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

Autor: Peipert JD; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Kulasekararaj AG; Department of Haematological Medicine, King's College Hospital, NIHR/Wellcome King's Clinical Research Facility, and King's College London, London, United Kingdom., Gaya A; Department of Hematology, Hospital Clinic de Barcelona, Spain., Langemeijer SMC; Department of Hematology, Radboud University Medical Center, Nijmegen, Netherlands., Yount S; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Gonzalez-Fernandez FA; Department of Hematology, Hospital Universitario Clinico San Carlos, Madrid, Spain., Ojeda Gutierrez E; Department of Hematology, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain., Martens C; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Sparling A; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Webster KA; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Cella D; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America., Tomazos I; Alexion Pharmaceuticals, Inc., Boston, MA, United States of America., Ogawa M; Alexion Pharmaceuticals, Inc., Boston, MA, United States of America., Piatek CI; Department of Medicine, Jane Anne Nohl Division of Hematology, Keck School of Medicine of University of Southern California, Los Angeles, CA, United States of America., Wells R; Department of Medicine, Sunnybrook Health Sciences Centre, Toronto, ON, Canada., Sicre de Fontbrune F; Centre de Référence Aplasie Médullaire, Service d'Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France., Röth A; Department of Hematology, West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany., Mitchell L; Department of Hematology, University Hospital Monklands, North Lanarkshire, Scotland., Hill A; Department of Haematology, St James' Institute of Oncology, Leeds, United Kingdom., Kaiser K; Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America.
Jazyk: angličtina
Zdroj: PloS one [PLoS One] 2020 Sep 04; Vol. 15 (9), pp. e0237497. Date of Electronic Publication: 2020 Sep 04 (Print Publication: 2020).
DOI: 10.1371/journal.pone.0237497
Abstrakt: 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.
Competing Interests: The authors of this paper have read the journal’s policy and have the following competing interests: Austin Kulasekararaj, Anna Gaya, F. Ataulfo Gonzalez-Fernandez, Emilio Ojeda Gutierrez, and Richard Wells have received honoraria and consulting fees from Alexion Pharmaceuticals, Inc. At the time of the study, Anita Hill had received honoraria and consulting fees from Alexion Pharmaceuticals, Inc. Currently she is an employee of Alexion Pharmaceuticals, Inc. (from 2020). David Cella is a consultant to, and has received grant support from, Alexion Pharmaceuticals, Inc. Caroline Piatek has received honoraria from Alexion Pharmaceuticals, Inc. Ioannis Tomazos and Masayo Ogawa are employees and stockholders of Alexion Pharmaceuticals, Inc. Flore Sicre de Fontbrune has received honoraria and research support (to St. Louis Hospital) from Alexion Pharmaceuticals, Inc. Alexander Röth has received consulting fees and honoraria from Alexion Pharmaceuticals, Inc., Apellis Pharmaceuticals, Roche, and Novartis and research funding from Alexion Pharmaceuticals. Inc. and Roche. Lindsey Mitchell has received honoraria from Alexion Pharmaceuticals, Inc. Eculizumab (Soliris®) and ravulizumab (Ultomiris®) are developed, manufactured, and marketed by Alexion Pharmaceuticals, Inc. This does not alter our adherence to PLOS ONE policies on sharing data and materials.
Databáze: MEDLINE
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