Cardiomyopathies in Propionic Aciduria are Reversible After Liver Transplantation

Propionic acidemia (PA) is a severe metabolic disorder with cardiac and neurologic complications and a poor quality of life. Liver transplantation (LT) was thus proposed in PA to increase enzyme activity. We studied retrospectively LT in PA in two European centers. Twelve patients underwent 17 LTs between 1991 and 2013. They developed severe, unusual and unexpected complications, with high mortality (58%). When present, the cardiomyopathy resolved and no acute metabolic decompensation occurred allowing dietary relaxation. Renal failure was present in half of the patients before LT and worsened in all of them. We suggest that cardiac and renal functions should be assessed before LT and monitored closely afterward. A renal sparing immunosuppression should be used. We speculate that some complications may be related to accumulated toxicity of the disease and that earlier LT could prevent some of these consequences. As kidney transplantation has been performed successfully in methylmalonic acidemia, a metabolic disease in the same biochemical pathway, the choice of the organ to transplant could be further discussed.

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“…As we previously reported, cardiomyopathy after LT was reversible (7). Thus it remains a main indication for LT.…”

Section: Discussionmentioning

confidence: 55%

Early and Late Complications After Liver Transplantation for Propionic Acidemia in Children: A Two Centers Study

Charbit‐Henrion

Lacaille

McKiernan

et al. 2015

American Journal of Transplantation

100

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“…Cardiomyopathy appears to develop independent of any specifi c metabolic profi le and appears to occur at any age [ 34 , 44 , 47 ]. The mean age of presentation in one series was 7 years [ 44 ]. Long QT syndrome (delayed repolarization of the heart) is also reported and may occur in as high as 70 % of patients with PROP [ 47 -51 ].…”

Section: Complicationsmentioning

confidence: 99%

Organic Acidemias

Thomas¹

2015

Nutrition Management of Inherited Metabolic Diseases

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“…Besides improved survival, patients are also at a much lower or even no risk of metabolic decompensation after liver transplantation [13]. In two patients, the cardiomyopathy was reversed during the year after orthotopic liver transplantation [14], and in another patient, neurologic prognosis was improved [15]. Liver transplantation can only correct the enzymatic deficiency in the liver, while the metabolic defect in other organs may maintain a significant production of toxic metabolites, thus questioning its indication for PA.…”

Section: Standard Treatmentmentioning

confidence: 99%

Understanding molecular mechanisms in propionic acidemia and investigated therapeutic strategies

Richard

Pérez

Pérez‐Cerdá

et al. 2015

Expert Opinion on Orphan Drugs

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Cardiomyopathies in Propionic Aciduria are Reversible After Liver Transplantation

Cited by 115 publications

References 32 publications

Early and Late Complications After Liver Transplantation for Propionic Acidemia in Children: A Two Centers Study

Early and Late Complications After Liver Transplantation for Propionic Acidemia in Children: A Two Centers Study

Organic Acidemias

Understanding molecular mechanisms in propionic acidemia and investigated therapeutic strategies

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