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

October 4, 2020

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Publicated to: Plos One. 15 (9): e0237497-e0237497 - 2020-09-04 15(9), DOI: 10.1371/journal.pone.0237497

Authors:

Peipert JD; Kulasekararaj AG; Gaya A; Langemeijer SMC; Yount S; Gonzalez-Fernandez FA; Gutierrez EO; Martens C; Sparling A; Webster KA; Cella D; Tomazos I; Ogawa M; Piatek CI; Wells R; de Fontbrune FS; Röth A; Mitchell L; Hill A; Kaiser K

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Affiliations

Alexion Pharmaceut Inc, Boston, MA USA - Author

Alexion Pharmaceuticals, Inc. - Author

Hop St Louis, AP HP, Serv Hematol Greffe, Ctr Reference Aplasie Medullaire, Paris, France - Author

Hopital Saint-Louis - Author

Hosp Clin Barcelona, Dept Hematol, Barcelona, Spain - Author

Hosp Univ Clin San Carlos, Dept Hematol, Madrid, Spain - Author

Hosp Univ Puerta de Hierro Majadahonda, Dept Hematol, Madrid, Spain - Author

Hospital Clinic Barcelona - Author

Hospital Clinico San Carlos de Madrid - Author

Hospital Universitario Puerta de Hierro - Author

Keck School of Medicine of USC - Author

King's College Hospital - Author

Kings Coll Hosp London, NIHR Wellcome Kings Clin Res Facil, Dept Haematol Med, London, England - Author

Kings Coll London, London, England - Author

Northwestern Univ, Feinberg Sch Med, Dept Med Social Sci, Chicago, IL 60611 USA - Author

Northwestern University Feinberg School of Medicine - Author

Radboud Univ Nijmegen, Dept Hematol, Med Ctr, Nijmegen, Netherlands - Author

Radboud University Nijmegen Medical Centre - Author

St James Inst Oncol, Dept Haematol, Leeds, W Yorkshire, England - Author

St James's Institute of Oncology - Author

Sunnybrook Health Sciences Centre - Author

Sunnybrook Hlth Sci Ctr, Dept Med, Toronto, ON, Canada - Author

Univ Duisburg Essen, Univ Hosp Essen, West German Canc Ctr, Dept Hematol, Essen, Germany - Author

Univ Hosp Monklands, Dept Hematol, North Lanarkshire, Scotland - Author

Univ Southern Calif, Keck Sch Med, Jane Anne Nohl Div Hematol, Dept Med, Los Angeles, CA 90007 USA - Author

Universitats Klinikum Essen und Medizinische Fakultat - Author

University Hospital Monklands - Author

Abstract

© 2020 Peipert et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. 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.

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Keywords

Responsible consumption and production

Quality index

Bibliometric impact. Analysis of the contribution and dissemination channel

The work has been published in the journal PLoS One due to its progression and the good impact it has achieved in recent years, according to the agency Scopus (SJR), it has become a reference in its field. In the year of publication of the work, 2020, it was in position , thus managing to position itself as a Q1 (Primer Cuartil), in the category Multidisciplinary. Notably, the journal is positioned above the 90th percentile.

Independientemente del impacto esperado determinado por el canal de difusión, es importante destacar el impacto real observado de la propia aportación.

Según las diferentes agencias de indexación, el número de citas acumuladas por esta publicación hasta la fecha 2026-04-04:

Open Alex: 35
WoS: 17
Scopus: 2

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Leadership analysis of institutional authors

This work has been carried out with international collaboration, specifically with researchers from: Canada; France; Germany; Gran Bretanya; Netherlands; United Kingdom; United States of America.

the author responsible for correspondence tasks has been Gaya Valls, Anna.

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Impact on the Sustainable Development Goals (SDGs)

Analysis of institutional authors

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