Abstract
Evidence on the prognostic value of autologous stem cell transplantation (ASCT) and minimal residual disease (MRD) dynamics of patients with newly diagnosed multiple myeloma (NDMM) in China is limited. Our objective in the current study was to understand the current care paradigm and outcomes of these patients. This longitudinal cohort study used historical data from three top-tier hematologic disease care hospitals that contributed to the National Longitudinal Cohort of Hematological Diseases-Multiple Myeloma. Treatment regimens [proteasome inhibitor (PI)-, immunomodulatory drug (IMiD)-, PI+IMiD-based, and conventional], post-induction response, ASCT and MRD status, and survival outcomes [progression-free survival (PFS) and overall survival (OS)] were evaluated. In total, 454 patients with NDMM were included (median age, 57 years; 59.0% males) with a median follow-up of 58.7 months. The overall response rate was 91.0%, 83.9%, 90.6%, and 60.9% for PI-, IMiD-, PI+IMiD-based, and conventional regimens, respectively. Patients with ASCT during first-line therapy (26.2%) had a longer PFS and OS than patients who did not receive ASCT [median PFS, 42.9 vs. 21.2 months, P < 0.001; median OS, not reached (NR) vs. 65.8 months, P < 0.001]. The median OS was NR, 71.5, and 56.6 months among patients with sustained MRD negativity, loss of MRD negativity, and persistent MRD, respectively (P < 0.001). Multivariate analysis revealed that the lactic dehydrogenase level, International Staging System stage, extra-medullary disease, and upfront ASCT were independent factors in predicting OS among NDMM patients. Our study showed that novel agent-based regimens, first-line ASCT, and sustained MRD negativity were associated with a superior outcome for patients with NDMM in China (Identifier: NCT04645199).
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