{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T13:57:04Z","timestamp":1791899824855,"version":"4.3.4"},"reference-count":30,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2007,1,1]],"date-time":"2007-01-01T00:00:00Z","timestamp":1167609600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2007,1,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>The effect of ERT with imiglucerase on BMD in type 1 GD was studied using BMD data from the International Collaborative Gaucher Group Gaucher Registry. Data were analyzed for 160 untreated patients and 342 ERT-treated patients. Imiglucerase significantly improves BMD in patients with GD, with 8 years of ERT leading to normal BMD.<\/jats:p>\n               <jats:p>Introduction: The objective was to determine the effect of enzyme replacement therapy (ERT; Cerezyme, imiglucerase) on BMD in type 1 Gaucher disease (GD).<\/jats:p>\n               <jats:p>Materials and Methods: The study population included all adults (men, 18\u201370 years; women, 18\u201350 years) enrolled in the International Collaborative Gaucher Group (ICGG) Gaucher Registry for whom lumbar spine BMD measurements were available. BMD data with up to 8 years of follow-up were analyzed for 160 patients who received no ERT and 342 patients treated with ERT alone. BMD was assessed by DXA of the lumbar spine. Z scores for patients with GD were compared with a reference population. From the model's estimate, percent of patients by age and sex with osteoporosis (T score \u2a7d \u22122.5) were calculated.<\/jats:p>\n               <jats:p>Results: DXA Z scores for patients with GD in the no ERT (untreated) group were significantly below normal (y intercept = \u22120.80 Z score units, p &amp;lt; 0.001) and remained \u02dc1 SD below the reference population over time (slope = \u22120.010 Z score units per year, p = 0.68). The DXA Z scores for patients with GD who received ERT at a dose of 60 U\/kg\/2 weeks were significantly lower than the reference population at baseline (y-intercept = \u22121.17 Z score units, p &amp;lt; 0.001), but improved significantly over time (slope = +0.132 Z score units per year, p &amp;lt; 0.001). A significant dose\u2013response relationship was noted for the ERT group, with the slopes for the three main dosing groups of 15, 30, and 60 U\/kg\/2 weeks of +0.064, +0.086, and +0.132 Z score units per year, respectively. The BMD of patients with GD treated with ERT increased to \u22120.12 (60 U\/kg\/2 weeks), \u22120.48 (30 U\/kg\/2 weeks), and \u22120.66 (15 U\/kg\/2 weeks) SD of the mean of the reference population after 8 years of ERT, approaching the reference population. Estimated risk of osteoporosis of this GD population, if left untreated, ranged from \u02dc10 to 30% in women and 10% to 25% in men.<\/jats:p>\n               <jats:p>Conclusions: ERT with imiglucerase (Cerezyme) may increase BMD in patients with GD. Response to treatment with imiglucerase is slower for BMD than for hematologic and visceral aspects of GD. A normal (age- and sex-adjusted) BMD should be a therapeutic goal for patients with type 1 GD.<\/jats:p>","DOI":"10.1359\/jbmr.061004","type":"journal-article","created":{"date-parts":[[2006,10,10]],"date-time":"2006-10-10T13:43:11Z","timestamp":1160487791000},"page":"119-126","source":"Crossref","is-referenced-by-count":127,"title":["Effect of Enzyme Replacement Therapy With Imiglucerase on BMD in Type 1 Gaucher Disease"],"prefix":"10.1093","volume":"22","author":[{"given":"Richard J","family":"Wenstrup","sequence":"first","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children's Hospital Research Foundation, Cincinnati, Ohio, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine A","family":"Kacena","sequence":"additional","affiliation":[{"name":"Biostatistics, Genzyme Corporation, Cambridge, Massachusetts, 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