Low-dose enzyme replacement therapy for Gaucher's disease: Effects of age, sex, genotype, and clinical features on response to treatment

Zimran, Ari; Elstein, Deborah; Kannai, Ruth; Zevin, Shoshana; Hadas‐Halpern, Irith; Levy‐Lahad, Ephrat; Cohen, Yaël; Horowitz, Mia; Abrahamov, Ayala

doi:10.1016/0002-9343(94)90042-6

Cited by 81 publications

(50 citation statements)

References 16 publications

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“…Although we have not seen any effect of genotype on response to enzyme therapy [13], the analysis of genotype based on the full ICGG cohort may elucidate the apparent conundrum where some investigators have recommended higher doses in patients ''resistant'' to lower doses [14] although these same authors have not found a real difference in clinically important disease parameters by virtue of higher doses [15]. More than a decade ago, Beutler [16] maintained that there are patients who will respond poorly to any dose while others will respond well regardless of dose.…”

contrasting

confidence: 60%

Using the International Gaucher Disease Registry data: Can we devise a virtuous circle for treated patients?

Elstein

Zimran

2008

American J Hematol

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contrasting

confidence: 60%

Using the International Gaucher Disease Registry data: Can we devise a virtuous circle for treated patients?

Elstein

Zimran

2008

American J Hematol

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“…1,2 In accordance with previous studies, enzyme supplementation therapy was found to improve the nonmetabolic symptoms of the disease. [6][7][8][9][10][11] In addition, we established that hypermetabolism, reflected in increased REE in GD patients improves during enzyme supplementation therapy, although normalization was not achieved. This improvement of REE was associated with an increase in body weight.…”

Section: Discussionmentioning

confidence: 85%

“…Alglucerase has proven to be effective in improving the manifestations of GD such as hepatosplenomegaly and cytopenia. [6][7][8][9][10][11] It is presently unknown whether the metabolic abnormalities improve similarly in relation to the other symptoms. Therefore the aim of the present study was to evaluate the effect of enzyme supplementation therapy on metabolic parameters in relation to changes in hepatic and splenic volumes, hemoglobin level, and platelet count.…”

mentioning

confidence: 99%

Differential Effects of Enzyme Supplementation Therapy on Manifestations of Type 1 Gaucher Disease

Hollak

Corssmit

Aerts

et al. 1997

The American Journal of Medicine

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Differential effects of enzyme supplementation therapy on manifestations of type 1 gaucher disease Hollak, C.E.M.; Corssmit, E.P.M.; Aerts, J.M.F.G.; Endert, E.; Sauerwein, H.P.; Romijn, J.A.; van Oers, M.H.J. Disclaimer/Complaints regulationsIf you believe that digital publication of certain material infringes any of your rights or (privacy) interests, please let the Library know, stating your reasons. In case of a legitimate complaint, the Library will make the material inaccessible and/or remove it from the website. Please Ask the Library: http://uba.uva.nl/en/contact, or a letter to: Library of the University of Amsterdam, Secretariat, Singel 425, 1012 WP Amsterdam, The Netherlands. You will be contacted as soon as possible. ) and hemoglobin and platelet count were obtained before and after 6 months of alglucerase therapy (15 U/kg per month). In 7 of the 12 patients hepatic glucose production was measured by infusing 3-3 H glucose. For comparison, REE and glucose metabolism were studied in 7 weight-and age-matched healthy subjects.RESULTS: REE and glucose production were increased in GD patients as compared with controls (REE: 29.8 kcal/kg/24 h { 3.6 and 23.1 { 2.3 kcal/kg/24 h, respectively, P õ 0.05; glucose production: 14.00 mmol/kg/min { 0.51 and 10.77 mmol/kg/min { 0.26, respectively, P õ 0.03). There were no differences in plasma glucose concentrations. Whereas the elevated REE decreased after 6 months of alglucerase therapy from 129% to 120% of predicted values (P õ 0.01), the increase in hepatic glucose production did not

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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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Low-dose enzyme replacement therapy for Gaucher's disease: Effects of age, sex, genotype, and clinical features on response to treatment

Cited by 81 publications

References 16 publications

Using the International Gaucher Disease Registry data: Can we devise a virtuous circle for treated patients?

Using the International Gaucher Disease Registry data: Can we devise a virtuous circle for treated patients?

Differential Effects of Enzyme Supplementation Therapy on Manifestations of Type 1 Gaucher Disease

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

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