Natalizumab treatment of multiple sclerosis in Spain: results of an extensive observational study
Natalizumab treatment of multiple sclerosis in Spain: results of an extensive observational study
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DOI：
10.1007/s00415-012-6414-9
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发表时间：
2012-09-01
影响因子：
6
通讯作者：
Montalban, X.
中科院分区：
文献类型：
--
作者：
Fernandez, O.;Oreja-Guevara, C.;Montalban, X.
Natalizumab已被证明在治疗多发性硬化症的关键临床试验中是有效的；然而，临床实践中表明治疗的患者的临床特征与临床试验中的患者不同。因此，这项研究的目的是收集西班牙日常临床实践中纳他珠单抗使用的数据。西班牙各地的86个参与中心提交了基线和治疗后的疾病特征数据。有效数据来自1364名患者(69.3%为女性，86.9%患有复发-缓解性疾病)。93%的人之前接受过多发性硬化症的治疗。在治疗至少一年的825例患者中，年化复发率(ARR)从他他珠单抗治疗前一年的中位数2.0[平均值2.01，95%可信区间1.92-2.11]下降到1年的0.0%(平均值0.25，95%可信区间0.21-0.29)(p<；0.001)。扩展残疾状态量表评分从基线的中位数3.5(平均值3.71，95%可信区间3.60~3.82)降至3.0(平均值3.37，95%可信区间3.25~3.49)(p<；0.0001)。停药率为14%。一名患者因进行性多灶性白质脑病(PML)和另一名可能的PML(随后确诊)而停用那他珠单抗。虽然我们的患者比关键研究中的患者有更严重的疾病，但ARR也有类似的降低。这一发现与之前的观察研究是一致的。这一效应与基线EDSS无关。
Natalizumab has been shown to be effective in pivotal clinical trials in multiple sclerosis; however, the patients in whom treatment is indicated in clinical practice have a different clinical profile from those included in the clinical trials. The aim of this study is therefore to collect data on natalizumab use in everyday clinical practice in Spain. The 86 participating centers throughout Spain submitted data on disease characteristics at baseline and after treatment. Valid data were available for 1,364 patients (69.3% women, 86.9% with relapsing-remitting disease). Ninety-three percent had received prior therapy for multiple sclerosis. For the 825 patients on treatment for at least a year, the annualized relapse rate (ARR) decreased from median 2.0 [mean 2.01, 95% confidence interval (CI) 1.92-2.11] in the year prior to natalizumab to 0.0 (mean 0.25, 95% CI 0.21-0.29) at 1 year (p < 0.001). The Expanded Disability Status Scale (EDSS) score decreased from median 3.5 at baseline (mean 3.71, 95% CI 3.60-3.82) to 3.0 (mean 3.37, 95% CI 3.25-3.49) (p < 0.0001). The discontinuation rate was 14%. One patient discontinued natalizumab due to progressive multifocal leukoencephalopathy (PML) and another due to probable PML (subsequently confirmed). Although our patients had more severe disease than those in the pivotal study, a similar reduction in ARR was observed. This finding is in line with previous observational studies. The effect was independent of baseline EDSS.