Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
dc.citation.volume | 11 | |
dc.contributor.author | Horovitz, Dafne Dain Gandelman | |
dc.contributor.author | Acosta, Angelina X. | |
dc.contributor.author | Giugliani, Roberto | |
dc.contributor.author | Hlavata, Anna | |
dc.contributor.author | Hlavata, Katarina | |
dc.contributor.author | Tchan, Michel C. | |
dc.contributor.author | Barth, Anneliese Lopes | |
dc.contributor.author | Cardoso Jr., Laercio | |
dc.contributor.author | Leao, Emilia Katiane Embirucu de Araujo | |
dc.contributor.author | Esposito, Ana Carolina | |
dc.contributor.author | Kyosen, Sandra Obikawa [UNIFESP] | |
dc.contributor.author | Souza, Carolina Fischinger Moura de | |
dc.contributor.author | Martins, Ana Maria [UNIFESP] | |
dc.coverage | London | |
dc.date.accessioned | 2020-07-22T13:23:06Z | |
dc.date.available | 2020-07-22T13:23:06Z | |
dc.date.issued | 2016 | |
dc.description.abstract | Background: Enzyme replacement therapy (ERT) with laronidase (recombinant human alpha-L-iduronidase, Aldurazyme (R)) is indicated for non-neurological signs and symptoms of mucopolysaccharidosis type I (MPS I). The approved laronidase dose regimen is weekly infusions of 0.58mg/kg, however, patients and caregivers may have difficulty complying with the weekly regimen. We examined clinical outcomes, tolerability, compliance, and satisfaction in a series of patients who switched to every other week infusions. Methods: This multinational, retrospective, chart review case series analyzed data from 20 patients who had undergone ERT with laronidase 0.58mg/kg weekly for more than one year, and who then switched to 1.2mg/kg every other week. Results: The majority of patients had attenuated MPS I phenotypes (9 with Hurler-Scheie and 8 with Scheie syndromes) and 3 patients had severe MPS I (Hurler syndrome). Most patients presented with organomegaly (17/ 20), umbilical and/or inguinal hernia (16/20), cardiac abnormalities (17/20), musculoskeletal abnormalities (19/20), and neurological and/or developmental deficits (15/20). Following laronidase treatment, signs stabilized or improved. No deterioration or reversal of clinical outcome was noted in any patient who switched from the weekly dose of 0.58mg.kg to 1.2mg/kg every other week. There were no safety issues during the duration of every other week dosing. Patient compliance and satisfaction with the dosing regimen were greater with every other week dosing than weekly dosing. Conclusions: An alternative dose regimen of 1.2mg/kg laronidase every other week was well tolerated and clinically similar to the standard dose for patients who were stabilized with weekly 0.58 mg/kg for one year or more. When an individualized approach to laronidase therapy is necessary, every other week dosing may be an alternative for patients with difficulty receiving weekly infusions. | en |
dc.description.affiliation | Fiocruz MS, Inst Nacl Saude Mulher Crianca & Adolescente Fern, BR-21045900 Rio De Janeiro, Brazil | |
dc.description.affiliation | Univ Fed Bahia, Dept Pediat, Serv Genet Med, Salvador, BA, Brazil | |
dc.description.affiliation | Hosp Clin Alegre, Med Genet Serv, Porto Alegre, RS, Brazil | |
dc.description.affiliation | Comenius Univ, Childrens Hosp, Dept Pediat 2, Bratislava, Slovakia | |
dc.description.affiliation | Westmead Hosp, Dept Med Genet, Sydney, NSW, Australia | |
dc.description.affiliation | Univ Sydney, Sydney, NSW 2006, Australia | |
dc.description.affiliation | Univ Fed Sao Paulo, Dept Pediat, Sao Paulo, Brazil | |
dc.description.affiliationUnifesp | Univ Fed Sao Paulo, Dept Pediat, Sao Paulo, Brazil | |
dc.description.source | Web of Science | |
dc.description.sponsorship | Sanofi Genzyme | |
dc.description.sponsorship | Sanofi Genzyme, Cambridge, MA, USA | |
dc.format.extent | - | |
dc.identifier | http://dx.doi.org/10.1186/s13023-016-0437-8 | |
dc.identifier.citation | Orphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016. | |
dc.identifier.doi | 10.1186/s13023-016-0437-8 | |
dc.identifier.file | WOS000375109300001.pdf | |
dc.identifier.issn | 1750-1172 | |
dc.identifier.uri | https://repositorio.unifesp.br/handle/11600/56035 | |
dc.identifier.wos | WOS:000375109300001 | |
dc.language.iso | eng | |
dc.publisher | Biomed Central Ltd | |
dc.relation.ispartof | Orphanet Journal Of Rare Diseases | |
dc.rights | info:eu-repo/semantics/openAccess | |
dc.subject | Enzyme replacement therapy | en |
dc.subject | alpha-L-iduronidase deficiency | en |
dc.subject | Clinical outcomes | en |
dc.subject | Tolerability | en |
dc.title | Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series | en |
dc.type | info:eu-repo/semantics/article |
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