Real-World Use of Ruxolitinib in Polycythemia Vera

Experts give real-world insight in using ruxolitinib in patients with polycythemia vera. 

Srdan Verstovsek, MD, PhD: If we all agree that the goals of therapy in polycythemia [P] vera are far beyond just controlling the red blood cell count, then we are talking about therapies like ruxolitinib that provide multiple benefits at the same time. In my experience with ruxolitinib, in my own clinic, we would certainly aim to eliminate a need for phlebotomy to control the red blood cell count. The white blood cells and platelets usually follow suit, because it is antiproliferative medication. You’ll get very good control of blood cell count, and we know from the studies that 60% of patients eliminate the need for phlebotomy. Over time, control of the white cells and platelets get better, to the same level, if not more. But the main benefit, in my experience, is the control of patient’s quality of life.

When a patient has resistant or refractory disease to hydroxyurea, the counts are not controlled very well. Whether spleen or symptoms, that is not a very good situation. Control of the blood cell count alone, while it’s of primary interest, does not do full service to the patient. Ruxolitinib, being an anti-inflammatory medication, does well on the quality-of-life issues. Nine of 10 patients eliminate itching. Night sweating, low-grade fevers, some have bone aches and pains, or related symptoms to a big spleen—all these are controlled in a great majority of the patients.

We look at the patients who by clinical study design were not responding very well. Even in patients who are nonresponders by not optimally controlling the blood cell count, the control of the quality of life is so good. One needs to balance these 2 benefits in every practice: control the counts, control the quality of life. It’s best to have both. But quality of life is really important, if not as important, as the blood cell count control in the second-line setting, for which ruxolitinib is approved.

Ruben A. Mesa, MD: My experience with ruxolitinib has been very favorable for its use in polycythemia vera, both as an investigator on the RESPONSE study but also in terms of clinical practice. One, it is helpful in terms of control of counts, helping individuals become phlebotomy independent, controlling leukocytosis and thrombocytosis. Second, it is almost uniquely situated in terms of its impact for improving P vera–related symptoms, like fatigue and pruritis. Indeed, for pruritis there’s probably nothing more effective for very difficult pruritis than ruxolitinib, and for night sweats as well as improving spleen-related symptoms when present. You wrap all those things together. It really is quite an effective therapy.

In utilizing ruxolitinib we do need to be mindful of a couple of things. One, risk of shingles is genuine. It’s not overwhelming; it’s less than 10%, but its real. I do counsel my patients to receive the current shingles vaccine, particularly the newest 1, which is a nonlive vaccine. Two, patients with MPNs [myeloproliferative neoplasms], in particular patients on ruxolitinib, have a higher rate of nonmelanoma skin cancers. Good skin care and use of sunscreen help, but also if they’re older or have had issues, involvement of a dermatologist as part of the health care team is important monitoring for these patients.

Pankit Vachhani, MD: I restrict my use of ruxolitinib in patients with polycythemia vera to those who are resistant, refractory or intolerant to the first-line treatment, which typically is hydroxyurea. This is, largely speaking, in line with the FDA-approved use of ruxolitinib in polycythemia vera. There are no large studies of real-world data of ruxolitinib use in polycythemia vera. However, from my own practice, I have seen that patients use their perceptions and their experience as well as my experience of ruxolitinib and use it in patients with polycythemia vera. The outcomes in those patients is, largely speaking, in line with that from the RESPONSE and RESPONSE-2 clinical trials.

When I’ve used it, I have seen that my patients have a remarkable improvement in their symptomatology. Typically, this happens within weeks. There are 2 areas where I’ve seen rapid improvement of their symptoms, 1 being the cytokine symptom cluster-related symptoms. Those include pruritus and daytime or nighttime sweats but also splenomegaly symptom cluster-related symptoms. Early satiety or abdominal discomfort patients are rather fast in noting improvement in these symptoms. In fact, I’m not seeing patients become worse on ruxolitinib with regard to symptoms.

Transcript edited for clarity.

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