Literature DB >> 29294253

Atypical adult-onset methylmalonic acidemia and homocystinuria presenting as hemolytic uremic syndrome.

David Navarro1, Ana Azevedo2, Sílvia Sequeira3, Ana Carina Ferreira4, Fernanda Carvalho4, Teresa Fidalgo5, Laura Vilarinho6, Maria Céu Santos7, Joaquim Calado4, Fernando Nolasco4.   

Abstract

Thrombotic microangiopathy (TMA) syndromes can be secondary to a multitude of different diseases. Most can be identified with a systematic approach and, when excluded, TMA is generally attributed to a dysregulation in the activity of the complement alternative pathways-atypical hemolytic uremic syndrome (aHUS). We present a challenging case of a 19-year-old woman who presented with thrombotic microangiopathy, which was found to be caused by methylmalonic acidemia and homocystinuria, a rare vitamin B12 metabolism deficiency. To our knowledge, this is the first time that an adult-onset methylmalonic acidemia and homocystinuria presents as TMA preceding CNS involvement.

Entities:  

Keywords:  Methylmalonic aciduria and homocystinuria; Thrombotic microangiopathy; Vitamin B12 metabolism

Year:  2018        PMID: 29294253      PMCID: PMC5886929          DOI: 10.1007/s13730-017-0298-6

Source DB:  PubMed          Journal:  CEN Case Rep        ISSN: 2192-4449


  13 in total

1.  Cobalamin C deficiency-associated thrombotic microangiopathy: uncommon or unrecognised?

Authors:  James N George
Journal:  Lancet       Date:  2015-09-05       Impact factor: 79.321

2.  Molecular picture of cobalamin C/D defects before and after newborn screening era.

Authors:  C Nogueira; A Marcão; H Rocha; C Sousa; H Fonseca; C Valongo; L Vilarinho
Journal:  J Med Screen       Date:  2016-07-07       Impact factor: 2.136

Review 3.  Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management.

Authors:  Nuria Carrillo-Carrasco; Randy J Chandler; Charles P Venditti
Journal:  J Inherit Metab Dis       Date:  2011-07-12       Impact factor: 4.982

Review 4.  Haemolytic uraemic syndrome.

Authors:  Fadi Fakhouri; Julien Zuber; Véronique Frémeaux-Bacchi; Chantal Loirat
Journal:  Lancet       Date:  2017-02-25       Impact factor: 79.321

5.  Identification of the gene responsible for methylmalonic aciduria and homocystinuria, cblC type.

Authors:  Jordan P Lerner-Ellis; Jamie C Tirone; Peter D Pawelek; Carole Doré; Janet L Atkinson; David Watkins; Chantal F Morel; T Mary Fujiwara; Emily Moras; Angela R Hosack; Gail V Dunbar; Hana Antonicka; Vince Forgetta; C Melissa Dobson; Daniel Leclerc; Roy A Gravel; Eric A Shoubridge; James W Coulton; Pierre Lepage; Johanna M Rommens; Kenneth Morgan; David S Rosenblatt
Journal:  Nat Genet       Date:  2005-11-27       Impact factor: 38.330

6.  Newborn screening and early biochemical follow-up in combined methylmalonic aciduria and homocystinuria, cblC type, and utility of methionine as a secondary screening analyte.

Authors:  James D Weisfeld-Adams; Mark A Morrissey; Brian M Kirmse; Bobbie R Salveson; Melissa P Wasserstein; Peter J McGuire; Sherlykutty Sunny; Jessica L Cohen-Pfeffer; Chunli Yu; Michele Caggana; George A Diaz
Journal:  Mol Genet Metab       Date:  2009-09-27       Impact factor: 4.797

7.  Spectrum of MMACHC mutations in Italian and Portuguese patients with combined methylmalonic aciduria and homocystinuria, cblC type.

Authors:  Célia Nogueira; Chiara Aiello; Roberto Cerone; Esmeralda Martins; Ubaldo Caruso; Isabella Moroni; Cristiano Rizzo; Luísa Diogo; Elisa Leão; Fernando Kok; Federica Deodato; Maria Cristina Schiaffino; Sara Boenzi; Olivier Danhaive; Clara Barbot; Sílvia Sequeira; Mattia Locatelli; Filippo M Santorelli; Graziella Uziel; Laura Vilarinho; Carlo Dionisi-Vici
Journal:  Mol Genet Metab       Date:  2007-12-27       Impact factor: 4.797

8.  The adolescent and adult form of cobalamin C disease: clinical and molecular spectrum.

Authors:  C Thauvin-Robinet; E Roze; G Couvreur; M-H Horellou; F Sedel; D Grabli; G Bruneteau; C Tonneti; A Masurel-Paulet; D Perennou; T Moreau; M Giroud; H Ogier de Baulny; S Giraudier; L Faivre
Journal:  J Neurol Neurosurg Psychiatry       Date:  2008-02-01       Impact factor: 10.154

9.  Clinical and renal biopsy findings predicting outcome in renal thrombotic microangiopathy: a large cohort study from a single institute in China.

Authors:  Xiao-Juan Yu; Feng Yu; Di Song; Su-Xia Wang; Yan Song; Gang Liu; Ming-Hui Zhao
Journal:  ScientificWorldJournal       Date:  2014-08-11

Review 10.  Renal thrombotic microangiopathy in patients with cblC defect: review of an under-recognized entity.

Authors:  Bodo B Beck; FrancJan van Spronsen; Arjan Diepstra; Rolf M F Berger; Martin Kömhoff
Journal:  Pediatr Nephrol       Date:  2016-06-11       Impact factor: 3.714

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  1 in total

1.  Clinical, phenotypic and genetic landscape of case reports with genetically proven inherited disorders of vitamin B12 metabolism: A meta-analysis.

Authors:  Arnaud Wiedemann; Abderrahim Oussalah; Nathalie Lamireau; Maurane Théron; Melissa Julien; Jean-Philippe Mergnac; Baptiste Augay; Pauline Deniaud; Tom Alix; Marine Frayssinoux; François Feillet; Jean-Louis Guéant
Journal:  Cell Rep Med       Date:  2022-06-27
  1 in total

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