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硼替佐米、沙利度胺和来那度胺在多发性骨髓瘤治疗中的作用:临床和经济信息概述。

The role of bortezomib, thalidomide and lenalidomide in the management of multiple myeloma: an overview of clinical and economic information.

机构信息

Laboratory of Pharmacoeconomics, co Area Vasta Centro, Regional Health System, Florence, Italy.

出版信息

Pharmacoeconomics. 2011 Apr;29(4):269-85. doi: 10.2165/11585930-000000000-00000.

DOI:10.2165/11585930-000000000-00000
PMID:21395348
Abstract

Bortezomib, thalidomide and lenalidomide can be aimed at treating patients with newly diagnosed multiple myeloma (both eligible and ineligible for transplantation) as well as those with relapsed or refractory disease. This review analysed the available clinical and economic data on these three drugs. Irrespective of which of the three agents is considered, the magnitude of the benefit in newly diagnosed cases (transplanted or non-transplanted) tends to be between 10 and 20 months per patient in terms of progression-free survival or survival; the survival benefit is smaller in relapsed or refractory disease. In addition, a single-institution observational analysis evaluated the outcomes in nearly 3000 consecutive patients examined between 1971 and 2006. The survival in patients diagnosed between 2001 and 2006 was longer than that observed in patients diagnosed between 1994 and 2000. This finding supports the conclusion that novel agents provide a survival improvement compared with traditional therapy. Formal cost-effectiveness studies on these three agents are still lacking. A MEDLINE search retrieved only four short papers or letters and no full-length analysis. Hence, the cost effectiveness of these agents needs further investigation, with separate assessments of the different therapeutic settings. In a simplified analysis, we tried to contrast the average cost of treatment for each of the novel agents versus their respective benefit, expressed in quality-adjusted survival. Despite its preliminary nature, our assessment indicates that the cost effectiveness of these three agents is likely to be within commonly accepted pharmacoeconomic thresholds.

摘要

硼替佐米、沙利度胺和来那度胺可用于治疗新诊断的多发性骨髓瘤患者(适合和不适合移植的患者)以及复发性或难治性疾病患者。本综述分析了这三种药物的现有临床和经济数据。无论考虑使用这三种药物中的哪一种,在新诊断的病例(移植或非移植)中,无进展生存期或总生存期的获益幅度通常在每个患者 10 至 20 个月之间;在复发性或难治性疾病中,生存获益较小。此外,一项单机构观察性分析评估了近 3000 例连续患者在 1971 年至 2006 年间的结果。2001 年至 2006 年诊断的患者的生存率长于 1994 年至 2000 年诊断的患者。这一发现支持这样的结论,即新型药物与传统治疗相比提供了生存改善。这三种药物的正式成本效益研究仍然缺乏。对这三种药物的 MEDLINE 搜索仅检索到四份短论文或信件,没有完整的分析。因此,这些药物的成本效益需要进一步研究,分别评估不同的治疗环境。在简化分析中,我们试图对比每种新型药物的平均治疗成本与其各自的生存效益,用质量调整生存来表示。尽管我们的评估具有初步性质,但表明这三种药物的成本效益可能在可接受的药物经济学范围内。

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