Marina Kremyanskaya, MD, PhD, assistant professor of medicine, hematology, and medical oncology, Mount Sinai Hospital, discusses the need for disease-modifying treatment approaches for patients with myeloproliferative neoplasms. 

Ruxolitinib (Jakafi) has been the standard-of-care treatment for patients with myelofibrosis, as well as polycythemia vera, since it received regulatory approval from the FDA, according to Kremyanskaya. Patients are often treated with the agent either soon after diagnosis, when they become symptomatic, or when they develop other disease characteristics that provide rationale to initiate therapy, Kremyanskaya adds. To achieve a longer duration of response and disease modification, novel agents are often studied in combination with ruxolitinib, Kremyanskaya says. 

Although ruxolitinib has been shown to improve both quality of life and survival for these patients, it does not modify disease with respect to providing a cure or halting disease progression, Kremyanskaya adds. Newer agents that may provide these benefits are currently being investigated on clinical trials, often in combination with ruxolitinib, to achieve optimal benefit, Kremyanskaya concludes.

Videos

Marina Kremyanskaya, MD, PhD, discusses the need for disease-modifying treatment approaches for patients with myeloproliferative neoplasms. 

Dr. Kremyanskaya on the Need for Disease-Modifying Approaches in MPNs

Marina Kremyanskaya, MD, PhD, assistant professor of medicine, hematology and medical oncology at Mount Sinai Hospital, discusses responses with CPI-0610 in patients with myelofibrosis.

Data from the phase II MANIFEST trial suggest that CPI-0610 with or without ruxolitinib (Jakafi) may have disease modifying effects in patients with myelofibrosis who have progressed on or had an inadequate response to ruxolitinib.

CPI-0610 alone and in combination with ruxolitinib led to improved hemoglobin levels in anemic patients, improved bone marrow fibrosis, spleen volume reduction, and symptom improvement in the majority of patients. Additionally, 2 patients who were transfusion-dependent at baseline achieved transfusion-independence with the combination of CPI-0610 and ruxolitinib, says Kremyanskaya.

Both the monotherapy and combination regimens appear to be well-tolerated in this patient population.

Marina Kremyanskaya, MD, PhD, discusses responses with CPI-0610 in patients with myelofibrosis.

Dr. Kremyanskaya on Responses With CPI-0610 in Patients With Myelofibrosis

Marina Kremyanskaya, MD, hematologist/oncologist at Mount Sinai Hospital, discusses the effect of CPI-0610 in patients with myelofibrosis.

In the MANIFEST trial, patients with myelofibrosis who are refractory to or intolerant of ruxolitinib (Jakafi) were treated with the BET inhibitor CPI-0610 in combination with ruxolitinib or CPI-0610 alone. Preclinical data with myelofibrosis models suggest that a combination of BET and JAK inhibitors can result in synergistic reduction of splenomegaly, bone marrow fibrosis, and improvement in anemia, says Kremyanskaya.

It is hopeful that this combination will demonstrate efficacy in the clinical setting, Kremyanskaya concludes.

Marina Kremyanskaya, MD, discusses the effect of CPI-0610 in patients with myelofibrosis. 
 

Marina Kremyanskaya, MD

Articles

Dr. Kremyanskaya on the Need for Disease-Modifying Approaches in MPNs

Dr. Kremyanskaya on Responses With CPI-0610 in Patients With Myelofibrosis

Dr. Kremyanskaya on Effect of CPI-0610 in Patients With Myelofibrosis