Assessment of Clinical Outcome of Patients With Primary Myelofibrosis Treated ‎With Ruxolitinib in the Real-World Practice

BACKGROUND: Primary myelofibrosis (PMF) is a disease that characterized by bone ‎marrow fibrosis which ‎may ‎result sometime in cytopenias and extramedullary ‎hematopoiesis causing massive ‎splenomegaly. Ruxolitinib (Rux) therapy that targeted ‎Janus Kinase-2 receptor and approved ‎for treatment of primary myelofibrosis. ‎

OBJECTIVE: evaluate the clinical ‎outcome of patients with ‎primary myelofibrosis ‎receiving Ruxolitinib treatment as ‎a compassionate use program ‎and compare this treatment with the best ‎available treatment (BAT).‎

METHODS: ‎This is a retrospective case series conducted at the national ‎center of hematology /‎‎Mustansiriyah University. The enrollment of patients started ‎in May 2014 and ended in May ‎‎2017. There were ‎‎22 patients diagnosed with PMF (7 on ‎the Ruxolitinib arm and 15 on best ‎available treatment arm). The ‎treatment response ‎was evaluated according to consensus criteria ‎of IWG-MRT 2013 in primary ‎‎myelofibrosis.

RESULTS: In this study 3 out of 7 patients on Ruxolitinib arm showed reduction in spleen size and reduction in anemia. In addition to ‎that 4 patients ‎showed clinical response specifically in spleen ‎size (with 65% reduction from baseline during the first three months of treatment).

CONCLUSION: Rux is effective and safe to treating ‎primary myelofibrosis with ‎symptomatic splenomegaly.