource savings and achievement of the primary endpoint are likely. J. Clin. Apheresis 31:434-442, 2016. © 2015 Wiley Periodicals, Inc.

摘要

普乐沙福是一种有效的造血干细胞动员剂,适用于自体移植的候选者,包括骨髓瘤和淋巴瘤患者。在此,我们将PHANTASTIC试验中的98例普乐沙福接受者与151例采用传统化疗(均使用粒细胞集落刺激因子,G-CSF)动员的历史对照者进行比较。70例(71.4%)接受普乐沙福动员的患者在≤2次单采中达到了≥4×10⁶ CD34⁺细胞/kg且无临床显著中性粒细胞减少的复合主要终点,相比之下,历史对照者为48例(31.8%)(P<0.001),并且在对该复合终点各组成部分进行测试的情景分析中,这一显著优势得以保持。对249例患者进行了患者层面的成本分析,其中包括对初始动员失败患者进行再次动员的成本。接受普乐沙福动员患者的联合平均治疗成本为12,679英镑,而历史对照者为11,694英镑。然而,普乐沙福使每位淋巴瘤患者平均节省3,828英镑,但使每位骨髓瘤患者平均成本增加5,245英镑。目前的数据表明,普乐沙福作为一线动员剂具有成本效益,对于淋巴瘤患者肯定如此,因为在淋巴瘤患者中可能会大量节省资源并实现主要终点。《临床血液成分单采杂志》31:434 - 442, 2016年。© 2015威利期刊公司

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引用本文的文献

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Bone Marrow Transplant. 2021 Aug;56(8):1876-1887. doi: 10.1038/s41409-021-01251-8. Epub 2021 Mar 22.
2
Efficacy and safety of plerixafor for hematopoietic stem cell mobilization for autologous transplantation in patients with non-Hodgkin lymphoma and multiple myeloma: A systematic review and meta-analysis.普乐沙福用于非霍奇金淋巴瘤和多发性骨髓瘤患者自体移植造血干细胞动员的疗效与安全性:一项系统评价和荟萃分析。
Exp Ther Med. 2019 Aug;18(2):1141-1148. doi: 10.3892/etm.2019.7691. Epub 2019 Jun 19.