Efficacy and Safety of Ruxolitinib in Primary Myelofibrosis Patients with Abdominal Thrombotic Complications: A Case Series

BACKGROUND. Primary myelofibrosis (PMF) is a clonal myeloproliferative neoplasm of hematopoietic stem cells characterized by excessive production of proinflammatory profibrogenic cytokines providing runaway proliferation and accumulation of mature blood cells along with developing reticulin/collagen bone marrow fibrosis. Long-term survival rates shown by PMF patients are considerably lower than population-based survival rates, not least because of life-threatening portal vein thrombotic complications and Budd-Chiari syndrome. Ruxolitinib is the first pathogenic targeted drug blocking the JAK2 kinase activity in PMF. This drug persistently reduces the spleen size and the intensity of constitutional symptoms. However, for this category of patients the role of ruxolitinib in thrombosis risk reduction has until now remained underresearched. AIM. To assess the efficacy and safety of ruxolitinib in PMF patients with abdominal thrombosis. MATERIALS & METHODS. Case reports of 4 PMF patients treated with ruxolitinib. At disease onset, portal vein thromboses were detected, that required liver transplantation in 2 out of 4 cases. RESULTS. The present paper reports success with ruxolitinib in 4 PMF patients. Ruxolitinib dose modification as well as side effect control, when administered concurrently with other immunosuppressive drugs, facilitates health status stabilization, spleen reduction, and improvement of constitutional symptoms. CONCLUSION. Ruxolitinib therapy in PMF patients with portal and hepatic vein thrombosis promotes better quality of life and reduces the risk of repeated thrombotic complications. After liver transplantation, PMF patients need further optimization of dosing schedule of chemotherapy and immunosuppressive therapy with most careful monitoring of adverse events.