Ruxolitinib as an emerging treatment in myelofibrosis

Robyn M Emanuel,1 Holly L Geyer,1 Ruben A Mesa2 1Department of Internal Medicine, 2Department of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ, USA Abstract: Essential thrombocythemia, polycythemia vera, and myelofibrosis belong to the class of Bcr-Abl negative hematologic neoplasms, which ar...

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Autores principales: Emanuel RM, Geyer HL, Mesa RA
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Publicado: Dove Medical Press 2013
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spelling oai:doaj.org-article:f8f59dfd22a74503ad171dbe561dd9e82021-12-02T06:25:08ZRuxolitinib as an emerging treatment in myelofibrosis1179-9889https://doaj.org/article/f8f59dfd22a74503ad171dbe561dd9e82013-03-01T00:00:00Zhttp://www.dovepress.com/ruxolitinib-as-an-emerging-treatment-in-myelofibrosis-a12419https://doaj.org/toc/1179-9889Robyn M Emanuel,1 Holly L Geyer,1 Ruben A Mesa2 1Department of Internal Medicine, 2Department of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ, USA Abstract: Essential thrombocythemia, polycythemia vera, and myelofibrosis belong to the class of Bcr-Abl negative hematologic neoplasms, which arise in part from varying Janus kinase-2 (JAK2) cellular deregulation. With the development of novel tyrosine kinase inhibitors capable of successfully inhibiting JAK in vivo, an influx of JAK2 inhibitors has come under clinical investigation. Ruxolitinib (Jakafi®; Incyte Corporation, Wilmington, DE, USA) was the first of these compounds to gain US Food and Drug Administration approval in late 2011 for the treatment of intermediate- and high-risk myelofibrosis. Two Phase III clinical trials – Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment-I and -II (COMFORT-I and -II) – played key roles in the US Food and Drug Administration approval of ruxolitinib with successful demonstration of spleen reduction and symptom palliation. Well tolerated in most patients, common side effects include cytopenias and gastrointestinal toxicities. The majority of preliminary data appears to suggest that if administered in a dose-titrated fashion, ruxolitinib can be used safely in a clinical practice setting. Additionally, patients most likely to benefit from ruxolitinib treatment are those with moderate to severe constitutional symptoms or splenomegaly. Future studies are ongoing in applying ruxolitinib to other hematologic and solid tumor malignancies. More clinical experience is recommended before the utility of this medication in a routine clinical practice setting can be fully determined. Keywords: myeloproliferative neoplasms, myelofibrosis, ruxolitinib, JAK2 inhibitors, INCB018424Emanuel RMGeyer HLMesa RADove Medical PressarticleDiseases of the blood and blood-forming organsRC633-647.5ENBlood and Lymphatic Cancer: Targets and Therapy, Vol 2013, Iss default, Pp 11-18 (2013)
institution DOAJ
collection DOAJ
language EN
topic Diseases of the blood and blood-forming organs
RC633-647.5
spellingShingle Diseases of the blood and blood-forming organs
RC633-647.5
Emanuel RM
Geyer HL
Mesa RA
Ruxolitinib as an emerging treatment in myelofibrosis
description Robyn M Emanuel,1 Holly L Geyer,1 Ruben A Mesa2 1Department of Internal Medicine, 2Department of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ, USA Abstract: Essential thrombocythemia, polycythemia vera, and myelofibrosis belong to the class of Bcr-Abl negative hematologic neoplasms, which arise in part from varying Janus kinase-2 (JAK2) cellular deregulation. With the development of novel tyrosine kinase inhibitors capable of successfully inhibiting JAK in vivo, an influx of JAK2 inhibitors has come under clinical investigation. Ruxolitinib (Jakafi®; Incyte Corporation, Wilmington, DE, USA) was the first of these compounds to gain US Food and Drug Administration approval in late 2011 for the treatment of intermediate- and high-risk myelofibrosis. Two Phase III clinical trials – Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment-I and -II (COMFORT-I and -II) – played key roles in the US Food and Drug Administration approval of ruxolitinib with successful demonstration of spleen reduction and symptom palliation. Well tolerated in most patients, common side effects include cytopenias and gastrointestinal toxicities. The majority of preliminary data appears to suggest that if administered in a dose-titrated fashion, ruxolitinib can be used safely in a clinical practice setting. Additionally, patients most likely to benefit from ruxolitinib treatment are those with moderate to severe constitutional symptoms or splenomegaly. Future studies are ongoing in applying ruxolitinib to other hematologic and solid tumor malignancies. More clinical experience is recommended before the utility of this medication in a routine clinical practice setting can be fully determined. Keywords: myeloproliferative neoplasms, myelofibrosis, ruxolitinib, JAK2 inhibitors, INCB018424
format article
author Emanuel RM
Geyer HL
Mesa RA
author_facet Emanuel RM
Geyer HL
Mesa RA
author_sort Emanuel RM
title Ruxolitinib as an emerging treatment in myelofibrosis
title_short Ruxolitinib as an emerging treatment in myelofibrosis
title_full Ruxolitinib as an emerging treatment in myelofibrosis
title_fullStr Ruxolitinib as an emerging treatment in myelofibrosis
title_full_unstemmed Ruxolitinib as an emerging treatment in myelofibrosis
title_sort ruxolitinib as an emerging treatment in myelofibrosis
publisher Dove Medical Press
publishDate 2013
url https://doaj.org/article/f8f59dfd22a74503ad171dbe561dd9e8
work_keys_str_mv AT emanuelrm ruxolitinibasanemergingtreatmentinmyelofibrosis
AT geyerhl ruxolitinibasanemergingtreatmentinmyelofibrosis
AT mesara ruxolitinibasanemergingtreatmentinmyelofibrosis
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