{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T23:21:17Z","timestamp":1790464877382,"version":"4.1.0"},"reference-count":24,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2017,5,2]],"date-time":"2017-05-02T00:00:00Z","timestamp":1493683200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100006396","name":"Alexion Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006396","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003478","name":"Ministry of Health, Labour and Welfare","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003478","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Clinical Endocrinology"],"published-print":{"date-parts":[[2017,7]]},"abstract":"<jats:title>Summary<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Hypophosphatasia (<jats:styled-content style=\"fixed-case\">HPP<\/jats:styled-content>) is a rare skeletal disease characterized by hypomineralization and low alkaline phosphatase activity. Asfotase alfa (<jats:styled-content style=\"fixed-case\">AA<\/jats:styled-content>) has been recently developed to treat <jats:styled-content style=\"fixed-case\">HPP<\/jats:styled-content> complications. This study evaluated its safety and efficacy in Japan.<\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p>Open\u2010label, multicentre, prospective trial. Patients were enrolled in 11 hospitals from June 2014 to July 2015.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients<\/jats:title><jats:p>Thirteen patients (9 females, 4 males) ages 0\u00a0days to 34\u00a0years at baseline were enrolled and treated with <jats:styled-content style=\"fixed-case\">AA<\/jats:styled-content> (2\u00a0mg\/kg three times weekly subcutaneously in all but one patient). All had <jats:italic><jats:styled-content style=\"fixed-case\">ALPL<\/jats:styled-content><\/jats:italic> gene mutations. <jats:styled-content style=\"fixed-case\">HPP<\/jats:styled-content> forms were perinatal (n=6), infantile (n=5), childhood (n=1) and adult (n=1).<\/jats:p><\/jats:sec><jats:sec><jats:title>Measurements<\/jats:title><jats:p>Safety determined from adverse events (<jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s) and laboratory data was the primary outcome measure. Efficacy was assessed as a secondary outcome measure from overall survival, respiratory status, rickets severity and gross motor development.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Injection site reactions were the most frequent <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s. Serious <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s possibly related to treatment were convulsion and hypocalcaemia observed in a patient with the perinatal form. In addition, hypercalcaemia and\/or hyperphosphatemia was observed in three patients with the infantile form and a low\u2010calcium and\/or low\u2010phosphate formula was given to these patients. With respect to efficacy, all patients survived and the radiographic findings, developmental milestones and respiratory function improved.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Asfotase alfa therapy improved skeletal, respiratory and physical symptoms with a few serious <jats:styled-content style=\"fixed-case\">AE<\/jats:styled-content>s in patients with <jats:styled-content style=\"fixed-case\">HPP<\/jats:styled-content>. Our results add support to the safety and efficacy of <jats:styled-content style=\"fixed-case\">AA<\/jats:styled-content> therapy for <jats:styled-content style=\"fixed-case\">HPP<\/jats:styled-content> patients.<\/jats:p><\/jats:sec>","DOI":"10.1111\/cen.13343","type":"journal-article","created":{"date-parts":[[2017,4,5]],"date-time":"2017-04-05T15:33:20Z","timestamp":1491406400000},"page":"10-19","source":"Crossref","is-referenced-by-count":66,"title":["Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial"],"prefix":"10.1111","volume":"87","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3531-884X","authenticated-orcid":false,"given":"Taichi","family":"Kitaoka","sequence":"first","affiliation":[{"name":"Department of Pediatrics Osaka University Graduate School of Medicine  Osaka 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