Suchergebnisse

The current state of research in environmental risk factors of ALS has provided many intriguing possible associations. Yet only one-smoking is at this time firmly established. The methodologic difficulties with studying a rare disease that occurs late in life, which could be related to exposures many decades ago, make relationships dauntingly difficult to prove. Despite continued improvement in methodology, significant challenges remain. The diagnostic criteria for ALS are complicated and there are continued efforts to improve them. As they are, the criteria do not yet capture all people with ALS, which further complicates epidemiologic studies. It is hoped that larger datasets with better characterization of different clinical features and laboratory markers will provide more robust estimates of risk factors in ALS in the years to come. A better understanding of environmental risk factors could help reduce exposures and it is hoped markedly reduce ALS incidence over time. Epidemiologic research is critical to advance this field, but the relative rarity of ALS and the current notion that exposures may affect the risk of ALS only decades later make such projects complex with many challenges. One US project of great potential is the National ALS Registry. It is a congressionally-mandated prospective population-based registry encompassing the entire US population. In addition to quantifying the incidence, prevalence, and demographics of ALS in the US, another main goal of the Registry is to examine the risk factors for the disease through online risk factor modules. There are currently 17 different risk factor modules that persons with ALS can complete including, but not limited to, cigarette smoking, alcohol consumption, military service history, occupational history, and a family history of ALS. Since the Registry's launch in October 2010, over 45,000 online risk factor modules have been completed. To our knowledge, this is the largest and most geographically diverse collection of risk factor data available about ...

Abstract
Objective
To characterize the participant demographics in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database compared with the web-portal National Amyotrophic Lateral Sclerosis (ALS) Registry (the Registry).

Methods
Demographics and ALS symptom information were compared between the self-reported registrant data in the Registry web portal (2010–2021) and the latest available PRO-ACT data (updated August 2022), which is a collection of clinical trials data.

Results
Greater percentages of younger (≤ 59 years old) but smaller percentages of older (60 + years old) participants were represented in PRO-ACT compared to Registry. Enrollment for minority race groups was greater in the Registry portal data, but race information was largely missing/unknown in PRO-ACT database. Median age at the time of diagnosis and age at the time of symptom onset were significantly higher for Registry enrollees compared to the participants of PRO-ACT. Symptom onset sites were similarly reported, but duration between self-noted symptom onset and diagnosis was slight, but significantly longer for the Registry enrollees (11 vs. 9 months). Hispanic were as likely as non-Hispanic to participate in research studies, based on the Registry data.

Conclusion
There was a notable difference in the age distribution and minority representation of enrollees between the PRO-ACT and Registry study populations. Age distribution in the PRO-ACT database skewed to a younger and less diverse cohort. Despite the clinical heterogeneity and complex disease mechanism of ALS, identifying the underrepresented demographic niche in the PRO-ACT and Registry study populations can help improve patient participation and criteria for patient selection to enhance generalizability.

Abstract
Background
Amyotrophic lateral sclerosis (ALS) is a progressive, fatal disease with largely unknown etiology. This study compares racial differences in clinical characteristics of ALS patients enrolled in the National ALS Registry (Registry).

Methods
Data from ALS patients who completed the Registry's online clinical survey during 2013–2022 were analyzed to determine characteristics such as site of onset, associated symptoms, time of symptom onset to diagnosis, and pharmacological and non-pharmacological interventions for White, Black, and other race patients.

Results
Surveys were completed by 4242 participants. Findings revealed that Black ALS patients were more likely to be diagnosed at a younger age, to have arm or hand initial site of onset, and to experience pneumonia than were White ALS patients. ALS patients of other races were more likely than White ALS patients to be diagnosed at a younger age and to experience twitching. The mean interval between the first sign of weakness and an ALS diagnosis for Black patients was almost 24 months, statistically greater than that of White (p = 0.0374; 16 months) and other race patients (p = 0.0518; 15.8 months). The mean interval between problems with speech until diagnosis was shorter for White patients (6.3 months) than for Black patients (17.7 months) and other race patients (14.8 months).

Conclusions and Relevance
Registry data shows racial disparities still exist in the diagnosis and clinical characteristics of ALS patients. Increased recruitment of non-White ALS patients and better characterization of symptom onset between races might aid clinicians in diagnosing ALS sooner, leading to earlier therapeutic interventions.