Introduction/Aims: The Midwest has the highest regional prevalence of self-reported amyotrophic lateral sclerosis (ALS) in the United States, but with limited epidemiological studies. We aimed to explore the characteristics of patients with ALS in the Midwest.Methods: This was a retrospective cohort study of participants with ALS deceased between January, 2010, and September, 2020, registered with the ALS Association Mid-America Chapter. Demographics and clinical variables included gender, race/ethnicity, military status, site of onset, interventions (gastrostomy, non-invasive ventilation, tracheostomy), and visits to ALS Association-registered clinics. Disease characteristics were compared to the National ALS Registry, and survival analysis was performed followed by sample augmentation with historical data to estimate survival with hypothetical censoring. Results:The database included 1447 participants with a mean age at diagnosis of 65.7 ± 11.9 y (>60 y at diagnosis: 72%). The median survival from symptom onset was 28.0 mo (95% confidence limit: 26.3, 29.7); sample augmentation increased this to 41.0 mo (38.5, 43.5). Bulbar onset disease and older age at diagnosis were associated with shorter survival. Participants not followed in ALS-Association registered clinics were more frequently male, had familial onset and tracheostomy. Veterans (N = 298) were older at diagnosis but had similar survival after adjustment for age. Discussions: Our cohort had an older age at onset and more frequent bulbar onset than the National ALS Registry, perhaps reflecting ascertainment biases in each registry. Prospective cohort studies with more clinical and functional data are needed to better characterize ALS in Midwest, veterans, and non-clinic populations, and to optimize care.
Background: Motor neuron disorders are rare, progressive neurodegenerative diseases which affect multiple domains of motor function. The ability to assess function from home using an electronic medical record (EMR) would facilitate pragmatic studies. Objective: To develop a Patient Activity of Daily Living scale for Amyotrophic Lateral Sclerosis and other motor neuron disorders (PADL-ALS) to support large pragmatic trials. Methods: The Greater Plains Collaborative Clinical Data Research Network (GPC) developed and tested the feasibility of using the PADL-ALS. We convened patient and caregiver focus groups and in-person meetings to recommend changes to the ALS Functional Rating Scale-Revised (ALSFRS-R), which clarified language and added questions about pseudobulbar affect, pain, and faith. Feasibility was determined by conducting a survey of participants identified using EMR-computable phenotypes and returned via patient-preferred modalities. Results: Surveys were distributed to 1079 participants at nine GPC health systems. The survey response rate was 44.4% (range 12.9-57.66%): male to female ratio 1.56; 84% self-identified as a patient with ALS. Patient respondents used computers or tablets more frequently than caregivers responding on their behalf. The PADL-ALS correlated to clinic-performed ALSFRS-R within 4 weeks of survey completion (n=33, rho=0.93, Kansas only). The pseudobulbar affect question correlated to functional motor burden. Over 80% agreed to be contacted for future research opportunities. Conclusion: We demonstrated the feasibility of determining functional burden with the PADL-ALS using an EMR-computable phenotype. Future directions include implementing the PADL-ALS to answer pragmatic questions about ALS care.
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