Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series

dc.citation.volume11
dc.contributor.authorHorovitz, Dafne Dain Gandelman
dc.contributor.authorAcosta, Angelina X.
dc.contributor.authorGiugliani, Roberto
dc.contributor.authorHlavata, Anna
dc.contributor.authorHlavata, Katarina
dc.contributor.authorTchan, Michel C.
dc.contributor.authorBarth, Anneliese Lopes
dc.contributor.authorCardoso Jr., Laercio
dc.contributor.authorLeao, Emilia Katiane Embirucu de Araujo
dc.contributor.authorEsposito, Ana Carolina
dc.contributor.authorKyosen, Sandra Obikawa [UNIFESP]
dc.contributor.authorSouza, Carolina Fischinger Moura de
dc.contributor.authorMartins, Ana Maria [UNIFESP]
dc.coverageLondon
dc.date.accessioned2020-07-22T13:23:06Z
dc.date.available2020-07-22T13:23:06Z
dc.date.issued2016
dc.description.abstractBackground: 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.affiliationFiocruz MS, Inst Nacl Saude Mulher Crianca & Adolescente Fern, BR-21045900 Rio De Janeiro, Brazil
dc.description.affiliationUniv Fed Bahia, Dept Pediat, Serv Genet Med, Salvador, BA, Brazil
dc.description.affiliationHosp Clin Alegre, Med Genet Serv, Porto Alegre, RS, Brazil
dc.description.affiliationComenius Univ, Childrens Hosp, Dept Pediat 2, Bratislava, Slovakia
dc.description.affiliationWestmead Hosp, Dept Med Genet, Sydney, NSW, Australia
dc.description.affiliationUniv Sydney, Sydney, NSW 2006, Australia
dc.description.affiliationUniv Fed Sao Paulo, Dept Pediat, Sao Paulo, Brazil
dc.description.affiliationUnifespUniv Fed Sao Paulo, Dept Pediat, Sao Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipSanofi Genzyme
dc.description.sponsorshipSanofi Genzyme, Cambridge, MA, USA
dc.format.extent-
dc.identifierhttp://dx.doi.org/10.1186/s13023-016-0437-8
dc.identifier.citationOrphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016.
dc.identifier.doi10.1186/s13023-016-0437-8
dc.identifier.fileWOS000375109300001.pdf
dc.identifier.issn1750-1172
dc.identifier.urihttps://repositorio.unifesp.br/handle/11600/56035
dc.identifier.wosWOS:000375109300001
dc.language.isoeng
dc.publisherBiomed Central Ltd
dc.relation.ispartofOrphanet Journal Of Rare Diseases
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectEnzyme replacement therapyen
dc.subjectalpha-L-iduronidase deficiencyen
dc.subjectClinical outcomesen
dc.subjectTolerabilityen
dc.titleAlternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case seriesen
dc.typeinfo:eu-repo/semantics/article
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