SIDEBAR: Novel Agents Are Still Needed to Address Cytopenias in Myelofibrosis

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Despite improving splenomegaly and symptoms in patients with myelofibrosis, ruxolitinib has not been helpful for managing cytopenias in this population, according to Wendy Stock, MD, of the University of Chicago.

“Additional trials are underway with novel [JAK] inhibitors, with some hints about improvements in cytopenias,” she said. For example, in a phase I/II trial, CYT387 improved anemia in half of patients, including 58% of those who were transfusion dependent.1

“Novel agents are still desperately need for the patients with disease progression and worsening cytopenias,” Dr. Stock contended. One showing promise in clinical trials is pomalidomide, which appears especially effective for anemia in patients with JAK2 mutations. And epigenetic modulators, such as decitabine (Dacogen), are showing impressive activity in small studies.

Future Trials

“Could you use these drugs in combination with the JAK inhibitors?” she asked. “I think those are the trials that we are going to see as the next generation of studies in these disorders.”

Dr. Stock concluded, “So we have hope for symptom management, some suggestion of actual change in natural history of the disease, but really a long way to go.” Nonetheless, this is “a very exciting time for the myeloproliferative disorders, which have been on the back burner for many years.” ■


1. Pardanani AD, Caramazza D, George G, et al: Safety and efficacy of CYT387, a JAK-1/2 inhibitor, for the treatment of myelofibrosis. 2011 ASCO Annual Meeting. Abstract 6514. Presented June 3, 2011.

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