Abstract
Abstract
Myelofibrosis, a rare, chronic myeloproliferative neoplasm associated with progressive bone marrow fibrosis and extramedullary hematopoiesis, is a life-shortening disease with a heterogeneous clinical presentation that poses significant challenges for the advanced practitioner in oncology in patient assessment and management. Common clinical manifestations of myelofibrosis are splenomegaly, various spleen-related and constitutional symptoms, and anemia. Optimal management includes regular spleen size assessment by palpation as well as monitoring symptoms and quality of life with validated instruments to evaluate therapeutic response and disease progression. The Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib, which has been shown to provide effective and lasting spleen size reduction and symptom mitigation as well as a survival advantage compared with placebo and best available therapy in randomized controlled clinical trials, has been approved for the treatment of myelofibrosis in more than 80 countries worldwide. However, ruxolitinib is associated with dose-dependent cytopenias, particularly thrombocytopenia, attributable to its mechanism of action; therefore, blood cell counts should be monitored and dose adjusted as necessary. Through continual patient education and support, advanced practitioners in oncology can help patients to remain on therapy and ultimately maximize overall treatment benefit.
