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- Title
A TERAPIA DE REPOSIÇÃO ENZIMÁTICA PODE MUDAR A FREQUÊNCIA DE INTERVENÇÕES MÉDICAS NA MUCOPOLISSACARIDOSE TIPO I? UM ESTUDO RETROSPECTIVO E EXPLORATÓRIO.
- Authors
de Bitencourt, Fernanda Hendges; de Ávila, Mayna Yaçanã Borges; de Souza, Mônica Vinhas; Vieira, Taiane Alves; Schwartz, Ida Vanessa Doederlein
- Abstract
BACKGROUND: Mucopolysaccharidosis type I (MPSI) is a lysosomal disorder (LSD) which can be treated with enzyme replacement therapy (ERT) with laronidase. AIM: To describe the effect of ERT on MPSI patients evaluated at a single referral center for LSD by assessing the frequency of medical interventions. METHODS: An exploratory, retrospective study with pre- and post-intervention assessments. We reviewed medical records to collect data on the number of medical appointments/year/patient, medications used, hospital admissions, surgeries, and exams performed. These variables were then compared between the pre- and the post-ERT periods. RESULTS: Nine patients (severe=3; attenuated=6) were included in the study. The median age for the start of ERT was 9 years, and the median time on ERT was 4 years. On average, patients received 90% of the infusions predicted for the study period. Only the number of surgeries/year/patient was found to be dependent on length of disease (p=0.0004) and on severity of phenotype (p=0.014). Regarding pre- and post-ERT comparisons, there was a significant difference (mean number/ year/patient in exams (pre-ERT, 10.2±2.7; post-ERT, 22.5±2.1; p=0.005) and hospital admissions (pre-ERT, 0.05±0.04; post-ERT, 0.30±0.11; p=0.013). CONCLUSION: Our data suggest ERT didn't alter the natural history of MPSI the outcomes assessed in this study. This may be due to the relatively advanced age of patients when they started treatment at our Center.
- Publication
Clinical & Biomedical Research, 2014, Vol 34, Issue 1, p11
- ISSN
2357-9730
- Publication type
Article