Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients
dc.contributor.author | Giugliani, Roberto | pt_BR |
dc.contributor.author | Harmatz, Paul | pt_BR |
dc.contributor.author | Jones, Simon A. | pt_BR |
dc.contributor.author | Mendelsohn, Nancy J. | pt_BR |
dc.contributor.author | Vellodi, Ashok | pt_BR |
dc.contributor.author | Qiu, Yongchang | pt_BR |
dc.contributor.author | Hendriksz, Christian J. | pt_BR |
dc.contributor.author | Vijayaraghavan, Suresh | pt_BR |
dc.contributor.author | Whiteman, David A.H. | pt_BR |
dc.contributor.author | Pano, Arian | pt_BR |
dc.date.accessioned | 2019-06-22T02:34:52Z | pt_BR |
dc.date.issued | 2017 | pt_BR |
dc.identifier.issn | 2214-4269 | pt_BR |
dc.identifier.uri | http://hdl.handle.net/10183/196044 | pt_BR |
dc.description.abstract | Objectives: This 109-week, nonrandomized, observational study ofmucopolysaccharidosis II (MPS II) patients already enrolled in the Hunter Outcome Survey (HOS) (NCT00882921), assessed the long-termimmunogenicity of idursulfase, and examined the effect of idursulfase-specific antibody generation on treatment safety (via infusion- related adverse events [IRAEs]) and pharmacodynamics (via urinary glycosaminoglycans [uGAGs]). Methods: Male patients ≥5 years, enrolled in HOS regardless of idursulfase treatment status were eligible. Blood/ urine samples for anti-idursulfase antibody testing and uGAG measurement were collected every 12 weeks. Results: Due to difficulties in enrolling treatment-naïve patients, data collection was limited to 26 enrolled patients of 100 planned patients (aged 5.1–35.5 years) all ofwhomwere non-naïve to treatment. Fifteen (58%) patients completed the study. There were 11/26 (42%) seropositive patients at baseline (Ab+), and 2/26 (8%) others developed intermittent seropositivity by Week 13. A total of 9/26 patients (35%) had ≥1 sample positive for neutralizing antibodies. Baseline uGAG levels were lowdue to prior idursulfase treatment and did not change appreciably thereafter. Ab+patients had persistently higher uGAG levels at entry and throughout the study than Ab− patients. Nine of 26 (34%) patients reported IRAEs. Ab+ patients appeared to have a higher risk of developing IRAEs than Ab−patients.However, the relative risk was not statistically significant and decreased after adjustment for age. Conclusions: 50% of study patients developed idursulfase antibodies. Notably Ab+ patients had persistently higher average uGAG levels. A clear association between IRAEs and antibodies was not established. | en |
dc.format.mimetype | application/pdf | pt_BR |
dc.language.iso | eng | pt_BR |
dc.relation.ispartof | Molecular genetics and metabolism reports. Amsterdam. vol. 12 (Sept. 2017), p. 2-7 | pt_BR |
dc.rights | Open Access | en |
dc.subject | Neutralizing antibodies | en |
dc.subject | Mucopolissacaridose II | pt_BR |
dc.subject | Idursulfase | en |
dc.subject | Terapia de reposição de enzimas | pt_BR |
dc.subject | Hunter syndrome | en |
dc.subject | Enzyme replacement therapy | en |
dc.subject | Cognitive impairment | en |
dc.subject | Immunogenicity | en |
dc.subject | Glycosaminoglycans | en |
dc.title | Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients | pt_BR |
dc.type | Artigo de periódico | pt_BR |
dc.identifier.nrb | 001089830 | pt_BR |
dc.type.origin | Estrangeiro | pt_BR |
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