CDC Grand Rounds: National Amyotrophic Lateral Sclerosis (ALS) Registry Impact, Challenges, and Future Directions

Mehta, Paul; Horton, D. Kevin; Kasarskis, Edward J.; Tessaro, Ed; Eisenberg, M; Laird, Susan; Iskander, John K.

doi:10.15585/mmwr.mm6650a3

Cited by 17 publications

(17 citation statements)

References 17 publications

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“…In general, most patients with ALS survive 2 to 5 years [4] . Nonetheless, there were patients who live for 10 years and 20 years or even longer [5] .…”

Section: Introductionmentioning

confidence: 99%

Predictors of survival in patients with amyotrophic lateral sclerosis: A large meta-analysis

Su¹,

Cheng²,

Jiang³

et al. 2021

eBioMedicine

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Background: The survival time of amyotrophic lateral sclerosis (ALS) is greatly variable and protective or risk effects of the potential survival predictors are controversial. Thus, we aim to undertake a comprehensive meta-analysis of studies investigating non-genetic prognostic and survival factors in patients with ALS. Methods: A search of relevant literature from PubMed, Embase, Cochrane library and other citations from 1 st January 1966 to 1 st December 020 was conducted. Random-effects models were conducted to pool the multivariable or adjusted hazard ratios (HR) by Stata MP 16.0. PROSPERO registration number: CRD42021256923. Findings: A total of 5717 reports were identified, with 115 studies meeting pre-designed inclusion criteria involving 55,169 ALS patients. Five dimensions, including demographic, environmental or lifestyle, clinical manifestations, biochemical index, therapeutic factors or comorbidities were investigated. Twenty-five prediction factors, including twenty non-intervenable and five intervenable factors, were associated with ALS survival. Among them, NFL (HR:3.70, 6.80, in serum and CSF, respectively), FTD (HR:2.98), ALSFRS-R change (HR:2.37), respiratory subtype (HR:2.20), executive dysfunction (HR:2.10) and age of onset (HR:1.03) were superior predictors for poor prognosis, but pLMN or pUMN (HR:0.32), baseline ALSFRS-R score (HR:0.95), duration (HR:0.96), diagnostic delay (HR:0.97) were superior predictors for a good prognosis. Our results did not support the involvement of gender, education level, diabetes, hypertension, NIV, gastrostomy, and statins in ALS survival. Interpretation: Our study provided a comprehensive and quantitative index for assessing the prognosis for ALS patients, and the identified non-intervenable or intervenable factors will facilitate the development of treatment strategies for ALS.

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“…In general, most patients with ALS survive 2 to 5 years [4] . Nonetheless, there were patients who live for 10 years and 20 years or even longer [5] .…”

Section: Introductionmentioning

confidence: 99%

Predictors of survival in patients with amyotrophic lateral sclerosis: A large meta-analysis

Su¹,

Cheng²,

Jiang³

et al. 2021

eBioMedicine

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“…Mehta [ 33 ] described the impacts, challenges, and subsequent directions of the National ALS Registry in the United States. In collaboration with the Centers for Disease Control and Prevention (CDC), this national registry was launched in October 2010.…”

Section: Resultsmentioning

confidence: 99%

“…We identified two ways of access, which can be: (1) open to anyone or (2) restricted to users who are previously registered by a health professional. In the American register, developed by Mehta [ 33 ], algorithms are used to verify data quality and veracity by comparing them with those from distinct sources. Such a factor points out the importance of identifying those that can feed register data.…”

Section: Discussionmentioning

confidence: 99%

National registry for amyotrophic lateral sclerosis: a systematic review for structuring population registries of motor neuron diseases

et al. 2021

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Background This article comprises a systematic review of the literature that aims at researching and analyzing the frequently applied guidelines for structuring national databases of epidemiological surveillance for motor neuron diseases, especially Amyotrophic Lateral Sclerosis (ALS). Methods We searched for articles published from January 2015 to September 2019 on online databases as PubMed - U.S. National Institutes of Health’s National Library of Medicine, Scopus, Science Direct, and Springer. Subsequently, we analyzed studies that considered risk factors, demographic data, and other strategic data for directing techno-scientific research, calibrating public health policies, and supporting decision-making by managers through a systemic panorama of ALS. Results 2850 studies were identified. 2400 were discarded for not satisfying the inclusion criteria, and 435 being duplicated or published in books or conferences. Hence, 15 articles were elected. By applying quality criteria, we then selected six studies to compose this review. Such researches featured registries from the American (3), European (2), and Oceania (1) continent. All the studies specified the methods for data capture and the patients’ recruitment process for the registers. Discussions From the analysis of the selected papers and reported models, it is noticeable that most studies focused on the prospect of obtaining data to characterize research on epidemiological studies. Demographic data (ID01) are present in all the registries, representing the main collected data category. Furthermore, the general health history (ID02) is present in 50% of the registries analyzed. Characteristics such as access control, confidentiality and data curation. We observed that 50% of the registries comprise a patient-focused web-based self-report system. Conclusion The development of robust, interoperable, and secure electronic registries that generate value for research and patients presents itself as a solution and a challenge. This systematic review demonstrated the success of a population register requires actions with well-defined development methods, as well as the involvement of various actors of civil society.

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“…The average survival of patients with ALS is 2-5 years. Approximately 20% of patients survive for five years, eventually succumbing to respiratory failure, whereas 5% of patients may live for 20 years [19,20].…”

Section: Amyotrophic Lateral Sclerosis (Als)mentioning

confidence: 99%

CRISPR/Cas9-Mediated Gene Correction to Understand ALS

Yun

2020

IJMS

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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease caused by the death of motor neurons in the spinal cord and brainstem. ALS has a diverse genetic origin; at least 20 genes have been shown to be related to ALS. Most familial and sporadic cases of ALS are caused by variants of the SOD1, C9orf72, FUS, and TARDBP genes. Genome editing using clustered regularly interspaced short palindromic repeats/CRISPR-associated system 9 (CRISPR/Cas9) can provide insights into the underlying genetics and pathophysiology of ALS. By correcting common mutations associated with ALS in animal models and patient-derived induced pluripotent stem cells (iPSCs), CRISPR/Cas9 has been used to verify the effects of ALS-associated mutations and observe phenotype differences between patient-derived and gene-corrected iPSCs. This technology has also been used to create mutations to investigate the pathophysiology of ALS. Here, we review recent studies that have used CRISPR/Cas9 to understand the genetic underpinnings of ALS.

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CDC Grand Rounds: National Amyotrophic Lateral Sclerosis (ALS) Registry Impact, Challenges, and Future Directions

Cited by 17 publications

References 17 publications

Predictors of survival in patients with amyotrophic lateral sclerosis: A large meta-analysis

Predictors of survival in patients with amyotrophic lateral sclerosis: A large meta-analysis

National registry for amyotrophic lateral sclerosis: a systematic review for structuring population registries of motor neuron diseases

CRISPR/Cas9-Mediated Gene Correction to Understand ALS

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