Classic BCR::ABL1 negative myeloproliferative neoplasms (MPN) include polycythemia vera, essential thrombocythemia, and myelofibrosis. These diseases arise from the clonal proliferation of a single hematopoietic stem cell which has acquired a driver mutation. They are considered chronic diseases, manifesting by symptoms listed in the MPN-10 score, thrombosis or, more rarely, hemorrhage. These diseases can progress to myelofibrosis or blast phase, with a guarded prognosis. Diagnosis requires bone marrow biopsy. First-line treatments consist of low-dose aspirin, phlebotomies in polycythemia vera, anticoagulation, and cytoreduction using hydroxyurea in certain situations. Treatments for myelofibrosis include allogeneic stem cell transplantation for curative purposes or JAK2 inhibitors to reduce both symptoms and spleen size. Recent research advances in a better understanding of the mutated protein have led to the development of new treatments aimed at eradicating the mutated cells, including human monoclonal antibodies and bispecific antibodies. These promising targeted treatments are currently undergoing clinical trials.
Keywords
Myeloproliferative neoplasm, MPN-10, thrombosis, diagnostic criteria, bone marrow biopsy, prognosis, treatments in clinical practice, future targeted therapies