Abstract

This pilot study compared outcomes of patients with severe myelofibrosis (MF) who received pre- and post-SCT Rux (Cohort A), pre-SCT Rux only (Cohort B), and never received Rux (Cohort C), between 6/2012 and 6/2017. The study consisted of 16 patients, 11 male and 5 female, with a median age of 59 years at the time of SCT. The MF scores ranged from 2 to 3 at diagnosis. All patients had constitutional symptoms and splenomegaly at diagnosis except 1 who had prior splenectomy. There were 4 patients in cohort A, 9 patients in cohort B, and 3 patients in cohort C. The median duration of Rux pre-SCT was 8 months; the median duration of Rux post-SCT was 20 months. JAK2V617F mutation was detected in 3/4, 7/9, and 4/4 patients in each cohort. All patients received reduced intensity conditioning regimens. The median time (days) for neutrophil engraftment (ANC>500) was 12.5 in cohort A, 15 in cohort B, and 17 in cohort C. CMV/EBV viremia was detected in 8 patients before day 100, 2 of them in cohort A. No fungal pneumonia or acute GVHD observed in cohort A. The median spleen size reduction one-year post-SCT was 32%, 11%, and 17% in each cohort. At the time of last follow up, all patients in cohort A achieved CR and are alive, only 2 patients in cohort B achieved CR and no one in cohort C achieved a remission. The patients treated with Rux pre- and post-SCT showed better overall outcomes and good tolerability.

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