Approval of pegcetacoplan for PNH patients

blood cells

On 13 December 2021 a new drug for the treatment of adult patients with PNH was approved in the EU: The substance pegcetacoplan which inhibits specific processes in the immune system will probably be available in Germany in the beginning of 2022. Its trade name is Aspaveli®.

The drug is a so-called C3 complement inhibitor: The complement system is part of the body's own immune system which in PNH attacks the red blood cells (erythrocytes) and destroys them. Pegcetacoplan inhibits a certain part of the complement system and thereby the destruction of red blood cells outside the blood vessels (extravascular), leading to an improved life cycle of the erythrocytes and improved haemoglobin levels.

Pegecetacoplan is approved for patients who remain anaemic despite treatment with a C5 inhibitor (eculizumab or ravulizumab) for at least 3 months. C5 is also part of the complement system. The drug is administered twice a week as a subcutaneous infusion. Among the most common side effects are reactions at the injection site, infections of the upper respiratory tract, headache, abdominal pain, and diarrhoea.

"Summary of product characteristics" by the European Medicines Agency EMA.

The drug is not entirely new: On 5 April of this year, we already reported in detail on the mechanism of action and the current studies on it in a blog post on our website.

Lectures of the Ulm Patient Day online

Rare Disease Day logo

In connection with the Day of Rare Diseases , the Ulm University Hospital held another AA / PNH patient seminar on March 6th, 2021. Due to the pandemic, it took place in virtual form for the first time and also attracted numerous participants in this format.

The lectures of the varied program are available for download on the website of the Center for Rare Diseases of the Ulm University Hospital .

Reminder: Survey on COVID-19 at AA and PNH

syringe with vials for vaccination © weyo

We read and hear it every day in the newspaper and on the radio - the COVID vaccination operation is in full swing and the number of first and second vaccinated people in Germany is steadily increasing. The proportion of vaccinated AA / PNH patients is also increasing, but whether and how they react to the vaccination compared to healthy people has not yet been scientifically recorded. Therefore we have created a short anonymous survey and ask you to tell us about your experience with vaccination. You can also take part in the survey if you cannot or do not want to be vaccinated or if you have contracted COVID-19. In doing so, you help to create a meaningful database to further improve the care of AA / PNH patients in the future.

We make the results of the survey available to science free of charge. Participating patients can also receive the evaluations on request.

The survey and further information can be found under the following link: https://aa-pnh.org/umfragen/irvap/de/ and in English at https://aa-pnh.org/umfragen/irvap/en/

Thank you for your help!

Workshop on AA / PNH on June 14th, 2021

Notebook und Becher © Chris Montgomery on Unsplash

A virtual workshop on aplastic anemia and PNH will take place on Monday, June 14th, 2021 at 5 p.m. Prof. Dr. Jörg Westermann from Charité - Universitätsmedizin Berlin will inform you about the origin, diagnosis and treatment options for both diseases and is available to answer any questions. Afterwards, the participants have the opportunity to exchange experiences with one another.

The workshop is part of the DLH patient congress ( Deutsche Leukämie- & Lymphom-Hilfe eV ), which also offers numerous other workshops on, in some cases, cross-disease topics. Take a look at the program and register at https://www.dlh-kongress.de/der-kongress/online-anmeldung/ .

*** Attention! The workshop is not recorded so you cannot watch it later! ***



Collection of COVID-19 vaccination data from AA / PNH patients

Dear fellow AA and PNH patients,

Some time ago the first AA / PNH patients received their COVID-19 vaccination. But what effects do the vaccination or the COVID-19 disease have on our relatively small group of patients? In order to collect knowledge about this and to be able to make the results of science available for better care in the future, we are carrying out an international survey in cooperation with the Lichter Zellen foundation . The data is collected completely anonymously, i.e. without any personal or personally identifiable information. There is no way that your data can be assigned to you afterwards.

If you would like to receive further information or results about this survey or the AA / PNH register from us, you can enter your e-mail address after the survey. This is saved separately from the survey data.

The survey can be accessed under the following link: https://aa-pnh.org/umfragen/irvap/de/

Thank you in advance for your participation! You are helping all AA and PNH patients.

We would be delighted if, after answering the survey, you would inform other AA / PNH patients about it. If you have any questions or suggestions, please contact us by email at info (at) aa-pnh.de .

Results of the PEGASUS study on APL-2

A recently published study from Leeds, England examines the effectiveness of the new drug pegcetacoplan on paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan has not yet been approved for PNH therapy in Europe, but approval is currently being examined by the European Medicines Agency (EMA). For this reason, we would like to briefly introduce the drug and the study to you and first take a brief look at the blood formation disorder underlying PNH in order to understand how Pegcetacoplan works:

Normally there are certain proteins on the surface of the red blood cells (erythrocytes), which transport oxygen through our body. Among other things, these regulate the immune system and prevent the erythrocytes from being broken down prematurely as they circulate through the body. At the PNHWEITER

20th anniversary of the association – a look back

Do you know this feeling? Wake up in the morning, maybe it smells like coffee, maybe breakfast is already ready and someone gives a bouquet of flowers or a first gift - someone calls and congratulates, a serenade at work or among friends and at the end of the day a nice, big one Celebrate with ... stop! It's February, it's the year 2021 and the corona pandemic still has a firm grip on our country and the world. The first lockdown was decided almost a year ago and it is very likely that you have already experienced it: It is your birthday in the pandemic .

Our association Aplastic Anemia & PNH eV also celebrated its birthday last week, namely its 20th anniversary. For known reasons, however, there was no question of “celebrating” in this context - even if we did not give up hope of being able to make up for it one day. Until then, however, we don't want to simply skip the club's anniversary: It's a wonderful opportunity to review the past 20 years. Perhaps you have been with us from the start, perhaps you only became aware of the association a few weeks ago: We are very pleased that you are part of it, we would like to thank you for your interest, your questions and suggestions, and we would like to take a look back at you invite you to 20 years of AA & PNH eV.

WEITER

EMAA trial

EMAA trail for patients with moderate Aplastic Anaemia Adult patients with acquired moderate Aplastic Anaemia (mAA) who have not received a specific treatment for their mAA may be included in the EMAA trial of Ulm University (Eltrombopag in Moderate Aplastic Anaemia). They receive either ciclosporin and eltrombopag or ciclosporin and a placebo. The aim of the trial is to evaluate the efficacy and safety of the combination therapy of ciclosporin and eltrombopag. Patients with or without prior transfusions can be included.
WEITER