Journal of Blood Disorders & Transfusion

What is new in myelofibrosis?

3^rd International Conference on Hematology & Blood Disorders

November 02-04, 2015 Atlanta, USA

Natalia Curto-Garcia

Royal Free London NHS Foundation Trust, UK

Posters-Accepted Abstracts: J Blood Disord Transfus

Abstract:

The discovery of JAK2 mutation a decade ago has marked a crucial change in our understanding and knowledge of the pathogenesis, prognosis and management of myeloproliferative disorders, particularly Myelofibrosis. Since then, new mutations have been identified and added as prognostic factors in the international scoring system. A new group of drugs have been developed to target the main pathways involved in Myelofibrosis. Ruxolitinib was the first JAK2 inhibitor that demonstrated rapid symptomatic improvement and spleen reduction as reflected in the COMFORT I-II studies. Furthermore, a recent study reported the efficacy and safety of Ruxolitinib therapy in COMFORT I patients after three years. In recent years, new medications are under investigation as single agent or in combination with Ruxolitinib such as Pacritinib (JAK2-FLT3 inhibitor), Panobinostat (Pan-Deacetylase inhibitor) or Smo inhibitors. The preliminary results of these drugs showed promising responses in splenomegaly reduction, improvement in symptoms and reduction in JAK2 mutant allele; although important side effects have also been described for these drugs. On the other hand, Myelofibrosis presents with significant fibrosis in the bone marrow that affects the haematopoiesis and as a consequence, patients develop marked cytopenias and related symptoms. Recently, a new specific anti-fibrotic activity agent is under investigation in a few clinical trials. The current landscape of therapy for Myelofibrosis is based on JAK2 inhibitors however, promising agents may contribute to achieve our final goal of cure in this disease, probably with a combination strategy.

Biography :

Email: natcurto@gmail.com