Update of PNH guideline

Photo: psdesign1_fotolia

 

Guidelines are summaries of the current scientific knowledge about a disease – in addition to a complete list of possible symptoms and the most targeted diagnostic methods, you can read about the best possible treatment options according to the current state of knowledge. They serve primarily as a non-binding guide for doctors, but also contain interesting information for patients. Every now and then, guidelines are updated so that the findings of new studies can be incorporated into everyday medical practice - this is exactly what happened this month for the PNH guideline of the German Society of Haematology and Medical Oncology (DGHO) . The last version was still from November 2019 and a revision was necessary in particular to bring recommendations on the use of new drug therapy options up to the status of 2022: Specifically, we are talking about pegcetacoplan, the new C3 complement inhibitor, which has been approved in the EU since December 2021 under the trade name Aspaveli® and, according to the manufacturer Swedish Orphan Biovitrum AB (Sobi), is expected to be available as a therapy option in Germany from April 2022. WEITER

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 .

Invitation to the Essen patient and family seminar

Virtual classroom © manfredsteger on pixabay

We are delighted that the Essen University Hospital is inviting you to its 12th PNH and AA seminar for patients and relatives . Due to the ongoing pandemic, it will take place virtually this year: On Saturday, September 25th, 2021 from 10 a.m. to 12 p.m. you will be brought up to date on the diseases and will have the opportunity to ask questions.


Invitation and link to participate

our virtual group

Mug and Notebook (c) Chris Montgomery on Unsplash

We introduced our regular virtual meetings when we had to cancel our local group meetings due to the pandemic. We didn't know whether anyone would even be interested in sitting in front of the screen and talking to people who were initially unknown about their experiences with aplastic anaemia and PNH. It has now been almost a year since we held our first "trial meeting", and our virtual group has established itself as an independent group.

We are very happy about this and thank you for your encouragement, empathy and loyalty.

The meetings continue to take place every third Thursday of the month at 7 p.m. and we look forward to meeting "old friends" as well as new faces.

next meeting

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!

Survey on COVID-19 at AA and PNH

Spritze mit Fläschchen für Impfung © weyo

Dear AA / PNH fellow patients,

Some time ago we informed you about a survey initiated by our association in cooperation with the lichter zellen foundation . The purpose of this survey is to record the effects that COVID-19 vaccination has on our patient group. At the same time, AA / PNH patients who have recovered from a COVID-19 disease can also take part in the survey.

We were able to make the survey known around the world, among other things, through the global network PNH Global Alliance . In the meantime, 72 patients from Germany, Austria and Switzerland and 12 patients from Europe have taken part in our survey. We are particularly pleased about the participation of 8 patients so far from Australia, Iran, Canada, Turkey and the USA.

This gives us the chance to make the results of this survey available to science in order to enable better care for our relatively small patient group in the future.

In order to achieve meaningful results, we are still dependent on the active participation of AA / PNH patients. Please pay attention to our survey and take a few minutes to answer the anonymous survey. You are helping all AA and PNH patients.

WEITER

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 .