Evaluation of agalsidase alfa treatment response in FOS patients from Argentina.

NEUMANN P; CACERES G; KISINOVSKI I; QUARIN A; GASTALDI A; CHOUA M; FRANCISCUTTI M; GHISOLFO H; ROA N; ROZENFELD PA

Playa del Carmen, Mexico

Simposio; III Congreso Latinoamericano de Enfermedades Lisosomales; 2010

Introduction Fabry Outcome Survey (FOS) is a longitudinal multicenter observational survey of Fabry patients. The objectives of this registry are to monitor natural history and treatment response in real-world patients. Materials and methods Fifty eight Fabry argentinian patients in ERT, 27 females and 31 males, registered in FOS were included in this study. Evaluation of agalsidase alfa treatment was assayed by periodical determination of the following parameters: serum creatinine, creatinine clearance, proteinuria, left ventricular (LV) mass, pain (BPI) and quality of life (EQ-5D). Results Renal function was stable in patients on ERT, with a mean change of GFR of 2.2 ml-min/y. Patients that started ERT in stages 2 showed a better response, as compared to patients on stage 3. LV mass assessment revealed a mean change of 0.27 mm/y in patients without LVH and an improvement of -0.46mm/y in patients with LVH at initiation of ERT. Patients showed an improvement in quality of life, as measured by EQ5D score and Health state today VAS, that was sustained over 5 years of treatment. Changes were slightly better in males than females. Patients had a reduction of pain, as measured by worst pain and average pain on BPI, over 5 years of ERT.  Conclusions ERT with agalsidase alfa in Argentinian patients showed a comparable effect to that reported in bibliography. Argentinian patients on ERT with agalsidase alfa showed an improvement in parameters assayed, revealing a beneficial effect of this therapy.