背景:免疫性血小板减少症(ITP)是一种自身免疫性疾病,其特征是血小板计数低和出血风险增加。在使用或不使用静脉注射免疫球蛋白(一线治疗)的皮质类固醇后,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-RAs),和Fostamatinib.在加拿大,二线治疗的选择受到药物治疗的影响.本叙述性审查的目标是:1)总结在ITP中使用TPO-RA和其他二线疗法的证据;2)强调加拿大各省和地区的TPO-RA公共资助标准的差异。
方法:我们对ITP的二线治疗进行了文献综述。我们向医疗保健提供者征求了有关加拿大TPO-RA公共资助计划的信息,药剂师,省卫生部。
结果:涉及TPO-RA的头对头试验,利妥昔单抗,脾切除术,和fostamatinib缺乏。有大量证据表明TPO-RAs在改善血小板计数水平方面的作用,与健康相关的生活质量,出血,以及安慰剂对照试验和观察性研究的疲劳;然而,通过加拿大省级资助计划获得TPO-RA的机会是可变的。脾切除术失败是安大略省TPO-RA资助的先决条件,曼尼托巴省,还有萨斯喀彻温省,但不是在艾伯塔省或魁北克.其他省份要么无法获得公共资金,要么根据具体情况提供资金。
结论:TPO-RA是治疗ITP的有效二线疗法;然而,访问在加拿大是可变的,这导致健康差异和国际治疗指南的吸收不足。
BACKGROUND: 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.
CONCLUSIONS: 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.