Clinical Profile of Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Treated With Complement Inhibitors: Results From a Real-World Study
Author(s)
Alexander Röth, MD1, Maria-Magdalena Balp, MASc, MD2, Anggie Wiyani, MSc3, Yasmin Taylor, MSc4, Brianne Kerr, BSc4, Jahnavi Yenamandra, M. Pharm.5, Silvia Sanz, MSc6, Eloise Beggiato, MD7.
1University Hospital Essen, Essen, Germany, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 4Adelphi Real World, Bollington, United Kingdom, 5Novartis Healthcare Private Limited, Hyderabad, India, 6NOVARTIS FARMACEUTICA, S.A., Barcelona, Spain, 7Città della Salute e della Scienza, Turin, Italy.
1University Hospital Essen, Essen, Germany, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 4Adelphi Real World, Bollington, United Kingdom, 5Novartis Healthcare Private Limited, Hyderabad, India, 6NOVARTIS FARMACEUTICA, S.A., Barcelona, Spain, 7Città della Salute e della Scienza, Turin, Italy.
OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder characterized by hemolysis, anemia, and thrombosis. Patients are managed by complement inhibitors (Ci) and/or supportive treatments. This study investigated clinical characteristics and patient-reported outcomes (PROs) of patients not treated with Ci (Ci-naïve).
METHODS: Data were drawn from Adelphi PNH II Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients (December 2023-May 2024) across France, Germany, Italy and Spain. Physicians reported on demographics, clinical parameters, symptoms, and treatments. PRO was collected with FACIT-Fatigue questionnaire (scored 0 - 52; higher score indicating less fatigue, general population mean [standard deviation; SD]: 43.5 [8.3], in Germany). Data collected at diagnosis and survey were analyzed with descriptive statistics without imputing missing data.
RESULTS: Thirty-one physicians provided data on 78 Ci-naive patients. Median (interquartile range [IQR]) age at survey, time since diagnosis, and time on supportive treatments was 46.0 (36.8-60.0), 1.2 (0.5-2.0), and 1.1 (0.7-1.9) years, respectively. Physicians perceived patients (83%) to have mild PNH, reporting 39% subclinical, 38% classical, and 23% PNH with bone marrow failure. Commonly prescribed supportive therapies at survey were dietary supplements (56%), anticoagulants (21%); 35% had ≥1 blood transfusions within 12-month prior survey, while 36% received no treatment. At diagnosis and at survey, median (IQR) hemoglobin (Hb; g/dL) was: 9.0 (8.0-10.0) and 11.0 (10.0-12.0), and lactate dehydrogenase (U/L): 395.0 (254.0-520.0) and 250.0 (187.0-324.0), respectively. At survey, majority patients had suboptimal Hb (<10 g/dL: 15%; ≥10-<12 g/dL: 57%; ≥12 g/dL: 28%) and experienced symptoms (76%); primarily anemia (45%) and fatigue (42%); considered mostly mild. Twenty-six patients reported mean (SD) FACIT-Fatigue score of 36.1 (10.1).
CONCLUSIONS: In this real-world study, Ci-naïve patients received supportive treatments as part of disease management. However, majority had suboptimal Hb and symptoms. These patients may need targeted treatments which could contribute to improved clinical and patient outcomes.
METHODS: Data were drawn from Adelphi PNH II Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients (December 2023-May 2024) across France, Germany, Italy and Spain. Physicians reported on demographics, clinical parameters, symptoms, and treatments. PRO was collected with FACIT-Fatigue questionnaire (scored 0 - 52; higher score indicating less fatigue, general population mean [standard deviation; SD]: 43.5 [8.3], in Germany). Data collected at diagnosis and survey were analyzed with descriptive statistics without imputing missing data.
RESULTS: Thirty-one physicians provided data on 78 Ci-naive patients. Median (interquartile range [IQR]) age at survey, time since diagnosis, and time on supportive treatments was 46.0 (36.8-60.0), 1.2 (0.5-2.0), and 1.1 (0.7-1.9) years, respectively. Physicians perceived patients (83%) to have mild PNH, reporting 39% subclinical, 38% classical, and 23% PNH with bone marrow failure. Commonly prescribed supportive therapies at survey were dietary supplements (56%), anticoagulants (21%); 35% had ≥1 blood transfusions within 12-month prior survey, while 36% received no treatment. At diagnosis and at survey, median (IQR) hemoglobin (Hb; g/dL) was: 9.0 (8.0-10.0) and 11.0 (10.0-12.0), and lactate dehydrogenase (U/L): 395.0 (254.0-520.0) and 250.0 (187.0-324.0), respectively. At survey, majority patients had suboptimal Hb (<10 g/dL: 15%; ≥10-<12 g/dL: 57%; ≥12 g/dL: 28%) and experienced symptoms (76%); primarily anemia (45%) and fatigue (42%); considered mostly mild. Twenty-six patients reported mean (SD) FACIT-Fatigue score of 36.1 (10.1).
CONCLUSIONS: In this real-world study, Ci-naïve patients received supportive treatments as part of disease management. However, majority had suboptimal Hb and symptoms. These patients may need targeted treatments which could contribute to improved clinical and patient outcomes.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO50
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)