Marina Kremyanskaya, MD, PhD, a hematologist-oncologist at Mount Sinai Hospital, discusses unique findings from the phase II trial investigating the effect of CPI-0610, a bromodomain and extra-terminal protein (BET) inhibitor, as treatment for patients with myelofibrosis (MF) who previously progressed on ruxolitinib (Jakafi).

Eighteen patients with MF who were previously treated with ruxolitinib and had either progressed or did not achieve an adequate response were given either CPI-0610 as monotherapy or in combination with ruxolitinib in this clinical trial. Kremyanskaya says it was interesting to see an improvement in hemoglobin levels in both arms.

In addition to seeing responses in anemia, there were transfusion-dependent patients at baseline who became transfusion independent after receiving the combination regimen. There was also a degree of improvement in splenomegaly across all patients, as well as symptom scores.

Kremyanskaya also notes an improvement in bone marrow fibrosis in patients that had sequential bone marrow studies available. This is exciting because the combination seems to have an effect on the bone marrow environment of patients with MF, she adds.

Overall, the most common adverse events included mild diarrhea, nausea, vomiting, and reversible and non-cumulative thrombocytopenia. Spleen volume was also reduced with CPI-0610 in 14 of 16 evaluable patients.

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