Jakafi Represents the Present — and a Window to the Future — in Myelofibrosis

Though patients with myelofibrosis still do not have a large number of therapy options, the approval and success of Jakafi is bringing optimism to those treating this population. 
Though there are few therapeutic options for patients with myelofibrosis, Jakafi (ruxolitinib) represents an important look towards the future, according to Maureen E. Thyne, a physician assistant in the Outpatient Leukemia Service at Weill Cornell Medical College.

Jakafi is an oral Janus-associated kinase 1 (JAK1) and JAK2 inhibitor medication approved for the treatment of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis.

To gain insight into the treatment of the disease and the clinical utility of Jakafi, CURE interviewed Thyne at the 2016 Oncology Nursing Society Congress.

What were you tasked with presenting here at the meeting?

I'm here presenting on Jakafi (ruxolitinib) and its use in myelofibrosis — specifically, its use in intermediate and high-risk disease. This is a rare disease. Myelofibrosis affects fewer than 20,000 people in the country, so it's not particularly common, and there hadn't been any effective, FDA-approved therapies until Jakafi came along. It's exciting and it's done a great job in this patient population.

Prior to Jakafi coming onto the scene, what did treatment for patients with myelofibrosis look like?

We were able to use some combinations of transfusions, supportive care and chemotherapeutics that were non-specific to myelofibrosis. The only potential for cure was through bone marrow transplant, and that remains to be the case. With Jakafi, we have a therapy that's been designed specifically to help symptom burden and specific side effects from the disease. These patients typically experience enlarged spleens, so Jakafi not only shrinks them but also improves symptom status.

How are patients doing on the drug?

It's been several years since the approval, but the results have been impressive. Overall survival has been improved, and we didn't expect to see the level improvement that we have so quickly. We'll have five-year data later this year, so I'm anxious to see those results.

What are the next steps in myelofibrosis?

It's tough with rare diseases across the board, and myelofibrosis certainly resides in that group. There are limited therapies directed at the diseases — Jakafi is the only one approved now, but there are several others in clinical trials for patients who have been exposed to Jakafi and have not responded or have progressed through their responses. It's an exciting time in the myeloproliferative neoplasm world.

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