Home infusion program for Fabry disease: experience with agalsidase alfa in Argentina

fabry disease is an x-linked lysosomal storage disorder caused by inherited deficiency of the enzyme α-galactosidase a. enzyme replacement treatment using agalsidase alfa significantly reduces pain, improves cardiac function and quality of life, and slows renal deterioration. nevertheless, it is a life-long treatment which requires regular intravenous infusions and entails a great burden for patients. our objective was to evaluate retrospectively the safety and tolerability of the home infusion of agalsidase alfa in patients with fabry disease in argentina. we evaluated all the patients with fabry disease who received home infusion with agalsidase alfa 0.2 mg/kg between january 2005 and june 2011. the program included 87 patients; 51 males (mean age: 30 years) and 36 females (mean age: 34 years). a total of 5229 infusions (mean: 59 per patient; range: 1-150) were administered. a total of 5 adverse reactions were seen in 5 patients (5.7% of patients and 0.9% of the total number of infusions). all were mild in severity and resolved by reducing the rate of infusion and by using antihistaminics. all these 5 patients were positive for igg antibodies, but none of them presented ige antibodies and none suffered an anaphylactic shock. in our group 18 patients were switched from agalsidase beta to agalsidase alfa without complications. home infusion with agalsidase alfa is safe, well tolerated and is associated to high compliance.