Paroxysmal Nocturnal Hemoglobinuria in Pregnancy Treated With Pegcetacoplan: Case Report and Pharmacokinetic Analysis [PDF]
EJHaemThere is an unmet clinical need for effective treatment of paroxysmal nocturnal hemoglobinuria (PNH) in pregnancy for patients with inadequate response to C5 inhibitors.
Benjamin Chin‐Yee +9 more
exaly +7 more sources
Geographic Atrophy Progression in Clinical Practice Before and After Pegcetacoplan Treatment [PDF]
Vision (Switzerland)This retrospective study evaluated changes in ocular characteristics and retinal pigment epithelium (RPE) and photoreceptor ellipsoid zone (EZ) depletion rates before and after intravitreal pegcetacoplan initiation in clinical practice.
Hasenin Al-Khersan +2 more
exaly +5 more sources
Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria
Therapeutic Advances in Hematology, 2022 Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by complement-mediated hemolysis, thrombosis, and various degrees of bone marrow dysfunction.
Raymond Wong
exaly +3 more sources
Efficacy and Safety of Pegcetacoplan in Kidney Transplant Recipients With Recurrent Complement 3 Glomerulopathy or Primary Immune Complex Membranoproliferative Glomerulonephritis [PDF]
Kidney International ReportsIntroduction: Complement 3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) have high risks for disease recurrence and allograft loss in transplant kidneys.
Matthew Pickering +2 more
exaly +9 more sources
Efficacy of Continuous Pegcetacoplan Treatment for Subfoveal Geographic Atrophy in Age-Related Macular Degeneration: 36-Month Results from OAKS, DERBY, and GALE Open-Label Extension [PDF]
Clinical OphthalmologyDilsher S Dhoot,1 Sunir J Garg,2 David M Brown,3 Ivan J Suñer,4 Ehsan Rahimy,5,6 David S Boyer,7 Kineta Naidu,8 Chao Li,8 Caroline R Baumal,8,9 Robyn H Guymer10,11 1California Retinal Consultants/Retina Consultants of America, Santa Barbara, CA, USA ...
Dhoot DS +9 more
doaj +2 more sources
Frontiers in Immunology, 2022 More than half of patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with complement fraction C5 inhibitors experience residual anemia and hemolysis.
Cristina Vercellati +2 more
exaly +3 more sources
Direct Switch From Iptacopan to Pegcetacoplan in a Patient With Paroxysmal Nocturnal Hemoglobinuria [PDF]
eJHaemIntroduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal hematologic disorder treated with complement inhibitors. Currently, patients with suboptimal response to C5 inhibitors may be switched to proximal complement inhibitors, such as ...
Vitalii Latyshev +3 more
doaj +2 more sources
Pegcetacoplan-induced remission in pediatric immune-complex membranoproliferative glomerulonephritis with comorbid autosomal recessive polycystic kidney disease: a case report [PDF]
Frontiers in MedicineBackgroundMembranoproliferative glomerulonephritis (MPGN) is a rare glomerular disorder characterized by immune complex or complement-mediated injury, often leading to nephrotic syndrome, hypertension, and progressive renal dysfunction.
Reem Alrasheed +5 more
doaj +2 more sources
Methodological pitfalls in indirect treatment comparisons: insights from a recent systematic review and analysis for C3 glomerulopathy [PDF]
Journal of Comparative Effectiveness ResearchAim: Indirect treatment comparisons (ITCs), as outlined in NICE and ISPOR guidance, require careful evaluation of cross-trial heterogeneity to ensure valid comparisons, particularly in rare diseases with limited evidence.
Brian Hutton +7 more
doaj +2 more sources
C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria [PDF]
Frontiers in ImmunologyIntroductionParoxysmal nocturnal hemoglobinuria (PNH) is treated with complement inhibitors, yet incomplete responses remain a challenge. Terminal inhibition with eculizumab prevents intravascular hemolysis but often leaves residual extravascular ...
Santiago Rodríguez de Córdoba +8 more
doaj +2 more sources