Abstract
OBJECTIVE: This systematic literature review analyzed real-world evidence on autologous stem cell transplantation (ASCT) for newly diagnosed multiple myeloma (ndMM) in China. METHODS: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, major English and Chinese databases were searched for observational studies published between 2015 and 2025 that included Chinese ndMM patients eligible for ASCT. Extracted data covered patient characteristics, treatment responses, and survival outcomes associated with ASCT and non-ASCT treatments. Single-arm meta-analysis with a random-effects model was used for evidence synthesis and pooled outcomes of the treatments for the same phase were compared using Cochran's Q or chi-square tests. RESULTS: Eighty-eight observational studies (2015-2025) were included. Data were pooled using single-arm meta-analysis and the pooled outcomes for ASCT and non-ASCT treatments were compared using Cochran's Q or chi-square tests. Compared to patients receiving non-ASCT treatments, ASCT recipients were younger (54.5 years vs 61.4 years), had more early-stage disease (International Staging System (ISS) I: 25.8% vs 15.1%), more t(11;14) (14.1% vs 6.0%), and fewer complex karyotypes (2.9% vs 31.7%). Plerixafor plus granulocyte colony-stimulating factor (G-CSF) significantly improved mobilization success over cyclophosphamide plus G-CSF (73.6% vs 49.5%), though with slightly delayed engraftment. ASCT was associated with superior treatment response (complete response: 78.5% vs 45.3%; very good partial response (⩾VGPR): 98.6% vs 75.0%) and higher 5-year overall survival rate (70.6% vs 41.3%) and 5-year progression-free survival rate (41.3% vs 18.0%). Advanced revised-ISS stage, IgD subtype, and suboptimal pre-ASCT response were associated with poorer survival prognosis in ASCT recipients. CONCLUSION: Chinese ASCT recipients had more favorable baseline profiles and achieved significantly better clinical outcomes than those receiving non-ASCT treatments. Plerixafor-based mobilization enhanced stem cell collection success. However, advanced stage, IgD subtype, and inadequate pre-ASCT response could discount the effectiveness of ASCT. INPLASY REGISTRATION NUMBER: INPLASY2025110069.