Centro Hospitalar Universitário de Coimbra, Coimbra, Portugal.
Fundação Champalimaud, Lisboa, Portugal.
Cancer Rep (Hoboken). 2024 Oct;7(10):e70027. doi: 10.1002/cnr2.70027.
Patients with multiple myeloma often require multiple treatment lines. The order in which treatments are sequenced has impact on clinical outcomes. This study aimed to estimate progression-free survival (PFS) and overall survival (OS) with common treatment sequences used in Portugal and the incremental benefit of an optimal sequence in transplant-ineligible patients with multiple myeloma.
A state-transition sequential model with a five-health state conceptual structure was developed to simulate and compare survival outcomes between treatment sequences up to four lines of treatments. Data sources included randomized clinical trials and indirect treatment comparisons. A panel of Portuguese hematologists listed four most common treatment sequences and optimal sequence of choice in transplant-ineligible patients.
Our simulation estimated an OS between 6.1 and 7.8 years using the most common sequences, with VMP + DRd + Pd + Kd as the most effective (7.8 years). Optimal sequence of choice (DRd + PVd + Kd + Vd) achieved OS of 9.8 years and may extend OS in 2.0-3.7 years vs. most common sequences (26%-61% increase). This benefit was mostly explained by extended PFS in the first line of treatment.
Model results demonstrate that choosing the most effective treatment upfront is crucial in delaying disease progression thus yielding better survival outcomes in transplant-ineligible patients. There was a clear survival benefit in using daratumumab-based regimens in first line. This modelling exercise highlights the need to raise awareness around the impact of sequencing strategies to improve patient's outcomes.
多发性骨髓瘤患者通常需要接受多线治疗。治疗顺序的安排会对临床结局产生影响。本研究旨在评估葡萄牙常用治疗方案的无进展生存期(PFS)和总生存期(OS),以及在不适合移植的多发性骨髓瘤患者中,最佳治疗方案的增量获益。
采用五状态概念结构的状态转移序贯模型,模拟并比较了四线治疗方案的治疗顺序的生存结局。数据来源包括随机临床试验和间接治疗比较。一组葡萄牙血液学家列出了四种最常见的治疗方案和不适合移植的患者中首选的最佳治疗方案。
我们的模拟估计,使用最常见的方案,OS 为 6.1 至 7.8 年,其中 VMP+DRd+Pd+Kd 方案最有效(7.8 年)。首选的最佳治疗方案(DRd+PVd+Kd+Vd)可获得 9.8 年的 OS,与最常见的方案相比,可能延长 OS 2.0-3.7 年(26%-61%的增加)。这一获益主要归因于一线治疗中 PFS 的延长。
模型结果表明,优先选择最有效的治疗方案对于延缓疾病进展至关重要,从而在不适合移植的患者中获得更好的生存结局。在一线治疗中使用达雷妥尤单抗方案具有明显的生存获益。这项建模研究强调了需要提高对治疗方案排序策略影响的认识,以改善患者的结局。