Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey
Rattachement africain : jp. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
Paroxysmal nocturnal hemoglobinuria (PNH) is a lifelong, clonal hematologic disease posing life-threatening risks if untreated. The prognosis for PNH has improved with the advent of C5 inhibitors, which are now the standard of care where available. As treatment options continue to expand, healthcare providers can better address both PNH management and the impact of treatment on patients' daily lives. To investigate the burden of disease and key factors taken into consideration when patients with PNH choose their preferred treatment, we conducted an in-depth patient interview survey. Of survey participants (N = 30), 70.0% were receiving intravenous C5 inhibitors. Notably, 56.7% of all patients reported needing ≥ 1 hospital visit per month, and 46.7% required a day off from work or school for visits (33.3% among those receiving intravenous C5 inhibitors). A frequently reported burden of PNH treatment was financial concern, including the cost of treatment, hospital visits, and the negative impact on income. Additionally, burden related to waiting times and distance from the hospital and the overall time spent on outpatient PNH care were identified with similar results for patients receiving intravenous C5 inhibitors. These results suggest that the time and effort to get to treatment centers and the time required to receive treatment for PNH were critical unmet needs in PNH care. Our study indicates that persistent burdens associated with current PNH care should be taken into account alongside therapeutic effectiveness when making treatment decisions. Further analysis with a larger sample size is required to confirm these findings.
Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.
Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey
- Date Crossref
- 01/07/2025
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Osaka University of Economics pays non établi dans la noticeUniversité ou école supérieure
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Osaka City University pays non établi dans la noticeUniversité ou école supérieure
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The University of Osaka Department of Hematology and Oncology pays non établi dans la noticeUniversité ou école supérieure
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University of Tsukuba Hospital Department of Hematology pays non établi dans la noticeÉtablissement de santé
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Kumamoto University Hospital Department of Transfusion Medicine and Cell Therapy pays non établi dans la noticeÉtablissement de santé
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Keio University Department of Medicine pays non établi dans la noticeUniversité ou école supérieure
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Kanazawa University pays non établi dans la noticeUniversité ou école supérieure
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Crecon Medical Assessment Inc. (Japan) pays non établi dans la noticeEntreprise
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Tokai University Department of Internal Medicine pays non établi dans la noticeUniversité ou école supérieure
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NTT Medical Center Department of Hematology pays non établi dans la noticeÉtablissement de santé
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Teikyo University Mizonokuchi Hospital pays non établi dans la noticeUniversité ou école supérieure
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Fukushima Medical University Department of Hematology pays non établi dans la noticeUniversité ou école supérieure
Osaka University of Economics, Osaka City University et Department of Hematology and Oncology — The University of Osaka, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.