Evolving treatments and outcomes in patients with newly diagnosed multiple myeloma undergoing autologous stem cell transplantation: a real-world study in China

This single-centre retrospective study aimed to evaluate trends in the clinical characteristics, treatment approaches, and outcomes of patients with newly diagnosed multiple myeloma (NDMM) who underwent initial autologous stem cell transplantation (ASCT) between 2007 and 2023 in China. A total of 390 patients with NDMM were grouped into three time cohorts: 2007–2012 (79 patients), 2013–2018 (143 patients), and 2019–2023 (168 patients). Clinical characteristics, treatment regimens, and survival outcomes were analysed over these periods. The results revealed increases in the proportions of patients older than 65 years, those with extramedullary disease (EMD), and those with secondary amyloidosis (systemic organ involvement by immunoglobulin light chain amyloid deposition with a diagnosis of MM). Treatment trends included the preference for bortezomib-based regimens, with the addition of daratumumab (Dara)-based therapies in the most recent cohort. The pre-ASCT MRD-negative rate increased from 8.5% in Cohort 1 to 29.8% in Cohort 3 (p = 0.001), with corresponding post-ASCT improvements in MRD-negative status (64.4% to 80.4%). Survival outcomes also improved, with the 5-year PFS rate increasing from 44.62% (Cohort 1) to 60.36% (Cohort 2) and 87.61% in the latest cohort (p < 0.001) and the 5-year OS rate increasing from 58.23% to 68.46% and 81.82% in the latest cohort (p = 0.011), respectively. High-risk cytogenetic abnormalities (HRCAs) were associated with poorer PFS and OS, although tandem ASCT for high-risk patients resulted in improved outcomes. This real-world study highlights substantial advancements in response depth and survival outcomes for patients with NDMM undergoing ASCT. These improvements were driven by the introduction of new drugs and refined risk stratification methods.