Literature DB >> 18923918

Home treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectively.

S Bagewadi1, J Roberts, J Mercer, S Jones, J Stephenson, J E Wraith.   

Abstract

Enzyme replacement therapy for lysosomal storage disorders has made an important contribution to improving the quality of life of affected patients. The treatment, however, is invasive and onerous, involving weekly or biweekly intravenous infusions of product over a 3-4 h period. Such therapy can be extremely disruptive of normal family life and the provision of a safe, home treatment regimen is greatly appreciated by affected families. In this report we demonstrate the safety of home treatment with Elaprase for mucopolysaccharidosis type II (17 patients) and Naglazyme for mucopolysaccharidosis type VI (6 patients). Careful patient selection, an experienced home care company and a detailed management plan for potential anaphylaxis and infusion-associated reactions are important components in a successful home treatment programme.

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Year:  2008        PMID： 18923918     DOI： 10.1007/s10545-008-0980-0

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


  5 in total

1.  Intravenous enzyme replacement therapy: better in home or hospital?

Authors:  A Milligan; D Hughes; S Goodwin; L Richfield; A Mehta
Journal:  Br J Nurs       Date:  2006 Mar 23-Apr 12

2.  Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study.

Authors:  Paul Harmatz; Roberto Giugliani; Ida Schwartz; Nathalie Guffon; Elisa Leão Teles; M Clara Sá Miranda; J Edmond Wraith; Michael Beck; Laila Arash; Maurizio Scarpa; Zi-Fan Yu; Janet Wittes; Kenneth I Berger; Mary S Newman; Ann M Lowe; Emil Kakkis; Stuart J Swiedler
Journal:  J Pediatr       Date:  2006-04       Impact factor: 4.406

3.  Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe.

Authors:  J Cox-Brinkman; R G M Timmermans; F A Wijburg; W E Donker; A T van de Ploeg; J M F G Aerts; C E M Hollak
Journal:  J Inherit Metab Dis       Date:  2007-09-21       Impact factor: 4.982

Review 4.  Home therapy for lysosomal storage disorders.

Authors:  Derryalynn A Hughes; Alan Mlilligan; Atul Mehta
Journal:  Br J Nurs       Date:  2007 Dec 13-2008 Jan 9

5.  A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome).

Authors:  Joseph Muenzer; James E Wraith; Michael Beck; Roberto Giugliani; Paul Harmatz; Christine M Eng; Ashok Vellodi; Rick Martin; Uma Ramaswami; Muge Gucsavas-Calikoglu; Suresh Vijayaraghavan; Susanne Wendt; Suzanne Wendt; Ana Cristina Puga; Antonio Puga; Brian Ulbrich; Marwan Shinawi; Maureen Cleary; Diane Piper; Anne Marie Conway; Ann Marie Conway; Alan Kimura
Journal:  Genet Med       Date:  2006-08       Impact factor: 8.822
  5 in total
  10 in total

Review 1.  Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.

Authors:  Marcela Junqueira Brunelli; Álvaro N Atallah; Edina Mk da Silva
Journal:  Cochrane Database Syst Rev       Date:  2021-09-17

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.  A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase.

Authors:  Regina P El Dib; Gregory M Pastores
Journal:  Biologics       Date:  2009-10-12

8.  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 9.  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

10.  Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses.

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

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