Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN): A Multi-Country Study

简要总结

详细描述

主要结局

次要结局

• Participant demographics (age, sex, race)(Day 1 of treatment/Baseline.)
• Clinical characteristics of renal disease (UPCR)(Prior to and - Day 1 of treatment/Baseline.)
• Clinical characteristics of renal disease (eGFR)(Prior to and - Day 1 of treatment/Baseline.)
• Prior and concomitant C3G and primary IC-MPGN treatment information(Day 1 of treatment/Baseline.)
• ≥50% proteinuria reduction(At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years.)
• <1 g/g UPCR(At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years.)
• <0.5 g/g UPCR among participants aged 18 years or older(At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years.)
• <0.2 g/g UPCR among participants less than 18 years of age(Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years.)
• Resolution of nephrotic syndrome in those with nephrotic syndrome(Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years.)
• Sustained doubling of serum creatinine(Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years.)
• Progression to Chronic Kidney Disease Stage 5 or ESRD(At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years.)
• Receipt of new kidney transplant(At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years.)
• New onset of maintenance dialysis (i.e., for at least 4 weeks)(At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years.)
• Death from kidney failure(At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years.)
• Change in urine protein-to-creatinine ratio (UPCR) from the most recent diagnostic test results(Prior to start of pegcetacoplan treatment, at 1, 3, 6, 12 months, and then every 6 months through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Achieving UPCR of <1 g/g(At 1, 3, 6, 12 months and then every 6 months through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Change in eGFR derived from the most recent diagnostic test results of creatinine and height (if applicable)(Prior to start of pegcetacoplan treatment to eGFR derived at 1, 3, 6, 12 months and then until the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Proportion of participants with annualised eGFR decline ≤5 mL/min/1.73 m2(Yearly during pegcetacoplan treatment up to the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Change in kidney biopsy findings(At occurrence, during pegcetacoplan treatment, through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Summary of pegcetacoplan treatment information(Throughout the whole study duration whenever pegcetacoplan is administered per standard of care, up to 5.5 years.)
• Exposure-adjusted incidence rate of SAEs(Ongoing from the start of pegcetacoplan treatment to study end, or 8 weeks post last dose of pegcetacoplan, whichever occurs earlier.)
• Exposure-adjusted incidence rate of AESIs(Ongoing from the start of pegcetacoplan treatment to study end, or 8 weeks post last dose of pegcetacoplan, whichever occurs earlier.)
• Summary scores of FACIT-Fatigue and WPAI(At the start of pegcetacoplan treatment, as available; at study enrolment; and every 6 months during treatment, through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Summary of TSQM among adult participants(At the start of pegcetacoplan treatment, as available, at study enrolment, and every 6 months during treatment through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised number of C3G and primary IC-MPGN-related inpatient or outpatient hospital visits(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised C3G-related length of hospital stays(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised primary IC-MPGN-related length of hospital stays(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised number of C3G-related length of ICU admissions(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised number of primary IC-MPGN-related ICU admissions(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised C3G-related length of ICU stay(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised primary IC-MPGN-related length of ICU stay(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised number of C3G-related emergency department visits(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)
• Annualised number of primary IC-MPGN-related emergency department visits(Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant.)