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

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 and Karen Kaiser

PLOS ONE, 2020, vol. 15, issue 9, 1-12

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

Date: 2020
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Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0237497

DOI: 10.1371/journal.pone.0237497

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