Haema 2012; 3(1): 99-108
by Despoina Pantelidou, Maria Papaioannou
Haematology Unit of 1stDepartment of Internal Medicine, AHEPA University Hospital, Thessaloniki
Abstract
Despite the dramatic improvement on the understanding of the pathophysiology of Philadelphia negative classical Myeloproliferative Neoplasms (Ph-MPN) following the description, in the last years, of recurrent molecular abnormalities represented mainly by the V617F mutation in JAK2 ex- on 14, the treatment of MPN remains a challenge. In practice, therapeutic recommendations are largely based on consensus of experts appointed by the European LeukemiaNet (ELN). Two consensus conferences were convened to produce recommendations on the management of Ph(-) MPN, including monitoring, response definition, first- and second-line therapy, and therapy for special issues. The first step for the management of polycythemia vera (PV) and essential thrombocythemia (ET) is to identify the potential risk to develop major thrombotic or hemorrhagic complications and should be defined as high risk if age is greater than 60 years or there is a history of previous thrombosis. High-risk patients with PV should be managed with phlebotomy, low-dose aspirin, and cytoreduction, with either hydroxyurea or interferon at any age. High-risk patients with ET should be managed with cytoreduction, using hydroxyurea at any age. Monitoring response in PV and ET should use the ELN clinicohematologic criteria. The treatment strategy of patients with primary myelofibrosis (PMF) starts with the risk stratification and depends on the clinical symptoms and signs. Corticosteroids, androgens, erythropoiesis-stimulating agents, and immunomodulators are recommended to treat anemia of PMF, whereas hydroxyurea is the first-line treatment of PMF-associated splenomegaly. Indications for splenectomy include symptomatic portal hypertension, drug-refractory painful splenomegaly, and frequent red blood cell transfusions. The risk of allogeneic stem-cell transplantation–related complications is justified in transplantation-eligible patients whose median survival time is expected to be less than 5 years.