Cardiomyopathies in Propionic Aciduria are Reversible After Liver Transplantation - 07/03/14
Reprint requests: Pr P de Lonlay, Centre de Référence des Maladies Métaboliques, Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75743 Paris cedex 15, France.Abstract |
Objective |
To evauluate the relationship between propionic acidemia (PA) and cardiomyopathy.
Study design |
We retrospectively compared clinical and metabolic results of patients with PA with and without cardiomyopathy.
Results |
Of 26 patients with PA who survived the first year of age, a dilated cardiomyopathy developed in 6 (group 1) at a median age of 7 years (range, 5-11 years). They were compared with 14 patients without cardiomyopathy for whom data were available (group 2). Their median age at the time of the study was 11 years (range, 3-21 years). PA was diagnosed in the neonatal period in 5 of 6 patients in group 1 and 11 of 14 patients in group 2. All patients received similar medical treatment. Two patients in group 1 died of cardiac arrest. In 2 patients, the cardiomyopathy was reversed during the year after orthotopic liver transplantation (OLT). In 2 other patients, OLT was contraindicated because of severe heart disease. The number of metabolic distress episodes was similar in both groups. Excretion of propionate metabolites in urine did not correlate with the occurrence of cardiomyopathy.
Conclusion |
Dilated cardiomyopathy, a frequent complication of PA, develops independent of any specific metabolic profile and is reversible after OLT.
Le texte complet de cet article est disponible en PDF.Mots-clés : 3-OHP, ACEI, MC, OLT, ONLCFA, PA, PCC, PG, SF, TG
Plan
| Supported by the Fondation pour la Recherche Médicale, the Association Française contre les Myopathies, the Fondation Lejeune, and Orphan industry. The authors declare no conflicts of interest, real or perceived. |
Vol 156 - N° 1
P. 128-134 - janvier 2010 Retour au numéro
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