Patient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional study.
Patient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional study.
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患者报告了症状在肌萎缩性侧索硬化症（Prism-Als）中的影响：一项国家，横断面研究。
DOI：
10.1016/j.eclinm.2022.101768
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发表时间：
2023-01
影响因子：
15.1
通讯作者：
Heatwole, Chad
中科院分区：
文献类型：
--
作者：
Zizzi, Christine;Seabury, Jamison;Rosero, Spencer;Alexandrou, Danae;Wagner, Ellen;Weinstein, Jennifer S.;Varma, Anika;Dilek, Nuran;Heatwole, John;Wuu, Joanne;Caress, James;Bedlack, Richard;Granit, Volkan;Statland, Jeffrey M.;Mehta, Paul;Benatar, Michael;Heatwole, Chad
随着新的治疗干预措施正在肌萎缩侧索硬化症（ALS）人群中开发和测试，有必要更好地了解对ALS患者生活影响最大的症状和问题。我们的目的是确定国家ALS样本中成年人经历的症状的频率和相对重要性，并确定与该人群中最大疾病负担相关的因素。我们对具有不同ALS表型的个体进行了15次定性访谈，并分析了2018年8月至2019年3月期间关于ALS症状性疾病负担的732条报价。我们随后在2019年7月至2019年12月期间通过疾病控制和预防中心（CDC）国家ALS登记处对497名ALS和ALS变体参与者进行了一项全国性的横断面研究。参与者报告了189个症状性问题的患病率和相对重要性，这些问题代表了先前通过定性访谈确定的17个症状性主题。进行分析，以确定年龄，性别，教育，就业，自症状发作以来的时间，症状发作的位置，饲管状态，呼吸状态和语言状态与症状和症状性主题患病率的关系。在我们的样本中，患病率最高的症状主题是无法进行活动（93.8%），疲劳（92.6%），手或手指问题（87.7%），活动或行走受限（86.7%）以及社交场合表现下降（85.7%）。参与者认为，无法进行活动和行动或行走的限制对他们的生活产生了最大的整体影响。ALS患者会经历各种影响他们生活的症状。这些症状的患病率和重要性在ALS人群中有所不同。最普遍和最重要的症状为未来的治疗干预提供了潜在的目标。研究经费由。
As novel therapeutic interventions are being developed and tested in the amyotrophic lateral sclerosis (ALS) population, there is a need to better understand the symptoms and issues that have the greatest impact on the lives of individuals with ALS. We aimed to determine the frequency and relative importance of symptoms experienced by adults in a national ALS sample and to identify factors that are associated with the greatest disease burden in this population. We conducted 15 qualitative interviews of individuals with varied ALS phenotypes and analyzed 732 quotes regarding the symptomatic disease burden of ALS between August 2018 and March 2019. We subsequently conducted a national, cross-sectional study of 497 participants with ALS and ALS variants through the Centers for Disease Control and Prevention's (CDC) National ALS Registry between July 2019 and December 2019. Participants reported on the prevalence and relative importance of 189 symptomatic questions representing 17 symptomatic themes that were previously identified through qualitative interviews. Analysis was performed to determine how age, sex, education, employment, time since onset of symptoms, location of symptom onset, feeding tube status, breathing status and speech status relate to symptom and symptomatic theme prevalence. Symptomatic themes with the highest prevalence in our sample were an inability to do activities (93.8%), fatigue (92.6%), problems with hands or fingers (87.7%), limitations with mobility or walking (86.7%), and a decreased performance in social situations (85.7%). Participants identified inability to do activities and limitations with mobility or walking as having the greatest overall effect on their lives. Individuals with ALS experience a variety of symptoms that affect their lives. The prevalence and importance of these symptoms differ among the ALS population. The most prevalent and important symptoms offer potential targets for improvements in future therapeutic interventions. Research funding was provided by .
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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：
9.9
作者：
Heatwole, Chad;Johnson, Nicholas;Moxley, Richard T., III
通讯作者：
Moxley, Richard T., III
影响因子：
9.9
作者：
Heatwole, Chad;Bode, Rita;Moxley, Richard, III
通讯作者：
Moxley, Richard, III
DOI：
10.1080/21678421.2016.1267768
发表时间：
2017-05
影响因子：
2.8
作者：
Strong MJ;Abrahams S;Goldstein LH;Woolley S;Mclaughlin P;Snowden J;Mioshi E;Roberts-South A;Benatar M;HortobáGyi T;Rosenfeld J;Silani V;Ince PG;Turner MR
通讯作者：
Turner MR
影响因子：
5.7
作者：
Talbott, E. O.;Malek, A. M.;Lacomis, D.
通讯作者：
Lacomis, D.