Skip to content

No Surgical Innovation Without Evaluation

  • Hirst A
  • Philippou Y
  • Blazeby J
  • et al.
Citations of this article
Mendeley users who have this article in their library.
Get full text


A randomized, controlled trial of miglustat indicated that miglustat (Zavesca??) stabilized neurological disease over 12 months in adult and juvenile patients with Niemann-Pick disease type C (NP-C). We report data from a non-controlled, open-label extension to this initial randomized trial. All patients completing the randomized trial were allowed to continue treatment in a 12-month, non-controlled open-label extension. Those completing 12 months of extension therapy could continue further on miglustat in a 'continued extension' phase. From a total of 29 patients in the randomized phase (mean [??SD] age 24.6 ?? 9.1 years; 52% female), 21 completed 12 months of therapy with miglustat (17 of whom received miglustat in the initial randomized phase, and four in the extension phase), and 15 patients (all from the miglustat-randomized group) completed 24 months on miglustat. Mean horizontal saccadic eye movement velocity (HSEM-??) indicated improvement in the 12-month miglustat group, and stabilization in the 24-month group; swallowing was improved or stable in 86% and in up to 93%, respectively. Ambulation was stabilized in both the 12- and 24-month groups. In an exploratory disease stability analysis of prospective data on key parameters of disease progression (HSEM-??, swallowing, ambulation and cognition), 13/19 (68%) patients receiving ???12 months' miglustat therapy had stable disease. Among all patients receiving ???1 dose of miglustat (n = 28), the most frequent adverse events were diarrhoea, weight decrease, flatulence and tremor. Overall, these data suggest that long-term miglustat therapy stabilizes neurological disease and is well tolerated in adult and juvenile patients with NP-C. ?? 2009 Elsevier Inc. All rights reserved.




Hirst, A., Philippou, Y., Blazeby, J., Campbell, B., Campbell, M., Feinberg, J., … McCulloch, P. (2019). No Surgical Innovation Without Evaluation. Annals of Surgery, 269(2), 211–220.

Register to see more suggestions

Mendeley helps you to discover research relevant for your work.

Already have an account?

Save time finding and organizing research with Mendeley

Sign up for free