Quality of Life Among Patients with Mucopolysaccharidoses

To investigate preference-based quality of life (i.e., utility) in patients with mucopolysaccharidoses, a cross-sectional survey was carried out among proxy respondents. Preference-based quality of life of the six main subtypes of mucopolysaccharidoses was evaluated by utility measurement tools: visual analogue scale, standard gamble and time trade-off. Subjects were 93 medical students in a medical school and 6 medical experts on mucopolysaccharidoses in Japan. Group and individual interviews were carried out respectively. In standard gamble measurement among medical students, the mean utility value of mucopolysaccharidosis IH was the lowest (0.24), followed by mucopolysaccharidosis IIs (0.30) and mucopolysaccharidosis III (0.35). That of mucopolysaccharidosis IIm was the highest (0.76). The utility values of visual analogue sacle were significantly lower than those of standard gamble in all subtypes. All the correlation coefficients of utility values in each subtype of mucopolysaccharidoses between the three methods, using standard gamble as a reference standard, were statistically significant. The results among medical experts were similar to those among medical students. The utility values of patients with mucopolysaccharidoses were at low or medium levels. These results indicate that mucopolysaccharidoses pose a significant burden on overall health in these patients.