Advances in the Therapy of Chronic Idiopathic Myelofibrosis

Abstract

Abstract Learning Objectives After completing this course, the reader will be able to: Discuss the clinical manifestations of myelofibrosis with myeloid metaplasia and recent developments in the understanding of its pathogenesis.Discuss the most recent clinical trials involving novel therapies for myelofibrosis with myeloid metaplasia.Propose an algorithm-based approach to the treatment of patients with myelofibrosis with myeloid metaplasia. Access and take the CME test online and receive 1 AMA PRA Category 1 Credit™ at CME.TheOncologist.com The molecular basis of chronic idiopathic myelofibrosis (CIMF) has remained elusive, thus hampering the development of effective targeted therapies. However, significant progress regarding the molecular mechanisms involved in the pathogenes is of this disease has been made in recent years that will likely provide ample opportunity for the investigation of novel therapeutic approaches. At the fore front of these advances is the discovery that 35%–55% of patients with CIMF harbor mutations in the Janus kinase 2 tyrosine kinase gene. Until very recently, the management of patients with CIMF involved the use of supportive measures, including growth factors, transfusions, or interferon, and the administration of cyto-reductive agents, such as hydroxyurea and anagrelide. However, several trials have demonstrated the efficacy of antiangiogenic agents alone or in combination with corticosteroids. In addition, the use of reduced-intensity conditioning allogeneic stem cell transplantation has resulted in prolonged survival and lower transplant-related mortality.