{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,9]],"date-time":"2026-05-09T04:30:08Z","timestamp":1778301008677,"version":"3.51.4"},"reference-count":38,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2018,3,30]],"date-time":"2018-03-30T00:00:00Z","timestamp":1522368000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Foundation for the Promotion of Health and Biomedical Research of Valencia, FISABIO","award":["UGP-15-222"],"award-info":[{"award-number":["UGP-15-222"]}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Basic Clin Pharma Tox"],"published-print":{"date-parts":[[2018,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>The quantification of enzyme activity in the patient treated with enzyme replacement therapy (<jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>) has been suggested as a tool for dosage individualization, so we conducted a study to evaluate the relationship between glucocerebrosidase activity and clinical response in patients with Gaucher disease type I (<jats:styled-content style=\"fixed-case\">GD<\/jats:styled-content>1) to <jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>. The study included patients diagnosed with <jats:styled-content style=\"fixed-case\">GD<\/jats:styled-content>1, who were being treated with <jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>, and healthy individuals. Markers based on glucocerebrosidase activity measurement in patients\u2019 leucocytes were studied: enzyme activity at 15 min. post\u2010infusion (Act<jats:sub>75<\/jats:sub>) reflects the amount of enzyme that is distributed in the body post\u2010<jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content> infusion, and accumulated glucocerebrosidase activity during <jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content> infusion (Act<jats:sub>75\u20100<\/jats:sub>) indicates the total drug exposure during infusion. The clinical response was evaluated based on criteria established by Pastores <jats:italic>et al<\/jats:italic>. and Gaucher Severity Score Index. Statistical analysis included <jats:styled-content style=\"fixed-case\">ROC<\/jats:styled-content> analysis and area under the curve test. Act<jats:sub>75<\/jats:sub> and Act<jats:sub>75\u20100<\/jats:sub> were found to be moderate predictive markers of an optimal clinical response (area under the <jats:styled-content style=\"fixed-case\">ROC<\/jats:styled-content> of Act<jats:sub>75<\/jats:sub> was 0.733 and Act<jats:sub>75\u20100<\/jats:sub> was 0.817). Act<jats:sub>75\u20100<\/jats:sub> showed statistical significance in its discriminative capacity (<jats:italic>p<\/jats:italic> &lt; 0.05) for obtaining an optimal response to <jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>. The cut\u2010off point was 58% (<jats:styled-content style=\"fixed-case\">RR<\/jats:styled-content> = 1.800; 95% CI: 1.003\u20133.229; <jats:italic>p<\/jats:italic> &lt; 0.05). Moreover, Act<jats:sub>75<\/jats:sub> showed a significant and inverse correlation with the Gaucher Severity Score Index, and Act<jats:sub>75<\/jats:sub> and Act<jats:sub>75\u20100<\/jats:sub> presented a significant correlation with residual enzyme activity at diagnosis. Markers based on glucocerebrosidase activity have a good correlation with clinical response to <jats:styled-content style=\"fixed-case\">ERT<\/jats:styled-content>. Therefore, it could provide supporting clinical data for dose management in <jats:styled-content style=\"fixed-case\">GD<\/jats:styled-content>1 patients.<\/jats:p>","DOI":"10.1111\/bcpt.12977","type":"journal-article","created":{"date-parts":[[2018,2,8]],"date-time":"2018-02-08T10:52:39Z","timestamp":1518087159000},"page":"65-71","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Relationship Between Glucocerebrosidase Activity and Clinical Response to Enzyme Replacement Therapy in Patients With Gaucher Disease Type I"],"prefix":"10.1111","volume":"123","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2148-3163","authenticated-orcid":false,"given":"Elena","family":"Gras\u2010Colomer","sequence":"first","affiliation":[{"name":"Department of Pharmacy University Hospital Doctor Peset of Valencia Valencia Spain"},{"name":"Foundation for the Promotion of Health and Biomedical Research of Valencia (FISABIO) Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mar\u00eda\u2010Amparo","family":"Mart\u00ednez\u2010G\u00f3mez","sequence":"additional","affiliation":[{"name":"Department of Pharmacy University Hospital Doctor Peset of Valencia Valencia Spain"},{"name":"Foundation for the Promotion of Health and Biomedical Research of Valencia (FISABIO) Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M\u00f3nica","family":"Climente\u2010Mart\u00ed","sequence":"additional","affiliation":[{"name":"Department of Pharmacy University Hospital Doctor Peset of Valencia Valencia Spain"},{"name":"Pharmacy and Pharmaceutical Technology University of Valencia Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel","family":"Fernandez\u2010Zarzoso","sequence":"additional","affiliation":[{"name":"Department of Hematology University Hospital Doctor Peset of Valencia Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mercedes","family":"Almela\u2010Tejedo","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Hospital Virgen de los Lirios of Alcoi Alcoi Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vicente","family":"Giner\u2010Galva\u00f1","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Hospital Virgen de los Lirios of Alcoi Alcoi Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jose\u2010Antonio","family":"Marcos\u2010Rodr\u00edguez","sequence":"additional","affiliation":[{"name":"Department of Pharmacy University Hospital Virgen Macarena Seville Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alicia","family":"Rodr\u00edguez\u2010Fern\u00e1ndez","sequence":"additional","affiliation":[{"name":"Department of Hematology University Hospital Virgen Macarena Seville Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel\u2010\u00c1ngel","family":"Torralba\u2010Cabeza","sequence":"additional","affiliation":[{"name":"Deparment of Internal Medicine University Clinical Hospital Lozano Blesa of Zaragoza Zaragoza Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matilde","family":"Merino\u2010Sanjuan","sequence":"additional","affiliation":[{"name":"Pharmacy and Pharmaceutical Technology University of Valencia Valencia Spain"},{"name":"Molecular Recognition and Technological Development Institute Mixed Unit Polytechnic University of Valencia University of Valencia Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2018,3,30]]},"reference":[{"key":"e_1_2_8_2_1","volume-title":"Gaucher Disease. 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