外推经验长期生存数据:更新后续数据和参数外推方法对多发性骨髓瘤生存估计的影响
L J Bakker1,2, F W Thielen3,4, W K Redekop3,4
1Erasmus School of Health Policy and Management, Erasmus University Rotterdam, P.O. Box 1738, Rotterdam, 3000 DR, The Netherlands. lytskebakker@gmail.com.
BMC medical research methodology
|May 29, 2023
概括
在多发性骨髓瘤 (MM) 中使用参数和脊柱模型推断长期存活是可行的. 然而,研究人员必须对潜在的不确定性保持谨慎,即使随着随访的增加和审查的减少.
科学领域:
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
- 在瘤学瘤学.
背景情况:
- 对生存率的推断对于经济评估至关重要,特别是对临床试验的数据进行了正确的审查.
- 外推生存率估计的准确性取决于随访时间和关于生存危险的假设.
- 这项研究评估了新诊断的多发性骨髓瘤 (MM) 长期存活的取值技术.
研究的目的:
- 分析MM患者不同生存抽取方法的准确性.
- 评估不同数据截止点对长期生存估计的影响.
- 为了比较参数和spline模型在MM的生存外推.
主要方法:
- 利用来自随机对照试验和MM患者登记册的经验数据.
- 应用了标准的参数和线条模型,模拟了使用数据库锁定 (3-13年) 的减少后续.
- 与Kaplan-Meier RMST进行比较,选择基于统计匹配的模型.
主要成果:
- 随着随访增加,事件数量增加和审查减少,RMST错误率下降.
- 当随访时间超过六年时,观察到RMST错误的最显著减少.
- 在推断的RMST中发现了相当大的偏差,即使在低审查和高事件计数的情况下.
结论:
- 标准的参数和支线模型对于MM的生存外推是可行的.
- 研究人员和决策者应该承认外推结果中固有的不确定性.
- 不管数据的完整性如何,建议对外推算的生存率估计保持谨慎.
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