Case Studies in Myeloproliferative Neoplasms - Episode 12

Case 2: Safety and Efficacy of Ruxolitinib in High Risk MF

Dr Jamile Shammo presents clinical trial data on the efficacy and safety of ruxolitinib in patients with high risk myelofibrosis.

Jamile M. Shammo, MD: Those are the grade 3 and 4 toxicities. These data have already been shown. I’m not showing you anything new. This is what would be expected for a JAK inhibitor. There was a study done by the Italian group. The idea of the EXPAND study was to define the maximum safe starting dose [MSSD] in patients who have a platelet count that is between 50,000 and 100,000 per mm3. In the COMFORT trials, patients had to have a platelet count above 100,000 per mm3 to be enrolled. In this analysis, they had it between 50,000 and 100,000 per mm3. They had 2 different strata to identify the ruxolitinib dose that could be used in this patient population. They split the patients into 2 groups. The numbers are somewhat small, and they did it in escalation-type fashion and looked at the heme toxicity. Even though the ruxolitinib dose was 10 mg, the ideal that they established, there was a great deal of dose reduction and modification in both strata in the order of 40% and 60%. Even though this is the dose that’s recommended for this patient population, you still need to be very careful with monitoring those patients and doing due diligence with dose reduction and holding. It was clear that if you use those doses, you could have decent spleen responses and changes in symptoms score. But it’s clear that patients who are in stratum 1 and have a higher platelet count can have more meaningful reduction in the symptom score then those who have a lower platelet count.

This transcript has been edited for clarity.