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

• DOI: 10.1371/journal.pone.0237497
• Publication Date: 2020-09-04T17:29:36Z
• Authors: John Devin Peipert; Austin G. Kulasek...; Anna Gaya; Saskia M. C. Lang...; Susan Yount; F. Ataulfo Gonzal...; Emilio Ojeda Guti...; Christa Martens; Amy Sparling; Kimberly A. Webster; David Cella; Ioannis Tomazos; Masayo Ogawa; Caroline I. Piatek; Richard Wells; Flore Sicre de Fo...; Alexander Röth; Lindsay Mitchell; Anita Hill; Karen Kaiser
• Keywords: Adult; Science; Q; Medizin; R; Hemoglobinuria, Paroxysmal; Patient Preference; Middle Aged; Antibodies, Monoclonal, Humanized; 3. Good health; Young Adult; 03 medical and health sciences; Radboudumc 2: Cancer development and immune defence RIHS: Radboud Institute for Health Sciences; Complement Inactivating Agents; 0302 clinical medicine; Quality of Life; Medicine; Humans; Female; Haematology - Radboud University Medical Center; Research Article; Aged

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.The aim of this study was to assess patient preference for ravulizumab or eculizumab.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.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%).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.