Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.
<h4>Background</h4>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...
| Main Authors: | , , , , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Public Library of Science (PLoS)
2020-01-01
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| Series: | PLoS ONE |
| Online Access: | https://doi.org/10.1371/journal.pone.0237497 |
| _version_ | 1829500854409887744 |
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| author | 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 |
| author_facet | 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 |
| author_sort | John Devin Peipert |
| collection | DOAJ |
| description | <h4>Background</h4>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.<h4>Objective</h4>The aim of this study was to assess patient preference for ravulizumab or eculizumab.<h4>Methods</h4>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.<h4>Results</h4>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%).<h4>Conclusion</h4>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. |
| first_indexed | 2024-12-16T09:04:16Z |
| format | Article |
| id | doaj.art-f886a2dbac2e4004b37122b11f34a4e1 |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-12-16T09:04:16Z |
| publishDate | 2020-01-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj.art-f886a2dbac2e4004b37122b11f34a4e12022-12-21T22:37:06ZengPublic Library of Science (PLoS)PLoS ONE1932-62032020-01-01159e023749710.1371/journal.pone.0237497Patient 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 PeipertAustin G KulasekararajAnna GayaSaskia M C LangemeijerSusan YountF Ataulfo Gonzalez-FernandezEmilio Ojeda GutierrezChrista MartensAmy SparlingKimberly A WebsterDavid CellaIoannis TomazosMasayo OgawaCaroline I PiatekRichard WellsFlore Sicre de FontbruneAlexander RöthLindsay MitchellAnita HillKaren Kaiser<h4>Background</h4>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.<h4>Objective</h4>The aim of this study was to assess patient preference for ravulizumab or eculizumab.<h4>Methods</h4>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.<h4>Results</h4>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%).<h4>Conclusion</h4>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.https://doi.org/10.1371/journal.pone.0237497 |
| spellingShingle | 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 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 |
| 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. |
| title_full | Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. |
| title_fullStr | Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. |
| title_full_unstemmed | Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. |
| title_short | Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. |
| title_sort | patient preferences and quality of life implications of ravulizumab every 8 weeks and eculizumab every 2 weeks for the treatment of paroxysmal nocturnal hemoglobinuria |
| url | https://doi.org/10.1371/journal.pone.0237497 |
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