Tagraxofusp Shows Encouraging Response Rate in Patients With BPDCN
Marlise R. Luskin, MD, MSCE, discusses the trial of tagraxofusp in BPDCN, which showed high response rates and overall survival.
Marlise R. Luskin, MD, MSCE, a member of the Adult Leukemia Program at Dana-Farber Cancer Institute, associate program director of the Dana-Farber/Mass General Brigham Hematology/Oncology Fellowship Program, and associate professor of medicine at Harvard Medical School, discusses the data observed with tagraxofusp (Elzonris) in patients with blastic plasmacytoid dendritic-cell neoplasm (BPDCN).
In the phase 1/2 study with 47 patients enrolled (NCT02113982), 32 patients had no prior treatment, and the rest had received previous treatments. The phase 2 dose from earlier in the trial showed a high response rate according to Luskin, with a 90% overall response rate (ORR) and a 72% complete remission rate. This led to a 2-year overall survival rate of 50%, which Luskin says is promising compared to historical controls.
Additionally, the treatment allowed half of the patients to proceed to stem cell transplantation, aiming for a more durable remission. Responses to the treatment were observed quickly, often after 1 to 2 cycles, and some responses were found to be durable, providing evidence for the drug's effectiveness in both transplant-eligible and ineligible patients.
TRANSCRIPTION:
0:10 | The initial publication reported on 47 patients [who] were mostly without previous treatment, 32 of the 47, with the rest having had previous treatment. For untreated patients who received the recommended phase 2 dose, there was a really high response rate, 90% ORR, and 72%, almost three quarters, achieving a complete remission. In those patients, that translated to an overall survival [rate] at 2 years of 50%, which is really encouraging based on historical controls.
0:44 | This regimen also allowed half of these patients to proceed to transplantation, which is offered to patients who are able to receive this treatment with the goal of a more durable remission and cure. The other interesting thing is that the responses in this study were seen really quickly, generally after 1 or 2 cycles, allowing patients to receive this treatment and then move, when possible, to transplantation relatively quickly. Long-term outcomes suggest that in a portion of patients, the responses are durable, which provides additional data for this drug in patients, whether or not they are transplant eligible or ineligible.
