一项多中心、前瞻性、随机、开放标签的实用性试验，旨在比较干扰素β-1a和醋酸格拉替雷对患有多发性硬化症的儿科患者的有效性和安全性。
A multicentre, prospective, randomized, open-label pragmatic trial to compare the effectiveness and safety of interferon beta-1a and glatiramer-acetate in paediatric patients affected by Multiple Sclerosis.
作者信息
Simone Marta, Palumbi Roberto, Achille Mariaclara, Micella Stefania, Pascali Mara, Peschechera Antonia, Gabellone Alessandra, Marzulli Lucia, De Giacomo Andrea, Maggi Giulia, Reggiardo Giorgio, Felisi Mariagrazia, Padula Rosa, Bonifazi Donato, Valeriani Massimiliano, Papetti Laura, Lanzillo Roberta, Clerici Valentina Torri, Margari Lucia
机构信息
Child Neuropsychiatry Unit, Department of Precision and Regenerative Medicine and Jonic Area (Dimepre-J), University of Bari "Aldo Moro, Bari, Italy.
Child Neuropsychiatry Unit, Department of Translational Biomedicines and Neurosciences (DiBrain), University of Bari "Aldo Moro, Bari, Italy.
出版信息
Neurol Sci. 2025 Aug 8. doi: 10.1007/s10072-025-08377-3.
BACKGROUND
Paediatric-onset Multiple Sclerosis (POMS) is a rare, highly active disease requiring timely disease-modifying therapy.
OBJECTIVES
This trial compared the efficacy and safety of intramuscular (i.m.) Interferon beta-1a (IFN-beta 1a) and subcutaneous (s.c.) Glatiramer Acetate (GA).
METHODS
A 96-week, Phase IIIb, multicenter, open-label randomized trial enrolled 30 participants (ages 12-17). Patients were randomized into two groups: 15 received i.m. IFN-beta 1a, and 15 received s.c. GA. The primary endpoint was MRI disease activity-free status; secondary outcomes included annualized relapse rate (ARR), time to first relapse, Expanded Disability Status Scale (EDSS), cognitive and fatigue scores, and quality of life.
RESULTS
The groups differed by age but not sex. MRI disease activity-free status did not differ (p = 0.09). ARR was significantly lower in the GA group (0.20; 95% CI 0.08-0.42) than in the IFN-beta 1a group (0.57; 95% CI 0.31-0.95, p = 0.02). EDSS, cognitive, fatigue, and quality-of-life scores were comparable. Notably, 50% of GA patients and 30% of IFN-beta 1a patients switched to higher efficacy treatments.
CONCLUSIONS
Both treatments showed similar efficacy and safety, but early high-efficacy therapy may be preferable for POMS management.
TRIAL REGISTRATION
EudraCT Number: 2017-005129-18. https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-005129-18/IT.
背景
儿童期多发性硬化症（POMS）是一种罕见的、高度活跃的疾病，需要及时进行疾病修正治疗。
目的
本试验比较了肌肉注射（i.m.）干扰素β-1a（IFN-β 1a）和皮下注射（s.c.）醋酸格拉替雷（GA）的疗效和安全性。
方法
一项为期96周的IIIb期多中心开放标签随机试验招募了30名参与者（年龄12 - 17岁）。患者被随机分为两组：15名接受肌肉注射IFN-β 1a，15名接受皮下注射GA。主要终点是MRI无疾病活动状态；次要结局包括年化复发率（ARR）、首次复发时间、扩展残疾状态量表（EDSS）、认知和疲劳评分以及生活质量。
结果
两组在年龄上存在差异，但在性别上无差异。MRI无疾病活动状态无差异（p = 0.09）。GA组的ARR（0.20；95%CI 0.08 - 0.42）显著低于IFN-β 1a组（0.57；95%CI 0.31 - 0.95，p = 0.02）。EDSS、认知、疲劳和生活质量评分相当。值得注意的是，50%的GA患者和30%的IFN-β 1a患者转而接受更高疗效的治疗。
结论
两种治疗方法显示出相似的疗效和安全性，但对于POMS的管理，早期采用高效治疗可能更可取。
试验注册
欧洲药品管理局临床试验编号：2017 - 005129 - 18。https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-005129-18/IT。
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