Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
Aplastic Anemia and MDS International Foundation
Summary
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.
Description
The Global PNH Patient Registry is a prospective, longitudinal, web-based, observational natural history study. Participants with PNH will be followed throughout the course of their lives with either the participant or authorized respondents contributing data at varying intervals throughout the course of the study. Data will be collected at the start of the study (baseline), at least once per year or can be updated by the participant as needed. Data will be collected on demographics, quality of life, medical history, disease phenotypes, disease-related events, personal experience with PNH, gen…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
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…
Location
- Aplastic Anemia and MDS International FoundationBethesda, Maryland