Impact of Tafamidis Use in Colombian Patients Diagnosed With ATTR-CM on Health-Related Quality of Life During One Year of Follow-up: Real-World Study in Colombia
Author(s)
Juan M. Reyes, MSc1, Nilson Lopez, MD2, Juan Molina, MD3, Maria Juliana Rodriguez, MD4, Gustavo Ortega, MD5, Mónica García, Researcher6, Andreina Jose Alamo, BSc7, Gina Gonzalez, MD8, Nelson Murillo9, Nelson Murillo, MD10, Eduardo Echeverry, MD11, Orlando Castaño, MD12, Juan Lopez, MD13.
1Pfizer SAS, Bogotá, Colombia, 2Hospital Pablo Tobon Uribe, Medellin, Colombia, 3Pfizer, Bogota, Colombia, 4Fundacion Cardioinfantil, Bogota, Colombia, 5SURA, Barranquilla, Colombia, 6Pfizer, Pfizer, Colombia, 7PFIZER COLOMBIA, Bogota, Colombia, 8Fundación Santa fe de Bogotá, Bogota, Colombia, 9Colombia, 10Clinica de Occidente, Cali, Colombia, 11Clinica Imbanaco, Cali, Colombia, 12Colsanitas, Bogota, Colombia, 13Fundación Valle del Lili-Centro de investigaciones clínicas, Cali, Colombia.
1Pfizer SAS, Bogotá, Colombia, 2Hospital Pablo Tobon Uribe, Medellin, Colombia, 3Pfizer, Bogota, Colombia, 4Fundacion Cardioinfantil, Bogota, Colombia, 5SURA, Barranquilla, Colombia, 6Pfizer, Pfizer, Colombia, 7PFIZER COLOMBIA, Bogota, Colombia, 8Fundación Santa fe de Bogotá, Bogota, Colombia, 9Colombia, 10Clinica de Occidente, Cali, Colombia, 11Clinica Imbanaco, Cali, Colombia, 12Colsanitas, Bogota, Colombia, 13Fundación Valle del Lili-Centro de investigaciones clínicas, Cali, Colombia.
OBJECTIVES: To describe the impact of Tafamidis in health-related quality of life in Colombian ATTR-CM patients during one year of follow-up through a comprehensive assessment protocol
METHODS: This descriptive, longitudinal prospective, non-interventional study enrolled ATTR-CM patients prescribed tafamidis before March 30th, 2024. Follow-up continued from index date until death, study withdrawal, treatment termination, loss to follow-up, or 12-month completion, whichever occurred first. HRQoL was assessed quarterly using Kansas City Cardiomyopathy Questionnaire (KCCQ) across five participating hospitals. Patient outcomes were systematically recorded and analyzed throughout the study period.
RESULTS: Twenty-nine patients were enrolled. Time from recruitment to first tafamidis dose was: <6 months (20.7%), 6-12 months (51.7%), >12 months (24.1%), no data (3.4%). Patients were predominantly male (85.7%), mean age 74.8 years (SD 6.9). Time from symptoms to diagnosis was 0-3 months in 39.3% of subjects, and >12 months in 32.1% of cases. Wild-type ATTR was the most common form (55.2%). Left ventricular ejection fraction was <60% in 82.8% of patients.In patients treated >12 months, highest scores were in symptom burden (89.3) and total symptom score (82.5). For 7-12 months treatment, they were symptom burden (88.3), self-efficacy (83.3), physical limitations (81.9), and social limitations (74.9). For these groups of patients, there were noticeable improvements across all KCCQ domains compared to the ATTR-ACT trial baseline data. For ≤6 months of treatment, highest scores were in symptom burden (75.0) and total symptom score (70.8), comparing these patients with the ATTR-ACT study, this group showed improvements only in QoL and symptom burden.
CONCLUSIONS: In this observational Colombian cohort, longer tafamidis treatment duration was associated with better patient-reported outcomes across KCCQ domains. Our real-world data suggest potential benefits of early tafamidis initiation in ATTR-CM patients, though further research with larger cohorts and longer follow-up is needed to confirm these preliminary findings.
METHODS: This descriptive, longitudinal prospective, non-interventional study enrolled ATTR-CM patients prescribed tafamidis before March 30th, 2024. Follow-up continued from index date until death, study withdrawal, treatment termination, loss to follow-up, or 12-month completion, whichever occurred first. HRQoL was assessed quarterly using Kansas City Cardiomyopathy Questionnaire (KCCQ) across five participating hospitals. Patient outcomes were systematically recorded and analyzed throughout the study period.
RESULTS: Twenty-nine patients were enrolled. Time from recruitment to first tafamidis dose was: <6 months (20.7%), 6-12 months (51.7%), >12 months (24.1%), no data (3.4%). Patients were predominantly male (85.7%), mean age 74.8 years (SD 6.9). Time from symptoms to diagnosis was 0-3 months in 39.3% of subjects, and >12 months in 32.1% of cases. Wild-type ATTR was the most common form (55.2%). Left ventricular ejection fraction was <60% in 82.8% of patients.In patients treated >12 months, highest scores were in symptom burden (89.3) and total symptom score (82.5). For 7-12 months treatment, they were symptom burden (88.3), self-efficacy (83.3), physical limitations (81.9), and social limitations (74.9). For these groups of patients, there were noticeable improvements across all KCCQ domains compared to the ATTR-ACT trial baseline data. For ≤6 months of treatment, highest scores were in symptom burden (75.0) and total symptom score (70.8), comparing these patients with the ATTR-ACT study, this group showed improvements only in QoL and symptom burden.
CONCLUSIONS: In this observational Colombian cohort, longer tafamidis treatment duration was associated with better patient-reported outcomes across KCCQ domains. Our real-world data suggest potential benefits of early tafamidis initiation in ATTR-CM patients, though further research with larger cohorts and longer follow-up is needed to confirm these preliminary findings.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO142
Topic
Clinical Outcomes, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Clinician Reported Outcomes
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Rare & Orphan Diseases