{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,9]],"date-time":"2025-10-09T13:08:26Z","timestamp":1760015306361,"version":"3.37.3"},"reference-count":23,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2018,7,31]],"date-time":"2018-07-31T00:00:00Z","timestamp":1532995200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2018,7,31]],"date-time":"2018-07-31T00:00:00Z","timestamp":1532995200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"Protalix BioPharmaceutics"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2018,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Preliminary data suggest a positive effect of taliglucerase alfa on the bone marrow infiltration of Gaucher cells. In this investigator\u2010initiated study, we report the impact of taliglucerase alfa on the bone marrow fat fraction (FF) in 26 patients assessed by quantitative chemical shift imaging (QCSI). Of 15 treatment\u2010na\u00efve patients (median age 48 [range 24\u201368] years), eight had baseline FF\u2009\u2264\u20090.3, six of those with a FF\u2009\u2264\u20090.23 (\u2018bone at risk\u2019). All significantly improved from a median baseline FF of 0.24 (0.15\u20130.32) to 1st year FF of 0.37 (0.25\u20130.54) and 2nd year FF of 0.42 (0.27\u20130.59) (<jats:italic>p<\/jats:italic>\u2009=\u20090.01). Among the 11 \u2018switch\u2010over\u2019 patients (median age 42 [range 33\u201369] years; median imiglucerase exposure 8 [range 1\u201317] years), eight had baseline FF\u2009\u2264\u20090.3, five of those with FF\u2009&lt;\u20090.23. All, but one, significantly improved from a median baseline FF of 0.17 (0.08\u20130.28) to 1st year FF of 0.3 (0.05\u20130.34) and 2nd year FF of 0.34 (0.08\u20130.44) (<jats:italic>p<\/jats:italic>\u2009=\u20090.03). Two elderly female patients (age 43 and 58 years, with 17 years imiglucerase exposure) who remained at the same enzyme replacement therapy dose, increased from baseline FF of 0.13 and 0.19 to 0.26 at 1 year. Although the number of observations is small, we hypothesize that switching to taliglucerase may result in an improved bone marrow response. A larger study is needed to assess the early benefit of taliglucerase alfa in adult patients with type 1 Gaucher disease on the bone marrow compartment.<\/jats:p>","DOI":"10.1007\/s10545-018-0195-y","type":"journal-article","created":{"date-parts":[[2018,7,31]],"date-time":"2018-07-31T20:58:32Z","timestamp":1533070712000},"page":"1259-1265","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Improvement in bone marrow infiltration in patients with type I Gaucher disease treated with taliglucerase alfa"],"prefix":"10.1002","volume":"41","author":[{"given":"Ari","family":"Zimran","sequence":"first","affiliation":[{"name":"Gaucher Clinic Shaare Zedek Medical Center, affiliated with Hebrew\u2010University Medical School Jerusalem Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tama","family":"Dinur","sequence":"additional","affiliation":[{"name":"Gaucher Clinic Shaare Zedek Medical Center, affiliated with Hebrew\u2010University Medical School Jerusalem Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9151-0337","authenticated-orcid":false,"given":"Shoshana","family":"Revel\u2010Vilk","sequence":"additional","affiliation":[{"name":"Gaucher Clinic Shaare Zedek Medical Center, affiliated with Hebrew\u2010University Medical School Jerusalem Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eric M.","family":"Akkerman","sequence":"additional","affiliation":[{"name":"Academic Medical Centre Amsterdam Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura","family":"van Dussen","sequence":"additional","affiliation":[{"name":"Academic Medical Centre Amsterdam Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carla E. 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