Clinical characteristics, prognosis and treatment of myelofibrosis patients with severe thrombocytopenia

Impact of ruxolitinib pretreatment on outcomes after allogeneic stem cell transplantation in patients with myelofibrosis

“…prevent relapse, which is a major drawback in ASCT for MF. 8,[12][13][14]17,[34][35][36][37][38][39] In our cohort, we found that out of 103 patients who had evaluable molecular markers for monitoring, 72.8% achieved molecular clearance in the first 100 days. There is no difference between patients who were exposed to ruxolitinib or not.…”

“…Minimal residual disease (MRD) detection is currently extensively investigated in order to achieve deep complete remission, hence prevent relapse, which is a major drawback in ASCT for MF . In our cohort, we found that out of 103 patients who had evaluable molecular markers for monitoring, 72.8% achieved molecular clearance in the first 100 days.…”

Significance of thrombocytopenia in patients with primary and postessential thrombocythemia/polycythemia vera myelofibrosis

“…However, recent reports (14, 15, 22, 23) suggested that patients with severe thrombocytopenia (<50 ×10 9 /L) have particularly short survival. Tam et al has previously reported poor OS (<12 months) of patients with MF and platelets < 50 ×10 9 /L, and considered it as one of the features of accelerated phase (AP) of MF alongside elevated blasts ≥ 10%, and chromosome 17 aberrations, with respective median OS of 12, 10, and 5 months.…”

Authors' reply to comment What should be the endpoints of a symptomatic drug and its control?

“…1 However, the word "inferior" has specific statistical meaning, and key differences in PERSIST-1, COMFORT-I, and COMFORT-II must be recog nised. For example, patients with baseline platelet count of less than 100×10⁹ platelets per L were excluded from COMFORT trials 3,4 but not from PERSIST-1, 2 in which 33% of patients had baseline platelet counts of less than 100×10⁹ platelets per L including 16% with less 50×10⁹ platelets per L, 2 a population with severe symptom burden and shorter survival 5 excluded from the approved indication for ruxolitinib. Secondary endpoints for PERSIST-1 and COMFORT-I included a reduction in total symptom score (TSS) of at least 50%; COMFORT-I used MF-SAF, a 20-item instrument including a quality-of-life measure, and PERSIST-1 used MPN-SAF (initially v1.0, amended to v2.0), which contains the entire MF-SAF plus additional measures.…”