A systematic review of non-motor symptom evaluation in clinical trials for amyotrophic lateral sclerosis.
A systematic review of non-motor symptom evaluation in clinical trials for amyotrophic lateral sclerosis.
复制标题
在肌萎缩性侧面硬化症的临床试验中，对非运动症状评估的系统评价。
DOI：
10.1007/s00415-021-10651-1
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发表时间：
2022-01
影响因子：
6
通讯作者：
Pal S
中科院分区：
文献类型：
--
作者：
Beswick E;Forbes D;Hassan Z;Wong C;Newton J;Carson A;Abrahams S;Chandran S;Pal S
肌萎缩侧索硬化症（ALS）越来越被认为是一种多系统疾病，表现为常见且有影响力的非运动症状，例如神经精神症状、认知和行为变化、疼痛、睡眠障碍、疲劳和有问题的唾液。我们的目的是系统地回顾25年的ALS临床试验数据，以确定除了传统的运动功能和生存率测量外，是否还评估了非运动功能，以及评估的情况，以描述用于评估的工具。我们假设，在试验设计中，非运动症状的评估在很大程度上被忽略了，并且没有使用ALS合适的工具进行评估。我们回顾了自1994年利鲁唑获得许可以来，ALS研究药物的临床试验。对试验登记数据库（包括WHO国际试验登记、欧洲临床试验登记、clinicaltrials.gov和PubMed）进行了系统性检索，以查找1994年1月1日至2020年9月16日期间注册、完成或发表的II、III或IV期试验。没有语言限制。237项临床试验，包括超过29，222名参与者，调查了他们使用的非运动结局指标。这些试验评估了神经精神症状（75，32%）、认知障碍（16，6.8%）、行为改变（34，14%）、疼痛（55，23%）、睡眠障碍（12，5%）和疲劳（18，8%）。在184项试验（78%）中，有问题的唾液被评估为综合ALS-FRS（R）评分的一部分，但没有将其作为一种孤立的症状。31项（13%）试验（包括3585名受试者）不包括任何非运动症状评估。非运动症状，如神经精神，认知和行为变化，疼痛，睡眠障碍，疲劳和有问题的唾液尚未在ALS患者的试验中得到一致的评估。在评估时，非症状主要使用不适合ALS患者的仪器和损伤阈值进行评估。未来的试验应包括非运动症状评估，以评估候选药物的额外潜在治疗益处。CRD 42020223648。在线版本包含补充材料，可通过10.1007/s 00415 -021-10651-1获得。
Amyotrophic lateral sclerosis (ALS) is increasingly recognised as a multi-system disorder, presenting with common and impactful non-motor symptoms, such as neuropsychiatric symtpoms, cognitive and behavioural changes, pain, disordered sleep, fatigue and problematic saliva. We aimed to systematically review 25 years of ALS clinical trials data to identify if non-motor features were evaluated, in addition to the traditional measures of motor functioning and survival, and where evaluated to describe the instruments used to assess. We hypothesised that assessment of non-motor symptoms has been largely neglected in trial design and not evaluated with ALS-suitable instruments. We reviewed clinical trials of investigative medicinal products in ALS, since the licensing of riluzole in 1994. Trial registry databases including WHO International Trials Registry, European Clinical Trials Register, clinicaltrials.gov, and PubMed were systematically searched for Phase II, III or IV trials registered, completed or published between 01/01/1994 and 16/09/2020. No language restrictions were applied. 237 clinical trials, including over 29,222 participants, were investigated for their use of non-motor outcome measures. These trials evaluated neuropsychiatric symptoms (75, 32%), cognitive impairment (16, 6.8%), behavioural change (34, 14%), pain (55, 23%), sleep disturbances (12, 5%) and fatigue (18, 8%). Problematic saliva was assessed as part of composite ALS-FRS(R) scores in 184 trials (78%) but with no focus on this as an isolated symptom. 31 (13%) trials including 3585 participants did not include any assessment of non-motor symptoms. Non-motor symptoms such as neuropsychiatric, cognitive and behavioural changes, pain, disordered sleep, fatigue, and problematic saliva have not been consistently evaluated in trials for people with ALS. Where evaluated, non-symptoms were primarily assessed using instruments and impairment thresholds that are not adapted for people with ALS. Future trials should include non-motor symptom assessments to evaluate the additional potential therapeutic benefit of candidate drugs. CRD42020223648. The online version contains supplementary material available at 10.1007/s00415-021-10651-1.
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影响因子：
4.4
作者：
Cedarbaum, JM;Stambler, N;Nakanishi, A
通讯作者：
Nakanishi, A
影响因子：
3
作者：
BERGNER, M;BOBBITT, RA;GILSON, BS
通讯作者：
GILSON, BS
DOI：
10.1163/15685690260494924
发表时间：
2002-01-01
期刊：
PAIN CLINIC
影响因子：
--
作者：
Aksu, S;Çitak-Karakaya, I
通讯作者：
Çitak-Karakaya, I
影响因子：
4
作者：
Hita-Contreras, Fidel;Martinez-Lopez, Emilio;Martinez-Amat, Antonio
通讯作者：
Martinez-Amat, Antonio
影响因子：
4.7
作者：
Carvajal, Ana;Hribernik, Nezka;Centeno, Carlos
通讯作者：
Centeno, Carlos