Clinical Scenario: Myelofibrosis Treatment in the Second-Line Setting


John Mascarenhas, MD, presents a clinical scenario of a 73-year-old man with myelofibrosis and a low platelet count.

John Mascarenhas, MD: We’ll move on to the second case. This is a 73-year-old man who presented with bone pain and increasing fatigue. He also noticed a change in his eating habits, and he has abdominal discomfort that’s vague in description. His past medical history is significant for a diagnosis of primary myelofibrosis, which was diagnosed approximately 1 year ago. His baseline platelet count was 181,000 [per mm3] and his hemoglobin was 10 g/dL.

He was started on ruxolitinib and is currently on 10 mg twice a day. Initially, his spleen was 7 cm below the left costal margin. It’s now down to 4 cm. So, he hasn’t had a nice spleen response. His platelet counts have dropped from 181,000 to 68,000 [per mm3], and his hemoglobin is down to 7.4 g/dL. His bone pain and abdominal pain have resolved and his energy level is improved. He’s had improvement in spleen and symptoms, but his platelet count and hemoglobin are lower. Srdan, I’ll start with you. What are your initial thoughts when you hear about a case like this?

Srdan Verstovsek, MD, PhD: It’s quite a common experience. You start with ruxolitinib in [patients] who have a relatively decent blood cell count. His platelets are 181,000 [per mm3] and a hemoglobin of 10 [g/dL]. You don’t need to worry much about the dose adjustments from the beginning, although he was given 10 mg twice a day, 1 level below the normal dose of 15 mg twice a day, based on platelet number. That would be satisfactory. His spleen reduced somewhat. Importantly, his quality of life improved a lot. His bone pain and abdominal pain resolved. His energy level is improved. That’s at the expense of lowering the blood cell count. Which one is more important? You have platelets of 68,000 [per mm3] and a hemoglobin of 7.4 [g/dL]. He may or may not be getting blood [transfusions], but his quality of life is improving or normalized, and his spleen is smaller.

You have a conundrum here. What should I do? Let’s assume that a hemoglobin of 7.4 [g/dL] calls for transfusions, which he didn’t need before. The quality of life is good. If I decide to change therapy today, I don’t know whether the next therapy will have that pronounced effect on the quality-of-life improvement. Should I combine ruxolitinib with an anemia drug and improve the hemoglobin from 7.4 to 8.4 or 9.4 [g/dL] and eliminate the need for occasional transfusions, accepting the platelet number of 68,000 [per mm3]? Because I have a quality-of-life benefit and I don’t want to discount that. If that isn’t going to be durable, and the abdominal pain is coming back and the spleen is growing, that would call for an automatic change. But I am OK with maintaining patients on ruxolitinib for the time being, as long as the quality of life is improved, and trying to figure out how to improve the hemoglobin to prevent any transfusions.

Transcript edited for clarity.

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