4CPS-122 Analysis of the appropriateness of tafamidis prescription for the treatment of patients with transthyretin cardiac amyloidosis

<h3>Background and Importance</h3> Tafamidis 61 mg has recently been authorised in our country to treat the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), considering the results of the ATTR-ACT trial. <h3>Aim and Objectives</h3> To analyse the appropriateness of initial prescribing of tafamidis in patients with ATTR-CM. <h3>Material and Methods</h3> An observational, descriptive, and prospective study of initial prescriptions of tafamidis for the treatment of patients with ATTR-CM. The study was conducted in a university hospital from September 2023 to September 2024. All patients for whom treatment was requested were included, based on selection criteria from the ATTR-ACT trial. Request evaluations were conducted by a pharmacist, and discrepancies were resolved by multidisciplinary consensus. Apart from baseline characteristics of the patients, other variables for not initiating treatment according to the centre’s protocol were considered: glomerular filtration rate (GFR) &lt;25 mL/min/1.73 m<sup>2</sup>, transaminase values &gt;2 times the upper limit of normal (ULN), and modified body mass index (mBMI) &lt;600 kg/m<sup>2</sup>*g/L. <h3>Results</h3> The study included 36 patients with ATTR-CM (79% male); 2/36 patients had a diagnosis of hereditary ATTR-CM, while the rest had wild-type ATTR-CM. The median age was 82 [range 79–86] years. The baseline characteristics of the patients who started treatment were: mean left ventricular ejection fraction (LVEF) 56% (SD: 8), New York Heart Association (NYHA) heart failure class: II (24/33) and III (3/33), median NT-proBNP biological marker 2,243 pg/mL [IQR 1,809–5,015], mean interventricular septum wall thickness 15 (SD: 3) mm, 6-minute walk test 273 (SD: 122) m, 29/33 had nuclear imaging grade 3 and 4/33 grade 2. Other recorded variables included: mean GFR 51 (SD: 18) mL/min/1.73 m<sup>2</sup> and mean mBMI 107 (SD: 17) kg/m<sup>2</sup>*g/L. Treatment with tafamidis was initiated in 33/36 patients, while 3/36 patients did not start treatment due to not meeting the established criteria: one patient had left ventricular ejection fraction (LVEF) &lt;50% and two had transaminases &gt;2 ULN. <h3>Conclusion and Relevance</h3> The majority of tafamidis 61 mg prescriptions in patients with ATTR-CM met the established criteria. Discrepancies during pharmaceutical validation were resolved by consensus among the multidisciplinary team members at the centre. A high percentage of patients (94%) treated with tafamidis were diagnosed of wild-type of ATTR-CM. <h3>References and/or Acknowledgements</h3> <h3>Conflict of Interest</h3> No conflict of interest

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Published
2025-03-01
DOI
https://doi.org/10.1136/ejhpharm-2025-eahp.190
Primary Topic
Amyloidosis: Diagnosis, Treatment, Outcomes
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article
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article

4CPS-122 Analysis of the appropriateness of tafamidis prescription for the treatment of patients with transthyretin cardiac amyloidosis

M Gallego Delgado, P. Fernandez, Alwyn Gomez, Eduardo Villacorta et al.
Amyloidosis: Diagnosis, Treatment, Outcomes
article

4CPS-122 Analysis of the appropriateness of tafamidis prescription for the treatment of patients with transthyretin cardiac amyloidosis

M Gallego Delgado, P. Fernandez, Alwyn Gomez, Eduardo Villacorta, R Eiros Bachiller, Gisselle Gallego, MJ Otero, A López-González, Trinidad Martin Cillero
article en

Abstract

<h3>Background and Importance</h3> Tafamidis 61 mg has recently been authorised in our country to treat the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), considering the results of the ATTR-ACT trial. <h3>Aim and Objectives</h3> To analyse the appropriateness of initial prescribing of tafamidis in patients with ATTR-CM. <h3>Material and Methods</h3> An observational, descriptive, and prospective study of initial prescriptions of tafamidis for the treatment of patients with ATTR-CM. The study was conducted in a university hospital from September 2023 to September 2024. All patients for whom treatment was requested were included, based on selection criteria from the ATTR-ACT trial. Request evaluations were conducted by a pharmacist, and discrepancies were resolved by multidisciplinary consensus. Apart from baseline characteristics of the patients, other variables for not initiating treatment according to the centre’s protocol were considered: glomerular filtration rate (GFR) &lt;25 mL/min/1.73 m<sup>2</sup>, transaminase values &gt;2 times the upper limit of normal (ULN), and modified body mass index (mBMI) &lt;600 kg/m<sup>2</sup>*g/L. <h3>Results</h3> The study included 36 patients with ATTR-CM (79% male); 2/36 patients had a diagnosis of hereditary ATTR-CM, while the rest had wild-type ATTR-CM. The median age was 82 [range 79–86] years. The baseline characteristics of the patients who started treatment were: mean left ventricular ejection fraction (LVEF) 56% (SD: 8), New York Heart Association (NYHA) heart failure class: II (24/33) and III (3/33), median NT-proBNP biological marker 2,243 pg/mL [IQR 1,809–5,015], mean interventricular septum wall thickness 15 (SD: 3) mm, 6-minute walk test 273 (SD: 122) m, 29/33 had nuclear imaging grade 3 and 4/33 grade 2. Other recorded variables included: mean GFR 51 (SD: 18) mL/min/1.73 m<sup>2</sup> and mean mBMI 107 (SD: 17) kg/m<sup>2</sup>*g/L. Treatment with tafamidis was initiated in 33/36 patients, while 3/36 patients did not start treatment due to not meeting the established criteria: one patient had left ventricular ejection fraction (LVEF) &lt;50% and two had transaminases &gt;2 ULN. <h3>Conclusion and Relevance</h3> The majority of tafamidis 61 mg prescriptions in patients with ATTR-CM met the established criteria. Discrepancies during pharmaceutical validation were resolved by consensus among the multidisciplinary team members at the centre. A high percentage of patients (94%) treated with tafamidis were diagnosed of wild-type of ATTR-CM. <h3>References and/or Acknowledgements</h3> <h3>Conflict of Interest</h3> No conflict of interest

Complejo Hospitalario de Salamanca (ES)
Good health and well-being
Openalex Percentile: Top 96%
Amyloidosis: Diagnosis, Treatment, Outcomes
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