The efficacy and safety of autologous hematopoietic stem cell transplantation (auto-HCT) performed during the era of novel agents was comparable between elderly and younger patients with multiple myeloma, according to results of a retrospective study published in Bone Marrow Transplantation.

Uncertainty remains regarding the risks and benefits of auto-HCT in elderly patients (≥ 65 years). Although there is evidence that select elderly patients with multiple myeloma can benefit from auto-HCT, most trials evaluating auto-HCT in multiple myeloma focused on younger patients (<65 years). This large, retrospective study was conducted to specifically examine the safety and efficacy of auto-HCT in elderly versus younger patients with multiple myeloma in a real-world setting during the era of novel agents.

A total of 2056 patients with multiple myeloma included in a Japanese national registry met the following study criteria: 16 years of age or older; first auto-HCT with peripheral blood stem cells; use of melphalan alone (100 mg/m2, 140 mg/m2,and 200 mg/m2) as a conditioning regimen; no planned tandem transplantation; and auto-HCT received between 2007 and 2017. Of these patients, 287 were 65 years or older.As a historical control, data from 766 patients with multiple myeloma who received auto-HCT between 1995 and 2006 (pre novel agent era) were also evaluated in the survival analysis. The primary and secondary endpoints of the study were treatment-related mortality due to disease relapse or progression and overall survival (OS), respectively.

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In the 2007 to 2014 cohort, the percentages of older (75.6%) and younger patients (95.0%) receiving high-dose (200 mg/m2) melphalan as a conditioning regimen were significantly different (P <.001).Using a propensity score-matched-pair analysis in which 263 patients from each of the 2 age groups were matched according to patient characteristics to adjust for selection bias, no differences were seen in 100-day treatment-related mortality for younger patients (0.4% [younger patients] vs 1.2% [older patients]; P =.32), or 1-year treatment related mortality (1.2% [younger patients] vs 1.6% [older patients]; P =.69). 

Analyses of rates of 5-year OS following auto-HCT in the 2007 to 2014 cohort showed no significant differences between age groups (62.5% [younger patient] vs 63.5% [older patients]; P =.56).However, in the 1995 to 2006 cohort, 5-year OS rates were significantly lower for older patients (42.6%) compared with younger patients (58.2%; P =.00003).

“We showed that auto-HCT is safe and effective for treating multiple myeloma in elderly patients, particularly in the era of novel agents,” the study authors wrote. “Elderly multiple myeloma patients who underwent auto-HCT showed comparable benefits, which highlights the fact that chronological age alone should not be used to determine transplantation eligibility.” The authors concluded that further research is warranted to determine optimal selection strategies as it relates to patient age as well as the optimal dose of melphalan for older patients in the era of novel agents.


  1. Mizuno S, Kawamura K, Hanamura I, et al. Efficacy and safety of autologous stem celltransplantation in patients aged ≥ 65 years with multiple myeloma in the era ofnovel agents
  2. . Bone Marrow Transplant. doi:10.1038/s41409-019-0478-4