P07—Ventricular Arrhythmias are the Major Cause of Death in Patients With the Classical Phenotype of Fabry Disease

Abstract

0.3 mg/kg back to 1 mg/kg, the normal approved dose for agalsidase beta. The patient immediately felt more at ease and was less anxious with this higher dose. His neuropathic pain has ceased, and the GL-3 levels normalized again: plasma GL-3 was 4.4 mol/mL, and urinary GL-3/creatinine was 0.8 mol/mol. Conclusions: Agalsidase beta at 1 mg/kg is the correct dose for this Fabry patient, who received this dose for years without any adverse events. A dose decrease imposed by delivery issues rendered the patient anxious and increased his neuropathic pain. Moreover, the urinary GL-3 increased dramatically. This suggests increased disease activity. When the 1-mg/kg dose could be reinitiated, the patient reported a clear improvement of anxiety and neuropathic pain. GL-3 levels normalized again. These observations emphasize the importance of a correct dose of 1 mg/kg to control disease activity in our patient.