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Myeloproliferative Neoplasms Case Studies

Case 1: A Patient with Disease Progression on Hydroxyurea

Published Online:Oct 12, 2016
Srdan Verstovsek, MD, provides information on the diagnosis and treatment of patients with polycythemia vera.

Srdan Verstovsek, MD, provides information on the diagnosis and treatment of patients with polycythemia vera: Case 1

Srdan Verstovsek, MD: When we talk about failing hydroxyurea, there are different ways of failing hydroxyurea. Either there is increased risk of phlebotomy, or there is a real need for phlebotomy, that means the red blood cells are starting to grow without control despite the optimal dose of hydroxyurea. There is a progressive leukocytosis, or platelets start to go up. Patients may also have increase in the spleen or develop new symptoms that are not controlled well with hydroxyurea. All of these factors would indicate a failure of hydroxyurea. In that setting, ruxolitinib would help patients a lot, and we have patients, a number of them, who come through MD Anderson for a second opinion in these clinical scenarios. In that case, we would suggest ruxolitinib because it has been shown to markedly improve the symptoms within a few weeks, increase their quality of life, decreasing the spleen if it’s enlarged, and controlling the red blood cells, white blood cells, and platelets. So, we would expect at least about 60% to 80% of the patients to have a real benefit in controlling all five aspects that we look at within polycythemia vera to say this is a real clinical benefit.

Practically, that means elimination of the phlebotomy need. That means elimination of our supportive medications for control of itching, or fatigue, or night sweating. That also means increasing weight in patients that are losing weight because of increasing in the spleen. So, you can imagine the whole spectrum of benefits with ruxolitinib beyond just controlling the red blood cell count.

The starting dose for polycythemia vera is 10 mg twice a day for all patients. This is different than in myelofibrosis for which ruxolitinib is also used. Here, everybody starts with 10 mg twice a day. Expectation is that about 10% of the patients may need less. That means 5 mg twice a day. And, about 60%, 65% of patients may need more, meaning 15 mg twice a day, 20 mg twice a day, and some even 25 mg twice a day. But, the medication should not be used daily. It has to be taken twice a day because it has a short life in the body, about three-and-a-half hours’ half-life. It doesn’t really work if it’s taken only once a day.

In terms of disease control with ruxolitinib after hydroxyurea, for which this medication now is approved and is widely available throughout the United States, my experience was excellent. Patients really rapidly developed improvements in quality of life. If they have an enlarged spleen, that would go away within 2 months usually. There would be significant clinical benefits in all aspects of polycythemia vera.

In terms of the symptom control in patients that have longstanding history of polycythemia vera, and do not do well on hydroxyurea for which ruxolitinib is used, that is one of the most important aspects that I see right away. The symptoms are really uncontrolled in patients that have advanced features. This is poor quality of life that limits their enjoyment of everyday activities, enjoyment with the family. When you can eliminate itching, fatigue, weakness, night sweating, and enlargement of the spleen within a few weeks, that is marked improvement that really is gratifying for everybody.

The performance status and quality of life of the patients on ruxolitinib in general improves markedly in a short order. Performance status, that means how they perform whether they can enjoy quality of life by doing activities that they used to do and cannot do anymore, like go fishing or golfing. This is the real benefit that you see. The quality of life improves within few weeks to the level that these patients have not experienced for years.

Ruxolitinib is an approved therapy for second-line therapy in polycythemia vera, patients that are intolerant or resistant to hydroxyurea. Now, there are some problems in developing…within that group of patients, patients that have some liver damage or have some kidney damage need to be monitored very closely. The dose of ruxolitinib needs to be adjusted because it is affected by these organ functions. So, a liver function test should be performed before therapy, and on therapy if necessary. There is also a need to look at the kidney function because it’s excreted through the kidney and dose adjustments are necessary. In patients that have a history of chronic hepatitis, or a history of TB or some other atypical chronic infections, one needs to be cognizant of those because there might be a reactivation of hepatitis or TB. And certainly a consult to an infectious disease specialist is in order if one contemplates therapy with ruxolitinib in a patient that is known to have exposure or a history of chronic infections.


Case 1: A Patient with Disease Progression on Hydroxyurea

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