Moulis Guillaume, Sailler Laurent, Adoue Daniel, Lapeyre-Mestre Maryse
Service de médecine interne, CHU de Toulouse, Toulouse, France - Université de Toulouse, UMR1027, Toulouse, France - Inserm, UMR1027, Toulouse, France.
Service de médecine interne, CHU de Toulouse, Toulouse, France.
Therapie. 2014 Sep-Oct;69(5):437-48. doi: 10.2515/therapie/2014056. Epub 2014 Oct 7.
Immune thrombocytopenia (ITP) is a rare condition. Its epidemiology is not well-known. First-line treatment is based on corticosteroids. ITP leads to persistency (lasting more than 3 months) in 70% of adult cases. Then, several second-line treatments (SLTs) are available, mainly splenectomy, rituximab (off-label) and thrombopoietin-receptor agonists. Their efficacy and safety have not been compared, particularly in the long-term. FAITH (French Adult primary Immune Thrombocytopenia: a pHarmacoepidemiological study) is dedicated to the building and follow-up of the cohort of all adults with primary ITP in France persistently treated (>3 months) through the database of French Health Insurance system (système national d'information interrégimes d'Assurance maladie, SNIIRAM), in order to assess the benefit-to-risk balance of SLTs in real-life practice. CARMEN (Cytopénies Auto-immunes : Registre Midi-PyrénéEN) is a clinical registry of all incident adult ITP patients in the Midi-Pyrénées region. It is aimed at describing ITP clinical features, assessing SLT benefit-to-risk balance and adherence to guidelines for ITP management. FAITH is registered n°ENCEPP/SDPP/4574.
免疫性血小板减少症(ITP)是一种罕见病。其流行病学情况尚不为人熟知。一线治疗以皮质类固醇为基础。在70%的成年患者中,ITP会导致病情持续(持续超过3个月)。然后,有几种二线治疗方法可供选择,主要是脾切除术、利妥昔单抗(超适应症用药)和血小板生成素受体激动剂。它们的疗效和安全性尚未得到比较,尤其是在长期使用方面。FAITH(法国成人原发性免疫性血小板减少症:一项药物流行病学研究)致力于通过法国医疗保险系统(全国跨医保信息系统,SNIIRAM)的数据库,建立并随访法国所有持续治疗(>3个月)的成年原发性ITP患者队列,以便评估二线治疗方法在实际临床中的风险效益比。CARMEN(自身免疫性血细胞减少症:南比利牛斯登记处)是南比利牛斯地区所有成年ITP新发病例患者的临床登记处。其目的是描述ITP的临床特征,评估二线治疗方法的风险效益比以及对ITP管理指南的依从性。FAITH的注册号为ENCEPP/SDPP/4574。