We propose to use the OHDSI Community Network to help us explore the natural history (NH) of the AA, AL, and ATTR forms.
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We want to understand how best to study these diseases using clinical trial and real world evidence data.
Our goal is to increase our knowledge about the disease process to help inform design and conduct of clinical trials in these disease spaces; decide upon the best study design, select enrollment criteria, endpoints, when to measure endpoints, etc. Ultimately, we would like to bring more interest from pharmaceutical companies to these disease spaces because approved therapies are lacking. The understanding of the NH of the diseases is key.
We have many scientific questions that want the OHDSI collaborative team to help us answer by tapping their network of Real World Evidence.
• What are the prognostic factors? Ideally, we would like to query the data to see if there are any correlations between different serum biomarkers, imaging biomarkers and clinical outcomes.
• What is the correlation between demographic factors (age, sex, race, underlying medical disorders, etc.) and different presentations of the disease, treatment responses, and outcomes?
• What is the relationship between treatments and favorable or unfavorable clinical outcomes and biomarkers?
• Could a prognostic disease model be constructed using clinical characteristics +/- biomarkers +/- histology?
• Which are the typical symptoms and the most frequent and higher than expected adverse events? These diseases have very heterogeneous in symptoms (i.e., GI (nausea, vomiting, diarrhea, constipation), motor/sensory neuropathy, brain fog, anxiety/depression, cardiomyopathy with preserved ejection fraction of ≥50% (HFpEF) (usually), renal failure, skin problems, etc.
• What is the prevalence of these different clinical presentations, and how do they evolve before and after treatment?
• Are any genetic factors pathognomonic and/or predictive of progression?