Myeloid disease: Edited by Martin S. TallmanNovel strategies for patients with chronic myeloproliferative disordersBarosi, Giovannia; Rosti, VittoriobAuthor Information aUnit of Clinical Epidemiology and Center for the Study of Myelofibrosis, Italy bLaboratory of Organ Transplantation, IRCCS Policlinico S. Matteo Foundation, Pavia, Italy Correspondence to Giovanni Barosi, MD, Unit of Clinical Epidemiology, IRCCS Policlinico S. Matteo, Viale Golgi 19, 27100 Pavia, Italy Tel: +39 0382 503636; fax: +39 0382 503917; e-mail: [email protected] Current Opinion in Hematology: March 2009 - Volume 16 - Issue 2 - p 129-134 doi: 10.1097/MOH.0b013e3283257a9e Buy Metrics Abstract Purpose of review This review focuses on new strategies for unmet clinical needs and on new targeted therapies in classical Philadelphia-negative myeloproliferative neoplasms. Recent findings Meta-analyses in essential thrombocythemia documented Janus kinase 2 (JAK2) V617F as being associated with increased risk of thrombosis. New studies reinforced the evidence of leukocytosis as an independent risk factor for thrombosis in polycythemia vera and essential thrombocythemia. In a phase II trial of pegylated interferon-alpha2a in polycythemia vera patients, a decrease of JAK2 mutant expression to undetectable levels was demonstrated. New trials documented that 5-azacytidine and bortezomib have negligible effect in primary myelofibrosis, whereas thalidomide and tipifarnib produce 22 and 44% response, respectively. In primary myelofibrosis, the JAK2 inhibitor, INCB018424, resulted in a rapid and marked reduction in splenomegaly and a clinical improvement, with a modest effect on JAK2 V617F burden. Summary Treating low-risk essential thrombocythemia and polycythemia vera patients presenting with leukocytosis or JAK2 V617F mutation in order to prevent thrombosis deserves a prospective validation. Pursuing clonal remission in polycythemia vera by interferon needs new evidence. Tipifarnib may be added to conventional therapeutic instruments for symptomatic primary myelofibrosis. The results of anti-JAK2-targeted therapies are encouraging as regards symptoms reduction but not clonal remission. © 2009 Lippincott Williams & Wilkins, Inc.