Author
Dr. Marlin Fromme and Prof. Pavel Strnad, Alpha1 Germany eV, as published in Alpha1 Journal 2/2022.
„"The follow-up study now has its first results, which impressively demonstrate the usefulness of the research methods used to date."“
Alpha-1 antitrypsin deficiency-associated liver disease (AATD-LD) is currently on the rise. Positive data on fazirsiran, an siRNA drug, were recently published, and consequently, many are now wondering whether this medication is also suitable for them. To answer this question, we need to be able to look into the future to predict the risk of developing clinically relevant liver disease. Fortunately, we are well-equipped for this, as this was the very question that motivated us to found the Alpha1 Consortium in 2015. Over the years, we have strived to comprehensively characterize the Alpha1 drugs clinically and in the laboratory. Last year, we contacted some of you by phone or mail to check on your progress and to see if any complications had arisen in the meantime.
This so-called longitudinal follow-up study has now yielded its first results, impressively demonstrating the usefulness of the examination methods used to date. The core of our investigations so far has been the measurement of liver stiffness using FibroScan. Initial analyses now show that values < 7.1 kPa can be classified as "not a cause for concern".
To date, we have been able to assess the course of more than 300 participants with these values for at least one year, and none of these participants experienced a life-threatening liver complication. Since the average follow-up period was almost four years, this result is an important reassurance. In contrast, there were 28 participants with liver stiffness ≥ 15 kPa, and almost half of them developed serious liver complications. Therefore, we would particularly like to ask participants with double-digit liver stiffness values to contact us and discuss treatment options within the framework of the ongoing clinical trials.
At the same time, we would like to ask for your support in these longitudinal studies. We will contact you again by phone or in writing in the coming months and ask you to complete the relevant questionnaires.
Even more important is to learn from test subjects who have developed serious complications, had to go to hospital, or even died in the meantime.
Only with such complete information can we provide you with expert advice and advance the drug trial. Help us gain a clearer view into the crystal ball!
For the liver team
Dr. Marlin Fromme and Prof. Pavel Strnad
