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Monitoring Clinical Course and Treatment Response in Chronic Inflammatory Demyelinating Polyneuropathy During Routine Care: A Review of Clinical and Laboratory Assessment Measures.
JAMA Neurology ( IF 29.0 ) Pub Date : 2020-09-01 , DOI: 10.1001/jamaneurol.2020.0781
Jeffrey A Allen 1 , Ingemar S J Merkies 2, 3 , Richard A Lewis 4
Affiliation  

Importance Identifying clinical change in many neurologic diseases, including chronic inflammatory demyelinating polyneuropathy (CIDP), can be challenging. At the same time, how change is defined heavily influences a patient's diagnostic and treatment pathway. It can be especially problematic when equivocal subjective observations are interpreted as clinically meaningful and then used to make diagnostic and treatment decisions. Change in clinical trials is strictly defined by a preselected metric, but there is a perception that formal outcomes collection during routine clinical care is neither feasible nor necessary. Given the importance placed on how change is interpreted, there is a need to select assessments that can be applied to routine care that are representative of the neurologic disease state.

Observations For an outcome measure to be useful during clinical trials, it must have good reliability, validity, be responsive to change, and have clinical meaning. To be useful during routine clinical care, the assessment must additionally be easy to collect without the need for extensive training or equipment and should provide an immediately available result that can be rapidly quantified and interpreted. Chronic inflammatory demyelinating polyneuropathy is clinically heterogeneous and so is best evaluated with a diverse group of assessment tools. Assessing strength impairment, disability, and quality of life is ideally suited for everyday practice when caring for patients with CIDP. While electrophysiologic studies, imaging, cerebrospinal fluid, and nodal/paranodal antibodies can provide diagnostic data, they are less practical and helpful longitudinal assessment tools.

Conclusions and Relevance Sound clinimetric outcome measures in CIDP are widely available and have the potential to help clinicians objectify treatment response and disease progression. Such data are critically important when justifying the need for ongoing or periodic immunotherapy, documenting relapse or deterioration, or providing reassurance of disease improvement, stability, or remission.



中文翻译:

在常规护理期间监测慢性炎性脱髓鞘性多发性神经病的临床过程和治疗反应:临床和实验室评估措施的综述。

重要性 鉴定包括慢性炎症性脱髓鞘性多发性神经病(CIDP)在内的许多神经系统疾病的临床变化可能具有挑战性。同时,如何定义更改会严重影响患者的诊断和治疗途径。当模棱两可的主观观察结果被解释为具有临床意义,然后用于做出诊断和治疗决策时,这可能尤其成问题。临床试验的变化是由预先选择的指标严格定义的,但是有一种看法认为,在常规临床护理过程中进行正式的结局收集既不可行,也没有必要。考虑到如何解释变化的重要性,有必要选择可以应用于常规治疗的代表神经系统疾病状态的评估。

观察结果 为了使结果度量在临床试验中有用,它必须具有良好的可靠性,有效性,对变化敏感并具有临床意义。为了在常规临床护理中有用,评估还必须易于收集而无需大量培训或设备,并且应提供可以快速量化和解释的立即可用的结果。慢性炎症性脱髓鞘性多发性神经病在临床上是异质性的,因此最好使用多种评估工具进行评估。评估CIDP患者时,评估力量障碍,残疾和生活质量非常适合日常练习。电生理研究,影像学检查,脑脊液和淋巴结/旁淋巴结抗体可以提供诊断数据,

结论与相关性 CIDP中合理的临床指标可广泛使用,并且有可能帮助临床医师确定治疗反应和疾病进展。当证明需要持续或定期进行免疫治疗,记录复发或恶化或保证疾病改善,稳定或缓解时,此类数据至关重要。

更新日期:2020-09-14
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