Amylo-AFFECT-QOL, a self-reported questionnaire to assess health-related quality of life and to determine the prognosis in cardiac amyloidosis

Mounira Kharoubi; Mélanie Bézard; Amaury Broussier; Arnault Galat; Romain Gounot; Elsa Poullot; Valérie Molinier-Frenkel; Pascale Fanen; Benoit Funalot; Emmanuel Itti; François Lemonnier; Gagan Deep Sing Chadha; Soulef Guendouz; Sophie Mallet; Amira Zaroui; Vincent Audard; Etienne Audureau; Philippe Le Corvoisier; Luc Hittinger; Violaine Planté Bordeneuve; Jean-Pascal Lefaucheur; Aurélien Amiot; Emilie Bequignon; Sophie Bartier; Vincent Leroy; Emmanuel Teiger; Silvia Oghina; Thibaud Damy

doi:10.3389/fcvm.2023.1124660

Amylo-AFFECT-QOL, a self-reported questionnaire to assess health-related quality of life and to determine the prognosis in cardiac amyloidosis

Front Cardiovasc Med. 2023 Mar 14:10:1124660. doi: 10.3389/fcvm.2023.1124660. eCollection 2023.

Authors

Mounira Kharoubi^{1

2

3

4}, Mélanie Bézard^{1

2

3

4}, Amaury Broussier^{5

6}, Arnault Galat^{1

2

3

4}, Romain Gounot^{2

3

7}, Elsa Poullot^{2

8}, Valérie Molinier-Frenkel^{2

7

9}, Pascale Fanen^{9

10}, Benoit Funalot^{2

10}, Emmanuel Itti^{2

3

9

11}, François Lemonnier^{2

3}, Gagan Deep Sing Chadha^{2

3

4}, Soulef Guendouz^{1

2

3

4}, Sophie Mallet^{1

2

3

4}, Amira Zaroui^{1

2

3

4}, Vincent Audard^{9

12}, Etienne Audureau¹³, Philippe Le Corvoisier¹⁴, Luc Hittinger^{1

2

3

4}, Violaine Planté Bordeneuve^{1

15}, Jean-Pascal Lefaucheur^{1

16}, Aurélien Amiot^{1

17}, Emilie Bequignon^{1

18}, Sophie Bartier^{1

18}, Vincent Leroy^{1

17}, Emmanuel Teiger^{1

2

3

4}, Silvia Oghina^{1

2

3}, Thibaud Damy^{1

2

3

4

14}

Affiliations

¹ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Cardiology, Henri Mondor University Hospital, Créteil, France.
² AP-HP (Assistance Publique-Hôpitaux de Paris), French National Referral Centre for Cardiac Amyloidosis, Cardiogen Network, Henri Mondor University Hospital, Créteil, France.
³ AP-HP (Assistance Publique-Hôpitaux de Paris), GRC Amyloid Research Institute, Henri Mondor University Hospital, Créteil, France.
⁴ AP-HP (Assistance Publique-Hôpitaux de Paris), DHU A-TVB, Henri Mondor University Hospital, Créteil, France.
⁵ Université Paris-Est Créteil (UPEC), Creteil, France.
⁶ AP-HP (Assistance Publique-Hôpitaux de Paris), Hopitaux Henri-Mondor/Emile Roux, Department of Geriatrics, Limeil-Brévannes, France.
⁷ AP-HP (Assistance Publique-Hôpitaux de Paris), Lymphoid Malignancies, Henri Mondor University Hospital, Créteil, France.
⁸ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Pathology, Henri Mondor University Hospital, Créteil, France.
⁹ University Paris Est Créteil, Institut National de la Santé et de la Recherche Médicale (INSERM) U955, Institut Mondor de Recherche Biomédicale (IMRB), Créteil, France.
¹⁰ AP-HP (Assistance Publique-Hôpitaux de Paris), Département de Génétique, Hôpital Universitaire Henri Mondor, Créteil, France.
¹¹ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Nuclear Medicine, Henri Mondor University Hospital, Créteil, France.
¹² AP-HP (Assistance Publique-Hôpitaux de Paris), Nephrology and Renal Transplantation Department, Henri Mondor Hospital University, Centre de Référence Maladie Rare « Syndrome Néphrotique Idiopathique », Fédération Hospitalo-Universitaire « Innovative Therapy for Immune Disorders », Créteil, France.
¹³ AP-HP (Assistance Publique-Hôpitaux de Paris), Public Health Department, Henri Mondor University Hospital, Créteil, France.
¹⁴ INSERM Clinical Investigation Centre 1430, AP-HP (Assistance Publique-Hôpitaux de Paris), Henri Mondor University Hospital, Créteil, France.
¹⁵ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Neurology, Henri Mondor University Hospital, Créteil, France.
¹⁶ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Neurophysiology, Henri Mondor University Hospital, Créteil, France.
¹⁷ AP-HP (Assistance Publique-Hôpitaux de Paris), Hepato Gastro Enterology Department, Henri Mondor University Hospital, Créteil, France.
¹⁸ AP-HP (Assistance Publique-Hôpitaux de Paris), Department of Otolaryngology, Henri Mondor University Hospital, Créteil, France.

Abstract

Background and aims: Self-reported questionnaires are useful for estimating the health-related quality of life (HR-QoL), impact of interventions, and prognosis. To our knowledge, no HR-QoL questionnaire has been developed for cardiac amyloidosis (CA). This study aimed to validate Amylo-AFFECT-QOL questionnaire to assess HR-QoL and its prognostic value in CA.

Methods: A self-reported questionnaire, "Amylo-AFFECT" had been designed and validated for CA symptoms evaluation and screening by physicians. It was adapted here to assess HR-QoL (Amylo-AFFECT-QOL) and its prognostic value in CA. To validate the theoretical model, internal consistency and convergent validity were assessed, particularly correlations between Amylo-AFFECT-QOL and the HR-QoL Minnesota Living Heart Failure (MLHF) questionnaire.

Results: Amylo-AFFECT-QOL was completed by 515 patients, 425 of whom (82.5%) had CA. Wild-type and hereditary transthyretin amyloidosis (ATTRwt and ATTRv) and immunoglobulin light-chain amyloidosis (AL) were diagnosed in 47.8, 14.7, and 18.8% of cases, respectively. The best HR-QoL evaluation was obtained with five dimensions: "Heart failure," "Vascular dysautonomia," "Neuropathy," "Ear, gastrointestinal, and urinary dysautonomia," and "Skin or mucosal involvement." The global Amylo-AFFECT-QOL and MLHF scores showed significant positive correlations (rs = 0.72, p < 0.05). Patients with a final diagnosis of CA had a global Amylo-AFFECT-QOL score significantly higher than the control group composed by patients with other diagnoses (22.2 ± 13.6 vs. 16.2 ± 13.8, respectively, p-value < 0.01). According to the Amylo-AFFECT-QOL global results, ATTRv patients' QoL was more affected than AL patients' QoL or ATTRwt patients' QoL. Patients with a higher HR-QoL score had a greater risk of death or heart transplant after 1 year of follow-up (log-rank < 0.01).

Conclusion: Amylo-AFFECT-QOL demonstrates good psychometric properties and is useful for quantifying HR-QoL and estimating CA prognosis. Its use may help to improve overall management of patients with CA.

Keywords: cardiac amyloidosis; prognosis; quality of life; self-reported questionnaire; transthyretin.

Copyright © 2023 Kharoubi, Bézard, Broussier, Galat, Gounot, Poullot, Molinier-Frenkel, Fanen, Funalot, Itti, Lemonnier, Sing Chadha, Guendouz, Mallet, Zaroui, Audard, Audureau, Le Corvoisier, Hittinger, Planté Bordeneuve, Lefaucheur, Amiot, Bequignon, Bartier, Leroy, Teiger, Oghina and Damy.

Grants and funding

The French National Reference Centre for Cardiac Amyloidosis (Henri Mondor Hospital, Créteil, France) has received an institutional grant from Pfizer.