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References from Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors. Local targets link to admitted publications; unresolved targets remain external evidence.
Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT
10.3389/fimmu.2019.01157 · 2019 · External reference
How I treat paroxysmal nocturnal hemoglobinuria
10.1182/blood.2019003812 · 2021 · External reference
Paroxysmal nocturnal hemoglobinuria: where are we going
2023 · External reference
Paroxysmal nocturnal hemoglobinuria: where we stand
2023 · External reference
Paroxysmal nocturnal hemoglobinuria
10.1182/blood-2014-02-522128 · 2014 · External reference
Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry
10.3324/haematol.2013.093161 · 2014 · External reference
Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry.
10.1007/s00277-020-04052-z · 2020 · External reference
Managing fatigue in patients with paroxysmal nocturnal hemoglobinuria: a patient-focused perspective
10.2147/jbm.s339660 · 2022 · External reference
Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria
10.1002/ajh.21757 · 2010 · External reference
Erratum: “Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinura” by Hillmen et al., Am J Hematol 553–559, 2010, DOI number 21757.
10.1002/ajh.21861 · 2010 · External reference
The kidneys in paroxysmal nocturnal hemoglobinuria
10.1182/blood.v57.1.83.83 · 1981 · External reference
Under-recognized complications in patients with paroxysmal nocturnal haemoglobinuria: raised pulmonary pressure and reduced right ventricular function
10.1111/j.1365-2141.2012.09166.x · 2012 · External reference
High incidence of progression to chronic renal insufficiency in patients with paroxysmal nocturnal hemoglobinuria (PNH).
10.1182/blood.v110.11.3678.3678 · 2007 · External reference
The complement alternative pathway in paroxysmal nocturnal hemoglobinuria: from a pathogenic mechanism to a therapeutic target
10.1111/imr.13137 · 2023 · External reference
The long-acting anti-C5 ravulizumab results in C3 binding to PNH red cells similar to its parental molecule eculizumab
10.1111/bjh.18662 · 2023 · External reference
Extravascular hemolysis due to C3-loading in patients with PNH treated with eculizumab: defining the clinical syndrome
2017 · External reference
The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey
10.1007/s00277-021-04715-5 · 2022 · External reference
Predictors for improvement in patient-reported outcomes: post-hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH).
10.1182/blood-2021-144554 · 2021 · External reference
Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria
10.1056/nejmoa031688 · 2004 · External reference
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria
10.1056/nejmoa061648 · 2006 · External reference
Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria
10.1007/s00277-022-04903-x · 2022 · External reference
Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria
10.1182/bloodadvances.2022009129 · 2023 · External reference
Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria
10.1056/nejmoa2029073 · 2021 · External reference
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.
10.1016/s2352-3026(22)00210-1 · 2022 · External reference
Unresolved reference
2022 · External reference
Unresolved reference
2023 · External reference
C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab
10.1002/ajh.25960 · 2020 · External reference
Fatigue in cancer patients compared with fatigue in the general United States population
10.1002/cncr.10245 · 2002 · External reference
Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial
10.1007/s12185-013-1404-y · 2013 · External reference
The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective.
10.1080/16078454.2022.2127630 · 2022 · External reference
The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors in France, Germany and the United Kingdom: patient-reported insights on symptoms and quality of life
10.1111/ejh.13816 · 2022 · External reference
Unresolved reference
2020 · External reference
Real-world eculizumab dosing patterns among patients with paroxysmal nocturnal hemoglobinuria in a US population.
10.2147/ceor.s346816 · 2022 · External reference
Effectiveness of eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) with or without aplastic anemia in the International PNH Registry.
2019 · External reference
Real-world experience of pegcetacoplan in paroxysmal nocturnal hemoglobinuria
2024 · External reference
Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH
2024 · External reference