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多发性骨髓瘤外周血干细胞动员策略的成本与疗效

Cost and efficacy of peripheral stem cell mobilization strategies in multiple myeloma.

作者信息

Van de Wyngaert Zoé, Nerich Virginie, Fouquet Guillemette, Chrétien Marie-Lorraine, Caillot Denis, Azar Nabih, Garderet Laurent, Lenain Pascal, Macro Margaret, Bourhis Jean-Henri, Belhocine Ramdane, Jaccard Arnaud, Karlin Lionel, Bobin Arthur, Moya Niels, Systchenko Thomas, Gruchet Cecile, Giraud Christine, Guidez Stéphanie, Darras Claire, Princet Isabelle, Touzeau Cyrille, Moreau Philippe, Hulin Cyrille, Deconinck Erik, Limat Samuel, Leleu Xavier

机构信息

CHU Lille, Service des Maladies du Sang, F-59000, Lille, France.

Hématologie et thérapie cellulaire, Hôpital Saint Antoine, APHP, Université Paris-Sorbonne, INSERM UMRs 938, Paris, France.

出版信息

Bone Marrow Transplant. 2020 Dec;55(12):2254-2260. doi: 10.1038/s41409-020-0940-3. Epub 2020 May 23.

DOI:10.1038/s41409-020-0940-3
PMID:32447348
Abstract

Mobilization of peripheral blood stem cells (PBSC) can be performed using plerixafor, which is expensive, or high-dose cyclophosphamide (HDCy). We hypothesized that the overall cost of mobilization with plerixafor might not be greater if the cost of complication management was considered. We performed a cost analysis of these two strategies. This multicentric observational study recruited patients with myeloma who underwent a first PBSC mobilization. We considered direct medical costs, including hospitalization, mobilization agents, apheresis, and supportive treatments. We included 111 patients, 54 and 57 in the HDCy and plerixafor groups, respectively. Cost of mobilization with HDCy was 5097 ± 2982€ vs. 10958 ± 1789€ for plerixafor (p < 0.0001). Cost of agents used was 1287 ± 779€ vs. 6552 ± 509€, respectively (p = 0.0009). The mean number of days of hospitalization was 2 and 2.1 days, respectively (p = 0.035). All patients achieved the minimum PBSC collection target (p = 1.0); however, ASCT was performed with HDCy in 67% patients and with plerixafor in 86% (p = 0.02). Plerixafor mobilization incurred a greater cost, mostly due to the greater cost of the drug. Hospitalization length in the two groups was similar in our series. Interestingly, plerixafor appeared to be a very effective and safe mobilizing approach translating into a greater ASCT success.

摘要

外周血干细胞(PBSC)动员可使用普乐沙福(价格昂贵)或大剂量环磷酰胺(HDCy)来进行。我们推测,如果考虑并发症管理成本,使用普乐沙福进行动员的总体成本可能不会更高。我们对这两种策略进行了成本分析。这项多中心观察性研究纳入了首次进行PBSC动员的骨髓瘤患者。我们考虑了直接医疗成本,包括住院、动员剂、血细胞分离术和支持性治疗。我们纳入了111例患者,HDCy组和普乐沙福组分别为54例和57例。HDCy动员的成本为5097±2982欧元,而普乐沙福为10958±1789欧元(p<0.0001)。所用药物的成本分别为1287±779欧元和6552±509欧元(p=0.0009)。平均住院天数分别为2天和2.1天(p=0.035)。所有患者均达到了最低PBSC采集目标(p=1.0);然而,67%接受HDCy的患者和86%接受普乐沙福的患者进行了自体造血干细胞移植(ASCT)(p=0.02)。普乐沙福动员的成本更高,主要是由于药物成本更高。在我们的系列研究中,两组的住院时间相似。有趣的是,普乐沙福似乎是一种非常有效且安全的动员方法,这转化为更高的ASCT成功率。

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1
Cost and efficacy of peripheral stem cell mobilization strategies in multiple myeloma.多发性骨髓瘤外周血干细胞动员策略的成本与疗效
Bone Marrow Transplant. 2020 Dec;55(12):2254-2260. doi: 10.1038/s41409-020-0940-3. Epub 2020 May 23.
2
A comparison of chemo-free strategy with G-CSF plus plerixafor on demand versus intermediate-dose cyclophosphamide and G-CSF as PBSC mobilization in newly diagnosed multiple myeloma patients: An Italian explorative cost Analysis.新诊断多发性骨髓瘤患者中无化疗策略与按需 G-CSF 联合普乐沙福与中剂量环磷酰胺和 G-CSF 作为 PBSC 动员的比较:意大利探索性成本分析。
Transfus Apher Sci. 2020 Oct;59(5):102819. doi: 10.1016/j.transci.2020.102819. Epub 2020 May 25.
3
Plerixafor plus granulocyte colony-stimulating factor versus placebo plus granulocyte colony-stimulating factor for mobilization of CD34(+) hematopoietic stem cells in patients with multiple myeloma and low peripheral blood CD34(+) cell count: results of a subset analysis of a randomized trial.培洛昔福联合粒细胞集落刺激因子与安慰剂联合粒细胞集落刺激因子动员多发性骨髓瘤且外周血 CD34+细胞计数低的患者中的 CD34+造血干细胞:一项随机试验的亚组分析结果。
Biol Blood Marrow Transplant. 2012 Oct;18(10):1564-72. doi: 10.1016/j.bbmt.2012.05.017. Epub 2012 Jun 6.
4
Peripheral blood stem cell mobilization in multiple myeloma patients treat in the novel therapy-era with plerixafor and G-CSF has superior efficacy but significantly higher costs compared to mobilization with low-dose cyclophosphamide and G-CSF.在新型治疗时代,使用普乐沙福和粒细胞集落刺激因子(G-CSF)动员多发性骨髓瘤患者的外周血干细胞,与使用低剂量环磷酰胺和G-CSF进行动员相比,具有更高的疗效,但成本显著更高。
J Clin Apher. 2013 Oct;28(5):359-67. doi: 10.1002/jca.21280. Epub 2013 Jun 14.
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Plerixafor on-demand combined with chemotherapy and granulocyte colony-stimulating factor: significant improvement in peripheral blood stem cells mobilization and harvest with no increase in costs.按需使用普乐沙福联合化疗和粒细胞集落刺激因子:在外周血干细胞动员和采集方面有显著改善,且不增加成本。
Br J Haematol. 2014 Jan;164(1):113-23. doi: 10.1111/bjh.12606. Epub 2013 Oct 21.
6
Are we choosing mobilization regimens for autologous stem cell transplantation in multiple myeloma wisely: A single center comparison of GCSF+/-plerixafor vs cyclophosphamide/GCSF+/-plerixafor.我们是否明智地选择了多发性骨髓瘤自体干细胞移植的动员方案:GCSF+/-plerixafor 与环磷酰胺/GCSF+/-plerixafor 的单中心比较。
J Clin Apher. 2022 Aug;37(4):348-353. doi: 10.1002/jca.21976. Epub 2022 Feb 26.
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Plerixafor: A chemokine receptor-4 antagonist for mobilization of hematopoietic stem cells for transplantation after high-dose chemotherapy for non-Hodgkin's lymphoma or multiple myeloma.普乐沙福:一种趋化因子受体-4 拮抗剂,用于高剂量化疗治疗非霍奇金淋巴瘤或多发性骨髓瘤后移植造血干细胞的动员。
Clin Ther. 2010 May;32(5):821-43. doi: 10.1016/j.clinthera.2010.05.007.
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[Cost-effectiveness of peripheral blood stem cell collection using plerixafor: a single-center study].
Rinsho Ketsueki. 2020;61(11):1563-1569. doi: 10.11406/rinketsu.61.1563.
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Plerixafor as first- and second-line strategies for autologous stem cell mobilization in patients with non-Hodgkin's lymphoma or multiple myeloma.培洛昔福作为非霍奇金淋巴瘤或多发性骨髓瘤患者自体干细胞动员的一线和二线策略。
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Comparable efficacy and lower cost of PBSC mobilization with intermediate-dose cyclophosphamide and G-CSF compared with plerixafor and G-CSF in patients with multiple myeloma treated with novel therapies.与培洛昔康和 G-CSF 相比,新型疗法治疗多发性骨髓瘤患者中,中剂量环磷酰胺和 G-CSF 动员 PBSC 的疗效相当,但成本更低。
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本文引用的文献

1
Evaluation of the predictive value of the hematopoietic progenitor cell count using an automated hematology analyzer for CD34+ stem cell mobilization and apheresis product yield.采用自动化血液分析仪评估造血祖细胞计数对 CD34+ 干细胞动员和单采产品产量的预测价值。
Int J Lab Hematol. 2020 Apr;42(2):170-179. doi: 10.1111/ijlh.13142. Epub 2019 Dec 12.
2
Peripheral blood stem cell mobilization in multiple myeloma patients treat in the novel therapy-era with plerixafor and G-CSF has superior efficacy but significantly higher costs compared to mobilization with low-dose cyclophosphamide and G-CSF.在新型治疗时代,使用普乐沙福和粒细胞集落刺激因子(G-CSF)动员多发性骨髓瘤患者的外周血干细胞,与使用低剂量环磷酰胺和G-CSF进行动员相比,具有更高的疗效,但成本显著更高。
J Clin Apher. 2013 Oct;28(5):359-67. doi: 10.1002/jca.21280. Epub 2013 Jun 14.
The Efficacy and Safety of Chemotherapy-Based Stem Cell Mobilization in Multiple Myeloma Patients Who Are Poor Responders to Induction: The Mayo Clinic Experience.基于化疗的干细胞动员在诱导治疗反应不佳的多发性骨髓瘤患者中的疗效和安全性:梅奥诊所的经验。
Transplant Cell Ther. 2021 Sep;27(9):770.e1-770.e7. doi: 10.1016/j.jtct.2021.06.016. Epub 2021 Jun 18.
4
Stem Cell Mobilization in Multiple Myeloma: Comparing Safety and Efficacy of Cyclophosphamide +/- Plerixafor versus Granulocyte Colony-Stimulating Factor +/- Plerixafor in the Lenalidomide Era.多发性骨髓瘤中的干细胞动员:来那度胺时代环磷酰胺 +/- 培洛昔芬与粒细胞集落刺激因子 +/- 培洛昔芬的安全性和疗效比较。
Transplant Cell Ther. 2021 Jul;27(7):590.e1-590.e8. doi: 10.1016/j.jtct.2021.04.016. Epub 2021 Apr 26.