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进行性共济失调的诊断和管理指南。

Guidelines on the diagnosis and management of the progressive ataxias.

机构信息

Department of Neurology, Essex Centre for Neurological Sciences, Queen's Hospital, Romford, RM7 0AG, UK.

Ataxia UK, 12 Broadbent Close, London, N6 5JW, UK.

出版信息

Orphanet J Rare Dis. 2019 Feb 20;14(1):51. doi: 10.1186/s13023-019-1013-9.

Abstract

The progressive ataxias are a group of rare and complicated neurological disorders, knowledge of which is often poor among healthcare professionals (HCPs). The patient support group Ataxia UK, recognising the lack of awareness of this group of conditions, has developed medical guidelines for the diagnosis and management of ataxia. Although ataxia can be a symptom of many common conditions, the focus here is on the progressive ataxias, and include hereditary ataxia (e.g. spinocerebellar ataxia (SCA), Friedreich's ataxia (FRDA)), idiopathic sporadic cerebellar ataxia, and specific neurodegenerative disorders in which ataxia is the dominant symptom (e.g. cerebellar variant of multiple systems atrophy (MSA-C)). Over 100 different disorders can lead to ataxia, so diagnosis can be challenging. Although there are no disease-modifying treatments for most of these entities, many aspects of the conditions are treatable, and their identification by HCPs is vital. The early diagnosis and management of the (currently) few reversible causes are also of paramount importance. More than 30 UK health professionals with experience in the field contributed to the guidelines, their input reflecting their respective clinical expertise in various aspects of ataxia diagnosis and management. They reviewed the published literature in their fields, and provided summaries on "best" practice, including the grading of evidence available for interventions, using the Guideline International Network (GIN) criteria, in the relevant sections.A Guideline Development Group, consisting of ataxia specialist neurologists and representatives of Ataxia UK (including patients and carers), reviewed all sections, produced recommendations with levels of evidence, and discussed modifications (where necessary) with contributors until consensus was reached. Where no specific published data existed, recommendations were based on data related to similar conditions (e.g. multiple sclerosis) and/or expert opinion. The guidelines aim to assist HCPs when caring for patients with progressive ataxia, indicate evidence-based (where it exists) and best practice, and act overall as a useful resource for clinicians involved in managing ataxic patients. They do, however, also highlight the urgent need to develop effective disease-modifying treatments, and, given the large number of recommendations based on "good practice points", emphasise the need for further research to provide evidence for effective symptomatic therapies.These guidelines are aimed predominantly at HCPs in secondary care (such as general neurologists, clinical geneticists, physiotherapists, speech and language therapists, occupational therapists, etc.) who provide care for individuals with progressive ataxia and their families, and not ataxia specialists. It is a useful, practical tool to forward to HCPs at the time referrals are made for on-going care, for example in the community.

摘要

进行性共济失调是一组罕见且复杂的神经退行性疾病,医护人员(HCPs)对其认识往往不足。患者支持组织共济失调英国(Ataxia UK)认识到对这类疾病缺乏认识,因此制定了用于诊断和管理共济失调的医学指南。虽然共济失调可能是许多常见病症的一种症状,但这里的重点是进行性共济失调,包括遗传性共济失调(例如脊髓小脑共济失调(SCA)、弗里德里希共济失调(FRDA))、特发性散发性小脑共济失调以及以共济失调为主要症状的特定神经退行性疾病(例如多系统萎缩(MSA-C)的小脑变异型)。有 100 多种不同的疾病可导致共济失调,因此诊断具有挑战性。尽管大多数这些疾病都没有改变疾病进程的治疗方法,但这些疾病的许多方面都是可治疗的,医护人员的识别至关重要。早期诊断和管理目前为数不多的可逆转病因也至关重要。超过 30 名在该领域具有丰富经验的英国卫生专业人员为指南做出了贡献,他们的意见反映了他们在共济失调诊断和管理各个方面的临床专业知识。他们在各自的领域中审查了已发表的文献,并根据指南国际网络(GIN)标准,就“最佳”实践提供了干预措施的证据分级摘要,包括相关章节。由共济失调专家神经病学家和共济失调英国代表(包括患者和照顾者)组成的指南制定小组审查了所有章节,提出了具有证据水平的建议,并与贡献者讨论了修改意见(如有必要),直到达成共识。在没有特定已发表数据的情况下,建议是基于与类似疾病(例如多发性硬化症)相关的数据和/或专家意见。该指南旨在协助医护人员在照顾患有进行性共济失调的患者时提供帮助,指出基于证据的(如果存在)最佳实践,并作为参与管理共济失调患者的临床医生的有用资源。然而,它们还强调了迫切需要开发有效的疾病修正治疗方法,并且鉴于基于“良好实践要点”的大量建议,强调需要进一步研究为有效的对症治疗提供证据。本指南主要针对提供进行性共济失调患者及其家属护理的二级保健医护人员(例如,一般神经病学家、临床遗传学家、物理治疗师、言语和语言治疗师、职业治疗师等),而不是针对共济失调专家。它是一个有用的实用工具,可以在进行转诊以进行后续社区护理时转发给医护人员。

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