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

doi:10.1371/journal.pone.0237497

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

Medical Social Sciences

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Abstract

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 language	English (US)
Article number	e0237497
Journal	PloS one
Volume	15
Issue number	9 September
DOIs	https://doi.org/10.1371/journal.pone.0237497
State	Published - Sep 2020

Funding

This study was funded by Alexion Pharmaceuticals, Inc. (Boston, MA, USA). Alexion Pharmaceuticals, Inc., provided support in the form of salaries and stocks to authors (Ioannis Tomazos and Masayo Ogawa) and contributors (Ji Yu, Ke Zu, Scott Rottinghaus, and Simu Thomas) of this study. The specific roles of the authors are articulated in the author contributions section, and the contributors are acknowledged in the Acknowledgments. The sponsor (Alexion Pharmaceuticals, Inc.) had no additional role and was not involved in interpretation of the data, reporting of the results, or preparation of the manuscript. The decision to submit the manuscript to PLoS One was made independently by the authors with no influence from the sponsor. Medical writing and editorial support were provided by ApotheCom (Yardley, PA, USA) and funded by Alexion Pharmaceuticals, Inc. (Boston, MA, USA). The authors thank all the patients and investigators who participated in and contributed to this study. Editorial review for scientific accuracy was provided by Shweta Rane, PhD, CMPP, of Alexion Pharmaceuticals, Inc. Ji Yu and Ke Zu of Alexion Pharmaceuticals, Inc., provided additional statistical support. We also thank Scott Rottinghaus and Simu Thomas of Alexion Pharmaceuticals, Inc., for their contributions to the implementation of this study.

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Peipert, J. D., Kulasekararaj, A. G., Gaya, A., Langemeijer, S. M. C., Yount, S., Gonzalez-Fernandez, F. A., Gutierrez, E. O., Martens, C., Sparling, A., Webster, K. A., Cella, D., Tomazos, I., Ogawa, M., Piatek, C. I., Wells, R., de Fontbrune, F. S., Röth, A., Mitchell, L., Hill, A., & Kaiser, K. (2020). Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. PloS one, 15(9 September), Article e0237497. https://doi.org/10.1371/journal.pone.0237497

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

abstract = "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{\textcopyright}). Of 98 patients, 95 completed PNH-PPQ{\textcopyright} 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.",

author = "Peipert, {John Devin} and Kulasekararaj, {Austin G.} and Anna Gaya and Langemeijer, {Saskia M.C.} and Susan Yount and Gonzalez-Fernandez, {F. Ataulfo} and Gutierrez, {Emilio Ojeda} and Christa Martens and Amy Sparling and Webster, {Kimberly A.} and David Cella and Ioannis Tomazos and Masayo Ogawa and Piatek, {Caroline I.} and Richard Wells and {de Fontbrune}, {Flore Sicre} and Alexander R{\"o}th and Lindsay Mitchell and Anita Hill and Karen Kaiser",

note = "Publisher Copyright: {\textcopyright} 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.",

year = "2020",

month = sep,

doi = "10.1371/journal.pone.0237497",

language = "English (US)",

volume = "15",

journal = "PloS one",

issn = "1932-6203",

publisher = "Public Library of Science",

number = "9 September",

}

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 2020, 'Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria', PloS one, vol. 15, no. 9 September, e0237497. https://doi.org/10.1371/journal.pone.0237497

TY - JOUR

T1 - Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria

AU - Peipert, John Devin

AU - Kulasekararaj, Austin G.

AU - Gaya, Anna

AU - Langemeijer, Saskia M.C.

AU - Yount, Susan

AU - Gonzalez-Fernandez, F. Ataulfo

AU - Gutierrez, Emilio Ojeda

AU - Martens, Christa

AU - Sparling, Amy

AU - Webster, Kimberly A.

AU - Cella, David

AU - Tomazos, Ioannis

AU - Ogawa, Masayo

AU - Piatek, Caroline I.

AU - Wells, Richard

AU - de Fontbrune, Flore Sicre

AU - Röth, Alexander

AU - Mitchell, Lindsay

AU - Hill, Anita

AU - Kaiser, Karen

N1 - Publisher Copyright: © 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.

PY - 2020/9

Y1 - 2020/9

N2 - 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.

AB - 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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U2 - 10.1371/journal.pone.0237497

DO - 10.1371/journal.pone.0237497

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VL - 15

JO - PloS one

JF - PloS one

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

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