Literature DB >> 19427803

Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses.

Barbara K Burton1, Chani Wiesman, Andrea Paras, Katherine Kim, Rachel Katz.   

Abstract

The transition to home infusion therapy is described in 11 patients with mucopolysaccharide (MPS) disorders initially treated with enzyme replacement therapy (ERT) in an out-patient infusion center. Home therapy was well tolerated with no infusion-related reactions (IRR) occurring in 671 patient-weeks of treatment. In addition, compliance with the weekly treatment regimen was improved in patients on home therapy.

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Year:  2009        PMID： 19427803     DOI： 10.1016/j.ymgme.2009.04.007

Source DB:  PubMed          Journal:  Mol Genet Metab        ISSN： 1096-7192            Impact factor:   4.797


  9 in total

1.  Receiving enzyme replacement therapy for a lysosomal storage disorder: a preliminary exploration of the experiences of young patients and their families.

Authors:  R Freedman; M Sahhar; L Curnow; J Lee; H Peters
Journal:  J Genet Couns       Date:  2013-03-28       Impact factor: 2.537

Review 2.  Mucopolysaccharidosis VI.

Authors:  Vassili Valayannopoulos; Helen Nicely; Paul Harmatz; Sean Turbeville
Journal:  Orphanet J Rare Dis       Date:  2010-04-12       Impact factor: 4.123

Review 3.  Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review.

Authors:  Linda A Bradley; Hamish R M Haddow; Glenn E Palomaki
Journal:  Genet Med       Date:  2017-05-18       Impact factor: 8.822

4.  The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus.

Authors:  Joseph Muenzer; Olaf Bodamer; Barbara Burton; Lorne Clarke; Gudrun Schulze Frenking; Roberto Giugliani; Simon Jones; Maria Verónica Muñoz Rojas; Maurizio Scarpa; Michael Beck; Paul Harmatz
Journal:  Eur J Pediatr       Date:  2011-10-29       Impact factor: 3.183

5.  Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease.

Authors:  Maurizio Scarpa; Zsuzsanna Almássy; Michael Beck; Olaf Bodamer; Iain A Bruce; Linda De Meirleir; Nathalie Guffon; Encarna Guillén-Navarro; Pauline Hensman; Simon Jones; Wolfgang Kamin; Christoph Kampmann; Christina Lampe; Christine A Lavery; Elisa Leão Teles; Bianca Link; Allan M Lund; Gunilla Malm; Susanne Pitz; Michael Rothera; Catherine Stewart; Anna Tylki-Szymańska; Ans van der Ploeg; Robert Walker; Jiri Zeman; James E Wraith
Journal:  Orphanet J Rare Dis       Date:  2011-11-07       Impact factor: 4.123

6.  Home treatment in paediatric patients with Hunter syndrome: the first Italian experience.

Authors:  Ferdinando Ceravolo; Italia Mascaro; Simona Sestito; Elisa Pascale; Antonino Lauricella; Elio Dizione; Daniela Concolino
Journal:  Ital J Pediatr       Date:  2013-09-09       Impact factor: 2.638

7.  Home infusion with Elosulfase alpha (VimizimR) in a UK Paediatric setting.

Authors:  Niamh Finnigan; Jane Roberts; Jean Mercer; Simon A Jones
Journal:  Mol Genet Metab Rep       Date:  2017-11-05

Review 8.  Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future.

Authors:  David Ah Whiteman; Alan Kimura
Journal:  Drug Des Devel Ther       Date:  2017-08-23       Impact factor: 4.162

9.  Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses.

Authors:  Yılmaz Yıldız; H Serap Sivri
Journal:  Turk Arch Pediatr       Date:  2021-11
  9 in total

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