eliglustat是否应为1型戈谢病患者的一线治疗方法？安全性和有效性的定义。 - Suppr

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Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy.

作者信息

Zimran Ari, Goldblatt Jack, Szer Jeff

机构信息

Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 91031, Israel.

Genetics Services of Western Australia, 374 Bagot Rd, Subiaco, WA 6008, Australia.

出版信息

Blood Cells Mol Dis. 2018 Feb;68:14-16. doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.

DOI:10.1016/j.bcmd.2017.09.003

PMID:28935503

Abstract

摘要

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Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy.

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Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy.

作者信息

Zimran Ari, Goldblatt Jack, Szer Jeff

机构信息

Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 91031, Israel.

Genetics Services of Western Australia, 374 Bagot Rd, Subiaco, WA 6008, Australia.

出版信息

Blood Cells Mol Dis. 2018 Feb;68:14-16. doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.

DOI:10.1016/j.bcmd.2017.09.003

PMID:28935503

Abstract

摘要

相似文献

Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy.

Blood Cells Mol Dis. 2018 Feb;68:14-16. doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.

Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Blood Cells Mol Dis. 2019 Jul;77:101-102. doi: 10.1016/j.bcmd.2019.04.003. Epub 2019 Apr 9.

Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.

Am J Hematol. 2017 Nov;92(11):1170-1176. doi: 10.1002/ajh.24877. Epub 2017 Oct 3.

Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States.

Mol Genet Metab. 2016 Feb;117(2):95-103. doi: 10.1016/j.ymgme.2015.09.002. Epub 2015 Sep 7.

A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration.

Blood Cells Mol Dis. 2018 Feb;68:185-191. doi: 10.1016/j.bcmd.2017.01.006. Epub 2017 Jan 13.

Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.

Eur J Intern Med. 2017 Jan;37:25-32. doi: 10.1016/j.ejim.2016.07.011. Epub 2016 Aug 10.

Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease.

Drug Des Devel Ther. 2015 Aug 18;9:4639-47. doi: 10.2147/DDDT.S77760. eCollection 2015.

Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna.

Wien Klin Wochenschr. 2022 Jun;134(11-12):471-477. doi: 10.1007/s00508-022-02021-2. Epub 2022 Apr 12.

Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results.

Am J Hematol. 2021 Sep 1;96(9):1156-1165. doi: 10.1002/ajh.26276. Epub 2021 Jul 11.

Long-term effects of eliglustat on skeletal manifestations in clinical trials of patients with Gaucher disease type 1.

Genet Med. 2023 Feb;25(2):100329. doi: 10.1016/j.gim.2022.10.011. Epub 2022 Dec 5.

引用本文的文献

Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy.

J Clin Med. 2022 Oct 24;11(21):6265. doi: 10.3390/jcm11216265.

Current and emerging pharmacotherapy for Gaucher disease in pediatric populations.

Expert Opin Pharmacother. 2021 Aug;22(11):1489-1503. doi: 10.1080/14656566.2021.1902989. Epub 2021 Mar 25.

A patient with Gaucher disease and plasma cell dyscrasia: bidirectional impact.

Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):389-394. doi: 10.1182/hematology.2020000123.

Small Molecule Chaperones for the Treatment of Gaucher Disease and -Associated Parkinson Disease.

Front Cell Dev Biol. 2020 May 19;8:271. doi: 10.3389/fcell.2020.00271. eCollection 2020.

mTOR hyperactivity mediates lysosomal dysfunction in Gaucher's disease iPSC-neuronal cells.

Dis Model Mech. 2019 Oct 16;12(10):dmm038596. doi: 10.1242/dmm.038596.

Lysosomal diseases: Overview on current diagnosis and treatment.

Genet Mol Biol. 2019;42(1 suppl 1):165-177. doi: 10.1590/1678-4685-GMB-2018-0159. Epub 2019 Apr 25.

Recent advances in the diagnosis and management of Gaucher disease.

Expert Rev Endocrinol Metab. 2018 Mar;13(2):107-118. doi: 10.1080/17446651.2018.1445524. Epub 2018 Mar 12.