{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,15]],"date-time":"2026-03-15T04:51:34Z","timestamp":1773550294837,"version":"3.50.1"},"reference-count":16,"publisher":"MDPI AG","issue":"18","license":[{"start":{"date-parts":[[2023,9,12]],"date-time":"2023-09-12T00:00:00Z","timestamp":1694476800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Pfizer"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Taliglucerase alfa is an enzyme replacement therapy approved for Gaucher disease. We assessed the duration\/compliance\/safety of such home infusions in commercial use in four countries where home infusion programs are available. The treatment duration\/compliance study included 173 patients (Israel, 58; US, 61; Brazil, 48; Australia, 6) who received \u22651 taliglucerase alfa home infusion through 6\/2021. The median age at home therapy initiation was 38 (range, 2\u201387) years; 58% were females. The median treatment duration (at home) was 2.7 (range, 0.04\u20139.0) years. The annual compliance rate was stable (\u226595%) throughout the study period. A search of the Pfizer global safety database (through 6\/2021), identified 19 adverse events (AEs) as related to \u201cdefinite home use\u201d and 14 to \u201cpossible home use\u201d of taliglucerase alfa; 42.4% of these AEs were serious; none were fatal. Twelve serious AEs in five separate case reports were considered treatment related: one case of chest discomfort\/pain and hypertension and one case of erythema associated with a toe blister, for which causality could not be excluded; pain in extremity; projectile vomiting and chills, alongside excessive eye blinking; and an infusion-related AE (pruritus). In conclusion, this real-life global study demonstrated that taliglucerase alfa home infusions are safe with high compliance rates.<\/jats:p>","DOI":"10.3390\/jcm12185913","type":"journal-article","created":{"date-parts":[[2023,9,12]],"date-time":"2023-09-12T03:54:06Z","timestamp":1694490846000},"page":"5913","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Real-World Experiences with Taliglucerase Alfa Home Infusions for Patients with Gaucher Disease: A Global Cohort Study"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9151-0337","authenticated-orcid":false,"given":"Shoshana","family":"Revel-Vilk","sequence":"first","affiliation":[{"name":"Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel"},{"name":"Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Royston","family":"Mansfield","sequence":"additional","affiliation":[{"name":"Pfizer R&D UK Ltd., Sandwich, Kent CT13 9NJ, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neta","family":"Feder-Krengel","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY 10001, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noya","family":"Machtiger-Azoulay","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY 10001, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David","family":"Kuter","sequence":"additional","affiliation":[{"name":"Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6783-2301","authenticated-orcid":false,"given":"Jeff","family":"Szer","sequence":"additional","affiliation":[{"name":"Clinical Haematology at Peter MacCallum Cancer Centre, The Royal Melbourne Hospital, Melbourne 3050, Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hanna","family":"Rosenbaum","sequence":"additional","affiliation":[{"name":"Hematology Day Care and Gaucher Clinic, The Center of Consulted Medicine, Clalit Services, Nazareth 1603701, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David Cavalcanti","family":"Ferreira","sequence":"additional","affiliation":[{"name":"Internal Medicine Department, Federal University of Santa Catarina, Florian\u00f3polis 88040-900, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-8725-2596","authenticated-orcid":false,"given":"Noa","family":"Ruhrman-Shahar","sequence":"additional","affiliation":[{"name":"Raphael Recanati Genetic Institute, Rabin Medical Center, Beilinson Hospital, Petach Tikva 4941492, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8211-9237","authenticated-orcid":false,"given":"Michael","family":"Wajnrajch","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY 10001, USA"},{"name":"Department of Pediatrics, Grossman School of Medicine, New York University, New York, NY 10016, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0077-8608","authenticated-orcid":false,"given":"Ari","family":"Zimran","sequence":"additional","affiliation":[{"name":"Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel"},{"name":"Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,9,12]]},"reference":[{"key":"ref_1","unstructured":"Revel-Vilk, S., Szer, J., and Zimran, A. 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Screen."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.hjdsi.2016.04.004","article-title":"Home infusion: Safe, clinically effective, patient preferred, and cost saving","volume":"5","author":"Polinski","year":"2017","journal-title":"Healthcare"},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"102478","DOI":"10.1016\/j.bcmd.2020.102478","article-title":"Direct and indirect effects of the sars-cov-2 pandemic on gaucher disease patients in spain: Time to reconsider home-based therapies?","volume":"85","author":"Giraldo","year":"2020","journal-title":"Blood Cells Mol. 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