Review of the safety and efficacy of imiglucerase treatment of Gaucher disease

Deborah Elstein, Ari ZimranGaucher Clinic, Shaare Zedek Medical Center, Jerusalem, IsraelAbstract: Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed...

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Autores principales:	Deborah Elstein, Ari Zimran
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2009
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/93035db7d71c4af793571bee8f3201da
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spelling	oai:doaj.org-article:93035db7d71c4af793571bee8f3201da2021-12-02T07:30:54ZReview of the safety and efficacy of imiglucerase treatment of Gaucher disease1177-54751177-5491https://doaj.org/article/93035db7d71c4af793571bee8f3201da2009-08-01T00:00:00Zhttp://www.dovepress.com/review-of-the-safety-and-efficacy-of-imiglucerase-treatment-of-gaucher-a3497https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Deborah Elstein, Ari ZimranGaucher Clinic, Shaare Zedek Medical Center, Jerusalem, IsraelAbstract: Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed developing antibodies; similarly, very few patients require pre-medication for allergic reactions. Within 3 to 5 years of imiglucerase therapy, best documented at doses of 30 to 60 units/kg/infusion, hepatosplenomegaly can be expected to be reduced so that the liver volume will be maintained at 1 to 1.5 times normal (30% to 40% reduction from advent of ERT) and spleen volume to &le;2 to 8 times normal (50% to 60% reduction from advent of ERT). For anemic and thrombocytopenic patients, with 2 to 5 years of imiglucerase, hemoglobin levels are expected to be &ge;11 g/dL for women and children and &ge;12 g/dL for men; and platelet counts in patients with an intact spleen, depending on the baseline value, should approximately be doubled. Bone crises and bone pain but not irreversible skeletal damage will improve in most patients. Nonetheless, some features and some symptomatic patients apparently do not respond equally well and/or perhaps inadequately. The benefit for patients with the neuronopathic forms is primarily in improved visceral and hematological signs and symptoms. There are still several unresolved issues, the high per-unit cost being an important one, which have spurred the development of biosimilar enzymes as well as chaperone therapies currently in clinical trials.Keywords: Gaucher disease, enzyme replacement therapy, imiglucerase, substrate reduction therapy, pharmacological chaperones, cost Deborah ElsteinAri ZimranDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2009, Iss default, Pp 407-417 (2009)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Deborah Elstein Ari Zimran Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
description	Deborah Elstein, Ari ZimranGaucher Clinic, Shaare Zedek Medical Center, Jerusalem, IsraelAbstract: Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed developing antibodies; similarly, very few patients require pre-medication for allergic reactions. Within 3 to 5 years of imiglucerase therapy, best documented at doses of 30 to 60 units/kg/infusion, hepatosplenomegaly can be expected to be reduced so that the liver volume will be maintained at 1 to 1.5 times normal (30% to 40% reduction from advent of ERT) and spleen volume to &le;2 to 8 times normal (50% to 60% reduction from advent of ERT). For anemic and thrombocytopenic patients, with 2 to 5 years of imiglucerase, hemoglobin levels are expected to be &ge;11 g/dL for women and children and &ge;12 g/dL for men; and platelet counts in patients with an intact spleen, depending on the baseline value, should approximately be doubled. Bone crises and bone pain but not irreversible skeletal damage will improve in most patients. Nonetheless, some features and some symptomatic patients apparently do not respond equally well and/or perhaps inadequately. The benefit for patients with the neuronopathic forms is primarily in improved visceral and hematological signs and symptoms. There are still several unresolved issues, the high per-unit cost being an important one, which have spurred the development of biosimilar enzymes as well as chaperone therapies currently in clinical trials.Keywords: Gaucher disease, enzyme replacement therapy, imiglucerase, substrate reduction therapy, pharmacological chaperones, cost
format	article
author	Deborah Elstein Ari Zimran
author_facet	Deborah Elstein Ari Zimran
author_sort	Deborah Elstein
title	Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
title_short	Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
title_full	Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
title_fullStr	Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
title_full_unstemmed	Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
title_sort	review of the safety and efficacy of imiglucerase treatment of gaucher disease
publisher	Dove Medical Press
publishDate	2009
url	https://doaj.org/article/93035db7d71c4af793571bee8f3201da
work_keys_str_mv	AT deborahelstein reviewofthesafetyandefficacyofimiglucerasetreatmentofgaucherdisease AT arizimran reviewofthesafetyandefficacyofimiglucerasetreatmentofgaucherdisease
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Review of the safety and efficacy of imiglucerase treatment of Gaucher disease

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