hematology and oncology
Primary Myelofibrosis (PMF)
Key Points
Myelofibrosis is excessive bone marrow fibrosis, often with loss of hematopoietic cells and consequently extramedullary hematopoiesis. Myelofibrosis is often primary but may occur secondary to a number of hematologic, malignant, and nonmalignant disorders, including polycythemia vera and essential thrombocytosis. Primary myelofibrosis is a clonal hematopoietic stem cell disorder and often involvesJAK2,CALR, orMPLmutations. Diagnose with blood count, examination of peripheral blood smear and bone marrow, and molecular testing forJAK2,MPL, and/orCALRmutations. Some patients have an indolent course and do not require therapy immediately, but some patients have a rapidly progressive downhill course with short survival. Ruxolitinibis the therapy of choice for control of symptoms; allogeneic stem cell transplantation is of benefit in selected cases. Early in the disease, pegylated interferon can be effective. 