EHA 2021 | Results from MANIFEST Phase II study of pelabresib for anemia associated with myelofibrosis
Srdan Verstovsek, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, discusses results from the MANIFEST (NCT04603495) Phase II study of pelabresib for anemia associated with myelofibrosis. Myelofibrosis patients in Arm 1 were treated with pelabresib monotherapy. In transfusion dependent (TD) patients, 21% of patients achieved transfusion independence (TI). In non-TD patients, 59.1% of patients achieved a mean hemoglobin (Hgb) increase ≥1.5g/dL sustained over a 12-week transfusion-free period. In patients with an anemia response, observed Hgb increase and TI achievement have been generally associated with an increase in reticulocyte count and/or increased CD71+ progenitor cells in the bone marrow. Pelabresib was generally well tolerated. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.
Transcript (edited for clarity)
Pelabresib, or CPI-0610, is the BET or bromodomain inhibitor with activity in myelofibrosis in different clinical settings. Primarily, it was studied for improvement in the spleen and symptoms, two out of three cardinal problems with patients with myelofibrosis. And it was active as a single agent in a second-line setting, active in the frontline setting in patients that are started on JAK inhibitors...
Pelabresib, or CPI-0610, is the BET or bromodomain inhibitor with activity in myelofibrosis in different clinical settings. Primarily, it was studied for improvement in the spleen and symptoms, two out of three cardinal problems with patients with myelofibrosis. And it was active as a single agent in a second-line setting, active in the frontline setting in patients that are started on JAK inhibitors. So, in combination, more active in patients who are already on the JAK inhibitor but have a suboptimal response.
What we are presenting at the European Hematology Association meeting is a very interesting additional benefit of pelabresib in patients with myelofibrosis, and that is on anemia. While we are preoccupied with spleen and symptoms, anemia is the third major problem, and this drug appears to be able to improve the red blood cell count number of patients that are treated with ruxolitinib JAK inhibitor, or even in the single agent setting on its own. That is a very valuable additional benefit that becomes overlooked by many.
The increase in red blood cell count eliminates transfusion requirements in many patients, which is the actual real clinical benefit that we want to see in our patients. Increasing just in number of red blood cell count without real clinical impact is questionable. Clinical impact of elimination or decreasing transfusion requirement is real. So, therefore in the future, if this drug has a future, and I think it does, significant future in armamentarium of medications for myelofibrosis, we should account for its ability to counteract the spleen and the symptoms and anemia, all the three cardinal problems with these patients.
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