Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells, characterized by hemolytic anemia, bone marrow failure, and thrombosis. A multinational phase III study reported that eculizumab, C5 monoclonal antibody therapy, effectively prevents complement attacks in patients with PNH who suffer from severe hemolytic attacks. As a result, both the levels of complement-induced hemolysis and quality of life (QOL) in patients with high disease activity have improved dramatically.

PNH and covid-19 research

Do PNH patients have the same resistance to Covid-19 as other people? If a PNH patient gets Covid, how does the disease progress? How does eculizumab affect this disease progression? How do PNH patients react to the corona vaccines? Here you can read the research we have found on these topics.

other pnh research

Recently, an increasing number of research in PNH and the burden of this illness has been conducted. In some of the research projects and publications, patient representatives were involved. These are some of the publications.

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quality of life

The PNH Global Alliance is committed to ensuring that the quality of life of patients is measured as accurately as possible in trials, patient registries and other studies. It is not self-evident that higher blood values (e.g. hemoglobin) lead to less fatigue and the ability to concentrate better; this must be demonstrated with a sophisticated measuring instrument, such as the QLQ-AA/PNH 54.  A report about this instrument has been created: Design and development of a disease specific quality of life tool for PNH patients. Read the document here

Patient representative Pascale Burmester from Germany was interviewed by Dr Jens Panse about the meaning of the AA and PNH specific Quality of Life Questionnaire (QLQ -AA/PNH 54). Watch the video of the interview for more information! Subtitles are available in different languages. Please select a language in the settings of the video.

Here is the current QLQ AA/PNH 54 quality of life questionnaire for patients with AA and/or PNH which measures how they have been feeling recently. Patients are asked to try to answer as many of the questions as possible by ticking one of the four boxes. Download the document with questions here.

Click the button below to read how the QLQ AA/PNH 54 is being used in Japan.

clinical trials

Three times a year we update our overview of clinical trials in PNH taking place in each country. For the latest information on clinical trials you may want to go to the source of our overview which can be found here.


Wellbeing encompasses many strands including diet, sleep, exercise and management of stress levels. A number of PNH patients report that stress makes their symptoms worse.