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, Karen Kaiser*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

22 Scopus citations


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 < 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%). Conclusion This 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.

Original languageEnglish (US)
Article numbere0237497
JournalPloS one
Issue number9 September
StatePublished - Sep 2020

ASJC Scopus subject areas

  • General


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