Clinical outcomes for 2788 patients with transthyretin amyloidosis: Tafamidis meglumine early access program in France - 19/10/24

Doi : 10.1016/j.acvd.2024.08.006

Olivier Lairez ^a, Patricia Réant ^b, Jocelyn Inamo ^c, Julien Jeanneteau ^d, Fabrice Bauer ^e, Gilbert Habib ^f, Jean-Christophe Eicher ^g, Benoit Lequeux ^h, Damien Legallois ⁱ, Constant Josse ^j, Aurelie Hippocrate ^k, Mathilde Bartoli ^k, Margaux Dubois ^k, Charlotte Noirot Cosson ^k, Pierre-Alexandre Squara ^k, Stephane Fievez ^k, Aurore Quinault ^k, Jeremie Rudant ^k, Mounira Kharoubi ^l, Thibaud Damy ^l,^{^{1
X (Twitter) handle: @ThibaudDamy.},}⁎
^a Cardiac Imaging Centre, Department of Cardiology, Toulouse University Hospital, 31059 Toulouse, France
^b Cardiology Department, Haut-Levêque Hospital, Bordeaux University Hospital, University of Bordeaux, Inserm 1045, IHU Lyric, CIC1401, 33600 Pessac, France
^c Reception Team 7525, Cardiology Department, Hospital P. Zobda-Quitman, Martinique University Hospital, University of Antilles, 97261 Fort-de-France, Martinique
^d Cardiology, Clinique Saint-Joseph, 49800 Trelazé, France
^e Department of Cardiac Surgery, Rouen University Hospital, Normandy University, UNIROUEN, Inserm U1096, 76000 Rouen, France
^f Cardiology Department, La Timone University Hospital, 13005 Marseille, France
^g Cardiology Department, François-Mittérrand Hospital, Dijon University Hospital, 21000 Dijon, France
^h Cardiology Department, Poitiers University Hospital, 86000 Poitiers, France
ⁱ Normandy University, UNICAEN, Inserm U1086 ANTICIPE, 14000 Caen, France
^j eXYSTAT, 92240 Malakoff, France
^k Pfizer, 75014 Paris, France
^l Cardiology Department and French National Reference Centre for Cardiac Amyloidosis, Henri-Mondor University Hospital, AP–HP, GRC Amyloid Research Institute and IMRB, Inserm, Université Paris Est Créteil, 94010 Créteil, France

^⁎Corresponding author. Cardiology Department, Henri-Mondor University Hospital, 1, rue Gustave-Eiffel, 94010 Créteil, France.Cardiology Department, Henri-Mondor University Hospital1, rue Gustave-EiffelCréteil94010France

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Graphical abstract

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Highlights

•	EAPs enable patients to access novel effective treatments for severe diseases.
•	EAPs operate in the context of unmet medical need.
•	This study reports patient characteristics and outcomes from a unique European EAP.
•	In total, 2788 patients given tafamidis meglumine for ATTR-CM in real-world clinical practice.
•	The population had a median age of 82years, and 88% were still alive at 18 months.
•	NYHA class progression & survival consistent with previous tafamidis efficacy data.

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Abstract

Background

Early access experience in France with tafamidis meglumine, a selective transthyretin stabilizer for transthyretin-related amyloidosis cardiomyopathy (ATTR-CM), following transthyretin-related amyloidosis (ATTR) polyneuropathy approval and positive ATTR-ACT study results.

Aim

To describe the characteristics and clinical outcomes for patients in the French ATTR-CM tafamidis meglumine early access programme (28 Nov 2018 to 01 Jun 2021).

Methods

Patients with confirmed ATTR-CM received tafamidis meglumine 20mg/day or 80mg/day. Demographic and clinical data were collected prospectively until patients discontinued treatment or died, or the programme ended.

Results

Overall, 222 physicians from 126 centres enrolled 2788 patients. The median age was 82years, 81.6% were male and New York Heart Association severity was class I for 12.8%, class II for 60.1% and class III for 27.0%. Overall, 1943 (74.6%) had genetic testing, and the results were available at tafamidis start for 1208 (62.2%) patients: 995 (82.4%) had wild-type ATTR and 213 (17.6%) had hereditary ATTR. Most patients started treatment≤12months after diagnosis (88.3%): 2268 (81.3%) at 20mg/day, with 401 (17.7%) increasing to 80mg/day. Median follow-up duration was 11.8months. New York Heart Association class improved or remained stable for 1299 (77.6%), whereas 376 (22.4%) worsened between inclusion and last follow-up. Among patients initiated at 80mg, 297 (81.1%) improved or remained stable and 69 (18.9%) worsened. New York Heart Association class progression did not vary with age. The 18-month survival rates were 89.8% (95% confidence interval: 87.0–92.0) among patients aged<80years, and 86.5% (95% confidence interval: 83.9–88.7) among those aged≥80years.

Conclusions

Early tafamidis meglumine access was given to 2788 patients with ATTR-CM. New York Heart Association class progression and survival were consistent with previously published data.

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Keywords : France, Heart failure, Real-world data, Tafamidis, Transthyretin cardiac amyloidosis

Plan

☆	X post (Tweet): New publication on demographic characteristics and clinical follow-up of the 2788 transthyretin amyloid cardiomyopathy patients treated with tafamidis during the French Early Access Programme (RTU) from Nov 2018 to Jun 2021 confirms tafamidis RWE effectiveness.