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IPIG PNH Registry

If you have been diagnosed with paroxysmal nocturnal hemoglobinuria (PNH), you may be invited by your treating specialist to participate in the IPIG PNH Registry in the coming months.

We would like to explain what the IPIG PNH Registry is and why it exists.

IPIG stands for ‘International PNH Interest Group’, which is a non-profit organisation registered in the United States and the only global professional society that focuses specifically on PNH and its treatment. The 400 members are represented by a board of directors made up of global experts in the treatment of PNH.  The aim of IPIG is to improve the care and treatment of patients with PNH by providing guidance, information and education to healthcare professionals. IPIG will manage the new PNH registry.

What is a registry?

A registry is a medical-scientific study. A registry is also called a ‘cohort study’. It collects disease data from as many people as possible with a specific, often rare disease over as long a period as possible in order to better understand the disease, compare disease progression, recognise influences on the disease and the effects of the disease. No drugs are tested in a registry.

What is the aim of the IPIG PNH Registry?

The aim of this registry is to learn more about PNH and to contribute to the understanding of the characteristics and complications of the disease. The registry will also collect information on the treatments/medications used in PNH patients, the impact on patients’ quality of life and healthcare utilisation (e.g. hospitalisations, emergency room visits, physician visits and outpatient care).

It is planned to include at least 2000 patients from over 20 countries around the world in this registry.

Participation is voluntary. By participating in the registry, you are supporting research into PNH.

What about the other PNH Registry I take part in?

You may have heard of the International PNH Registry or have been participating in it for some time. This registry was founded by the company Alexion in response to guidelines from the health authorities in order to conduct further research into the drug eculizumab. It will be transferred to the new IPIG PNH registry, as it is no longer scientifically sufficient due to the limitation of data of patients receiving eculizumab or ravulizumab. This is because other PNH drugs are now available and others are being developed to further investigate the course of the disease.

Advantages of the new IPIG PNH Registry:

  • The data is collected by IPIG (International PNH Interest Group), an academic community of many medical professionals specialising in PNH worldwide
  • Every patient with PNH can participate, regardless of whether which medication/s are prescribed
  • The data is not collected by one pharmaceutical company as several pharmaceutical companies participate in the funding of the IPIG registry, so that no single company has more influence
  • 12 specialised specialists and patient representatives (members of the PNH Global Alliance) make decisions on the analysis of the data, so that the findings benefit patients worldwide
  • A single study reduces the risk of data fragmentation and is easier for centres to manage than multiple competing studies
  • The progression of PNH and safety under different therapies can be monitored
  • Academic accountability through IPIG leads to greater acceptance of published findings

What does taking part in the IPIG PNH Registry involve?

  • If you wish to participate in the IPIG Registry, you will be asked by your treating specialist to sign a consent form. If you decide to participate in the IPIG Registry, you can change your mind and withdraw your participation at any time.
  • Your doctor will then complete questionnaires every six months with your data and blood values that are relevant to the study. This includes, for example, information about you (such as your age and gender, ethnicity), your disease, your treatments, the results of routine examinations and tests, and any health changes that may occur. If you are being treated with a particular PNH treatment, your study doctor may also record additional safety data in the IPIG Registry, and your data will be made available to the manufacturer of the PNH treatment.
  • You will also be asked to complete questionnaires about your quality of life every six months. You can complete the questionnaires on a tablet at the clinic or on your personal electronic device such as a computer, tablet or smartphone, so you can decide for yourself where and when to complete the questionnaires. These will take approximately 25 minutes to complete.
  • If you decide to participate in an interventional clinical trial while participating in the IPIG Registry, data collection in the IPIG Registry will be paused during your participation in the clinical trial.