Tandem Versus Single Autologous Stem Cell Transplantation for High‐Risk Multiple Myeloma in the Era of Novel Agents: A Real‐World Study of China

ABSTRACT Background This study compares the efficacy and safety of single autologous stem cell transplantation (ASCT) versus tandem ASCT for multiple myeloma (MM) patients in the era of novel agents. Methods A total of 112 high‐risk MM patients were included (single ASCT, (n = 57) or tandem ASCT (n = 55) in this retrospective multicenter study. Responses and outcomes were evaluated. Results At 100 days after ASCT1 and ASCT2, 36 (63.2%) versus 45 (81.8%) patients achieved sCR/CR, 16 (28.1%) versus 7 (12.7%) patients achieved VGPR, and 5 (8.8%) versus 1 (1.8%) patient achieved PR, respectively, in the single and tandem ASCT cohorts. The 3‐year cumulative incidence of non‐relapse mortality and disease progression was 0% versus 7.3% ( p = 0.083), and 45.8% versus 25.8% ( p = 0.039), respectively, for the single and tandem ASCT cohort. The tandem ASCT cohort showed a trend of better 3‐year probability of PFS (58.1% vs. 64.7%, p = 0.064) compared with the single ASCT cohort. In multivariate analysis, ultra high‐risk and achieving<VGPR response after ASCT1 were associated with an inferior PFS. Ultra high‐risk was also associated with an inferior OS. Conclusions Tandem ASCT demonstrated improved outcomes compared to single ASCT in high‐risk MM patients receiving triplet or quadruplet induction and maintenance therapy. However, patients with ultra high‐risk cytogenetics may require innovative therapeutic approaches, as tendem ASCT does not overcome their adverse prognosis.