Replacement therapy with imiglucerase for type 1 Gaucher's disease

Zimran, Ari; Elstein, Deborah; Levy-Lahad, E; Zevin, Shoshana; Hadas‐Halpern, Irith; Abrahamov, Ayala; Bar-Ziv, Y; Schwartz, Alexander; Földes, J

doi:10.1016/s0140-6736(95)91038-7

Cited by 105 publications

(62 citation statements)

References 8 publications

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“…Lower scores imply less severity (see Table 2 for further details). Many studies 55,71,72,74,77,93,94,96,100,101,120,121 report the SSI for patients at baseline, but only Alfonso 70 and Caubel 89 report on SSI before and after ERT; both studies used the early SSI version, 16 which included age at diagnosis as a scoring factor. Caubel 89 also compared this result with that using their own alternative clinical global score.…”

Section: Effects Of Ert On Global Scores Of Disease Severitymentioning

confidence: 99%

The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review

Connock¹,

Burls²,

Frew³

et al. 2006

Health Technol Assess

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Section: Effects Of Ert On Global Scores Of Disease Severitymentioning

confidence: 99%

The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review

Connock¹,

Burls²,

Frew³

et al. 2006

Health Technol Assess

View full text Add to dashboard Cite

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“…This recombinant, infusible enzyme is produced in Chinese hamster ovary cells and differs from the wild-type human enzyme sequence by a single amino acid substitution at position 495 [3,4].…”

Section: Introductionmentioning

confidence: 99%

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

et al. 2013

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Type 1 Gaucher disease (GD1), resulting from glucocerebrosidase deficiency, leads to splenomegaly, hepatomegaly, anemia, thrombocytopenia, and bone involvement. Current standard treatment is enzyme replacement therapy. Velaglucerase alfa is an enzyme replacement product for GD1, with the same amino acid sequence as naturally occurring human glucocerebrosidase. This multinational, Phase 3 trial evaluated the efficacy and safety of two doses of velaglucerase alfa in 25 treatment‐naïve, anemic patients with GD1 (4–62 years of age), randomized to intravenous velaglucerase alfa 60 U/kg (n=12) or 45 U/kg body weight (n=13) every other week for 12 months. The primary endpoint was change from baseline in hemoglobin concentration in the 60 U/kg arm. At 12 months, mean hemoglobin concentrations increased from baseline [60 U/kg: +23.3%; +2.43 g/dL (P<0.001); 45 U/kg: +23.8%; +2.44 g/dL (P<0.001)], as did mean platelet counts [60 U/kg: +65.9%; +50.9 × 109/L (P=0.002); 45 U/kg: +66.4%; +40.9 × 109/L(P=0.01)]. Mean splenic volume decreased from baseline [60 U/kg: −50.4%, from 14.0 to 5.8 multiples of normal (MN) (P=0.003); 45 U/kg: −39.9%, from 14.5 to 9.5 MN (P=0.009)]. No drug‐related serious adverse events or withdrawals were observed. One patient developed antibodies. Velaglucerase alfa was generally well tolerated and effective for adults and children with GD1 in this study. All disease‐specific parameters measured demonstrated clinically meaningful improvements after 12 months. Am. J. Hematol. 88:166–171, 2013. © 2012 Wiley Periodicals, Inc.

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“…Imiglucerase (Cerezyme; Genzyme), a recombinant analogue of β-glucocerebrosidase produced in Chinese hamster ovary cells 2,4 , was approved by the FDA in 1994 and largely replaced alglucerase. In addition, miglustat (Zavesca; Actelion), a small-molecule drug that inhibits the synthesis of sphingolipids, such as glucocerebroside 1 , was approved by the FDA in 2003 for patients with mild to moderate type 1 Gaucher's disease for whom ERT is not an option.…”

Section: Basis Of Discoverymentioning

confidence: 99%

“…In addition, the overall N-linked glycan composition of the two products differ, with velaglucerase alfa largely having natural high-mannose-type oligosaccharides, whereas the glycans in imiglucerase have been enzymatically trimmed to (man) 3 (Glcnac) 2 structures 5 . Despite these differences, clinical studies have so far not shown major differences between the two enzymes with respect to pharmacokinetics and efficacy 4,8,10 . In addition, a recent study that systematically compared the two enzymes in a mouse model of Gaucher's disease concluded that both enzymes show similar therapeutic effects at several different doses and at different ages, with differing degrees of disease involvement 11 .…”

Section: Indicationsmentioning

confidence: 99%