Definition:
What is PNH?
- PNH is a rare and potentially life-threatening acquired hematologic disorder, that arises as a consequence of a somatic mutation in a gene (PIGA); in this context, uncontrolled activation of the terminal complement pathway leads to intravascular hemolysis, hemolytic anemia, and a prothrombotic state. Complement-mediated intravascular hemolysis is the major contributor to morbidities and premature mortality associated with PNH.
What is the PNH Registry?
- The International PNH Registry is a prospective, non-interventional study that compiles important data on the natural history and management of patients diagnosed or with signs of PNH.
Following a post marketing commitment requested by the EMA (European Medicine Agency) the PNH Registry has been collaboratively designed and developed with an Executive Committee comprised of a team of international experts in PNH.
The aim of the PNH Registry is to collect data to characterise the progression of PNH as well as associated clinical outcomes, mortality and morbidity. Results from the PNH Registry will provide a better understanding of PNH and its real-world outcomes.
Patient inclusion: All your patients with a detected PNH clone at least 1% may be enrolled in the PNH Registry. Patient eligibility
Data collection: Clinical and patient reported outcomes will be collected by physicians and patients throughout the PNH Registry’s duration. Data collection