Abstract

VILLALOBOS-AZUAJE, Jacobo José  and  PADRON-NIEVES, Maritza del Carmen. Classic Fabry disease in asymptomatic pediatric patients: Bioethical aspects of enzyme therapy. rev.latinoam.bioet. [online]. 2020, vol.20, n.2, pp.25-40.  Epub Apr 23, 2021. ISSN 1657-4702.  https://doi.org/10.18359/rlbi.4642.

This paper aims to propose a bioethical framework for the administration of enzyme therapy in children with asymptomatic Fabry disease. A cross-sectional, observational, descriptive field study was conducted with quantitative and qualitative analysis, in the light of bioethics with physicians who have diagnosed and treated patients with classic Fabry disease at the local and international level, using a survey with closed questions validated by expert judgment. The application of the principles of non-maleficence, justice, and sacredness of life in the administration of enzymatic treatment for these patients was evaluated. The information was grouped and processed with descriptive statistics. 83,33 % of respondents considered that a child with asymptomatic Fabry disease should receive specific enzymatic treatment, regardless of its cost (justice), and that treatment should be funded by the State in order to prevent complications of the disease. This was recognized by 75% of the physicians surveyed (not maleficence). 66,66 % considered that the sacredness of life must prevent the complications that condition death. Thus, based on the principles of bioethics, initiating specific enzymatic therapy for the treatment of children with asymptomatic Fabry disease is sustained.

Keywords : Fabry's disease; Fabry's disease in children; enzyme therapy; bioethics.

        · abstract in Spanish | Portuguese     · text in Spanish     · Spanish ( pdf )