Literature DB >> 20436523

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach.

J Halter1, Wmm Schüpbach2,3, A Gratwohl1, M Hirano4, C Casali5, R Elhasid6, K Fay7, S Hammans8, I Illa9, L Kappeler3, S Krähenbühl10, T Lehmann1, H Mandel11, R Marti12, H Mattle2,3, K Orchard13, D Savage14, C M Sue15, D Valcarcel16.   

Abstract

Allogeneic hematopoietic SCT (HSCT) has been proposed as a treatment for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). HSCT has been performed in nine patients using different protocols with varying success. Based on this preliminary experience, participants of the first consensus conference propose a common approach to allogeneic HSCT in MNGIE. Standardization of the transplant protocol and the clinical and biochemical assessments will allow evaluation of the safety and efficacy of HSCT as well as optimization of therapy for patients with MNGIE.

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Year:  2010        PMID： 20436523      PMCID： PMC4578692          DOI： 10.1038/bmt.2010.100

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN： 0268-3369            Impact factor:   5.483


  42 in total

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2.  Effect of tacrolimus on the production of oxygen free radicals in hepatic mitochondria.

Authors:  S Y Han; E J Chang; H J Choi; C S Kwak; S I Suh; J H Bae; S B Park; H C Kim; K C Mun
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3.  Thymidine phosphorylase mutations cause instability of mitochondrial DNA.

Authors:  Michio Hirano; Clotilde Lagier-Tourenne; Maria L Valentino; Ramon Martí; Yutaka Nishigaki
Journal:  Gene       Date:  2005-07-18       Impact factor: 3.688

4.  Cognitive dysfunction and hypogonadotrophic hypogonadism in a Brazilian patient with mitochondrial neurogastrointestinal encephalomyopathy and a novel ECGF1 mutation.

Authors:  F J Carod-Artal; M D Herrero; M C Lara; E López-Gallardo; E Ruiz-Pesini; R Martí; J Montoya
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5.  Antiviral efficacy and toxicity of ribavirin and foscarnet each given alone or in combination in the murine AIDS model.

Authors:  R F Omar; P Harvie; P Gourde; A Désormeaux; M Tremblay; D Beauchamp; M G Bergeron
Journal:  Toxicol Appl Pharmacol       Date:  1997-03       Impact factor: 4.219

Review 6.  Subacute mitochondrial liver disease in a patient with AIDS: possible relationship to prolonged fluconazole administration.

Authors:  M P Guillaume; C De Prez; E Cogan
Journal:  Am J Gastroenterol       Date:  1996-01       Impact factor: 10.864

7.  Improvement in rejection, engraftment rate and survival without increase in graft-versus-host disease by high marrow cell dose in patients transplanted for aplastic anaemia.

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Journal:  Br J Haematol       Date:  1988-05       Impact factor: 6.998

8.  Aromatic antiepileptic drugs and mitochondrial toxicity: effects on mitochondria isolated from rat liver.

Authors:  N A G Santos; W S G Medina; N M Martins; F E Mingatto; C Curti; A C Santos
Journal:  Toxicol In Vitro       Date:  2008-03-15       Impact factor: 3.500

9.  Thymidine phosphorylase gene mutations in MNGIE, a human mitochondrial disorder.

Authors:  I Nishino; A Spinazzola; M Hirano
Journal:  Science       Date:  1999-01-29       Impact factor: 47.728

10.  Unbalanced deoxynucleotide pools cause mitochondrial DNA instability in thymidine phosphorylase-deficient mice.

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  52 in total

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2.  Clinical and biochemical improvement following HSCT in a patient with MNGIE: 1-year follow-up.

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Journal:  J Neurol       Date:  2011-06-12       Impact factor: 4.849

5.  MNGIE Syndrome: Liver Cirrhosis Should Be Ruled Out Prior to Bone Marrow Transplantation.

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Journal:  JIMD Rep       Date:  2012-12-29

Review 6.  Mitochondrial matters: Mitochondrial bottlenecks, self-assembling structures, and entrapment in the female germline.

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Review 7.  Erythrocyte-mediated delivery of recombinant enzymes.

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Review 8.  Mitochondrial DNA depletion syndromes: review and updates of genetic basis, manifestations, and therapeutic options.

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Review 9.  Gastrointestinal and hepatic manifestations of mitochondrial disorders.

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10.  The role of brain MRI in mitochondrial neurogastrointestinal encephalomyopathy.

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Journal:  Neuroradiol J       Date:  2013-11-07
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