两项研究合二为一：利用来自德国独立研究PANGEA和PEARL的真实世界数据，对芬戈莫德与干扰素及醋酸格拉替雷进行倾向评分匹配比较。
Two studies in one: A propensity-score-matched comparison of fingolimod versus interferons and glatiramer acetate using real-world data from the independent German studies, PANGAEA and PEARL.
作者信息
Alsop Jonathan, Medin Jennie, Cornelissen Christian, Vormfelde Stefan Viktor, Ziemssen Tjalf
机构信息
Numerus Ltd, Wokingham, United Kingdom.
Novartis Pharma AG, Basel, Switzerland.
出版信息
PLoS One. 2017 May 5;12(5):e0173353. doi: 10.1371/journal.pone.0173353. eCollection 2017.
BACKGROUND
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.
METHODS AND FINDINGS
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).
CONCLUSIONS
Fingolimod appears to be more effective than BRACE in improving clinical and physician-/patient-reported outcomes in individuals with active MS.
背景
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。
方法与结果
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）。
结论
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。
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