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

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

Author Horovitz, Dafne Dain Gandelman Google Scholar
Acosta, Angelina X. Google Scholar
Giugliani, Roberto Google Scholar
Hlavata, Anna Google Scholar
Hlavata, Katarina Google Scholar
Tchan, Michel C. Google Scholar
Barth, Anneliese Lopes Google Scholar
Cardoso Jr., Laercio Google Scholar
Leao, Emilia Katiane Embirucu de Araujo Google Scholar
Esposito, Ana Carolina Google Scholar
Kyosen, Sandra Obikawa Autor UNIFESP Google Scholar
Souza, Carolina Fischinger Moura de Google Scholar
Martins, Ana Maria Autor UNIFESP Google Scholar
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 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.
Keywords Enzyme replacement therapy
alpha-L-iduronidase deficiency
Clinical outcomes
xmlui.dri2xhtml.METS-1.0.item-coverage London
Language English
Sponsor Sanofi Genzyme
Sanofi Genzyme, Cambridge, MA, USA
Date 2016
Published in Orphanet Journal Of Rare Diseases. London, v. 11, p. -, 2016.
ISSN 1750-1172 (Sherpa/Romeo, impact factor)
Publisher Biomed Central Ltd
Extent -
Access rights Open access Open Access
Type Article
Web of Science ID WOS:000375109300001

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