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

Detalhes bibliográficos
Autor(a) principal: Horovitz, Dafne Dain Gandelman
Data de Publicação: 2016
Outros Autores: Acosta, Angelina X., Giugliani, Roberto, Hlavata, Anna, Hlavata, Katarina, Tchan, Michel C., Barth, Anneliese Lopes, Cardoso Jr., Laercio, Leao, Emilia Katiane Embirucu de Araujo, Esposito, Ana Carolina, Kyosen, Sandra Obikawa [UNIFESP], Souza, Carolina Fischinger Moura de, Martins, Ana Maria [UNIFESP]
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UNIFESP
Texto Completo: http://dx.doi.org/10.1186/s13023-016-0437-8
https://repositorio.unifesp.br/handle/11600/56035
Resumo: 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.
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spelling Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case seriesEnzyme replacement therapyalpha-L-iduronidase deficiencyClinical outcomesTolerabilityBackground: 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.Fiocruz MS, Inst Nacl Saude Mulher Crianca & Adolescente Fern, BR-21045900 Rio De Janeiro, BrazilUniv Fed Bahia, Dept Pediat, Serv Genet Med, Salvador, BA, BrazilHosp Clin Alegre, Med Genet Serv, Porto Alegre, RS, BrazilComenius Univ, Childrens Hosp, Dept Pediat 2, Bratislava, SlovakiaWestmead Hosp, Dept Med Genet, Sydney, NSW, AustraliaUniv Sydney, Sydney, NSW 2006, AustraliaUniv Fed Sao Paulo, Dept Pediat, Sao Paulo, BrazilUniv Fed Sao Paulo, Dept Pediat, Sao Paulo, BrazilWeb of ScienceSanofi GenzymeSanofi Genzyme, Cambridge, MA, USABiomed Central Ltd2020-07-22T13:23:06Z2020-07-22T13:23:06Z2016info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion-application/pdfhttp://dx.doi.org/10.1186/s13023-016-0437-8Orphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016.10.1186/s13023-016-0437-8WOS000375109300001.pdf1750-1172https://repositorio.unifesp.br/handle/11600/56035WOS:000375109300001engOrphanet Journal Of Rare DiseasesLondoninfo:eu-repo/semantics/openAccessHorovitz, Dafne Dain GandelmanAcosta, Angelina X.Giugliani, RobertoHlavata, AnnaHlavata, KatarinaTchan, Michel C.Barth, Anneliese LopesCardoso Jr., LaercioLeao, Emilia Katiane Embirucu de AraujoEsposito, Ana CarolinaKyosen, Sandra Obikawa [UNIFESP]Souza, Carolina Fischinger Moura deMartins, Ana Maria [UNIFESP]reponame:Repositório Institucional da UNIFESPinstname:Universidade Federal de São Paulo (UNIFESP)instacron:UNIFESP2024-08-11T09:44:33Zoai:repositorio.unifesp.br/:11600/56035Repositório InstitucionalPUBhttp://www.repositorio.unifesp.br/oai/requestbiblioteca.csp@unifesp.bropendoar:34652024-08-11T09:44:33Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)false
dc.title.none.fl_str_mv Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
title Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
spellingShingle Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
Horovitz, Dafne Dain Gandelman
Enzyme replacement therapy
alpha-L-iduronidase deficiency
Clinical outcomes
Tolerability
title_short Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
title_full Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
title_fullStr Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
title_full_unstemmed Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
title_sort Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
author Horovitz, Dafne Dain Gandelman
author_facet Horovitz, Dafne Dain Gandelman
Acosta, Angelina X.
Giugliani, Roberto
Hlavata, Anna
Hlavata, Katarina
Tchan, Michel C.
Barth, Anneliese Lopes
Cardoso Jr., Laercio
Leao, Emilia Katiane Embirucu de Araujo
Esposito, Ana Carolina
Kyosen, Sandra Obikawa [UNIFESP]
Souza, Carolina Fischinger Moura de
Martins, Ana Maria [UNIFESP]
author_role author
author2 Acosta, Angelina X.
Giugliani, Roberto
Hlavata, Anna
Hlavata, Katarina
Tchan, Michel C.
Barth, Anneliese Lopes
Cardoso Jr., Laercio
Leao, Emilia Katiane Embirucu de Araujo
Esposito, Ana Carolina
Kyosen, Sandra Obikawa [UNIFESP]
Souza, Carolina Fischinger Moura de
Martins, Ana Maria [UNIFESP]
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Horovitz, Dafne Dain Gandelman
Acosta, Angelina X.
Giugliani, Roberto
Hlavata, Anna
Hlavata, Katarina
Tchan, Michel C.
Barth, Anneliese Lopes
Cardoso Jr., Laercio
Leao, Emilia Katiane Embirucu de Araujo
Esposito, Ana Carolina
Kyosen, Sandra Obikawa [UNIFESP]
Souza, Carolina Fischinger Moura de
Martins, Ana Maria [UNIFESP]
dc.subject.por.fl_str_mv Enzyme replacement therapy
alpha-L-iduronidase deficiency
Clinical outcomes
Tolerability
topic Enzyme replacement therapy
alpha-L-iduronidase deficiency
Clinical outcomes
Tolerability
description 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.
publishDate 2016
dc.date.none.fl_str_mv 2016
2020-07-22T13:23:06Z
2020-07-22T13:23:06Z
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://dx.doi.org/10.1186/s13023-016-0437-8
Orphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016.
10.1186/s13023-016-0437-8
WOS000375109300001.pdf
1750-1172
https://repositorio.unifesp.br/handle/11600/56035
WOS:000375109300001
url http://dx.doi.org/10.1186/s13023-016-0437-8
https://repositorio.unifesp.br/handle/11600/56035
identifier_str_mv Orphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016.
10.1186/s13023-016-0437-8
WOS000375109300001.pdf
1750-1172
WOS:000375109300001
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Orphanet Journal Of Rare Diseases
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv -
application/pdf
dc.coverage.none.fl_str_mv London
dc.publisher.none.fl_str_mv Biomed Central Ltd
publisher.none.fl_str_mv Biomed Central Ltd
dc.source.none.fl_str_mv reponame:Repositório Institucional da UNIFESP
instname:Universidade Federal de São Paulo (UNIFESP)
instacron:UNIFESP
instname_str Universidade Federal de São Paulo (UNIFESP)
instacron_str UNIFESP
institution UNIFESP
reponame_str Repositório Institucional da UNIFESP
collection Repositório Institucional da UNIFESP
repository.name.fl_str_mv Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)
repository.mail.fl_str_mv biblioteca.csp@unifesp.br
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