Ilene Ceil Weitz, MD
de Latour RP et al. Oral monotherapy with iptacopan, a proximal complement inhibitor of factor b, has superior efficacy to intravenous terminal complement inhibition with standard of care eculizumab or ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: Results from the randomized, active-comparator-controlled, open-label, multicenter, phase III Apply-PNH study. ASH 2022;Abstract LBA2.
de Latour RP et al. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. Lancet Haematol 2022;9(9):e648-59. Abstract
Hill A et al. Eculizumab in paroxysmal nocturnal hemoglobinuria (PNH): A report of all 153 patients treated in the UK. ASH 2012;Abstract 3472.
Hillmen P et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2021;18;384(11):1028-37. Abstract
Hillmen P et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006;355(12):1233-43. Abstract
Kulasekararaj AG and Lazana I. Paroxysmal nocturnal hemoglobinuria: Where are we going. Am J Hematol 2023;98 Suppl 4:S33-43. Abstract
Risitano AM et al. Factor b inhibition with oral iptacopan monotherapy demonstrates sustained long-term efficacy and safety in anti-C5-treated patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and persistent anemia: Final 48-week results from the multicenter, phase III APPLY-PNH trial. ASH 2023;Abstract 571.
Risitano AM et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: Time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol 2019;10:1157. Abstract
Professor Alexander Röth, MD
Badri P et al. Pharmacokinetic and pharmacodynamic properties of cemdisiran, an RNAi therapeutic targeting complement component 5, in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria. Clin Pharmacokinet 2021;60(3):365-78. Abstract
Buatois S et al. Pharmacokinetic characterization and exposure-response relationship of crovalimab in the COMPOSER and COMMODORE 3 trials of patients with paroxysmal nocturnal hemoglobinuria (PNH). ASH 2022;Abstract 1247.
Gavriilaki E et al. Advancing therapeutic complement inhibition in hematologic diseases: PNH and beyond. Blood 2022;139(25):3571-82. Abstract
Lee JW et al. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): A double-blind, randomised, phase 3 trial. Lancet Haematol 2023;10(12):e955-65. Abstract
Liu H et al. Efficacy and safety of the C5 inhibitor crovalimab in complement inhibitor-naive patients with PNH (COMMODORE 3): A multicenter, phase 3, single-arm study. Am J Hematol 2023;98(9):1407-14. Abstract
Liu H et al. Six-month crovalimab extension in the phase 3 Commodore 3 study: Updated efficacy and safety results in complement inhibitor-naive patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH). EHA 2023;Abstract P785.
Röth A et al. The phase III, randomized Commodore 2 trial: Results from a multicenter study of crovalimab vs eculizumab in paroxysmal nocturnal hemoglobinuria (PNH) patients naive to complement inhibitors. EHA 2023;Abstract S181.
Röth A et al. The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. Blood 2020;135(12):912-20. Abstract
Röth A and Dührsen U. Treatment of paroxysmal nocturnal hemoglobinuria in the era of eculizumab. Eur J Haematol 2011;87(6):473-9. Abstract
Scheinberg P et al. Phase III randomized, multicenter, open-label Commodore 1 trial: Comparison of crovalimab vs eculizumab in complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria (PNH). EHA 2023;Abstract S183.
Sostelly A et al. Exposure-response relationship of the SMART-Ig anti-hC5 antibody crovalimab (SKY59): Results from the umbrella phase 1/2 composer trial in healthy volunteers and PNH patients. ASH 2019;Abstract 3745.
Tang G-Q et al. Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation. Front Immunol 2023;14:1213920. Abstract
Versmold K et al. Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting. Eur J Haematol 2023;111(1):84-95. Abstract
Carlos M de Castro III, MD
de Castro CM et al. Efficacy and safety is maintained in adult patients with paroxysmal nocturnal hemoglobinuria receiving pegcetacoplan for up to 3 years. ASH 2023;Abstract 574.
Hillmen P et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006;21;355(12):1233-43. Abstract
Kulasekararaj A et al. Danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: Phase 3 long-term data. ASH 2023;Abstract 576.
Kulasekararaj AG et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol 2022;109(3):205-14. Abstract
Risitano AM et al. Factor b inhibition with oral iptacopan monotherapy demonstrates sustained long-term efficacy and safety in anti-C5-treated patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and persistent anemia: Final 48-week results from the multicenter, phase III APPLY-PNH trial. ASH 2023;Abstract 571.
Risitano AM et al. Danicopan: An oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica 2021;106(12):3188-97. Abstract
Versmold K et al. Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting. Eur J Haematol 2023;111(1):84-95. Abstract