OBJECTIVE: Intravenous enzyme replacement therapy (ERT) with recombinant alpha-L-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based ERT for Gaucher and Fabry diseases has been reported to be safe and successful, we investigated the feasibility and safety of home therapy in patients with MPS I. SETTING: This two-centre study included 17 ERT-treated MPS I patients between 1 and 35 years of age. A patient was allowed to transfer to home treatment after a minimum period of 6 months of in-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. RESULTS: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 months). Two patients preferred to continue ERT in the hospital, whereas for two other patients the transfer to home was hampered for practical reasons. All patients who received ERT at home were assisted by either a relative or a nurse. In total over 1000 home infusions were performed and no serious complications were observed. Two infusion-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions. CONCLUSION: ERT for MPS I applied at home is safe and might alleviate the burden of life-long intravenous treatment in these patients.
OBJECTIVE: Intravenous enzyme replacement therapy (ERT) with recombinant alpha-L-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based ERT for Gaucher and Fabry diseases has been reported to be safe and successful, we investigated the feasibility and safety of home therapy in patients with MPS I. SETTING: This two-centre study included 17 ERT-treated MPS Ipatients between 1 and 35 years of age. A patient was allowed to transfer to home treatment after a minimum period of 6 months of in-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. RESULTS: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 months). Two patients preferred to continue ERT in the hospital, whereas for two other patients the transfer to home was hampered for practical reasons. All patients who received ERT at home were assisted by either a relative or a nurse. In total over 1000 home infusions were performed and no serious complications were observed. Two infusion-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions. CONCLUSION: ERT for MPS I applied at home is safe and might alleviate the burden of life-long intravenous treatment in these patients.
Authors: Roberto Giugliani; Andressa Federhen; Maria Verônica Muñoz Rojas; Taiane Vieira; Osvaldo Artigalás; Louise Lapagesse Pinto; Ana Cecília Azevedo; Angelina Acosta; Carmen Bonfim; Charles Marques Lourenço; Chong Ae Kim; Dafne Horovitz; Denize Bonfim; Denise Norato; Diane Marinho; Durval Palhares; Emerson Santana Santos; Erlane Ribeiro; Eugênia Valadares; Fábio Guarany; Gisele Rosone de Lucca; Helena Pimentel; Isabel Neves de Souza; Jordão Correa; José Carlos Fraga; José Eduardo Goes; José Maria Cabral; José Simionato; Juan Llerena; Laura Jardim; Liane Giuliani; Luiz Carlos Santana da Silva; Mara L Santos; Maria Angela Moreira; Marcelo Kerstenetzky; Márcia Ribeiro; Nicole Ruas; Patricia Barrios; Paulo Aranda; Rachel Honjo; Raquel Boy; Ronaldo Costa; Carolina Souza; Flavio F Alcantara; Silvio Gilberto A Avilla; Simone Fagondes; Ana Maria Martins Journal: Genet Mol Biol Date: 2010-12-01 Impact factor: 1.771