PNH

Update of PNH guideline

 

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

Bringing together AA/PNH patients

 

Most people who suffer from a rare disease such as aplastic anaemia (AA) or PNH are initially alone with this diagnosis: Since only a few thousand people in Germany suffer from AA and PNH , hardly anyone of their family and friends has ever heard of these diseases, let alone suffers from them themselves. This often makes it difficult for those affected to exchange questions, worries, tips and experiences.

At this point, we as an association, together with the lichterzellen foundation , would like to bring together locally as many AA and PNH patients and relatives as possible , so that they do not only meet on the internet, but maybe soon face to face with other affected people in their vicinity. We keep a list of patients and relatives in which you can register if you wish. Please fill out this form and send it to us.

WEITER

Approval of pegcetacoplan for PNH patients

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.

Recommendations regarding COVID-19 vaccination in AA/PNH updated

 

The SARS-CoV-2 pandemic continues to have us in its grip. Many AA/PNH patients are seeking advice on whether to get vaccinated (again) against the virus. Two of Germany's AA/PNH specialists, Dr Britta Höchsmann (UK Ulm) and Dr Jens Panse (UK Aachen), have analysed the scientific data available so far and updated their recommendations on vaccination. The two doctorsemphasise that their letter refelcts their personal recommendations and is not an official statement of the university hospitals or theDeutschen German Society of Haematologoy and Medical Oncology (DGHO). The letter should be understood as an aid until the statement of the DGHO expert group (non-malignant haematology working party) is available.

In general, Dr Höchsmann and Dr Panse consider the risk of infection with the SARS-CoV-2 ("Corona") virus to be more serious than the possible consequences of vaccination. The two experts emphasise that depending on the disease and status, some things should be taken into account, e.g. time of vaccination, type of vaccine etc.

For information is available in German in the recommendation letter regading COVID-19 vaccination in AA/PNH by Dr Höchsmann and Dr Panse.

12th Essen PNH/AA Seminar – Summary

WEITER

Studying with an invisible disability – a student explains

 

A disability - what is it actually? This question is not so easy to answer, because what is commonly understood as a disability can come in many forms and expressions - and then there is also the type of disability that is not noticed at first glance: we are talking about the so-called "invisible disability" . Examples of this are chronic illnesses and disabilities, which are associated with fatigue and rapid exhaustion and thus impair social participation. Aplastic anemia and paroxysmal nocturnal hemoglobinuria can also be included. While in the case of invisible disabilities the possible stigmatization by others does not apply from minute one of getting to know each other, this non-recognition of the disability also leads to expectations that are sometimes overwhelming. Ly Tran has to struggle with this again and again: Ly is a linguistics student and reports in the podcast seriesWEITER

Lectures of the Ulm Patient Day online

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

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

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.

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