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阿仑单抗治疗多发性硬化症:多中心队列的长期随访

Alemtuzumab for multiple sclerosis: Long term follow-up in a multi-centre cohort.

作者信息

Willis M D, Harding K E, Pickersgill T P, Wardle M, Pearson O R, Scolding N J, Smee J, Robertson N P

机构信息

Institute of Psychological Medicine and Clinical Neuroscience, Cardiff University, University Hospital of Wales, UK/Department of Neurology, University Hospital of Wales, UK.

Department of Neurology, University Hospital of Wales, UK.

出版信息

Mult Scler. 2016 Aug;22(9):1215-23. doi: 10.1177/1352458515614092. Epub 2015 Oct 29.

Abstract

BACKGROUND

Alemtuzumab has recently been approved for treatment of relapsing MS, but concerns remain about its use since long-term studies of adverse events remain limited. Furthermore, a clear understanding of its application and durability of effect in clinical practice has yet to evolve.

OBJECTIVES

To investigate long-term efficacy and safety outcomes in a multicentre cohort of patients treated with alemtuzumab.

METHODS

Patients treated from 2000 and followed-up at three regional centres were identified. Baseline and prospective data were obtained and validated by clinical record review.

RESULTS

One hundred patients were identified with a mean follow-up of 6.1 years (range 1-13). Forty patients were retreated with at least one further treatment cycle. Annualized relapse rates fell from 2.1 to 0.2 (p<0.0001) post-treatment and were sustained for up to eight years of follow-up. Mean change in EDSS score was +0.14. Forty-seven patients developed secondary autoimmunity.

CONCLUSION

Observed reduction in relapse rates reflected those reported in clinical trials, but we were unable to corroborate previous observations of disability reversal. 40% of patients required additional treatment cycles. Autoimmune adverse events were common, occurring at a higher rate than previously reported, but were largely predictable, and could be managed effectively within a rigorous monitoring regime.

摘要

背景

阿仑单抗最近已被批准用于治疗复发型多发性硬化症(MS),但由于对不良事件的长期研究仍然有限,其使用仍存在担忧。此外,对其在临床实践中的应用和疗效持久性尚未有清晰的认识。

目的

调查接受阿仑单抗治疗的多中心队列患者的长期疗效和安全性结果。

方法

确定2000年开始治疗并在三个地区中心进行随访的患者。通过临床记录审查获取并验证基线和前瞻性数据。

结果

确定了100例患者,平均随访6.1年(范围1 - 13年)。40例患者至少接受了一个额外的治疗周期。治疗后年化复发率从2.1降至0.2(p<0.0001),并在长达八年的随访中持续。扩展残疾状态量表(EDSS)评分的平均变化为+0.14。47例患者出现继发性自身免疫。

结论

观察到的复发率降低与临床试验报告的结果相符,但我们无法证实先前关于残疾逆转的观察结果。40%的患者需要额外的治疗周期。自身免疫性不良事件很常见,发生率高于先前报告,但在很大程度上是可预测的,并且可以在严格的监测方案内有效管理。

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