Taliglucerase alfa: Rio de Janeiro experience at Hemorio

CO RR EC TE D P RO OF non-inferior to imiglucerase. After 12 months of treatment, 85% of patients treated with eliglustat and 94% of patients treated with imiglucerase maintained all 4 stability goals (lower bound of 95% CI of difference [−17.6%] within the pre-specified [−25%] noninferiority margin). Of the 159 patients treated in this study, 145 (91%) completed 24 months of treatment. During the 12-month extension period, most patients in both groups maintained stability. Among 99 of 106 patients who continued on eliglustat, all 4 goals were maintained by 87% of patients, with stable spleen volume in 96% of patients, stable hemoglobin in 97% of patients, stable platelet count in 94% of patients, and stable liver volume in 96% of patients. Among 47 of 53 patients who received imiglucerase in the primary analysis period and then eliglustat in the trial extension, 86% of patients maintained all 4 goals, with stable spleen volume in 97% of patients, stable hemoglobin in 100% of patients, stable platelet count in 90% of patients, and stable liver volume in 95% of patients. Most adverse events were mild or moderate in severity. No new safety concerns have arisen after 24 months. Eliglustat was non-inferior to imiglucerase in maintaining stability after 12 months of treatment in patients previously stabilized on enzyme replacement therapy. In the 12-month trial extension, clinical stability by both composite and individual measures was maintained by more than 85% of all patients, both those who remained on eliglustat for 24 months and those who switched from imiglucerase to eliglustat.