Last modified: 2018-09-09
Abstract
Introduction: Despite the great advances in specific therapy in DMD, the pillars of the treatment are still as recommended in consensus (support therapies, cardioprotection). To assess access to such care, in 2012 a questionnaire was created by a European group, currently available in translated and validated version for the Brazilian population.
Objective: to define the profile of access to health care in DMD in a Brazilian sample.
METHODS: A total of 44 patients with a diagnosis of DMD were interviewed in an university neuropediatrics clinic in Rio de Janeiro, between December 2015 and January 2018. The answers were organized and analyzed in Excel.
Results: The mean age at diagnosis was 5 years and a half, with a median of 6 years. 32 (74.4%) patients had already lost gait capacity, on average, at 9 years of age. 62.8% were or had been in physical therapy and most did not require any type of ventilatory support.
Conclusions: Compared to previously published national data, there was an improvement in the time of diagnosis. However, when compared to international data, the Brazilian patient with DMD shows loss of gait earlier and finds it more difficult to perform the recommended therapies regularly. This difference could be explained by phenotypic variability among populations or by difficulties related to the lack of effective public strategies that guarantee accessibility to the dystrophic patient. It is necessary to formulate public policies that improve patient care with DMD in our population.