Journal d'hématologie et des maladies thromboemboliques
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Descriptive, Comparative Study of Filipino Myelofibrosis Patients: An Agematched Comparison of Patients on Ruxolitinib vs. Best Available Therapy

Flordeluna Zapata-Mesin, Ma Rosario Irene D. Castillo, Priscilla B. Caguioa, Honorata G. Baylon, Narcisa Sonia C. Comia4, Catherine C. Rosales and Susie L. Ponce

Introduction: Myelofibrosis is characterized by bone marrow fibrosis, cytopenias and extramedullary hematopoiesis. Ruxolitinib is a Janus Kinase-2 inhibitor recently approved for myelofibrosis. In the Philippines, it was made available through the compassionate use program.

Methods: This is a multicentre, retrospective case series of eleven patients in the compassionate use program and eleven age-matched historical controls on best available therapy.

Results: The median age was 54.5+7.33, 82% females. Using the International Working Group on Myelofibrosis Research and Treatment (IWG-MRT) consensus criteria of 2013, 1 out of the 11 patients had both spleen response and anemia response, achieving transfusion independence for > 12 weeks. Nine patients experienced clinical improvement specifically spleen response. Decrease in spleen size was seen in the first 3 months of ruxolitinib use with a mean of 53.5% from baseline. Hematologic toxicities were anemia and thrombocytopenia while nonhematologic were gastrointestinal disorders 5(45%) and hepatic dysfunction 4(36%). These patients were matched by age with historical controls on best available therapy. There was a remarkable decrease in spleen size with ruxolitinib (53.5%) compared to BAT group (18% from baseline). Thrombocytopenia and anemia occurred more frequently in the ruxolitinib group.

Conclusion: Ruxolitinib is a viable treatment option for Intermediate-2 to high risk myelofibrosis with massive splenomegaly. Anemia and thrombocytopenia were common hematologic toxicities during therapy.