Prognosis of Right Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

Abdallah Fayssoil (1, 2, 3) , Nicolas Mansencal (4, 3) , Lee S. Nguyen (5, 6, 7) , Olivier Nardi (2, 8) , Rabah Yaou (9, 10, 11, 12) , France Leturcq (9, 10, 11, 12) , Helge Amthor (8, 2) , Karim Wahbi (12, 13) , Henri Marc Becane (8, 2) , Frédéric Lofaso (8, 2) , Hélène Prigent (8, 2) , Guillaume Bassez (9, 10) , Anthony Behin (9, 10) , Tanya Stojkovic (9, 10) , Bertrand Fontaine (9, 10) , Denis Duboc (12, 13) , Olivier Dubourg (8, 3) , Bernard Clair (2, 8) , Pascal Laforet (8, 2) , Djillali Annane (2, 8) , David Orlikowski (2, 14)

1 END-ICAP - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologies appliquées
2 Hôpital Raymond Poincaré (Garches) [GHU AP-HP Université Paris-Saclay]
3 Hôpital Ambroise Paré [AP-HP]
4 CESP - Centre de recherche en épidémiologie et santé des populations
5 CIC Paris-Est - Centre d'investigation clinique Paris Est [CHU Pitié Salpêtrière]
6 IHU ICAN - Institut de Cardiométabolisme et Nutrition = Institute of Cardiometabolism and Nutrition [CHU Pitié Salpêtrière]
7 CMC Ambroise Paré [Neuilly-sur-Seine]
8 UVSQ - Université de Versailles Saint-Quentin-en-Yvelines
9 Institut de Myologie
10 CHU Pitié-Salpêtrière [AP-HP]
11 Centre de recherche en Myologie – U974 SU-INSERM
12 Hôpital Cochin [AP-HP]
13 Université Paris Descartes, Sorbonne Paris Cité
14 CIC 1429 - Centre d’Investigation Clinique 1429 [Garches]

Background Chronic respiratory failure and heart involvement may occur in Duchenne muscular dystrophy. We aimed to assess the prognostic value of the right ventricular (RV) systolic dysfunction in patients with Duchenne muscular dystrophy. Methods and Results We studied 90 genetically proven patients with Duchenne muscular dystrophy from 2010 to 2019, to obtain respiratory function and Doppler echocardiographic RV systolic function. Prognostic value was assessed in terms of death and cardiac events. The median age was 27.5 years, and median forced vital capacity was at 10% of the predicted value: 83 patients (92%) were on home mechanical ventilation. An RV systolic dysfunction was found in 46 patients (51%). In patients without RV dysfunction at inclusion, a left ventricular systolic dysfunction at inclusion was associated with a higher risk of developing RV dysfunction during follow-up with an odds ratio of 4.5 (P=0.03). RV systolic dysfunction was significantly associated with cardiac events, mainly acute heart failure (62%) and cardiogenic shock (23%). In a multivariable Cox model, the adjusted hazard ratio was 4.96 (95% CI [1.09-22.6]; P=0.04). In terms of death, we found a significant difference between patients with RV dysfunction versus patients without RV dysfunction in the Kaplan-Meier curves (log-rank P=0.045). Conclusions RV systolic dysfunction is frequently present in patients with Duchenne muscular dystrophy and is associated with increased risk of cardiac events, irrespective of left ventricular dysfunction and mechanical ventilation. Registration URL: https://www.clinicaltrials.org; unique identifier: NCT0250108

Mots clés: Duchenne muscular dystrophy; mortality; prognosis; right ventricle; ventilation

Domaines: Sciences du Vivant [q-bio]

Abdallah Fayssoil, Nicolas Mansencal, Lee S. Nguyen, Olivier Nardi, Rabah Yaou, et al.. Prognosis of Right Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy. Journal of the American Heart Association, 2023, 12 (16), ⟨10.1161/JAHA.122.027231⟩. ⟨hal-04190838⟩