Journal of Neurology

, Volume 254, Issue 4, pp 478–481

MRI derived brain atrophy in PSP and MSA-P

Determining sample size to detect treatment effects
  • Dominic C. Paviour
  • Shona L. Price
  • Andrew J. Lees
  • Nick C. Fox
ORIGINAL COMMUNICATION

DOI: 10.1007/s00415-006-0396-4

Cite this article as:
Paviour, D.C., Price, S.L., Lees, A.J. et al. J Neurol (2007) 254: 478. doi:10.1007/s00415-006-0396-4

Abstract

Progressive supranuclear palsy (PSP) and multiple system (MSA) atrophy are associated with progressive brain atrophy. Serial MRI can be applied in order to measure this change in brain volume and to calculate atrophy rates.

We evaluated MRI derived whole brain and regional atrophy rates as potential markers of progression in PSP and the Parkinsonian variant of multiple system atrophy (MSA-P).

17 patients with PSP, 9 with MSA-P and 18 healthy controls underwent two MRI brain scans. MRI scans were registered, and brain and regional atrophy rates (midbrain, pons, cerebellum, third and lateral ventricles) measured. Sample sizes required to detect the effect of a proposed disease-modifying treatment were estimated. The effect of scan interval on the variance of the atrophy rates and sample size was assessed.

Based on the calculated yearly rates of atrophy, for a drug effect equivalent to a 30% reduction in atrophy, fewer PSP subjects are required in each treatment arm when using midbrain rather than whole brain atrophy rates (183 cf. 499). Fewer MSA-P subjects are required, using pontine/cerebellar, rather than whole brain atrophy rates (164/129 cf. 794). A reduction in the variance of measured atrophy rates was observed with a longer scan interval.

Regional rather than whole brain atrophy rates calculated from volumetric serial MRI brain scans in PSP and MSA-P provide a more practical and powerful means of monitoring disease progression in clinical trials.

Key words

longitudinal MRI PSP MSA-P power calculations 

Copyright information

© Steinkopff-Verlag 2007

Authors and Affiliations

  • Dominic C. Paviour
    • 1
    • 2
  • Shona L. Price
    • 2
  • Andrew J. Lees
    • 1
    • 3
  • Nick C. Fox
    • 2
  1. 1.The Sara Koe PSP Research CentreInstitute of NeurologyLondon WC1NUK
  2. 2.Dementia Research CentreInstitute of Neurology UCLLondonUK
  3. 3.Reta Lila WestonInstitute of Neurological UCLLondonUK

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