Health-Related Quality of Life in Patients with Alpha-1 Antitrypsin Deficiency: The French Experience

Abstract

The French registry of patients with alpha-1 antitrypsin deficiency (AATD)-associated emphysema was launched in 2006. Here, we aimed to report on the baseline characteristics of these patients, their health-related quality of life (HRQoL) and factors associated with HRQoL. Another goal was to survey the practices of French physicians regarding augmentation therapy. We included 273 patients with AATD, emphysema, obstructive-pattern [forced expiratory volume in 1 sec/forced volume capacity (FEV₁/FVC) < 0.7], FEV₁ ≤ 80% predicted. Mean (SD) age was 51.8 (11.1) years, 240 (87.9%) of patients were smokers or ex-smokers, mean (SD) FEV₁ was 40.5% (15.7) predicted. Mean (SD) SGRQ score was 49.0 (20.0) and was higher for females than males (52.7 [20.7] vs 46.8 [18.2]; p = 0.01). Dyspnea showed the strongest association with SGRQ score (r = 0.65; p < 0.0001), followed by chronic bronchitis (r = 0.33; p < 0.0001) and wheezing (r = 0.32; p < 0.0001). Number of exacerbations in the year before inclusion was also significantly associated with SGRQ score (r = 0.36; p < 0.0001). The SGRQ score was associated with the 6-min walking distance (r = –0.53, p < 0.0001), FEV₁ (% predicted, r = –0.53, p < 0.0001) and DLCO (% predicted, r = –0.52, p < 0.0001). It was also associated with the GOLD 2006 (r = 0.53; p < 0.0001) and GOLD 2011 (r = 0.63; 
p < 0.0001) classifications and with the BODE index (r = 0.37; p < 0.0001). Age, history of tobacco smoking or current smoking did not show any association with SGRQ total scores. On multivariate analysis, a model including age, chronic bronchitis, dyspnea (MRC scale), diffusing lung capacity and 6-min walking distance explained 57% of the variation in the score. The French registry provides important insights into the clinical characteristics of French patients with AATD-related emphysema.

Keywords:

• antitrypsin deficiency
• COPD
• registry

Funding

This study was supported by a grant from the Laboratoire français du Fractionnement et des Biotechnologies. The funding source had no role in the design, conduct, or analysis of the study or the decision to submit the manuscript for publication.

This study was supported by a grant from the LFB. The funder had no role in the design, conduct, or analysis of the study or the decision to submit the manuscript for publication.

Declaration of Interest Statement

Pr. Cuvelier has received speaker fees from Novartis, Boehringer Ingelheim and consulting fees from LFB and Boehringer Ingelheim. Pr. Mornex has received consulting consulting fees from LFB and CSL ­Berhing, speaker fees from Boehringer Ingelheim, Pfizer, GSK, Actelion, LFB and research grants from LFB and ­Actelion.

Pr. Pison reports personal fees from LFB, non-financial support from GSK, Novartis, Pfizer, Actelion, Astra-Zeneca and Boerhinger-Ingelheim. Pr. Thabut has received speaking fees from Laboratoire Français du Fractionnement et des Biotechnologies (LFB), consulting fees from LFB and Novartis, and research grants from LFB. Dr. MC Pujazon has received consulting fees from LFB, Novartis, Stallergenes, and Chiesi.

The other authors have no interest to disclose.

Investigators

The investigators of the CONEDAT whose patients participated in this study are as follows (medical centers are in italics):

Thabut G, Fournier M, Mal H, Piperaud M, Jebrak G, Dauriat G, Biondi G, Ait Ilalne B, Hôpital Bichat, Paris; Mornex J-F, Hôpital Louis Pradel, Lyon; Pison C, Cherion C, Hôpital de Grenoble, Grenoble; Cuvelier A, Muir J-F, Hôpital de Bois-Guillaume, Rouen; Pujazon M-C, Carles P, Hôpital Larrey, Toulouse; Laffite J, Balduyck M, Grammont M, Wasielewsky E, Hôpital Calmette, Lille; Diot P, Marchand-Adam S, Pichon E, Henriet A-C, Hôpital Bretonneau, Tours; Chabot J-F, Guillaumot A, Beuraud N, Hôpital Brabois, Vandoeuvre les Nancy; Reynaud-Gaubert M, Ramadour M, Nieves A, Hôpital Sainte Marguerite, Marseille; Delaval P, Hôpital Pontchaillou, Rennes; Leroyer C, Hôpital Cavale Blanche, Brest; Quieffin J, Hôpital Jean Monod, Le Havre; Broussier P-M, Hôpital Charles Nicolle, Rouen; Mehdaoui A, Centre Hospitalier Général, Evreux; Morel H, Hôpital Broussais, Saint-Malo; Clément B, Nevers; Chiappa A-M, Hôpital de Quimper, Quimper; Angebault M, Coudray S, Centre Hospitalier Spécialisé en Pneumologie, Chevilly-Larue; Martin F, Lineau C, Hôpital Bretagne-Atlantique, Vannes; Charbonneau J, Hôpital Montbéliard, Montbéliard; Godard P, Hôpital Arnaud De Villeneuve, Montpellier; Kessler R, Hôpital Hautepierre, Strasbourg; Caillaud D, Hôpital Gabriel Montpied, Clermont-Ferrand; Lebas F-X, Goupil F, Latrouite L, Hôpital du Mans, Le Mans; Grignet J-P, Hôpital de Denain, Denain; Brun O, Perpignan; Perche A, Orléans; Gillet Juvin K, Hôpital Européen Georges Pompidou, Paris; Chanez P, Hôpital Nord, Marseille; Crequit J, Hôpital de Beauvais, Beauvais; Raymond S, Hôpital Belle Isle, Metz; Dot J-M, Hôpital d’Instruction des Armées Legouest, Metz-Armées; Steenhouwer F, Hôpital Victor Provo, Roubaix; Vaylet F, Hôpital d’instruction des armées, Clamart; Roa M, Hôpital Fréjus-Saint-Raphaël, Fréjus; Zalcman G, Hôpital Côte de Nacre, Caen; Guillou-Bideau B, Centre DELTA, Quimper; Soyez F, Bagneux; Iglesias E, Hôpital de la Source, Orléans; Bogdan G, Hôpital François Quesnay, Mantes la Jolie; Jounieaux V, Magois E, Hôpital d’Amiens, Amiens; Ouksel H, Hôpital d’Angers, Angers; Freymond N, Hôpital Lyon Sud, Pierre Benite; Eveilleau C, Brest; Terrioux P, Meaux; Bertholet C, Chambéry; Girard F, Hôpital les escartons, Briançon; Valcke J, Hôpital Privé Armand Brillard, Nogent sur Marne; Moutaux G, Tours; Tavernier J-Y, Hôpital de Douai, Douai; Briens E, Hôpital Yves Le Foll, Saint Brieuc; Caron F, Hôpital Poitiers, Poitiers; Claussner Paulignan M, Forbach; Lerolle U, Trélazé; Boudoux L, Hôpital Saint Vincent de Paul, Lille; Pourtout F, Courbevoie.