Blommestein Hedwig M, Verelst Silvia G R, de Groot Saskia, Huijgens Peter C, Sonneveld Pieter, Uyl-de Groot Carin A
Department of Health Policy & Management/institute for Medical Technology Assessment, Erasmus University, Rotterdam, The Netherlands.
Comprehensive Cancer Organisation, Utrecht, The Netherlands.
Eur J Haematol. 2016 Feb;96(2):198-208. doi: 10.1111/ejh.12571. Epub 2015 May 7.
To study the impact of novel treatments for elderly (≥66 yr) patients with multiple myeloma (MM) in daily practice by comparing real-world effects [overall survival (OS) and quality-adjusted life years (QALYs)] and costs over time. Also, we calculate cost-effectiveness of treatment sequences commonly prescribed to predict effects and costs if patients had received a different treatment sequence.
Real-world data including patient and disease characteristics, treatment information and resource use were collected from 1054 elderly patients with MM. Patients received first-line treatment during 2004-2007 (cohort 1) and 2008-2013 (cohort 2). The two cohorts were compared using a patient-level simulation (PLS) model comprising regression models which used patient and disease characteristics to estimate time to next treatment and death. Effects and costs from cohort 2 were compared to 4 commonly prescribed real-world sequences.
Utilisation of novel agents was higher for cohort 2 compared to cohort 1. Modelled average OS for cohort 1 was 38 months (median 25) and total costs €44,200. OS for cohort 2 was 42 months (median 28) and total costs €69,017. The model identified potential OS gains if all patients were to be treated using combinations containing thalidomide, lenalidomide and bortezomib in that particular order. This sequence had, compared to real-world treatment, the most favourable incremental cost-effectiveness ratio, €24,618 per life year gained and €34,875 per QALY.
Our patient-level model enabled to study the effects and costs of entire treatment sequences and to compare real-world treatment patterns over time. Increased utilisation of novel agents improved survival and increased costs for real-world patients with MM in the Netherlands.
通过比较实际效果[总生存期（OS）和质量调整生命年（QALY）]及不同时间的成本，研究新型治疗方法对老年（≥66岁）多发性骨髓瘤（MM）患者在日常临床实践中的影响。此外，我们计算了常用治疗方案的成本效益，以预测如果患者接受不同治疗方案时的效果和成本。
收集了1054例老年MM患者的实际数据，包括患者和疾病特征、治疗信息及资源使用情况。患者在2004 - 2007年（队列1）和2008 - 2013年（队列2）接受一线治疗。使用患者水平模拟（PLS）模型对两个队列进行比较，该模型包含回归模型，利用患者和疾病特征估计下次治疗时间和死亡时间。将队列2的效果和成本与4种常用的实际治疗方案进行比较。
与队列1相比，队列2新型药物的使用比例更高。队列1的模拟平均总生存期为38个月（中位数25个月），总成本44,200欧元。队列2的总生存期为42个月（中位数28个月），总成本69,017欧元。该模型确定，如果所有患者按照特定顺序使用包含沙利度胺、来那度胺和硼替佐米的联合方案进行治疗，总生存期可能会延长。与实际治疗相比，该方案的增量成本效益比最有利，每获得一个生命年为24,618欧元，每获得一个QALY为34,875欧元。
我们的患者水平模型能够研究整个治疗方案的效果和成本，并比较不同时间的实际治疗模式。新型药物使用比例的增加提高了荷兰MM实际患者的生存率，但也增加了成本。
