New recommendation regarding alpha-1 antitrypsin deficiency
Author
Marion Wilkens
In the summer of 2018, we wrote to the German Society for Pneumology and Respiratory Medicine (DGP) requesting a statement regarding alpha-1 antitrypsin deficiency replacement therapy. The reason for our inquiry was the uncertainty many doctors and patients felt about the purpose and necessity of this therapy. Wherever we found someone willing to listen, we asked for both a statement and a position paper, which already existed in many other countries and languages. Lo and behold, many drops wear away the stone, and we succeeded in getting the DGP to commission a position paper. It was written by many alpha-1 experts we know well and covers the entire spectrum of the disease.
We have also been advocating for a dedicated guideline on alpha-1 antitrypsin deficiency. Unfortunately, this won't be available anytime soon. Therefore, we are all the more pleased with the position paper we can now refer to, which provides physicians with the most important information about our condition.
The observational study from Ireland mentioned in the last newsletter was not available when the position paper was prepared; therefore, the RECOMMENDATION 7C: In patients with severe AATD and severe functional impairment (FEV1 < 30 % predicted), initiating augmentation therapy cannot generally be recommended; the decision to continue therapy should be made on a case-by-case basis. From our perspective, they certainly need to be reconsidered.
Let's stay on the topic of substitution:
Since May of this year, Grifols has published new product information for its product Prolastin©. Grifols has received extended approval to administer the substitution therapy in a home setting with the support of a healthcare professional. The new and relevant sections for us can be found under 4.2 and 4.4.
Both available products (Prolastin© and Respreeza©) are derived from human plasma. Plasma products are entirely dependent on blood donors. Up to 900 plasma donations are required to treat one Alpha-1 patient for a year. Currently, there is also a significant reliance on the USA, where the majority of the blood plasma needed in Europe is donated. For this reason, we have... Letter to our Health Minister Jens Spahn We have written a statement calling for more plasma donations. We also see a need to invest more in research into recombinant products.
New research findings:
A few days ago, our advisory board member Prof. Sabina Janciauskiene, together with other experts, published an article that may provide new avenues for the treatment of AATM in the future:
In simple terms, the idea is this: Preliminary data suggest that alpha-1 antitrypsin can be applied to the skin as a cream instead of undergoing replacement therapy. Using a model of human skin, the authors demonstrate that alpha-1 antitrypsin has no harmful effects and can penetrate the skin barrier. In cases of inflammation, alpha-1 antitrypsin appears to enhance regeneration and healing. It's conceivable that topical treatment could be particularly helpful for panniculitis and/or other skin conditions, but also for people starting replacement therapy, those with poor veins, or those who simply require a higher dose due to infection—many scenarios are possible, and this approach is worth exploring further.
Aside from that:
Our advisory board member, Dr. Pavel Strnad, also recently published an article on alpha-1 antitrypsin deficiency, focusing on the liver, and we would like to congratulate him. We hope to be able to provide a translation or summary of it soon, but for those who can't wait:
A note from us: Postponement of the anniversary
Having had to cancel so many wonderful and interesting events this year, we are currently working to ensure the safety of our 2021 anniversary information day. This event is particularly important to us, and we want to do everything we can to make sure we can celebrate 20 years of Alpha1 Germany e.V. together in 2021.
After careful consideration, we have decided to postpone the Alpha1 Information Day 2021. This will give international researchers more time to search for a vaccine against Covid-19.
A consultation with the Hotel Grand La Strada in Kassel revealed that we Information day with a large anniversary celebration The event can be postponed from April 23rd to 25th to September. Please note the weekend of [date missing]. 24.-26.09.2021 in your calendar.
We would be absolutely delighted if you could join us for our big anniversary celebration in September. Since we hold our regular anniversary celebration in April – and this time online - our General Meeting Since the conference will not be held in September, there will be much more time for good information, enriching conversations, and lively celebrations. Therefore, we ask you to also note the date. 24.04.2021 to be kept clear for Alpha1 Germany.
Corona Update
On December 31, 2019, cases of the disease from China were officially reported to the WHO. Even more than six months later, the coronavirus SARS-CoV-2 still hasn't let go of us.
- Here is a graphic explanation of how the virus infects us.
- And further explanations of what happens in the body.
- Here is a study that confirms that COVID-19 is generally a mild illness in children, including infants.
- Do you know your blood type? You should, but not only for the reason of this article.
And finally, some good news: We have not received any reports of new cases of Alphas being infected with COVID-19. Stay healthy and enjoy the weather – get plenty of fresh air!
