实践指南推荐摘要：多发性硬化症成人的疾病修正治疗：美国神经病学学会指南制定、传播和实施小组委员会的报告。

Practice guideline recommendations summary: Disease-modifying therapies for adults with multiple sclerosis: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology.

机构信息

From the Department of Neurology (A.R.-G.), Cleveland Clinic, OH; Department of Neurology (G.S.D.), Charles F. and Joanne Knight Alzheimer Disease Research Center, Washington University in St. Louis, MO; Department of Community Health Sciences (R.A.M.), Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Canada; Department of Neurology (A.R.), Mayo Clinic, Rochester, MN; UCSF Weill Institute for Neurosciences, Department of Neurology (B.A.C.C.), University of California, San Francisco; Department of Neurology (G.S.G.), Kansas University Medical Center, Kansas City; Department of Neurology (M.H.), School of Medicine, University of Louisville, KY; Consortium of Multiple Sclerosis Centers (J.H.), Hackensack, NJ; Department of Neuroscience (J.P.H.), St. Luke's University Health Network, Bethlehem, PA; Department of Neurology (D.E.J.), University of Virginia, Charlottesville; Consortium of Multiple Sclerosis Centers (R.L.), Hackensack, NJ; Department of Neurology (R.L.), School of Medicine, Wayne State University, Detroit, MI; Department of Neurology (D.P.), Keck School of Medicine, University of Southern California; Neurology Department (S.P.), Southern California Permanente Medical Group, Kaiser, Los Angeles; National Multiple Sclerosis Society (C.S.), Arlington, VA; National Multiple Sclerosis Society (R.S.), New York, NY; Santa Fe (J.S.), NM; Heart Rhythm Society (T.S.D.G.), Washington, DC; American Academy of Neurology (S.A.M.), Minneapolis, MN; and Department of Clinical Neurosciences, Psychiatry, Pediatrics and Community Health Sciences, Cumming School of Medicine (T.P.), University of Calgary, Alberta, Canada.

出版信息

Neurology. 2018 Apr 24;90(17):777-788. doi: 10.1212/WNL.0000000000005347.

DOI:10.1212/WNL.0000000000005347

PMID:29686116

Abstract

OBJECTIVE

To develop recommendations for disease-modifying therapy (DMT) for multiple sclerosis (MS).

METHODS

A multidisciplinary panel developed DMT recommendations, integrating findings from a systematic review; followed an Institute of Medicine-compliant process to ensure transparency and patient engagement; and developed modified Delphi consensus-based recommendations concerning starting, switching, and stopping DMTs pertinent to people with relapsing-remitting MS, secondary progressive MS, primary progressive MS, and clinically isolated syndromes of demyelination. Recommendations were supported by structured rationales, integrating evidence from one or more sources: systematic review, related evidence (evidence not from the systematic review), principles of care, and inference from evidence.

RESULTS

Thirty recommendations were developed: 17 on starting DMTs, including recommendations on who should start them; 10 on switching DMTs if breakthrough disease develops; and 3 on stopping DMTs. Recommendations encompassed patient engagement strategies and individualization of treatment, including adherence monitoring and disease comorbidity assessment. The panel also discussed DMT risks, including counseling about progressive multifocal leukoencephalopathy risk in people with MS using natalizumab, fingolimod, rituximab, ocrelizumab, and dimethyl fumarate; and made suggestions for future research to evaluate relative merits of early treatment with higher potency DMTs vs standard stepped-care protocols, DMT comparative effectiveness, optimal switching strategies, long-term effects of DMT use, definitions of highly active MS, and effects of treatment on patient-specified priority outcomes. This guideline reflects the complexity of decision-making for starting, switching, or stopping MS DMTs. The field of MS treatment is rapidly changing; the Academy of Neurology development process includes planning for future updates.

摘要

目的

制定多发性硬化症（MS）的疾病修正治疗（DMT）建议。

方法

一个多学科小组制定了 DMT 建议，整合了系统评价的结果；遵循符合医学研究所要求的流程，以确保透明度和患者参与；并制定了有关开始、转换和停止 DMT 的修改后的德尔菲共识建议，这些建议与复发缓解型 MS、继发进展型 MS、原发进展型 MS 和脱髓鞘的临床孤立综合征患者有关。建议得到了结构化理由的支持，整合了来自一个或多个来源的证据：系统评价、相关证据（非来自系统评价的证据）、护理原则和从证据中推断出的证据。

结果

制定了 30 条建议：17 条关于开始 DMT 的建议，包括谁应该开始使用它们的建议；10 条关于如果突破性疾病发生时如何转换 DMT 的建议；以及 3 条关于停止 DMT 的建议。建议包括患者参与策略和治疗个体化，包括药物依从性监测和疾病合并症评估。小组还讨论了 DMT 的风险，包括对使用那他珠单抗、芬戈莫德、利妥昔单抗、奥瑞珠单抗和富马酸二甲酯的 MS 患者进行进行性多灶性白质脑病风险的咨询；并提出了未来研究的建议，以评估早期使用更高效力的 DMT 与标准阶梯式治疗方案、DMT 比较效果、最佳转换策略、DMT 使用的长期效果、高度活跃性 MS 的定义以及治疗对患者指定的优先结果的影响。本指南反映了开始、转换或停止 MS DMT 决策的复杂性。MS 治疗领域正在迅速变化；神经病学学会的发展过程包括对未来更新的计划。