Healthcare Resource Utilization for Patients With Transthyretin Amyloid Cardiomyopathy: A Real-World Study in Europe, Canada, and Japan

Author(s)

Teresa Kauf, PhD1, Jade Garratt-Wheeldon, BSc2, Alisha Braid, BSc2, Katherine Smethers, BSc2, Mark Oellerich, MD3.
1Alnylam Pharmaceuticals, Cambridge, MA, USA, 2Adelphi Real World, Bollington, United Kingdom, 3Alnylam Switzerland GMBH, Zug, Switzerland.
OBJECTIVES: Describe healthcare resource utilization (HCRU) across New York Heart Association (NYHA) classes for transthyretin amyloidosis cardiomyopathy (ATTR-CM) patients in a real-world setting.
METHODS: Secondary analyses using data from the Adelphi ATTR Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients in Europe (France, Germany, Italy, Portugal, Spain and the UK), Canada and Japan, September 2024-February 2025. Physicians reported patient demographics, NYHA class, consultation history, and hospitalizations. Patients were grouped by NYHA class at survey as I, II, III, or IV. Analyses were descriptive.
RESULTS: Overall, 166 physicians provided data for 865 patients (n=645 Europe, n=120 Japan, n=100 Canada). Mean (SD) patient age was 72.7 (12.0) years, 73.5% were male and 12.8% had a mixed cardiomyopathy-polyneuropathy phenotype. At survey, 17.2% (n=149) were NYHA class I, 63.7% (n=551) II, 17.1% (n=148) III and 2.0% (n=17) IV. During the 12 months prior to survey, 31.8% of patients had been hospitalized (I 12.3%, II 31.0%, III 47.8%, IV 81.3%), of which, 66.7% of patients had been emergency room (ER) admissions for at least one hospitalization. Patients had a mean (SD) 8.2 (11.3) consultations with any HCP during the prior 12 months regarding their ATTR-CM (I 6.8 [5.5], II 7.6 [10.4], III 10.9 [16.8], IV 15.1 [13.1]). HCPs included cardiologists (91.6%), general practitioners (36.1%) and internists (13.9%). The mean (SD) number of tests/assessments conducted to monitor ATTR-CM during the prior 12 months was 19.2 (13.9) (I 17.5 [12.9], II 18.7 [13.7], III 22.2 [15.3], IV 22.3 [14.3]).
CONCLUSIONS: HCRU among ATTR-CM patients was high. Almost a third of NYHA II and half of NYHA III patients had been hospitalized, with many requiring ER admissions. Patients were closely monitored with frequent tests and consultations, highlighting complex disease management and potential burden for patients and healthcare systems. Optimizing treatment strategies may help to reduce HCRU and relieve burden.

Conference/Value in Health Info

2025-11, ISPOR Europe 2025, Glasgow, Scotland

Value in Health, Volume 28, Issue S2

Code

EE517

Topic

Clinical Outcomes, Economic Evaluation

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies

Disease

Cardiovascular Disorders (including MI, Stroke, Circulatory), Rare & Orphan Diseases

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