the international pnh interest group pnh registry

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Summary: 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.

the international pnh interest group (ipig) pnh registry is an international, observational database collecting real-world health information about pnh patients over time. the registry is owned and managed by ipig, a not-for-profit network of international pnh experts dedicated to improving care and treatment for pnh patients. several pharmaceutical partners are collaborating with ipig to fund the registry, which will provide data to support their post-approval regulatory commitments. the ipig pnh registry aims to collect data about all pnh patients including clinical outcomes, patient reported outcomes and health resource usage, as well as long term safety data for patients treated with anti-complement therapies. the ipig pnh registry is comprised of a 'core' pnh disease registry collecting a defined set of core variables on all patients with pnh. in addition, there are several product-specific 'silos' initiated by marketing authorization holders including patients treated with pnh-specific therapies. the silos collect both the core dataset and additional variables to address specific objectives or requests from regulatory authorities (e.g. post-authorization safety data). data is collected at enrollment and at 6 monthly intervals during follow-up. this clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (that is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(a) of the public health service act and 42 cfr 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the public health service act or 42 cfr 11.24 and 11.44).

Eligibility criteria:
inclusion criteria: * patients with pnh confirmed by flow cytometry. * patient and/or parent/legally authorized representative provide written informed consent/assent to participate in the registry in a manner approved by the institutional review board/independent ethics committee and local regulations. exclusion criteria: * participating in an interventional pnh clinical trial. note: a patient included in the registry, who enrolls in an interventional pnh clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial/extension study. data collection in the registry will continue after patient involvement in the clinical trial/extension study has ended or trial protocol mandated data collection ceases.

Keywords: pnh registry patient registry paroxysmal nocturnal hemoglobinuria anemia, hemolytic anemia hematologic diseases myelodysplastic syndromes bone marrow diseases hemoglobinuria hemoglobinuria, paroxysmal