Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
Autor(a) principal: | |
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Data de Publicação: | 2016 |
Outros Autores: | , , , , , , , , , , , |
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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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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1814268309294546944 |