Gärtner Jutta, Brück Wolfgang, Weddige Almuth, Hummel Hannah, Norenberg Christiane, Bugge Jörg-Peter
Department of Paediatrics and Adolescent Medicine, University Medical Center Göttingen, Germany.
Department of Neuropathology, University Medical Center Göttingen, Germany.
Mult Scler J Exp Transl Clin. 2017 Dec 26;3(4):2055217317747623. doi: 10.1177/2055217317747623. eCollection 2017 Oct-Dec.
Study evaluating Betaferon(R)'s safety and tolerability in paediatric patients with multiple sclerosis (BETAPAEDIC) is a prospective, open-label observational multicentre study to assess the safety and effectiveness of interferon beta-1b in paediatric patients with relapsing-remitting multiple sclerosis.
Treatment-naïve patients (12-16 years) scheduled to start interferon beta-1b were enrolled with follow-up visits every six months for two years. Effectiveness was evaluated by annualised relapse rate, Expanded Disability Status Scale progression, cranial magnetic resonance imaging and cognitive testing. Fatigue was assessed by the Fatigue Severity Scale.
Sixty-eight patients were screened and 67 enrolled, with mean (standard deviation) age 14.2 (1.3) years (=65 in the effectiveness analysis). Mean disease duration was 11 months before study enrolment; at baseline, mean (standard deviation) Expanded Disability Status Scale was 0.6 (1.0); T2 lesion number 18.3 (15.1). Mean annualised relapse rate during the study was 0.7 (=57), 28/57 patients (49.1%) had no relapses and for 40/52 (76.9%) no Expanded Disability Status Scale progression was observed; 23/56 (41.1%) were relapse- and progression-free to last follow-up. Neuropsychological test and fatigue scores were within normal ranges (baseline and last follow-up). Eighteen patients had fatigue at some point. New T2 and gadolinium-enhancing (Gd+) lesions were seen in 43/55 (66.2%) and 29/55 (52.7%) patients respectively. Most frequent adverse events were influenza-like illness, headache, injection-site reactions and elevated liver enzymes.
Interferon beta-1b is an effective treatment with a favourable safety profile for paediatric patients.
评估β-干扰素（Betaferon(R)）在儿童多发性硬化症患者中的安全性和耐受性的研究（BETAPAEDIC）是一项前瞻性、开放标签的观察性多中心研究，旨在评估干扰素β-1b在复发缓解型儿童多发性硬化症患者中的安全性和有效性。
计划开始使用干扰素β-1b治疗的初治患者（12 - 16岁）入组，每六个月进行一次随访，为期两年。通过年化复发率、扩展残疾状态量表进展、头颅磁共振成像和认知测试来评估有效性。通过疲劳严重程度量表评估疲劳情况。
共筛查68例患者，67例入组，平均（标准差）年龄14.2（1.3）岁（有效性分析中n = 65）。研究入组前平均病程为11个月；基线时，平均（标准差）扩展残疾状态量表评分为0.6（1.0）；T2病灶数为18.3（15.1）。研究期间平均年化复发率为0.7（n = 57），28/57例患者（49.1%）无复发，40/52例患者（76.9%）未观察到扩展残疾状态量表进展；23/56例患者（41.1%）在最后一次随访时无复发且无进展。神经心理学测试和疲劳评分在正常范围内（基线和最后一次随访）。18例患者在某些时候出现疲劳。分别在43/55例患者（66.2%）和29/55例患者（52.7%）中观察到新的T2病灶和钆增强（Gd +）病灶。最常见的不良事件为流感样疾病、头痛、注射部位反应和肝酶升高。
干扰素β-1b对儿童患者是一种有效的治疗方法，且安全性良好。
