{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,5]],"date-time":"2026-06-05T23:05:11Z","timestamp":1780700711705,"version":"3.54.1"},"reference-count":17,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2017,12,14]],"date-time":"2017-12-14T00:00:00Z","timestamp":1513209600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","award":["G00423"],"award-info":[{"award-number":["G00423"]}],"id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Acta Ophthalmologica"],"published-print":{"date-parts":[[2018,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Purpose<\/jats:title><jats:p>To determine whether the ocular phenotype in patients with mucopolysaccharidosis type I (MPSI) Hurler is affected by the efficacy of previous haemopoietic stem cell transplantation (HSCT).<\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p>A retrospective cohort study of patients with MPSI who had undergone treatment with HSCT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Ocular phenotype was documented for each patient and compared to levels of biomarkers representing efficacy of previous transplantation. Main outcome measures: Assessment of visual acuity (VA), severity of corneal clouding and the presence of optic neuropathy or retinopathy. Biomarker assessment included dermatan sulphate\/chondroitin sulphate (DS\/CS) ratio and iduronidase enzyme level.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Severe corneal clouding was significantly greater in patients with lower iduronidase levels (p\u00a0=\u00a00.023) and raised DS\/CS ratio (<jats:italic>R<\/jats:italic><jats:sup>2<\/jats:sup>\u00a0=\u00a00.28 p\u00a0=\u00a00.043). Better VA was related to a higher iduronidase levels (<jats:italic>R<\/jats:italic><jats:sup>2<\/jats:sup>\u00a0=\u00a00.15, p\u00a0=\u00a00.004) and lower DS\/CS ratio (<jats:italic>R<\/jats:italic><jats:sup>2<\/jats:sup>\u00a0=\u00a00.38, p\u00a0=\u00a00.001).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Improved ocular phenotypes in MPSI are associated with markers signifying efficacy of prior transplant. Early and effective HSCT may result in a better visual prognosis and reduction in ocular complications for patients with MPSI.<\/jats:p><\/jats:sec>","DOI":"10.1111\/aos.13627","type":"journal-article","created":{"date-parts":[[2017,12,14]],"date-time":"2017-12-14T13:46:51Z","timestamp":1513259211000},"page":"494-498","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler)"],"prefix":"10.1111","volume":"96","author":[{"given":"Ahmed","family":"Javed","sequence":"first","affiliation":[{"name":"Manchester Royal Eye Hospital Manchester Academic Health Science Centre Manchester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tariq","family":"Aslam","sequence":"additional","affiliation":[{"name":"Manchester Royal Eye Hospital Manchester Academic Health Science Centre Manchester 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