Efficacy of RUX treatment
liver dysfunction, PET-MF, subentities, PPV-MF, Kidney dysfunction, PMF
KIDNEY DYSFUNCTION, THROMBOCYTOPENIA
The median RUX dose administered was 23.77 mg/day. Patients were started on a median dose of 30 mg/day (equivalent to 15 mg twice daily [BID]), not differing between the three subentities. 39.5% and 25.4% of patients received 30 mg/day or 40 mg/day, respectively; however, 35.1% of patients were dosed with 20 mg/day or less, and 20.8% of patients received 10 mg/day or less. There were no relevant differences between PMF, PPV-MF, or PET-MF patients regarding average, start, or last doses of RUX. Median treatment duration was 1.5 years, with a slightly shorter duration in patients with PMF and PET-MF than in PPV-MF patients. As expected, patients who dropped out showed a lower median average dose and a lower median treatment duration than patients who had completed the trial (20.8 mg/day vs 25.7 mg/day, 67% of patients remained on RUX continuously while 33% of patients interrupted RUX between one and nine times during the trial. The most frequent reasons for RUX interruption were AEs, particularly thrombocytopenia. Interestingly, patients who dropped out had not interrupted RUX significantly more frequently than those completing the trial. On the other hand, only 29.5% of patients received the same dose throughout the trial, while fewer than 20% each were subjected to at least one or two dose adjustments, respectively, with the rest experiencing up to 18 dose adjustments. Most dose adjustments were due to AEs, particularly thrombocytopenia. Kidney dysfunction was not reported as a reason for dose adjustment at all, and liver dysfunction was cited as the reason for dose adjustment once.Only 31.5% of patients completed the full 36 months on trial, while 54.3% of patients dropped out before reaching this time point. At the time of analysis, 14.2% of patients were still receiving treatment. Reasons for early drop out from the trial can be found in Table Study completion status at data-cutoff and reasons for early study discontinuation (Abbreviations: Among patients who dropped out, the combined fraction of high-risk and int-2–risk patients was over-represented (68.4% of drop-out patients); however, median study duration was comparable between the IPSS risk groups: (low: 17.5 months, int-1: 17.9 months, int-2: 17.7 months, high: 17.9 months; total: 17.8 months).