Thrombopoietin Receptor Agonists and Other Second-Line Therapies for Immune Thrombocytopenia: A Narrative Review With a Focus on Drug Access in Canada.

IF 1.2 4区 医学 Q4 MEDICINE, RESEARCH & EXPERIMENTAL
Joanne Britto, Anne Holbrook, Haowei Sun, Christine Cserti-Gazdewich, Oksana Prokopchuk-Gauk, Cyrus Hsia, Karima Khamisa, Paul R Yenson, Michelle Sholzberg, Harold J Olney, Sudeep Shivakumar, David Jones, Hayley Merkeley, Jacqueline Costello, Erin Jamula, Donald M Arnold
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引用次数: 0

Abstract

Introduction: Immune thrombocytopenia (ITP) is an autoimmune disease characterized by low platelet counts and increased risk of bleeding. After corticosteroids with or without intravenous immune globulin (first-line treatment), second-line treatment options include rituximab, splenectomy, thrombopoietin receptor agonists (TPO-RAs), and fostamatinib. In Canada, the choice of second-line therapy is influenced by access to medications. The goals of this narrative review are to 1) summarize the evidence for the use of TPO-RAs and other second-line therapies in ITP and 2) highlight differences in public funding criteria for TPO-RAs across provinces and territories in Canada.

Methods: We conducted a literature review of second-line therapies for ITP. We solicited information on public funding programs for TPO-RAs in Canada from health care providers, pharmacists, and provincial ministries of health.

Results: Head-to-head trials involving TPO-RAs, rituximab, splenectomy, and fostamatinib are lacking. There is substantial evidence of effect for TPO-RAs in improving platelet count levels, health-related quality of life, bleeding, and fatigue from placebo-controlled trials and observational studies; however, access to TPO-RAs through provincial funding programs in Canada is variable. Splenectomy failure is a prerequisite for the funding of TPO-RAs in Ontario, Manitoba, and Saskatchewan, but not in Alberta or Quebec. Other provinces either do not have access to public funding or funding is provided on a case-by-case basis.

Discussion: TPO-RAs are effective second-line therapies for the treatment of ITP; however, access is variable across Canada, which results in health disparities and poor uptake of international treatment guidelines.

治疗免疫性血小板减少症的血小板生成素受体激动剂和其他二线疗法:以加拿大药物获取情况为重点的叙述性综述》。
简介免疫性血小板减少症(ITP)是一种自身免疫性疾病,其特点是血小板计数低和出血风险增加。在皮质类固醇联合或不联合静脉注射免疫球蛋白(一线治疗)之后,二线治疗方案包括利妥昔单抗、脾切除术、血小板生成素受体激动剂(TPO-RA)和福司他替尼。在加拿大,二线疗法的选择受到药物可及性的影响。本叙述性综述的目的是:1)总结在ITP中使用TPO-RAs和其他二线疗法的证据;2)强调加拿大各省和地区在TPO-RAs公共资助标准上的差异:我们对治疗 ITP 的二线疗法进行了文献综述。方法:我们对治疗ITP的二线疗法进行了文献综述,并向医疗服务提供者、药剂师和各省卫生部征集有关加拿大TPO-RAs公共资助项目的信息:缺乏涉及TPO-RAs、利妥昔单抗、脾切除术和福他马替尼的正面试验。安慰剂对照试验和观察性研究有大量证据表明,TPO-RAs 在改善血小板计数水平、与健康相关的生活质量、出血和疲劳方面有显著效果;但是,加拿大各省的资助计划对 TPO-RAs 的使用情况不一。在安大略省、马尼托巴省和萨斯喀彻温省,脾切除术失败是资助 TPO-RAs 的先决条件,但在阿尔伯塔省或魁北克省则不是。其他省份要么无法获得公共资助,要么根据具体情况提供资助:讨论:TPO-RAs 是治疗 ITP 的有效二线疗法;然而,加拿大各地的使用情况各不相同,这导致了健康差异和对国际治疗指南的接受度较低。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Clinical and Investigative Medicine
Clinical and Investigative Medicine 医学-医学:研究与实验
CiteScore
1.50
自引率
12.50%
发文量
18
审稿时长
>12 weeks
期刊介绍: Clinical and Investigative Medicine (CIM), publishes original work in the field of Clinical Investigation. Original work includes clinical or laboratory investigations and clinical reports. Reviews include information for Continuing Medical Education (CME), narrative review articles, systematic reviews, and meta-analyses.
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