Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey
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Le résumé fourni par la source
INTRODUCTION: Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare condition, characterised by complement-mediated red blood cell breakdown, resulting in haemolytic anaemia. Associated symptoms, such as fatigue, impact on patient quality of life. Little is known about the comparative perceptions of symptom burden and treatment preferences among patients and physicians. METHODS: This study analysed secondary data from the Adelphi Real World PNH Disease Specific Programme™, a cross-sectional survey of physicians and patients with PNH in France, Germany, Italy, Spain, China, and Japan conducted January-November 2022. Haematologists and haematologist-oncologists reported demographic and clinical data for ≤10 consecutive consulting adult patients with PNH. Only patients with both physician-reported and patient-reported data were included in the study. Patients and physicians reported on clinical signs and symptom burden, treatment satisfaction, and preferred route of administration. Bivariate analyses were used to compare physician and patient responses. RESULTS: Forty-two physicians reported data for 225 patients. Median (interquartile range) patient age was 44.0 (35.0-56.0), 56.9% were male and 96.0% received treatment at survey. Most common clinical signs and symptoms were fatigue, anaemia, haemoglobinuria, dyspnoea, and headaches. All common signs/symptoms, except haemoglobinuria, were reported as significantly more frequent and severe by patients than physicians (p < 0.05). Most bothersome signs/symptoms reported were anaemia (physicians: 31.3%, patients: 21.3%) and fatigue (physicians: 29.9%, patients: 45.5%). Physicians were significantly more satisfied with treatment than patients (p < 0.001), regardless of treatment. Although oral administration was preferred by both physicians (60.2%) and patients (74.2%), there was significant difference in overall preference (p < 0.001), with 30.8% of physicians preferring intravenous administration, compared to 16.3% of patients. CONCLUSION: There was significant patient-physician disconnect in perception of PNH symptoms burden, treatment satisfaction, and administration preferences. Improved communication and patient involvement in disease management may contribute to optimised patient outcomes and quality of life.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey
- Date Crossref
- 25/08/2026
- 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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Chinese Academy of Medical Sciences & Peking Union Medical College pays non établi dans la noticeUniversité ou école supérieure
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Peking Union Medical College Hospital Department of Haematology pays non établi dans la noticeÉtablissement de santé
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Novartis (Switzerland) pays non établi dans la noticeEntreprise
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Novartis (United Kingdom) pays non établi dans la noticeEntreprise
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Novartis (United States) pays non établi dans la noticeEntreprise
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Adelphi Group (United Kingdom) pays non établi dans la noticeEntreprise
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St James's University Hospital pays non établi dans la noticeÉtablissement de santé
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Novartis Pharma AG pays non établi dans la noticeInstitution
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Novartis Pharmaceuticals UK pays non établi dans la noticeInstitution
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Novartis Pharmaceuticals Corporation pays non établi dans la noticeInstitution
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Adelphi Real World pays non établi dans la noticeInstitution
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IQVIA Australia and New Zealand pays non établi dans la noticeInstitution
Chinese Academy of Medical Sciences & Peking Union Medical College, Department of Haematology — Peking Union Medical College Hospital et Novartis (Switzerland), avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.