To assess the efficacy of enzyme replacement therapy, Dutch researchers and clinicians are proposing a new methodology:
- the clinical and paraclinical data of 102 patients with Pompe’s disease who had been receiving enzyme replacement therapy for a year were retrospectively analysed,
- an index, MCID (for minimal clinically important difference), described as the minimal difference producing a perceived clinical effect, was constructed on the basis of subjective parameters of clinical improvement and quality of life,
- used in this cohort, the MCID has proved useful in determining thresholds for which the objective improvements observed in respiratory function and the walking test translate into improved patient experience.
The authors advocate the use of this new index in patient follow-up, as well as in clinical trials.
