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Japanese patient preferences between ravulizumab and eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria
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- ISHIYAMA Ken
- Department of Hematology, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University
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- USUKI Kensuke
- Department of Hematology, NTT Medical Center Tokyo
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- IKEZOE Takayuki
- Department of Hematology, Fukushima Medical University
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- GOTOH Akihiko
- Department of Hematology, Tokyo Medical University
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- MYREN Karl-Johan
- Alexion Pharma Nordics AB
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- TOMAZOS Ioannis
- Alexion AstraZeneca Rare Disease
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- SHIMONO Akihiko
- Alexion Pharma G.K.
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- NINOMIYA Haruhiko
- Department of Medical Sciences, Faculty of Medicine, University of Tsukuba
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- SAKURAI Masatoshi
- Division of Hematology, Department of Medicine, Keio University School of Medicine
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- NAKAO Shinji
- Department of Hematology, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University
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- NISHIMURA Jun-ichi
- Department of Hematology and Oncology, Osaka University
Bibliographic Information
- Other Title
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- 日本人発作性夜間ヘモグロビン尿症患者におけるravulizumabおよびeculizumabの選択
- ニホンジン ホッサセイ ヤカン ヘモグロビン ニョウショウ カンジャ ニ オケル ravulizumab オヨビ eculizumab ノ センタク
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Description
<p>Ravulizumab is the first long-acting complement inhibitor approved for paroxysmal nocturnal hemoglobinuria (PNH) treatment. We evaluated patient preference for ravulizumab or eculizumab among Japanese adults with PNH. The ALXN1210-PNH-301 (NCT02946463) and ALXN1210-PNH-302 (NCT03056040) studies included 23 Japanese adults who are enrolled in complement inhibitor treatment-naive and eculizumab (≥6 months) treatment. Patient preference was assessed using the PNH-specific patient preference questionnaire (PNH-PPQ©). Most patients preferred ravulizumab (19/23, 82.6%), none preferred eculizumab, and four (17.4%) reported no preference (χ2 test, p<0.005). The preference for ravulizumab was driven by its lower infusion frequency (every 8 weeks) compared with eculizumab (every 2 weeks). The included Japanese patients with PNH preferred ravulizumab because of its reduced infusion frequency, which increases activity planning ability, treatment convenience, and overall quality of life, as compared with eculizumab. These data provide useful insight into patient perspectives and may aid decision-making for PNH treatment.</p>
Journal
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- Rinsho Ketsueki
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Rinsho Ketsueki 64 (1), 9-17, 2023
The Japanese Society of Hematology