PNH

23/03/2022

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. READ MORE

By Ulrike
06/03/2022

Bringing together AA/PNH patients

Photo: Guido Grochowski_Adobe Stock

 

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.

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By admin
22/12/2021

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.

By admin
09/12/2021

Recommendations regarding COVID-19 vaccination in AA/PNH updated

syringe with vials for vaccination © weyo

 

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.

By admin
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