A Less Costly Regimen of Alglucerase to Treat Gaucher's Disease

Figueroa, Michael L.; Rosenbloom, Barry E.; Kay, Andrea; Garver, Paul; Thurston, Dennis; Koziol, James A.; Gelbart, Terri; Beutler, Ernest

doi:10.1056/nejm199212033272304

Cited by 139 publications

(44 citation statements)

References 14 publications

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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%

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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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Section: Discussionmentioning

confidence: 85%

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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“…34,[82][83][84] HCT is not currently regarded as first-line treatment because of the low morbidity of ERT. 85,86 This approach may change in type III disease as ERT results are closely scrutinized and if HCT techniques improve. 87 The only experimental indication for HCT at present is in type III children who deteriorate neurologically and/or have pulmonary compromise while on ERT.…”

Section: Fucosidosismentioning

confidence: 99%

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

Peters

Steward

2003

Bone Marrow Transplant

286

178

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Summary:For the past two decades, hematopoietic cell transplantation (HCT) has been used as effective therapy for selected inherited metabolic diseases (IMD) including Hurler (MPS IH) and Maroteaux-Lamy (MPS VI) syndromes, childhood-onset cerebral X-linked adrenoleukodystrophy (X-ALD), globoid-cell leukodystrophy (GLD), metachromatic leukodystrophy (MLD), a-mannosidosis, osteopetrosis, and others. Careful pre-HCT evaluation is critical and coordinated, multidisciplinary follow-up is essential in this field of transplantation. The primary goals of HCT for these disorders have been to promote long-term survival with donor-derived engraftment and to optimize the quality of life. Guidelines for HCT and monitoring are provided; a brief overview of long-term results is also presented.

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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%

“…Twelve data sets (in 11 included studies 61,[82][83][84]87,93,95,96,99,107,116 ) reported change in spleen volume during ERT in terms of degree of enlargement or provided sufficient data for the appropriate calculation to be made. The results are summarised in the scatterplot shown in Figure 4.…”

Section: Spleenmentioning

confidence: 99%

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

2007

Clinical Governance: An International Journal

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A Less Costly Regimen of Alglucerase to Treat Gaucher's Disease

Cited by 139 publications

References 14 publications

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

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

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

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

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A Less Costly Regimen of Alglucerase to Treat Gaucher's Disease

Cited by 139 publications

References 14 publications

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

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

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

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

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