Alsop Jonathan, Medin Jennie, Cornelissen Christian, Vormfelde Stefan Viktor, Ziemssen Tjalf
Numerus Ltd, Wokingham, United Kingdom.
Novartis Pharma AG, Basel, Switzerland.
PLoS One. 2017 May 5;12(5):e0173353. doi: 10.1371/journal.pone.0173353. eCollection 2017.
This study compared outcomes following fingolimod or BRACE treatments (beta-interferons/glatiramer acetate) in patients with active MS (≥ 1 relapse in the previous year) following previous BRACE treatment.
Patients with active MS who previously received BRACE were identified from German prospective, observational studies, PANGAEA and PEARL. A novel methodology was developed to compare outcomes between propensity-score-matched cohorts (3:1 ratio) from the independent single-arm studies. Patients in PANGAEA (n = 1287) experienced 48% fewer relapses per year than those in PEARL (n = 429; annualized relapse rate ratio: 0.52; p < 0.001). The risk of 3-month or 6-month confirmed disability progression (CDP) was reduced in PANGAEA versus PEARL (3-month: 37% reduction; hazard ratio [HR], 0.63; p < 0.001; 6-month: 47% reduction; HR, 0.53; p < 0.001). A higher proportion of patients in PANGAEA (n = 1234) than PEARL (n = 401) were free from relapses and 3-month (65.7% vs 38.7%; p < 0.001) or 6-month (68.2% vs 39.2%; p < 0.001) CDP. The probability of confirmed disability improvement was higher in PANGAEA (n = 1163) than PEARL (n = 372; 3-month: 175% increase; HR, 2.75; p < 0.001; 6-month: 126% increase; HR, 2.26; p < 0.001). Patients in PANGAEA (n = 149) were less likely than those in PEARL (n = 307) to have taken sick leave (proportion with 0 days off work: 62.4% vs 44.6%; p = 0.0005). For change in disease severity from baseline (assessed by clinicians using the Clinical Global Impressions scale; PANGAEA, n = 1207; PEARL, n = 427), a larger proportion of patients had subjective improvement and a smaller proportion had worsening status in PANGAEA than PEARL (improvement: 28.2% vs 15.2%; worsening: 16.4% vs 30.4%; p < 0.0001).
Fingolimod appears to be more effective than BRACE in improving clinical and physician-/patient-reported outcomes in individuals with active MS.
本研究比较了在先前接受过BRACE治疗的活动性多发性硬化症（MS）患者（前一年至少复发1次）中，使用芬戈莫德或BRACE治疗（β-干扰素/醋酸格拉替雷）后的疗效。
从德国前瞻性观察性研究PANGAEA和PEARL中，识别出先前接受过BRACE治疗的活动性MS患者。开发了一种新方法，用于比较来自独立单臂研究的倾向评分匹配队列（3:1比例）之间的疗效。PANGAEA组（n = 1287）的患者每年复发次数比PEARL组（n = 429）少48%（年化复发率比：0.52；p < 0.001）。与PEARL组相比，PANGAEA组3个月或6个月确诊残疾进展（CDP）的风险降低（3个月：降低37%；风险比[HR]，0.63；p < 0.001；6个月：降低47%；HR，0.53；p < 0.001）。PANGAEA组（n = 1234）中无复发以及无3个月（65.7%对38.7%；p < 0.001）或6个月（68.2%对39.2%；p < 0.001）CDP的患者比例高于PEARL组（n = 401）。PANGAEA组（n = 1163）确诊残疾改善的概率高于PEARL组（n = 372；3个月：增加175%；HR，2.75；p < 0.001；6个月：增加126%；HR，2.26；p < 0.001）。PANGAEA组（n = 149）的患者比PEARL组（n = 307）请病假的可能性更小（无工作日比例：62.4%对44.6%；p = 0.0005）。对于从基线开始的疾病严重程度变化（由临床医生使用临床总体印象量表评估；PANGAEA组，n = 1207；PEARL组，n = 427），与PEARL组相比，PANGAEA组主观改善的患者比例更高，病情恶化的患者比例更小（改善：28.2%对15.2%；恶化：16.4%对30.4%；p < 0.0001）。
在改善活动性MS患者的临床及医生/患者报告结局方面，芬戈莫德似乎比BRACE更有效。
