5,205
Views
15
CrossRef citations to date
0
Altmetric
Hematology

Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison

ORCID Icon, , , , , , , , & show all
Pages 1913-1923 | Received 26 Apr 2021, Accepted 14 Aug 2021, Published online: 03 Sep 2021

References

  • Brodsky RA. Stem cell transplantation for paroxysmal nocturnal hemoglobinuria. Haematologica. 2010;95(6):855–856.
  • Jalbert JJ, Chaudhari U, Zhang H, et al. Epidemiology of PNH and real-world treatment patterns following an incident PNH diagnosis in the US. Blood. 2019;134(Supplement_1):3407–3407.
  • Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4):1840–1847.
  • Hill A, Hillmen P, Richards SJ, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005;106(7):2559–2565.
  • Hillmen P, Elebute M, Kelly R, et al. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2010;85(8):553–559.
  • Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123–4128.
  • Hillmen P, Muus P, Roth A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013;162(1):62–73.
  • Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233–1243.
  • Alexion Pharmaceuticals, Inc. Ultomiris [prescribing information]. Boston (MA): Alexion Pharmaceuticals, Inc.; 2018.
  • Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540–549.
  • Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530–539.
  • DeZern AE, Dorr D, Brodsky RA. Predictors of hemoglobin response to eculizumab therapy in paroxysmal nocturnal hemoglobinuria. Eur J Haematol. 2013;90(1):16–24.
  • Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804–2811.
  • McKinley CE, Richards SJ, Munir T, et al. Extravascular hemolysis due to C3-Loading in patients with PNH treated with eculizumab: defining the clinical syndrome. Blood. 2017;130(Supplement 1):3471–3471.
  • Nakayama H, Usuki K, Echizen H, et al. Eculizumab dosing intervals longer than 17 days may be associated with greater risk of breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria. Biol Pharm Bull. 2016;39(2):285–288.
  • Peffault de Latour R, Fremeaux-Bacchi V, Porcher R, et al. Assessing complement blockade in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab. Blood. 2015;125(5):775–783.
  • Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384(11):1028–1037.
  • de Castro C, Grossi F, Weitz IC, et al. C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab. Am J Hematol. 2020;95(11):1334–1243.
  • Phillippo D, Ades A, Dias S, et al. NICE DSU technical support document 18: methods for population-adjusted indirect comparisons in submissions to NICE. Decision Support Unit, ScHARR, University of Sheffield: NICE Decision Support Unit; 2016.
  • Jang JH, Okamoto S, Sakurai M, et al. Comparison of baseline clinical characteristics between Asian Vs. Non-Asian patients with paroxysmal nocturnal hemoglobinuria (PNH) from international PNH registry. Blood. 2017;130(Supplement 1):3472–3472.
  • Sakurai M, Jang JH, Chou WC, et al. Comparative study on baseline clinical characteristics of Asian versus non-Asian patients with paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2019;110(4):411–418.
  • Signorovitch JE, Sikirica V, Erder MH, et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940–947.
  • Barcellini W, Fattizzo B. Clinical applications of hemolytic markers in the differential diagnosis and management of hemolytic anemia. Dis Markers. 2015;2015:635670.
  • Risitano AM, Marotta S, Ricci P, 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.
  • Schrezenmeier H, Muus P, Socie G, et al. Baseline characteristics and disease burden in patients in the international paroxysmal nocturnal hemoglobinuria registry. Haematologica. 2014;99(5):922–929.
  • Lee JW, Jang JH, Kim JS, et al. Clinical signs and symptoms associated with increased risk for thrombosis in patients with paroxysmal nocturnal hemoglobinuria from a Korean registry. Int J Hematol. 2013;97(6):749–757.
  • Jang JH, Kim JS, Yoon SS, et al. Predictive factors of mortality in population of patients with paroxysmal nocturnal hemoglobinuria (PNH): results from a Korean PNH registry. J Korean Med Sci. 2016;31(2):214–221.
  • Yenerel MN, Muus P, Wilson A, et al. Clinical course and disease burden in patients with paroxysmal nocturnal hemoglobinuria by hemolytic status. Blood Cells Mol Dis. 2017;65:29–34.
  • Cella D, Eton DT, Lai J-S, Peterman AH, et al. Combining anchor and distribution-based methods to derive minimal clinically important differences on the Functional Assessment of Cancer Therapy (FACT) anemia and fatigue scales. J Pain Symptom Manage. 2002;24(6):547–561.
  • Cella D, Lai J, Chang CH, et al. Fatigue in cancer patients compared with fatigue in the general United States population. Cancer. 2002;94(2):528–538.
  • Snyder CF, Blackford AL, Sussman J, et al. Identifying changes in scores on the EORTC-QLQ-C30 representing a change in patients’ supportive care needs. Qual Life Res. 2015;24(5):1207–1216.
  • Scott NW, Fayers PM, Aaronson NK, et al. EORTC QLQ-C30 reference values. Brussels (Belgium): EORTC Quality of Life Group; 2008.
  • Cheng D, Ayyagari R, Signorovitch J. The statistical performance of matching-adjusted indirect comparisons. 2019: arXiv:1910.06449. [cited 2019 Oct 1]. https://ui.adsabs.harvard.edu/abs/2019arXiv191006449C.