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Long‐term outcome of methylmalonic aciduria after kidney, liver, or combined liver‐kidney transplantation: The French experience
Author(s) -
Brassier Anaïs,
Krug Pauline,
Lacaille Florence,
Pontoizeau Clément,
Krid Saoussen,
Sissaoui Samira,
Servais Aude,
Arnoux JeanBaptiste,
Legendre Christophe,
Charbit Marina,
Scemla Anne,
Francoz Claire,
Benoist JeanFrançois,
Schiff Manuel,
Mochel Fanny,
Touati Guy,
Broué Pierre,
Cano Aline,
Tardieu Marine,
Querciagrossa Stefania,
Grévent David,
Boyer Olivia,
Dupic Laurent,
Oualha Mehdi,
Girard Muriel,
Aigrain Yves,
Debray Dominique,
Capito Carmen,
Ottolenghi Chris,
Salomon Rémi,
Chardot Christophe,
Lonlay Pascale
Publication year - 2020
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1002/jimd.12174
Subject(s) - medicine , methylmalonic aciduria , transplantation , kidney transplantation , dialysis , liver transplantation , gastroenterology , kidney , methylmalonic acid , hepatoblastoma , surgery , vitamin b12
Organ transplantation is discussed in methylmalonic aciduria (MMA) for renal failure, and poor quality of life and neurological outcome. We retrospectively evaluated 23 French MMA patients after kidney (KT), liver‐kidney (LKT), and liver transplantation (LT). Two patients died, one after LKT, one of hepatoblastoma after KT. One graft was lost early after KT. Of 18 evaluable patients, 12 previously on dialysis, 8 underwent KT (mean 12.5 years), 8 LKT (mean 7 years), and 2 LT (7 and 2.5 years). At a median follow‐up of 7.3 (KT), 2.3 (LKT), and 1.0 years (LT), no metabolic decompensation occurred except in 1 KT. Plasma and urine MMA levels dramatically decreased, more after LKT. Protein intake was increased more significantly after LKT than KT. Enteral nutrition was stopped in 7/8 LKT, 1/8 KT. Early complications were frequent after LKT. Neurological disorders occurred in four LKT, reversible in one. Five years after KT, four patients had renal failure. The metabolic outcomes were much better after LKT than KT. LKT in MMA is difficult but improves the quality of life. KT will be rarely indicated. We need more long‐term data to indicate early LT, in the hope to delay renal failure and prevent neurodevelopmental complications.