Second-Line Treatment Options

Gary Schiller, MD: What would you consider in terms of stem cell transplantation? Who gets that and when?

Salman Fazal, MD: For the fit patients, if they achieve complete remission, allogeneic stem cell transplantation should be offered for those fit patients who are in remission. In the clinical study that was published in the New England Journal of Medicine, 47% of patients who were newly diagnosed with BPDCN [blastic plasmacytoid dendritic cell neoplasm] were able to undergo transplantation. Interestingly, there were 13 patients who underwent transplantation, and 3 of 13 patients underwent autologous stem cell transplantation, and there have been some data about use of autologous stem cell transplantation. However, allogeneic stem cell transplantation offers better results as compared with autologous stem cell transplant, and there is a scarcity of data for use of autologous stem cell transplantation.

Getting back to the median age of the disease, it is important to realize that it is in the late 60s, and we are not able to offer allogeneic stem cell transplantation for most of those patients. That is a big limitation in terms of performing stem cell transplantation for BPDCN. In terms of the outcome, the best outcome that we have for this disease is based on the patients who were able to undergo transplantation for BPDCN. I certainly would offer for patients who are fit and are in first remission.

Things may change. There are clinical trials for the anti-CD123 CAR [chimeric antigen receptor] T, which is something on the horizon. CAR T-cell therapy is an attractive option for a lot of B-cell malignancies and is 1 myeloid disease where the CAR T cell has a promising option. That would be something that I’m looking forward to in the future, to see the results from the clinical trial.

Gary Schiller, MD: You were careful. You almost said a promising potential, and that’s all you could say about a CD123 CAR because there might be a lot of off-target toxicity. Even with a novel agent, you see some toxicity that is capillary leak, and that’s why the albumin threshold was placed on the original trial. I hope you’re correct that engineered cell therapy will have a role, but that’s something completely investigational now.

Salman Fazal, MD: The University of Texas MD Anderson Cancer Center trials with anti-CD126 CAR T have been closed for enrollment because they had some undesired results in their early data. But it’s a promising option for patients who have relapsed disease and enrolling in a clinical trial allows us to evaluate other options.

Transcript Edited for Clarity