A small set of multiple myeloma (MM) patients will show exceptional response to autologous stem cell transplantation (ASCT) and remain progression-free for years without maintenance therapy, a recent study has found.
“Further studies are needed to pre-emptively identify this subset of patients who may not need aggressive, life-long therapy to maintain disease control, in order to decrease the long-term risk of financial and physical toxicity,” researchers said.
Of the 509 MM patients who received ASCT during the study period, 51 showed excellent long-term response. These patients showed no evidence of disease progression for at least 96 months. However, five were eventually excluded: two received maintenance therapy after transplantation, three patients showed developed smouldering MM.
Overall, 46 patients (median age at diagnosis, 57 years; 61 percent female) were deemed to be exceptional responders to ASCT, yielding a rate of 9 percent. In this group, the median duration of follow-up from diagnosis was 16.2 years.
Most of the exceptional responders (74 percent; n=34) showed complete response or better to ASCT, and another six patients (13 percent) had very good partial response. The poorest response status was achieving a stable disease, which was reported in one patient. The median time to best response after ASCT was 3 months.
At the time of the final haematology assessment, more than half (52 percent; n=24) of the participants were still in remission, and 19 still showed complete response. The median progression-free and overall survival post-ASCT were 13.8 and 19.9 years, respectively.