Multiple myeloma patients show excellent response to stem cell treatment

13 Sep 2020
Scientists fear new regulations would limit the potential of stem cell therapies.Scientists fear new regulations would limit the potential of stem cell therapies.

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.

Blood Cancer J 2020;10:87