Burden of Transfusion in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): Results From a Real-World Study in European Countries
Author(s)
Talha Munir, PhD1, Maria-Magdalena Balp, MASc, MD2, Anggie Wiyani, MSc3, Jahnavi Yenamandra, M. Pharm.4, Yasmin Taylor, MSc5, Sam Williamson, Bsc Human Biosciences6, Silvia Sanz, MSc7.
1St James Hospital, Leeds, United Kingdom, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 4Novartis Healthcare Private Limited, Hyderabad, India, 5Adelphi Real World, Bollington, United Kingdom, 6Adelphi Real World, Bollington, United Kingdom, 7NOVARTIS FARMACEUTICA, S.A., Barcelona, Spain.
1St James Hospital, Leeds, United Kingdom, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 4Novartis Healthcare Private Limited, Hyderabad, India, 5Adelphi Real World, Bollington, United Kingdom, 6Adelphi Real World, Bollington, United Kingdom, 7NOVARTIS FARMACEUTICA, S.A., Barcelona, Spain.
Presentation Documents
OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with hemolysis, anemia, and thrombosis. PNH is managed by complement-inhibitors (Ci) and supportive treatments including blood transfusions. This study investigated the real-world experience of PNH patients who are transfusion-dependent.
METHODS: Data were collected from Adelphi PNH Disease Specific Programme™ (Dec 2023-May 2024), a cross-sectional survey in France, Germany, Italy, Spain, and the United Kingdom. Transfusion dependency was defined as having ≥1 blood transfusions in ≤12 months prior to survey. Physicians reported patient demographics, clinical parameters, healthcare resource use; patients reported outcomes including FACIT-Fatigue (score 0-52, higher score indicating less fatigue), EQ-5D-VAS (score 0-100), EQ-5D-5L (utility score 0-1; with Germany tariff). Descriptive statistics were reported; missing data were not imputed.
RESULTS: This study included 64 transfusion-dependent patients (median; interquartile-range [IQR] age: 50.5 [38.0-64.0] years, 56% male), with 83% receiving Ci with a median (IQR) duration of 1.0 (0.7-2.4) years. Median (IQR) for most recent hemoglobin was 11.0 (9.9-11.9) g/dL, and lactate dehydrogenase was 254.0 (210.0-350.0) U/L. In 0-6 months, prior to survey, 47% of patients had ≥1 transfusion (mean standard-deviation [SD]: 1.1 [1.9]); in 6-12 months: 91% (2.2 [2.0]). Common reasons for recent transfusion were routine-transfusions (36%), hemolytic crisis (31%), and infections (16%). Within 12 months prior to survey, 28% patients had ≥1 hospitalization, of those 35% were admitted via the emergency room. Mean (SD) length-of-stay for hospitalization was 4.6 (1.8) nights. Patients (n=12, median [IQR] age: 49.5 [39.3-61.8] years) reported mean (SD) FACIT-Fatigue score of 35.8 (6.0). Mean (SD) EQ-5D-VAS and EQ-5D utility score was 63.8 (14.8), and 0.78 (0.17), respectively.
CONCLUSIONS: Majority of transfusion-dependent patients in this study were treated with Ci, yet had suboptimal hemoglobin levels, requiring regular transfusions. This affected healthcare resource use and quality of life, highlighting the need for treatments which can reduce transfusion-dependency.
METHODS: Data were collected from Adelphi PNH Disease Specific Programme™ (Dec 2023-May 2024), a cross-sectional survey in France, Germany, Italy, Spain, and the United Kingdom. Transfusion dependency was defined as having ≥1 blood transfusions in ≤12 months prior to survey. Physicians reported patient demographics, clinical parameters, healthcare resource use; patients reported outcomes including FACIT-Fatigue (score 0-52, higher score indicating less fatigue), EQ-5D-VAS (score 0-100), EQ-5D-5L (utility score 0-1; with Germany tariff). Descriptive statistics were reported; missing data were not imputed.
RESULTS: This study included 64 transfusion-dependent patients (median; interquartile-range [IQR] age: 50.5 [38.0-64.0] years, 56% male), with 83% receiving Ci with a median (IQR) duration of 1.0 (0.7-2.4) years. Median (IQR) for most recent hemoglobin was 11.0 (9.9-11.9) g/dL, and lactate dehydrogenase was 254.0 (210.0-350.0) U/L. In 0-6 months, prior to survey, 47% of patients had ≥1 transfusion (mean standard-deviation [SD]: 1.1 [1.9]); in 6-12 months: 91% (2.2 [2.0]). Common reasons for recent transfusion were routine-transfusions (36%), hemolytic crisis (31%), and infections (16%). Within 12 months prior to survey, 28% patients had ≥1 hospitalization, of those 35% were admitted via the emergency room. Mean (SD) length-of-stay for hospitalization was 4.6 (1.8) nights. Patients (n=12, median [IQR] age: 49.5 [39.3-61.8] years) reported mean (SD) FACIT-Fatigue score of 35.8 (6.0). Mean (SD) EQ-5D-VAS and EQ-5D utility score was 63.8 (14.8), and 0.78 (0.17), respectively.
CONCLUSIONS: Majority of transfusion-dependent patients in this study were treated with Ci, yet had suboptimal hemoglobin levels, requiring regular transfusions. This affected healthcare resource use and quality of life, highlighting the need for treatments which can reduce transfusion-dependency.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR34
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)