Information for Patients
The PNH Registry is an international research database that collects anonymised data on patients with PNH worldwide. It is non-interventional which means that participation will not affect patients’ normal medical care. It is intended to gather information about the natural history and management of patients with PNH and to obtain safety data on those treated with eculizumab.
All patients with a diagnosis of PNH or a detected PNH clone are eligible to participate in the registry providing written informed consent is given. Therefore the registry enrols all patients with PNH regardless of whether specific therapies are required are not. It is anticipated that patients will be followed for a minimum of 5 years and participation is completely voluntary.
After written consent has been provided, baseline forms are completed to document demographic details, medical history, and PNH relevant medications. Patients are asked to complete a health assessment questionnaire at this point and then every 6 months thereafter. Clinicians will also complete further forms every 6 months to document the development of disease complications and hospital stays. Information will also be collected about any blood transfusions patients have received, blood test results, new or changed medications and pregnancy. Patients treated with eculizumab will also have information about protection against meningococcal septicaemia, any reactions to the drug and the eculizumab dosage collected.
The Registry forms and questionnaires are completed during normal outpatient visits (or sometimes over the telephone) and all data is collected confidentially, as each patient enrolled is provided with a unique identification number. The data are therefore anonymised.
Outcomes of the Registry
The information collected is improving our understanding of the disease and this should help to improve treatments in the future. The Registry will also be used to provide efficacy and safety data about patients who are treated with eculizumab and other therapies for PNH.
Current status of the Registry (as of October 2013)
The Registry is active and entering patients in over 380 institutions throughout 28 countries worldwide. At present over 2,500 patients with PNH have been included in the Registry providing a unique resource for understanding PNH. The United Kingdom is the second largest recruiting country having entered over 350 patients to date.