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| |  Enzyme Replacement Therapy Improves Outcomes in Fabry’s Disease NEW YORK, NY -- December 2, 2009 -- Enzyme replacement therapy (ERT) improves clinical outcomes, including reducing pain and improving quality of life, in patients with Fabry’s disease, according to a 5-year study reported online (www.thelancet.com) and in this week’s edition of The Lancet. Fabry’s disease is a rare progressive multisystem disorder resulting from lack of the enzyme alpha-galactosidase A, which is essential in various metabolic pathways. Renal failure, cardiomyopathy, and cerebrovascular disease are the main causes of disease and premature death. Atul Mehta, FRCP, FRCPath, Royal Free Hospital, London, United Kingdom, and colleagues assessed the effect of the ERT agalsidase alfa on heart mass and function, renal function, pain, and quality of life. The Fabry Outcome Survey observational database -- comprising data from Europe, North America, and South America -- was used for the study. The research team found that in patients with baseline cardiac hypertrophy, treatment resulted in a sustained reduction in left ventricular mass (LVM) index after 5 years of around 10%; and a significant increase in midwall fractional shortening (MFS) from 14.3% to 16.0% after 3 years. In patients without baseline hypertrophy, LVM index and MFS remained stable. Kidney function was assessed using mean yearly fall in estimated glomerular filtration rate versus baseline after 5 years of ERT, and this was found to be -3.17 mL/min per 1.73 m² for men and -0.89 mL/min per 1.73 m² for women. Average pain, measured by Brief Pain Inventory score, improved significantly, from 3.7 at baseline to 2.5 after 5 years. Quality of life scores also improved in patients given replacement therapy. The authors say: “Our results show sustained benefits of agalsidase alfa during 5 years of treatment.” Dr. Mehta adds: “These long term ‘real life’ registry data confirm the hopes raised by short term clinical trials, commenced over 10 years ago. ERT has the potential to prolong quality life in this often fatal illness. ” In an accompanying comment, Ravi Thadhani, MD, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, and colleagues write: “Enzyme-replacement therapy has only been available typically since 2001 and, thus, duration of exposure might be too short to assess whether this strategy prolongs life. Keeping the high cost of this lifelong treatment front and centre, we await confirmatory evidence of a reduction of clinical events with an extension of life.” They conclude: “We are registered for success, and confirmation of this achievement will hopefully follow.”
SOURCE: The Lancet
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