Individualised low-dose alglucerase therapy for type 1 Gaucher's disease

Previous studies have shown that enzyme supplementation therapy with alglucerase for type 1 Gaucher's disease is effective at doses of 30-130 U/kg per month. Since both the clinical presentation and the response to therapy in Gaucher's disease are highly variable, individual dosing seems i...

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Bibliographic Details
Published in:The Lancet (British edition) 1995-06, Vol.345 (8963), p.1474-1478
Main Authors: Hollack, C.E.M., Goudsmith, R., Ek, M., von dem Borne, A.E.R.Kr, van Oers, M.H.J., Aerts, J.M.F.G., van Meely, S., Phoa, S.S.K.S.
Format: Article
Language:English
Subjects:
Adult
Aged
Biological and medical sciences
Disease
Drug Administration Schedule
Drug therapy
Enzymes
Errors of metabolism
Female
Gaucher Disease - blood
Gaucher Disease - classification
Gaucher Disease - drug therapy
Glucosylceramidase - administration & dosage
Humans
Infusions, Intravenous
Lipids (lysosomal enzyme disorders, storage diseases)
Liver - anatomy & histology
Liver - drug effects
Male
Medical research
Medical sciences
Metabolic diseases
Middle Aged
Platelet Count - drug effects
Spleen - anatomy & histology
Spleen - drug effects
Treatment Outcome
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Summary:Previous studies have shown that enzyme supplementation therapy with alglucerase for type 1 Gaucher's disease is effective at doses of 30-130 U/kg per month. Since both the clinical presentation and the response to therapy in Gaucher's disease are highly variable, individual dosing seems indicated. This notion, as well as the high costs of alglucerase and the unknown long-term side-effects, led us to investigate the efficacy of an individualised very low dose of alglucerase. Twenty-five adults with symptomatic type 1 Gaucher's disease (thirteen splenectomised) received alglucerase 1·15 U/kg three times a week (15 U/kg per month). Every 6 months, the dose was halved, maintained, or doubled, according to the response (based on haematological variables and liver and spleen volume). After 6 months of treatment, eighteen (72%) patients had a response (seventeen moderate, one good). After 12 months (in nineteen patients) and 18 months (in seven patients), all had sustained improvement. Severe splenomegaly resulted in slower haematological responses. Our results are similar to those obtained by others with higher-dose regimens and better than a low-dose regimen of 10 U/kg every 2 weeks. We conclude that very low initial doses of alglucerase, when administered frequently, are effective and cost-saving in the treatment of type 1 Gaucher's disease.
ISSN:0140-6736
1474-547X
DOI:10.1016/S0140-6736(95)91037-9