Literature DB >> 17096763

Current role of liver transplantation for methylmalonic acidemia: a review of the literature.

Mureo Kasahara1, Reiko Horikawa, Manabu Tagawa, Shinji Uemoto, Satoshi Yokoyama, Yumiko Shibata, Takafumi Kawano, Tatsuo Kuroda, Toshiro Honna, Koichi Tanaka, Morihiro Saeki.   

Abstract

To evaluate the current role of liver transplantation (LT) for methylmalonic acidemia (MMA), we reviewed the literature on outcomes of this treatment, and describe three of our own cases of living-donor liver transplantation (LDLT). The total number of LT cases identified was 18. Transplantation mode was deceased donor LT in 12, including five combined liver-kidney transplantations (CLKT) from deceased donors, and LDLT in six. Three hospital mortalities were noted, because of metabolic decompensation, sepsis and aspergillosis. Although mean postoperative serum MMA level decreased to 13.8% +/- 9.2% (range 1.25-26.1%) of preoperative levels, four patients (22.2%) had renal insufficiency after isolated LT and three (16.7%) had postoperative neurological disability. Continuing metabolic damage to the kidney and brain may occur even after successful LT. Further evaluation is required to determine the long-term suitability of this treatment modality.

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Year:  2006        PMID: 17096763     DOI: 10.1111/j.1399-3046.2006.00585.x

Source DB:  PubMed          Journal:  Pediatr Transplant        ISSN: 1397-3142


  28 in total

1.  FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.

Authors:  Irini Manoli; Justin R Sysol; Madeline W Epping; Lina Li; Cindy Wang; Jennifer L Sloan; Alexandra Pass; Jack Gagné; Yiouli P Ktena; Lingli Li; Niraj S Trivedi; Bazoumana Ouattara; Patricia M Zerfas; Victoria Hoffmann; Mones Abu-Asab; Maria G Tsokos; David E Kleiner; Caterina Garone; Kristina Cusmano-Ozog; Gregory M Enns; Hilary J Vernon; Hans C Andersson; Stephanie Grunewald; Abdel G Elkahloun; Christiane L Girard; Jurgen Schnermann; Salvatore DiMauro; Eva Andres-Mateos; Luk H Vandenberghe; Randy J Chandler; Charles P Venditti
Journal:  JCI Insight       Date:  2018-12-06

2.  Correction of methylmalonic aciduria in vivo using a codon-optimized lentiviral vector.

Authors:  Edward S Y Wong; Chantelle McIntyre; Heidi L Peters; Enzo Ranieri; Donald S Anson; Janice M Fletcher
Journal:  Hum Gene Ther       Date:  2014-04-02       Impact factor: 5.695

Review 3.  Renal dysfunction in methylmalonic acidurias: review for the pediatric nephrologist.

Authors:  Marina A Morath; Friederike Hörster; Sven W Sauer
Journal:  Pediatr Nephrol       Date:  2012-07-20       Impact factor: 3.714

4.  Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction.

Authors:  Nuria Carrillo-Carrasco; Randy J Chandler; Suma Chandrasekaran; Charles P Venditti
Journal:  Hum Gene Ther       Date:  2010-09       Impact factor: 5.695

Review 5.  Liver transplantation for pediatric inherited metabolic disorders: Considerations for indications, complications, and perioperative management.

Authors:  Kimihiko Oishi; Ronen Arnon; Melissa P Wasserstein; George A Diaz
Journal:  Pediatr Transplant       Date:  2016-06-21

Review 6.  Transplantation as disease modifying therapy in adults with inherited metabolic disorders.

Authors:  Sandra Sirrs; Fady Hannah-Shmouni; Stephen Nantel; James Neuberger; Eric M Yoshida
Journal:  J Inherit Metab Dis       Date:  2018-02-01       Impact factor: 4.982

7.  Successful Domino Liver Transplantation from a Patient with Methylmalonic Acidemia.

Authors:  A Khanna; R Gish; S C Winter; W L Nyhan; B A Barshop
Journal:  JIMD Rep       Date:  2015-07-29

8.  Pediatric combined liver-kidney transplantation: a single-center experience of 18 cases.

Authors:  Rémi Duclaux-Loras; Justine Bacchetta; Julien Berthiller; Christine Rivet; Delphine Demède; Etienne Javouhey; Rémi Dubois; Frédérique Dijoud; Alain Lachaux; Lionel Badet; Olivier Boillot; Pierre Cochat
Journal:  Pediatr Nephrol       Date:  2016-04-08       Impact factor: 3.714

Review 9.  Combined liver-kidney transplant for the management of methylmalonic aciduria: a case report and review of the literature.

Authors:  Peter J Mc Guire; Elizabeth Lim-Melia; George A Diaz; Kimiyo Raymond; Alexandra Larkin; Melissa P Wasserstein; Claude Sansaricq
Journal:  Mol Genet Metab       Date:  2007-10-26       Impact factor: 4.797

10.  Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.

Authors:  Randy J Chandler; Charles P Venditti
Journal:  Mol Ther       Date:  2009-10-27       Impact factor: 11.454

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