Global PNH Patient Registry

The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following:Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH.Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials).Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics.Assist the PNH community with the development of recommendations and standards of care.Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.

Inclusion Criteria:- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. PNH is defined as a genetic mutation in the PIG-A gene.Individuals must be willing to provide informed consent. Participants can be:legal adult participants who are able to provide their own consent;children and adults unable to provide their own consent, for whom consent must be provided by a Legally Authorized Representative (LAR) who is a legal adult.Individuals must have at least periodic access to the internet and be able to comply with web-based study procedures and data collectionsExclusion Criteria:Individuals not able to read and understand English.