Tasks and Objectives
Articles of Association / Membership Application (German)
Function of the bone marrow
Aplastic Anaemia
Information brochure
Fatigue – Handouts for assessors (German)
Anaesthetics in AA/PNH
lichterzellen forum
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
Studying with an invisible disability – one of the victims tells us
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
Information on corona vaccination for aplastic anaemia
In a DGHO paper, Prof. Dr. Alexander Röth from the Essen University Hospital points out suspected cases in patients with aplastic anemia in connection with the SARS-CoV-2 ("Corona") vaccination. At the West German Tumor Center (WTZ), relapses of the disease (recurrences) have been observed in 3 patients after vaccination. In 3 other patients, the number of blood platelets (thrombocytes) continued to fall, and 2 patients received the initial diagnosis of aplastic anemia shortly after the vaccination. Patients with aplastic anemia should therefore consult their treating hematologist regarding (further) SARS-CoV-2 ("Corona") vaccinations. Download DGHO paper
Interview on the work of self-help
Rainer Göbel in an interview with Stephan Pregizer © Cancer Survivor
Some people may still have the idea that self-help is nothing more than sitting in a circle of chairs and only talking or even moaning about the topic in which the corresponding self-help group is concerned, in our case about illnesses.
In fact, one of our core tasks is to enable patients and their relatives to exchange ideas with one another, be it in the said circle of chairs, when visiting a restaurant together, in virtual meetings or in some other way. But our range of tasks is diverse and we offer much more than just an exchange. We publish information material, have information stands at events, attend congresses and are involved in (political) lobbying. Rainer Göbel, member of our board and chairman of our umbrella association German Leukemia & Lymphoma Aid (DLH), gives an insight into the work of self-help using the example of blood cancer in an interview with Stephan Pregizer from the Cancer Survivor initiative - take a look for yourself:
Ravulizumab approved for children and adolescents
Ravulizumab (trade name Ultomiris®) has been approved for the treatment of PNH in adults since July 2019, according to the manufacturer Alexion Pharmaceuticals, Inc. also the approval of the European Commission for use in children from 10 kg body weight and adolescents.
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 .
Third vaccination seems to increase protection against corona in immunosuppressive therapy
Vaccination, vaccination, vaccination - there are few other topics that dominate the media, politics, but also our conversations with family and friends so much at the moment: Rightly, because the development of vaccines against the coronavirus and the increasing "vaccination coverage" of the population feed the hope of overcoming this crisis at some point.
For a few months, however, there have been indications that the corona vaccination does not work equally well for all people: A study from March of this year found that people taking immunosuppressive therapy are less able to develop protection against a COVID-19 infection than people without this medication. This can be easily explained:WEITER
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
To our survey on COVID-19 vaccination / disease in AA/PNH
activities Aplastic anemia disability Exchange Experience report Infections Information booths Medication Meeting opinion poll PNH report society studies Studies TdSE TV Typing vaccination Workshop
Phone: +49 30 54 909 40 80
Fax: +49 30 54 909 40 89
Email: info(at)aa-pnh.org