ScholarWorks@성균관대학교

초록

Purpose: To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II, compared with a historical placebo from a previous idursulfase trial (TKT024). Methods: The study comprised 2 sequential parts. In part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks. In the open, single-cohort part 2 study, additional participants received idursulfase beta for 52 weeks. Data from the idursulfase beta groups from parts 1 and 2 were pooled for comparisons with the historical placebo group (n = 32). The primary end point was a change in the 6-minute walk test at week 53. Results: Participants in the idursulfase beta groups (n = 24) were male Asians (mean age, 12.0 years). Idursulfase beta was superior to placebo in 6-minute walk test improvement (62.2 vs 7.3 m, P <.0001). Decrease in urine glycosaminoglycan excretion (−71.13% vs 21.39%, P <.0001) and reduction in the liver (−26.67% vs −0.80%, P <.0001) and spleen volumes (−26.46% vs 7.2%, P <.0001) were significant. The safety profile of idursulfase beta was satisfactory. Conclusion: Idursulfase beta is a safe and effective treatment option in mucopolysaccharidosis II, addressing crucial somatic ailments presented by patients. © 2025 The Authors

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