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Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I.
J Pediatr. 2016 Nov; 178:219-226.e1.JPed

Abstract

OBJECTIVE

To evaluate long-term outcomes of laronidase enzyme replacement therapy in patients with attenuated mucopolysaccharidosis type I.

STUDY DESIGN

Retrospective analyses of case notes, laboratory results, and data from clinical trials were used to evaluate urinary glycosaminoglycans, forced vital capacity (FVC), 6-minute walk test (6MWT), height-for-age Z score, cardiac valve function, corneal clouding, and visual acuity in 35 patients with attenuated mucopolysaccharidosis type I (Hurler-Scheie and Scheie syndromes) for up to 10 years following the initiation of laronidase therapy.

RESULTS

Statistically significant (P < .001) reductions in mean urinary glycosaminoglycan levels relative to baseline were observed 6 months after treatment initiation and were sustained throughout follow-up. Disease remained stable after treatment initiation with no statistically significant changes in mean FVC, 6MWT, or height-for-age Z score. At last assessments, mitral and aortic valve function remained stable in 65% (22/34) of patients; corneal clouding remained stable in 78% (18/23); visual acuity remained stable in 33% (8/24) and improved in 42% (10/24) of patients. Younger patients (<10 years at treatment initiation) maintained disease measures closer to norms for age for FVC, 6MWT, and height and showed fewer deteriorations in mitral and aortic valve disease and corneal clouding compared with patients aged ≥10 years at treatment initiation.

CONCLUSION

Laronidase treatment resulted in disease stabilization in the majority of patients with a mean follow-up of 6.1 years. Data suggest that early treatment may result in better outcomes.

Authors+Show Affiliations

Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK. Electronic address: sarah.laraway@elht.nhs.uk.Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK.Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK.Manchester Royal Eye Hospital, Central Manchester University Hospitals National Health Service Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK; Centre for Ophthalmology and Vision Sciences, Institute of Human Development, Faculty of Medical and Human Sciences, University of Manchester, Manchester, UK.Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK.Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

27788836

Citation

Laraway, Sarah, et al. "Outcomes of Long-Term Treatment With Laronidase in Patients With Mucopolysaccharidosis Type I." The Journal of Pediatrics, vol. 178, 2016, pp. 219-226.e1.
Laraway S, Mercer J, Jameson E, et al. Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I. J Pediatr. 2016;178:219-226.e1.
Laraway, S., Mercer, J., Jameson, E., Ashworth, J., Hensman, P., & Jones, S. A. (2016). Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I. The Journal of Pediatrics, 178, 219-e1. https://doi.org/10.1016/j.jpeds.2016.08.033
Laraway S, et al. Outcomes of Long-Term Treatment With Laronidase in Patients With Mucopolysaccharidosis Type I. J Pediatr. 2016;178:219-226.e1. PubMed PMID: 27788836.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I. AU - Laraway,Sarah, AU - Mercer,Jean, AU - Jameson,Elisabeth, AU - Ashworth,Jane, AU - Hensman,Pauline, AU - Jones,Simon A, PY - 2016/03/11/received PY - 2016/06/20/revised PY - 2016/08/09/accepted PY - 2016/10/30/pubmed PY - 2017/5/18/medline PY - 2016/10/30/entrez KW - MPS I KW - enzyme replacement therapy KW - laronidase KW - mucopolysaccharidosis type I SP - 219 EP - 226.e1 JF - The Journal of pediatrics JO - J. Pediatr. VL - 178 N2 - OBJECTIVE: To evaluate long-term outcomes of laronidase enzyme replacement therapy in patients with attenuated mucopolysaccharidosis type I. STUDY DESIGN: Retrospective analyses of case notes, laboratory results, and data from clinical trials were used to evaluate urinary glycosaminoglycans, forced vital capacity (FVC), 6-minute walk test (6MWT), height-for-age Z score, cardiac valve function, corneal clouding, and visual acuity in 35 patients with attenuated mucopolysaccharidosis type I (Hurler-Scheie and Scheie syndromes) for up to 10 years following the initiation of laronidase therapy. RESULTS: Statistically significant (P < .001) reductions in mean urinary glycosaminoglycan levels relative to baseline were observed 6 months after treatment initiation and were sustained throughout follow-up. Disease remained stable after treatment initiation with no statistically significant changes in mean FVC, 6MWT, or height-for-age Z score. At last assessments, mitral and aortic valve function remained stable in 65% (22/34) of patients; corneal clouding remained stable in 78% (18/23); visual acuity remained stable in 33% (8/24) and improved in 42% (10/24) of patients. Younger patients (<10 years at treatment initiation) maintained disease measures closer to norms for age for FVC, 6MWT, and height and showed fewer deteriorations in mitral and aortic valve disease and corneal clouding compared with patients aged ≥10 years at treatment initiation. CONCLUSION: Laronidase treatment resulted in disease stabilization in the majority of patients with a mean follow-up of 6.1 years. Data suggest that early treatment may result in better outcomes. SN - 1097-6833 UR - https://www.unboundmedicine.com/medline/citation/27788836/Outcomes_of_Long_Term_Treatment_with_Laronidase_in_Patients_with_Mucopolysaccharidosis_Type_I_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0022-3476(16)30700-4 DB - PRIME DP - Unbound Medicine ER -