Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives

Theodora Anagnostou, Mark R Litzow Mayo Clinic Rochester, Rochester, MN, USA Abstract: Ponatinib, a third-generation tyrosine kinase inhibitor that inhibits BCR/ABL independent of the mutation status, is currently approved for the treatment of patients with chronic myeloid leukemia or acute...

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Autores principales:	Anagnostou T, Litzow MR
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2017
Materias:	Ponatinib acute lymphoblastic leukemia chronic myeloid leukemia patient selection Diseases of the blood and blood-forming organs RC633-647.5
Acceso en línea:	https://doaj.org/article/bc6b72eeec174e29879a26b2e6c2ecc7
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spelling	oai:doaj.org-article:bc6b72eeec174e29879a26b2e6c2ecc72021-12-02T00:27:22ZSpotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives1179-9889https://doaj.org/article/bc6b72eeec174e29879a26b2e6c2ecc72017-12-01T00:00:00Zhttps://www.dovepress.com/spotlight-on-ponatinib-in-the-treatment-of-chronic-myeloid-leukemia-an-peer-reviewed-article-BLCTThttps://doaj.org/toc/1179-9889Theodora Anagnostou, Mark R Litzow Mayo Clinic Rochester, Rochester, MN, USA Abstract: Ponatinib, a third-generation tyrosine kinase inhibitor that inhibits BCR/ABL independent of the mutation status, is currently approved for the treatment of patients with chronic myeloid leukemia or acute lymphoblastic leukemia that are either resistant or unable to tolerate another tyrosine kinase inhibitor. Its US Food and Drug Administration approval was based on results from long-term follow-up of the pivotal Phase II PACE trial, which demonstrated deep and durable molecular responses in the treated patients. Despite the remarkable responses, ponatinib has been associated with high frequency of severe vascular events, which led to its withdrawal from the market in 2013. Following analysis of the risk factors of patients who developed vascular side effects, ponatinib was reintroduced in the market 1 year later with specific dose-reduction recommendations and carrying a black box warning. Thus, careful patient selection with identification of patients whose potential benefit from ponatinib exceeds the potential risks associated with its use is crucial. Ongoing and future studies are focusing on earlier detection of mutations, strategies to minimize side effects, and potential expansion of the treatment indications. Clinical trials testing the safety and efficacy of ponatinib as frontline therapy are ongoing. Keywords: ponatinib, acute lymphoblastic leukemia, chronic myeloid leukemia, patient selectionAnagnostou TLitzow MRDove Medical PressarticlePonatinibacute lymphoblastic leukemiachronic myeloid leukemiapatient selectionDiseases of the blood and blood-forming organsRC633-647.5ENBlood and Lymphatic Cancer: Targets and Therapy, Vol Volume 8, Pp 1-9 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Ponatinib acute lymphoblastic leukemia chronic myeloid leukemia patient selection Diseases of the blood and blood-forming organs RC633-647.5
spellingShingle	Ponatinib acute lymphoblastic leukemia chronic myeloid leukemia patient selection Diseases of the blood and blood-forming organs RC633-647.5 Anagnostou T Litzow MR Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
description	Theodora Anagnostou, Mark R Litzow Mayo Clinic Rochester, Rochester, MN, USA Abstract: Ponatinib, a third-generation tyrosine kinase inhibitor that inhibits BCR/ABL independent of the mutation status, is currently approved for the treatment of patients with chronic myeloid leukemia or acute lymphoblastic leukemia that are either resistant or unable to tolerate another tyrosine kinase inhibitor. Its US Food and Drug Administration approval was based on results from long-term follow-up of the pivotal Phase II PACE trial, which demonstrated deep and durable molecular responses in the treated patients. Despite the remarkable responses, ponatinib has been associated with high frequency of severe vascular events, which led to its withdrawal from the market in 2013. Following analysis of the risk factors of patients who developed vascular side effects, ponatinib was reintroduced in the market 1 year later with specific dose-reduction recommendations and carrying a black box warning. Thus, careful patient selection with identification of patients whose potential benefit from ponatinib exceeds the potential risks associated with its use is crucial. Ongoing and future studies are focusing on earlier detection of mutations, strategies to minimize side effects, and potential expansion of the treatment indications. Clinical trials testing the safety and efficacy of ponatinib as frontline therapy are ongoing. Keywords: ponatinib, acute lymphoblastic leukemia, chronic myeloid leukemia, patient selection
format	article
author	Anagnostou T Litzow MR
author_facet	Anagnostou T Litzow MR
author_sort	Anagnostou T
title	Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
title_short	Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
title_full	Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
title_fullStr	Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
title_full_unstemmed	Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
title_sort	spotlight on ponatinib in the treatment of chronic myeloid leukemia and philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives
publisher	Dove Medical Press
publishDate	2017
url	https://doaj.org/article/bc6b72eeec174e29879a26b2e6c2ecc7
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Spotlight on ponatinib in the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia: patient selection and perspectives

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