Literature DB >> 7492172

Cardiac involvement in mucopolysaccharidoses: effects of allogeneic bone marrow transplantation.

M A Gatzoulis1, A Vellodi, A N Redington.   

Abstract

Echocardiography was performed in 16 children undergoing bone marrow transplantation (BMT) for mucopolysaccharidoses. Cardiac involvement before BMT was detected in seven (44%). One year after BMT (11 patients/five with cardiac involvement), left ventricular restriction resolved in 2/3 patients and hypertrophy in one. In the remainder, at mean follow up of 2.5 years, no progression of preexisting or development of new cardiac involvement was noted. It is concluded that in a significant proportion of patients with mucopolysaccharidoses, cardiac involvement improved after BMT.

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Year:  1995        PMID： 7492172      PMCID： PMC1511298          DOI： 10.1136/adc.73.3.259

Source DB:  PubMed          Journal:  Arch Dis Child        ISSN： 0003-9888            Impact factor:   3.791


  6 in total

1.  Echocardiographic abnormalities in type IV mucopolysaccharidosis.

Authors:  R M John; D Hunter; R H Swanton
Journal:  Arch Dis Child       Date:  1990-07       Impact factor: 3.791

2.  Demonstration of restrictive ventricular physiology by Doppler echocardiography.

Authors:  C P Appleton; L K Hatle; R L Popp
Journal:  J Am Coll Cardiol       Date:  1988-04       Impact factor: 24.094

3.  Marrow transplantation in genetic disease.

Authors:  J A Barranger
Journal:  N Engl J Med       Date:  1984-12-20       Impact factor: 91.245

4.  Hypertrophic cardiomyopathy in mucopolysaccharidoses: regression after bone marrow transplantation.

Authors:  X Viñallonga; N Sanz; A Balaguer; L Miro; J J Ortega; J Casaldaliga
Journal:  Pediatr Cardiol       Date:  1992-04       Impact factor: 1.655

5.  Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype.

Authors:  J J Hopwood; A Vellodi; H S Scott; C P Morris; T Litjens; P R Clements; D A Brooks; A Cooper; J E Wraith
Journal:  J Inherit Metab Dis       Date:  1993       Impact factor: 4.982

6.  Echocardiographic abnormalities in the mucopolysaccharide storage diseases.

Authors:  D M Gross; J C Williams; C Caprioli; B Dominguez; R R Howell
Journal:  Am J Cardiol       Date:  1988-01-01       Impact factor: 2.778
  6 in total
  11 in total

1.  Prevalence and development of orthopaedic symptoms in the dutch hurler patient population after haematopoietic stem cell transplantation.

Authors:  F J Stoop; M C Kruyt; M H van der Linden; R J B Sakkers; P M van Hasselt; R M C Castelein
Journal:  JIMD Rep       Date:  2012-09-19

2.  Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.

Authors:  A Vellodi; E P Young; A Cooper; J E Wraith; B Winchester; C Meaney; U Ramaswami; A Will
Journal:  Arch Dis Child       Date:  1997-02       Impact factor: 3.791

3.  Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience.

Authors:  Daniel H Wiseman; Jean Mercer; Karen Tylee; Nilima Malaiya; Denise K Bonney; Simon A Jones; J Edmond Wraith; Robert F Wynn
Journal:  J Inherit Metab Dis       Date:  2012-06-21       Impact factor: 4.982

4.  Cardiac manifestations in the mouse model of mucopolysaccharidosis I.

Authors:  Maria C Jordan; Yi Zheng; Sergey Ryazantsev; Nora Rozengurt; Kenneth P Roos; Elizabeth F Neufeld
Journal:  Mol Genet Metab       Date:  2005-06-24       Impact factor: 4.797

5.  Bone marrow transplantation for Maroteaux-Lamy syndrome (MPS VI): long-term follow-up.

Authors:  E Herskhovitz; E Young; J Rainer; C M Hall; V Lidchi; K Chong; A Vellodi
Journal:  J Inherit Metab Dis       Date:  1999-02       Impact factor: 4.982

6.  Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I.

Authors:  Jakub Tolar; Elizabeth Braunlin; Megan Riddle; Brandon Peacock; Ron T McElmurry; Paul J Orchard; Bruce R Blazar
Journal:  J Heart Valve Dis       Date:  2009-09

7.  Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantation.

Authors:  A J T Schuldt; T J Hampton; V Chu; C A Vogler; N Galvin; M D Lessard; J E Barker
Journal:  Proc Natl Acad Sci U S A       Date:  2004-01-02       Impact factor: 11.205

Review 8.  Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA.

Authors:  Christian J Hendriksz; Maisoon Al-Jawad; Kenneth I Berger; Sara M Hawley; Rebecca Lawrence; Ciarán Mc Ardle; C Gail Summers; Elizabeth Wright; Elizabeth Braunlin
Journal:  J Inherit Metab Dis       Date:  2012-02-23       Impact factor: 4.982

Review 9.  Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management.

Authors:  Elizabeth A Braunlin; Paul R Harmatz; Maurizio Scarpa; Beatriz Furlanetto; Christoph Kampmann; James P Loehr; Katherine P Ponder; William C Roberts; Howard M Rosenfeld; Roberto Giugliani
Journal:  J Inherit Metab Dis       Date:  2011-07-09       Impact factor: 4.982

Review 10.  Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.

Authors:  Christiane S Hampe; Jacob Wesley; Troy C Lund; Paul J Orchard; Lynda E Polgreen; Julie B Eisengart; Linda K McLoon; Sebahattin Cureoglu; Patricia Schachern; R Scott McIvor
Journal:  Biomolecules       Date:  2021-01-29
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