학술논문
Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.
Document Type
article
Author
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
Source
PLoS ONE, Vol 15, Iss 9, p e0237497 (2020)
Subject
Language
English
ISSN
1932-6203
Abstract
BackgroundEculizumab 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.ObjectiveThe aim of this study was to assess patient preference for ravulizumab or eculizumab.MethodsStudy 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.ResultsOverall, 93% of patients preferred ravulizumab whereas 7% of patients either had no preference (6%) or preferred eculizumab (1%) (P < 0.001). For specific aspects of treatment, ravulizumab was preferred (in comparison to no preference or eculizumab) on infusion frequency (98% vs. 0% vs. 2%), ability to plan activities (98% vs. 0% vs. 2%), and overall quality of life (88% vs. 11% vs. 1%), among other aspects. Most participants selected frequency of infusions as the most important factor determining preference (43%), followed by overall quality of life (23%).ConclusionThis study shows that a substantial proportion of patients preferred ravulizumab over eculizumab and provides an important patient perspective on PNH treatment when there is more than one treatment option.