Pano/GemBuMel is Safe and Effective in High-Risk or Relapsed/Refractory Myeloma

Yago Nieto, MD, PhD, The University of Texas MD Anderson Cancer Center, Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine, discusses the findings from a phase 2 trial (NCT02506959) evaluating treatment with panobinostat, gemcitabine, busulfan, and melphalan (Pano/GemBuMel) for patients with high-risk or relapsed/refractory myeloma.

During the 2023 Transplantation and Cellular Therapy Meeting, Nieto presented data from the trial which showed the combination to be safe and effective in this patient population. Patients were enrolled in 2 separate cohorts, including autologous stem cell transplant (ASCT)-1 which consisted of 2 subgroups receiving a first frontline ASCT or a first salvage ASCT, and ASCT-2, which included those given second salvage ASCT.

Among patients, the overall response rate/complete response with measurable disease at ASCT were 67%/40% for patients receiving a frontline ASCT-1, and 93%/64% for second salvage ASCT. MRD negativity improved after ASCT-1 from 8.5 to 23% (P < .0001) and ASCT-2 from 34% to 55% (P = .02). Additionally, at a median follow-up of 51 months (range, 8-78), the median progression-free survival among patients receiving a frontline ASCT-1 was 45 compared with 21 for those with a 1st salvage ASCT-1, and 32 months with a second ASCT, respectively.

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0:08 | What we saw was that Pano/GemBuMel significantly improved progression-free survival when done as a first transplant compared with matched controls, but the outcomes were very similar when done as the second transplant. For overall survival, there were some numerical differences in favor of pano and GemBuMel, but they didn't reach statistical significance for overall survival.

0:38 | Our conclusion is that the panobinostat and GemBuMel is a safe regimen and is highly active. It leads to MRD negativity in a significant number of patients, even patients with relapsed and refractory disease, and it seems better in this non-randomized but concurrent match comparison with concurrent controls, and seems better when done as a first transplant.