SOHO State of the Art Updates and Next Questions | Updates on Myelofibrosis With Cytopenia.

Abstract

Myelofibrosis (MF) is a rare hematologic malignancy that is characterized by dysregulation of the JAK-STAT pathway resulting in fibrosis of the bone marrow, splenomegaly, and abnormalities in peripheral blood counts including anemia, leukocytosis, and thrombocytopenia. This disease has 2 phenotypic extremes - myeloproliferative and cytopenic. Cytopenic myelofibrosis presents with pronounced cytopenia and a different landscape of genetic mutations which results in worse clinical outcomes and a poor prognosis. Patients with cytopenic MF are at high risk of developing various complications like bleeding, infections, and transfusion dependency. Historically, the only Federal Drug Administration (FDA) approved therapy was ruxolitinib, a JAK1/2 inhibitor, which improved constitutional symptoms and splenomegaly, however, exacerbated anemia and thrombocytopenia.^1,2 There were very few options for patients with anemia and thrombocytopenia, and supportive treatments for these problems lack efficacy. Fortunately, there are newer treatment options which may allow for treatment of the symptoms and splenomegaly in the setting of cytopenias and even improve cytopenias. This up-to-date review not only highlights the prevalent options in therapeutic marketplace, but also sheds light on the significant unmet need of addressing anemia and thrombocytopenia in cytopenic MF.