This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.
Patients ≥18 years of age with a documented PNH diagnosis.
Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.
Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.
Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.
Initiated current treatment with pegcetacoplan in an interventional study.
Adult patients (≥18 years) with PNH, who started routine treatment with pegcetacoplan up to 12 months prior to enrollmentor at enrollment
Cohort
Other
Pegcetacoplan
DrugPegcetacoplan will be prescribed according to the label in patients with PNH.
Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months
Hemoglobin level in g/dL.
Change of LDH values from initiation of pegcetacoplan treatment to 6 months
Lactate Dehydrogenase (LDH) in U/L
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months
Absolute Reticulocyte Count (ARC) in 10\^9/L
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months
Indirect/ total bilirubin in umol/L
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months
Haptoglobin in mg/dL
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Swedish Orphan Biovitrum
Lead sponsor
IQVIA Pty Ltd
Collaborator
