PDF(Pubmed)
Abstract：
Disease-modifying therapies (DMTs) for multiple sclerosis (MS) reduce relapse frequency, magnetic resonance imaging (MRI) activity, and slow disability progression. Numerous DMTs are approved for relapsing forms of MS although real-world data on patient-reported outcomes (PROs) and quality of life (QoL) are needed to inform treatment choice. Immune reconstitution therapy with cladribine tablets is a highly effective treatment for relapsing MS (RMS). We present the protocol for an observational study to prospectively assess the effectiveness of cladribine tablets on clinical and MRI parameters as well as on PROs, including treatment satisfaction, QoL, sleep quality, self-perceived health, fatigue, and physical function. Enrolled patients at study sites in Italy will be adults with RMS (including relapsing-remitting and active secondary progressive MS) who are either treatment naïve or have received at least one first-line disease modifying DMT or no more than one second-line DMT. The primary objective will be change in global treatment satisfaction measured with the Treatment Satisfaction Questionnaire for Medication Version 1.4 approximately 24 months after initiating cladribine tablets in patients switching from previous DMTs. Secondary objectives will include global treatment satisfaction at earlier timepoints, will comprise treatment naïve patients, and will quantify treatment effectiveness and tolerability. We will also assess relapses, disability progression, MRI activity, and other PROs at approximately 12 and 24 months. The findings will provide insight from daily clinical practice into the patient\'s experience to complement data from controlled trials and inform treatment choice. EU PAS Registration Number EUPAS49334 filed 17/10/2022.
摘要：
多发性硬化症（MS）的疾病改良疗法（DMT）可降低复发频率，磁共振成像（MRI）活动，和缓慢的残疾进展。许多DMT被批准用于复发形式的MS，尽管需要有关患者报告的结果（PRO）和生活质量（QoL）的实际数据来告知治疗选择。克拉屈滨片的免疫重建疗法是复发性MS（RMS）的高效治疗方法。我们提出了一项观察性研究的方案，以前瞻性地评估克拉屈滨片对临床和MRI参数以及PRO的有效性。包括治疗满意度，QoL,睡眠质量,自我感知的健康，疲劳,和身体功能。在意大利的研究中心注册的患者将是患有RMS的成年人（包括复发缓解性和活动性继发性进行性MS），他们要么是未接受治疗的，要么已经接受了至少一种一线疾病修饰性DMT或不超过一种二线DMT。主要目标将是在从以前的DMT转换的患者开始服用克拉屈滨片剂后约24个月，通过1.4版药物治疗满意度问卷测量的全球治疗满意度的变化。次要目标将包括早期时间点的全球治疗满意度，将包括治疗初治患者，并将量化治疗有效性和耐受性。我们还将评估复发，残疾进展，MRI活动,和其他专业人员，大约12个月和24个月。这些发现将为患者的日常临床实践提供见解，以补充对照试验的数据并告知治疗选择。欧盟PAS注册号EUPAS49334于2022年10月17日提交。
