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Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria

Author

Listed:
  • John Devin Peipert
  • Austin G Kulasekararaj
  • Anna Gaya
  • Saskia M C Langemeijer
  • Susan Yount
  • F Ataulfo Gonzalez-Fernandez
  • Emilio Ojeda Gutierrez
  • Christa Martens
  • Amy Sparling
  • Kimberly A Webster
  • David Cella
  • Ioannis Tomazos
  • Masayo Ogawa
  • Caroline I Piatek
  • Richard Wells
  • Flore Sicre de Fontbrune
  • Alexander Röth
  • Lindsay Mitchell
  • Anita Hill
  • Karen Kaiser

Abstract

Background: Eculizumab has transformed management of paroxysmal nocturnal hemoglobinuria (PNH) since its approval. However, its biweekly dosing regimen remains a high treatment burden. Ravulizumab administered every 8 weeks demonstrated noninferiority to eculizumab in two phase 3 trials. In regions where two PNH treatment options are available, it is important to consider patient preference. Objective: The aim of this study was to assess patient preference for ravulizumab or eculizumab. Methods: Study 302s (ALXN1210-PNH-302s) enrolled PNH patients who participated in the extension period of phase 3 study ALXN1210-PNH-302. In the parent study, eculizumab-experienced adult PNH patients received ravulizumab or eculizumab during a 26-week primary evaluation period. All patients in the extension period received ravulizumab. In study 302s, patient treatment preference was evaluated using an 11-item PNH-specific Patient Preference Questionnaire (PNH-PPQ©). Of 98 patients, 95 completed PNH-PPQ© per protocol for analysis. Results: Overall, 93% of patients preferred ravulizumab whereas 7% of patients either had no preference (6%) or preferred eculizumab (1%) (P

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  • John Devin Peipert & Austin G Kulasekararaj & Anna Gaya & Saskia M C Langemeijer & Susan Yount & F Ataulfo Gonzalez-Fernandez & Emilio Ojeda Gutierrez & Christa Martens & Amy Sparling & Kimberly A Web, 2020. "Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria," PLOS ONE, Public Library of Science, vol. 15(9), pages 1-12, September.
    • Handle: RePEc:plo:pone00:0237497
    DOI: 10.1371/journal.pone.0237497
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    Cited by:

    1. S. W. Quist & A. J. Postma & K. J. Myrén & L. A. Jong & M. J. Postma, 2023. "Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(9), pages 1455-1472, December.

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