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Enzyme replacement improves cardiomyopathy in Anderson-Fabry disease
Monday, 29 November 1999 19:00 | 
| Radiology News - Radiology Articles |
Enzyme replacement with agalsidase alfa results in regression of hypertrophic cardiomyopathy in patients with Anderson-Fabry disease, according to a report in the February issue of Heart.
Enzyme replacement with agalsidase alfa results in regression of hypertrophic cardiomyopathy in patients with Anderson-Fabry disease, an X-linked glycosphingolipid storage disorder caused by alpha-galactosidase A deficiency, according to a report in the February issue of Heart.
"Agalsidase alfa resulted in regression of the cardiomyopathy of Fabry disease with ongoing response or stabilization during the follow-up period," Dr. Derralynn A. Hughes from Royal Free Hospital and University College Medical School, London, told Reuters Health.
Dr. Hughes and colleagues assessed the efficacy of agalsidase alfa on left ventricular mass and myocardial globotriaosylceramide (Gb3) content in 15 men with Anderson-Fabry disease.
Myocardial Gb3 content decreased approximately 20 per cent in the group receiving agalsidase alfa but increased about ten per cent in the placebo group during the six months of the study, the authors report. Plasma and urinary sediment Gb3 also decreased significantly with agalsidase alfa treatment.
Left ventricular mass decreased by an average of 11.5 g in the agalsidase group, the report indicates, whereas left ventricular mass increased by a mean 21.8 g in the placebo group.
Among 11 patients who received uninterrupted therapy with agalsidase alfa in a two-year open extension to the trial, seven demonstrated a reduction in left ventricular mass, while four showed increases compared to their pre-treatment values.
There were no significant changes in left ventricular function associated with treatment, the investigators say. Infusions were well tolerated, they note, and ten patients received their final 12 to 18 study infusions in their homes without significant adverse events.
The Fabry Outcome Survey is an international database collecting information on untreated and agalsidase alfa-treated Fabry patients, Dr. Hughes added. "It is the best source of information on the long-term effects of agalsidase alfa.
"Agalsidase alfa resulted in regression of the cardiomyopathy of Fabry disease with ongoing response or stabilization during the follow-up period," Dr. Derralynn A. Hughes from Royal Free Hospital and University College Medical School, London, told Reuters Health.
Dr. Hughes and colleagues assessed the efficacy of agalsidase alfa on left ventricular mass and myocardial globotriaosylceramide (Gb3) content in 15 men with Anderson-Fabry disease.
Myocardial Gb3 content decreased approximately 20 per cent in the group receiving agalsidase alfa but increased about ten per cent in the placebo group during the six months of the study, the authors report. Plasma and urinary sediment Gb3 also decreased significantly with agalsidase alfa treatment.
Left ventricular mass decreased by an average of 11.5 g in the agalsidase group, the report indicates, whereas left ventricular mass increased by a mean 21.8 g in the placebo group.
Among 11 patients who received uninterrupted therapy with agalsidase alfa in a two-year open extension to the trial, seven demonstrated a reduction in left ventricular mass, while four showed increases compared to their pre-treatment values.
There were no significant changes in left ventricular function associated with treatment, the investigators say. Infusions were well tolerated, they note, and ten patients received their final 12 to 18 study infusions in their homes without significant adverse events.
The Fabry Outcome Survey is an international database collecting information on untreated and agalsidase alfa-treated Fabry patients, Dr. Hughes added. "It is the best source of information on the long-term effects of agalsidase alfa.
