Modelling and Simulation, Evidera, London, England, UK.
Value Evidence and Outcomes, GlaxoSmithKline, Upper Providence, PA, USA.
Value Health. 2022 Dec;25(12):1977-1985. doi: 10.1016/j.jval.2022.05.011. Epub 2022 Aug 11.
Estimates on the distribution of patients with multiple myeloma (MM) by line of therapy (LOT) are scarce and get outdated quickly as new treatments become available. The objective of this study was to estimate the number of patients with MM by LOT and the number of patients who have received at least 4 previous LOTs including proteasome inhibitors, immunomodulatory agents, and anti-CD38 monoclonal antibodies (mAbs).
A compartmental model was developed to calculate the number of patients by LOT. Two pathways were considered based on stem cell transplant eligibility, and at each pathway, treatments were stratified in 2 types: anti-CD38 mAbs or other. The model population was stratified into 4 subgroups based on age and cytogenetic risk. Model inputs were informed from real-world evidence.
The model estimated that, in 2020, 126 869 patients were living with MM in the United States. Of these, 105 701 received treatment in any LOT, with 56 959, 27 252, 11 258, and 5217 in lines 1 to 4, respectively, and 5015 in line 5 or beyond. The model estimated that 3497 patients received at least 4 previous LOTs including proteasome inhibitors, immunomodulatory agents, and anti-CD38 mAbs. The model overall prevalence predictions aligned well with publicly available estimates.
This study proposes a novel framework to estimate MM prevalence. It can assist clinicians to understand future trends in MM epidemiology, healthcare systems to plan for future resource use allocation, and payers to quantify the budget impact of new treatments.
按治疗线（LOT）估计多发性骨髓瘤（MM）患者的分布情况的相关数据十分有限，且由于新疗法的不断出现，这些数据很快就会过时。本研究旨在估计按 LOT 划分的 MM 患者数量以及至少接受过 4 次包括蛋白酶体抑制剂、免疫调节剂和抗 CD38 单克隆抗体（mAb）在内的既往 LOT 治疗的患者数量。
采用房室模型来计算 LOT 患者数量。基于干细胞移植资格，考虑了两条途径，在每条途径中，将治疗方案分为抗 CD38 mAb 或其他两种类型。模型人群按年龄和细胞遗传学风险分为 4 个亚组。模型输入信息来源于真实世界的数据。
模型估计 2020 年美国有 126869 名 MM 患者仍在世。其中，有 105701 名患者接受了 LOT 治疗，分别有 56959、27252、11258 和 5217 名患者在第 1 至 4 线治疗中，第 5 线或更后线治疗的患者有 5015 名。模型估计有 3497 名患者接受了至少 4 次包括蛋白酶体抑制剂、免疫调节剂和抗 CD38 mAb 的既往 LOT 治疗。总体而言，模型对流行率的预测与公开的估计数据较为一致。
本研究提出了一种估计 MM 流行率的新框架。它可以帮助临床医生了解 MM 流行病学的未来趋势，帮助医疗体系规划未来资源利用分配，帮助支付方量化新疗法的预算影响。
