Early and late complications after liver transplantation for propionic acidemia in children: A two centers study

American Journal of Transplantation

Volumn 15, Issue 3, 2015, Pages 786-791

  Charbit Henrion, Fabienne ^a   Lacaille, F ^b   McKiernan, P ^c   Girard, M ^b   De Lonlay, P ^b   Valayannopoulos, V ^b   Ottolenghi, C ^b   Chakrapani, A ^c   Preece, M ^c   Sharif, K ^c   Chardot, C ^b   Hubert, P ^b   Dupic, L ^b  

^a IMAGINE INSTITUTE   (France)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

^c BIRMINGHAM CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Clinical research practice; kidney failure injury; liver transplantation hepatology; metabolism metabolite; pediatrics

Indexed keywords

AZATHIOPRINE; BASILIXIMAB; CREATININE; CYCLOSPORIN; HYDRACRYLIC ACID; MYCOPHENOLIC ACID; PROPIONIC ACID; STEROID; TACROLIMUS; IMMUNOSUPPRESSIVE AGENT;

ABSORPTIOMETRY; ACUTE BRAIN DISEASE; ACUTE DISSEMINATED ENCEPHALOMYELITIS; ADULT RESPIRATORY DISTRESS SYNDROME; ARTICLE; ARTIFICIAL VENTILATION; CARDIOMYOPATHY; CHILD; CLINICAL ARTICLE; COMA; CONGESTIVE CARDIOMYOPATHY; DRUG SUBSTITUTION; DRUG WITHDRAWAL; ECHOCARDIOGRAPHY; FEMALE; FOLLOW UP; HEART FUNCTION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; KIDNEY FAILURE; KIDNEY FUNCTION; LIVER DYSFUNCTION; LIVER GRAFT REJECTION; LIVER TRANSPLANTATION; MALE; MEDICAL RECORD REVIEW; METHYLMALONIC ACIDEMIA; NEPHROTOXICITY; NEUROTOXICITY; POSTOPERATIVE COMPLICATION; PRIORITY JOURNAL; PROPIONIC ACIDEMIA; QUALITY OF LIFE; RETROSPECTIVE STUDY; ADVERSE EFFECTS; CLINICAL TRIAL; MULTICENTER STUDY; PATHOPHYSIOLOGY;

CHILD; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; LIVER TRANSPLANTATION; MALE; PROPIONIC ACIDEMIA;

EID: 84924019146     PISSN: 16006135     EISSN: 16006143     Source Type: Journal    
DOI: 10.1111/ajt.13027     Document Type: Article

References (24)

1. Chapman KA, Gropman A, MacLeod E, et al. Acute management of propionic acidemia. Mol Genet Metab 2012; 105: 16-25.
2. Saudubray JM, Nassogne MC, de Lonlay P, Touati G., Clinical approach to inherited metabolic disorders in neonates: An overview. Semin Neonatol 2002; 7: 3-15.
3. Leonard JV, Vijayaraghavan S, Walter JH., The impact of screening for propionic and methylmalonic acidaemia. Eur J Pediat 2003; 162: S21-S24.
4. Saudubray JM, Touati G, Delonlay P, et al. Liver transplantation in propionic acidaemia. Eur J Pediatr 1999; 158: S65-S69.
5. de Baulny HO, Benoist JF, Rigal O, Touati G, Rabier D, Saudubray JM., Methylmalonic and propionic acidaemias: Management and outcome. J Inherit Metab Dis 2005; 28: 415-423.
6. Nizon M, Ottolenghi C, Valayannopoulos V, et al. Long-term neurological outcome of a cohort of 80 patients with classical organic acidurias. Orphanet J Rare Dis 2013; 8: 148.
7. Romano S, Valayannopoulos V, Touati G, et al. Cardiomyopathies in propionic aciduria are reversible after liver transplantation. J Pediat 2010; 156: 128-134.
8. Davison JE, Davies NP, Wilson M, et al. MR spectroscopy-based brain metabolite profiling in propionic acidaemia: Metabolic changes in the basal ganglia during acute decompensation and effect of liver transplantation. Orphanet J Rare Dis 2011; 6: 19.
9. Lacaille F., Liver transplantation and liver cell transplantation. Clin Res Hepatol Gastroenterol 2012; 36: 304-337.
10. Lacaille FP-TF, Laurent J, Jan D, et al. Liver transplantation for propionic acidemia in children: Prudence about a new indication. Hepatology 1994; 20: 122A.
11. Barshes NR, Vanatta JM, Patel AJ, et al. Evaluation and management of patients with propionic acidemia undergoing liver transplantation: A comprehensive review. Pediatr Transplant 2006; 10: 773-781.
12. Schwartz GJ, Haycock GB, Edelmann CM Jr, Spitzer A., A simple estimate of glomerular filtration rate in children derived from body length and plasma creatinine. Pediatrics 1976; 58: 259-263.
13. McKiernan P., Liver transplantation and cell therapies for inborn errors of metabolism. J Inherit Metab Dis 2013; 36: 675-680.
14. Rela M, Battula N, Madanur M, et al. Auxiliary liver transplantation for propionic acidemia: A 10-year follow-up. Am J Transplant 2007; 7: 2200-2203.
15. Kasahara M, Sakamoto S, Kanazawa H, et al. Living-donor liver transplantation for propionic acidemia. Pediatr Transplant 2012; 16: 230-234.
16. Vara R, Turner C, Mundy H, et al. Liver transplantation for propionic acidemia in children. Liver Transpl 2011; 17: 661-667.
17. de Keyzer Y, Valayannopoulos V, Benoist JF, et al. Multiple OXPHOS deficiency in the liver, kidney, heart, and skeletal muscle of patients with methylmalonic aciduria and propionic aciduria. Pediatr Res 2009; 66: 91-95.
18. Campbell K, Ng V, Martin S, et al. Glomerular filtration rate following pediatric liver transplantation-The SPLIT experience. Am J Transplant 2010; 10: 2673-2682.
19. Gijsen VM, Hesselink DA, Croes K, Koren G, de Wildt SN., Prevalence of renal dysfunction in tacrolimus-treated pediatric transplant recipients: A systematic review. Pediatr Transplant 2013; 17: 205-215.
20. Kelly D., Safety and efficacy of tacrolimus in pediatric liver recipients. Pediatr Transplant 2011; 15: 19-24.
21. Lam C, Desviat LR, Perez-Cerda C, Ugarte M, Barshop BA, Cederbaum S., 45-Year-old female with propionic acidemia, renal failure, and premature ovarian failure; late complications of propionic acidemia ? Mol Genet Metab 2011; 103: 338-340.
22. Dejean de la Batie C, Barbier V, Valayannopoulos V, et al. Acute psychosis in propionic acidemia: 2 case reports. J Child Neurol 2014; 29: 274-279.
23. Brassier A, Boyer O, Valayannopoulos V, et al. Renal transplantation in 4 patients with methylmalonic aciduria: A cell therapy for metabolic disease. Mol Genet Metab 2013; 110: 106-110.
24. Andrews E, Jansen R, Crane AM, Cholin S, McDonnell D, Ledley FD., Expression of recombinant human methylmalonyl-CoA mutase: In primary mut fibroblasts and Saccharomyces cerevisiae. Biochem Med Metab Biol 1993; 50: 135-144.