Description
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition. Prospective longitudinal data in large populations are limited. The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data. In addition, information on the progression of disease in the PNH population, including those patients who discontinue treatment, will be collected. This registry is intended to increase knowledge about PNH in the medical community and patient population. The IPIG PNH registry will be comprised of the core PNH disease registry (“core PNH registry”), including all patients enrolled in the registry with collection of core variables at enrollment and during follow-up, and several product-specific silo protocols (“silos”) initiated by marketing authorization holders (MAHs) and including patients treated with PNH-specific therapies, in order to collect specific data (e.g., post-authorization safety data) and address specific objectives or requests from regulatory authorities.
