Barbara K Burton1, Nathalie Guffon, Jane Roberts, Ans T van der Ploeg, Simon A Jones. 1. Northwestern University Feinberg School of Medicine and Division of Genetics, Birth Defects and Metabolism, Children's Memorial Hospital, 2300 Children's Plaza, Chicago, IL 60614, USA. BBurton@childrensmemorial.org
Abstract
OBJECTIVE: To use our experience with patients in the observational database HOS - the Hunter Outcome Survey - to evaluate the feasibility of home infusions of idursulfase for patients with mucopolysaccharidosis type II (MPS II). STUDY DESIGN: Data were collected on or before 16 October 2009. Of 421 patients receiving idursulfase, 92 (21.9%) had received infusions at home or in an alternative, approved non-hospital environment. Information in HOS relating to the transition to home therapy was analyzed. RESULTS: Patients started home therapy after a median of 9.0months of idursulfase. Most were aged 5-11years at transfer (45.7%; median age, 8.0years), but many (19.6%) were <5years of age. Patients had disease manifestations typical of the wider population; over one-third had cognitive impairment, in some cases severe. Illness was the most frequent cause of missed home infusions. Six patients stopped home therapy; four subsequently resumed home infusions. Five infusion-related reactions occurred in 2 of the 59 patients who had received home therapy for at least 12months. Reactions were classified as mild-to-moderate. All reactions that occurred at home were readily managed at home. CONCLUSIONS: Providing appropriate factors are considered, it should be feasible for patients with MPS II, including those who are severely affected, to receive infusions at home.
OBJECTIVE: To use our experience with patients in the observational database HOS - the Hunter Outcome Survey - to evaluate the feasibility of home infusions of idursulfase for patients with mucopolysaccharidosis type II (MPS II). STUDY DESIGN: Data were collected on or before 16 October 2009. Of 421 patients receiving idursulfase, 92 (21.9%) had received infusions at home or in an alternative, approved non-hospital environment. Information in HOS relating to the transition to home therapy was analyzed. RESULTS:Patients started home therapy after a median of 9.0months of idursulfase. Most were aged 5-11years at transfer (45.7%; median age, 8.0years), but many (19.6%) were <5years of age. Patients had disease manifestations typical of the wider population; over one-third had cognitive impairment, in some cases severe. Illness was the most frequent cause of missed home infusions. Six patients stopped home therapy; four subsequently resumed home infusions. Five infusion-related reactions occurred in 2 of the 59 patients who had received home therapy for at least 12months. Reactions were classified as mild-to-moderate. All reactions that occurred at home were readily managed at home. CONCLUSIONS: Providing appropriate factors are considered, it should be feasible for patients with MPS II, including those who are severely affected, to receive infusions at home.
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
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
Authors: Joseph Muenzer; Simon A Jones; Anna Tylki-Szymańska; Paul Harmatz; Nancy J Mendelsohn; Nathalie Guffon; Roberto Giugliani; Barbara K Burton; Maurizio Scarpa; Michael Beck; Yvonne Jangelind; Elizabeth Hernberg-Stahl; Maria Paabøl Larsen; Tom Pulles; David A H Whiteman Journal: Orphanet J Rare Dis Date: 2017-05-02 Impact factor: 4.123