Clinical studies

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. WEITER

By Ulrike
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
28/05/2021

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! ***



By Ulrike
05/04/2021

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

By Ulrike
02/02/2021

RECOMMENDATIONS OF THE ULM UNIVERSITY HOSPITAL ON COVID-19 VACCINATION IN AA/PNH

A few weeks ago, vaccinations against the SARS-CoV-2 virus began in Germany. As announced in our February virtual group meeting, the Ulm University Hospital, as one of the most important AA/PNH centres in Germany, has now published a recommendation letter (German) on this topic. If you need assistance with details contained in the recommendation letter please email us at info(at)aa-pnh.org or give us a call: +49 30 549094080. We briefly summarise the most important contents for you below:WEITER
By Ulrike
01/10/2020

Results of RACE trial

The EBMT (European Society for Blood and Marrow Transplantation) conducts the RACE clinical trial to evaluate the combined therapy of equine ATG, ciclosporin and eltrombopag in patients with severe or very severe Aplastic Anaemia. "RACE" means "A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients". Results of the trial (in the Netherlands by way of example) show good response rates in the patients who received all three medicines. In Germany, the trial must not be conducted because of missing admissions. However, we consider it to be important to watch the trial data in order to use it ind admission discussions with authorities.
By Ulrike
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