Treatment of immune thrombocytopenic purpura: focus on eltrombopag

Lawrence RiceWeill Cornell Medical College, The Methodist Hospital, Houston, Texas, USAAbstract: Immune thrombocytopenic purpura (ITP) is a relatively common autoimmune disorder in which antibodies are produced to circulating platelets. Symptoms can be mild, but for most patients the risk of severe...

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Autor principal:	Lawrence Rice
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2009
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/6c28fca0d9f74d4d9876a2b6c8052885
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spelling	oai:doaj.org-article:6c28fca0d9f74d4d9876a2b6c80528852021-12-02T03:31:39ZTreatment of immune thrombocytopenic purpura: focus on eltrombopag1177-54751177-5491https://doaj.org/article/6c28fca0d9f74d4d9876a2b6c80528852009-03-01T00:00:00Zhttp://www.dovepress.com/treatment-of-immune-thrombocytopenic-purpura-focus-on-eltrombopag-a2984https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Lawrence RiceWeill Cornell Medical College, The Methodist Hospital, Houston, Texas, USAAbstract: Immune thrombocytopenic purpura (ITP) is a relatively common autoimmune disorder in which antibodies are produced to circulating platelets. Symptoms can be mild, but for most patients the risk of severe bleeding is unacceptable and treatment is required. Glucocorticoids followed by splenectomy had been the mainstays of therapy. High dose intravenous immunoglobulin and anti-RhD therapy are available for patients with severe illness, but produce only temporary benefit. Rituximab may provide more durable responses, danazol may be underutilized, and immunosuppressants and cytotoxic agents are less often required. Recently the pathophysiology of ITP has been more clearly elucidated, particularly the importance of decreased production of platelets in most patients and the very blunted rise that occurs in serum thrombopoietin (TPO). The isolation of TPO and better understanding of its role in thrombopoiesis has led to the development of new highly effective treatments. TPO analogs had some successes in treating highly refractory ITP patients but were taken out of development due to TPO-antibody induction. Two second-generation TPO-mimetics, romiplostim and the orally available eltrombopag, have recently been licensed in some territories for the treatment of ITP. Approval of eltrombopag was based on results from Phase II and III placebo-controlled clinical trials and a long-term extension study. About 80% of patients achieve significant increases in platelet count (11% of placebo patients), with reduced bleeding and reduced use of concomitant medications; responses are often durable with no tachyphylaxis. The side effects of eltrombopag are generally mild and not worse than placebo, although there are concerns about hepatic dysfunction, and the potentials for thromboses, marrow reticulin fibrosis, rebound thrombocytopenia and cataracts. This is an important new option for highly refractory patients, and its niche in earlier treatment (and for other thrombocytopenic disorders) is yet to be defined.Keywords: eltrombopag, immune thrombocytopenic purpura, autoimmune disorder, serum thrombopoietin Lawrence RiceDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2009, Iss default, Pp 151-157 (2009)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Lawrence Rice Treatment of immune thrombocytopenic purpura: focus on eltrombopag
description	Lawrence RiceWeill Cornell Medical College, The Methodist Hospital, Houston, Texas, USAAbstract: Immune thrombocytopenic purpura (ITP) is a relatively common autoimmune disorder in which antibodies are produced to circulating platelets. Symptoms can be mild, but for most patients the risk of severe bleeding is unacceptable and treatment is required. Glucocorticoids followed by splenectomy had been the mainstays of therapy. High dose intravenous immunoglobulin and anti-RhD therapy are available for patients with severe illness, but produce only temporary benefit. Rituximab may provide more durable responses, danazol may be underutilized, and immunosuppressants and cytotoxic agents are less often required. Recently the pathophysiology of ITP has been more clearly elucidated, particularly the importance of decreased production of platelets in most patients and the very blunted rise that occurs in serum thrombopoietin (TPO). The isolation of TPO and better understanding of its role in thrombopoiesis has led to the development of new highly effective treatments. TPO analogs had some successes in treating highly refractory ITP patients but were taken out of development due to TPO-antibody induction. Two second-generation TPO-mimetics, romiplostim and the orally available eltrombopag, have recently been licensed in some territories for the treatment of ITP. Approval of eltrombopag was based on results from Phase II and III placebo-controlled clinical trials and a long-term extension study. About 80% of patients achieve significant increases in platelet count (11% of placebo patients), with reduced bleeding and reduced use of concomitant medications; responses are often durable with no tachyphylaxis. The side effects of eltrombopag are generally mild and not worse than placebo, although there are concerns about hepatic dysfunction, and the potentials for thromboses, marrow reticulin fibrosis, rebound thrombocytopenia and cataracts. This is an important new option for highly refractory patients, and its niche in earlier treatment (and for other thrombocytopenic disorders) is yet to be defined.Keywords: eltrombopag, immune thrombocytopenic purpura, autoimmune disorder, serum thrombopoietin
format	article
author	Lawrence Rice
author_facet	Lawrence Rice
author_sort	Lawrence Rice
title	Treatment of immune thrombocytopenic purpura: focus on eltrombopag
title_short	Treatment of immune thrombocytopenic purpura: focus on eltrombopag
title_full	Treatment of immune thrombocytopenic purpura: focus on eltrombopag
title_fullStr	Treatment of immune thrombocytopenic purpura: focus on eltrombopag
title_full_unstemmed	Treatment of immune thrombocytopenic purpura: focus on eltrombopag
title_sort	treatment of immune thrombocytopenic purpura: focus on eltrombopag
publisher	Dove Medical Press
publishDate	2009
url	https://doaj.org/article/6c28fca0d9f74d4d9876a2b6c8052885
work_keys_str_mv	AT lawrencerice treatmentofimmunethrombocytopenicpurpurafocusoneltrombopag
_version_	1718401732651778048

Treatment of immune thrombocytopenic purpura: focus on eltrombopag

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