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多发性骨髓瘤患者中基于化疗与非化疗的干细胞动员(±普乐沙福):一项意大利成本效益分析

Chemotherapy-based versus chemotherapy-free stem cell mobilization (± plerixafor) in multiple myeloma patients: an Italian cost-effectiveness analysis.

作者信息

Lazzaro Carlo, Castagna Luca, Lanza Francesco, Laszlo Daniele, Milone Giuseppe, Pierelli Luca, Saccardi Riccardo

机构信息

Health Economist and Research Director, Studio di Economia Sanitaria, Milan, Italy.

Oncology and Haematology Unit, BMT section, Istituto Clinico Humanitas, Rozzano, Italy.

出版信息

Bone Marrow Transplant. 2021 Aug;56(8):1876-1887. doi: 10.1038/s41409-021-01251-8. Epub 2021 Mar 22.

DOI:10.1038/s41409-021-01251-8
PMID:33753907
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8338551/
Abstract

Given the availability and efficacy of the mobilizing agent plerixafor in augmenting hematopoietic progenitor cell mobilization with granulocyte colony-stimulating factor (G-CSF), there is a strong case for comparing the cost-effectiveness of mobilization with G-CSF + cyclophosphamide versus G-CSF alone. This study investigated the cost and effectiveness (i.e., successful 4 million-CD34 collection) of G-CSF alone versus high-dose cyclophosphamide (4 g/m) + G-CSF mobilization (± on-demand plerixafor) in patients with multiple myeloma (MM) eligible for autograft in Italy. A decision tree-supported cost-effectiveness analysis (CEA) model in MM patients was developed from the societal perspective. The CEA model compared G-CSF alone with cyclophosphamide 4 g/m + G-CSF (± on-demand plerixafor) and was populated with demographic, healthcare and non-healthcare resource utilization data collected from a questionnaire administered to six Italian oncohematologists. Costs were expressed in Euro (€) 2019. The CEA model showed that G-CSF alone was strongly dominant versus cyclophosphamide + G-CSF ( ± on-demand plerixafor), with incremental savings of €1198.59 and an incremental probability of a successful 4 million-CD34 apheresis (+0.052). Sensitivity analyses confirmed the robustness of the base-case results. In conclusion, chemotherapy-free mobilization (± on-demand plerixafor) is a "good value for money" option for MM patients eligible for autograft.

摘要

鉴于动员剂普乐沙福在增强造血祖细胞动员与粒细胞集落刺激因子(G-CSF)联合使用时的可用性和有效性,很有必要比较G-CSF联合环磷酰胺与单独使用G-CSF进行动员的成本效益。本研究调查了在意大利适合自体移植的多发性骨髓瘤(MM)患者中,单独使用G-CSF与高剂量环磷酰胺(4g/m²)+G-CSF动员(±按需使用普乐沙福)的成本和有效性(即成功采集400万个CD34细胞)。从社会角度建立了一个支持决策树的MM患者成本效益分析(CEA)模型。该CEA模型将单独使用G-CSF与4g/m²环磷酰胺+G-CSF(±按需使用普乐沙福)进行了比较,并填充了从对六位意大利血液肿瘤学家进行问卷调查收集的人口统计学、医疗保健和非医疗保健资源利用数据。成本以2019年欧元(€)表示。CEA模型显示,单独使用G-CSF与环磷酰胺+G-CSF(±按需使用普乐沙福)相比具有显著优势,节省增量为1198.59欧元,成功进行一次采集400万个CD34细胞的增量概率为+0.052。敏感性分析证实了基础案例结果的稳健性。总之,对于适合自体移植的MM患者,无化疗动员(±按需使用普乐沙福)是一种“性价比高”的选择。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/4782a461bb1b/41409_2021_1251_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/8d981059fdc9/41409_2021_1251_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/cca1ecbd2534/41409_2021_1251_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/a534684b10c2/41409_2021_1251_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/4782a461bb1b/41409_2021_1251_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/8d981059fdc9/41409_2021_1251_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/cca1ecbd2534/41409_2021_1251_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/a534684b10c2/41409_2021_1251_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e26c/8338551/4782a461bb1b/41409_2021_1251_Fig4_HTML.jpg

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Transfus Apher Sci. 2020 Oct;59(5):102819. doi: 10.1016/j.transci.2020.102819. Epub 2020 May 25.
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Expert Rev Pharmacoecon Outcomes Res. 2018 Aug;18(4):435-446. doi: 10.1080/14737167.2018.1464394. Epub 2018 Apr 20.
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The Efficacy and Safety of Chemotherapy-Based Stem Cell Mobilization in Multiple Myeloma Patients Who Are Poor Responders to Induction: The Mayo Clinic Experience.
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