Parkinsonism & related disorders · 2019
Combined cerebral atrophy score in Huntington's disease based on atlas-based MRI volumetry: Sample size calculations for clinical trials.
СтатьяМногоцентровое исследованиеНаблюдательное исследованиеПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
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Müller Hans-Peter, Huppertz Hans-Jürgen, Dreyhaupt Jens, Ludolph Albert C, Tabrizi Sarah J, Roos Raymund A C, Durr Alexandra, Landwehrmeyer G Bernhard, Kassubek Jan
Аннотация
INTRODUCTION: A volumetric MRI analysis of longitudinal regional cerebral atrophy in Huntington's disease (HD) was performed as a read-out of disease progression to calculate sample sizes for future clinical trials.
METHODS: This study was based on MRI data of 59 patients with HD and 40 controls recruited within the framework of the PADDINGTON study and investigated at baseline and follow-up after 6 and 15 months. Automatic atlas-based volumetry (ABV) of structural T1-weighted scans was used to calculate longitudinal volume changes of brain structures relevant in HD and to assess standardized effect sizes and sample sizes required for potential future studies.
RESULTS: Atrophy rates were largest in the caudate (-3.4%), putamen (-2.8%), nucleus accumbens (-1.6%), and the parietal lobes (-1.7%); the lateral ventricles showed an expansion by 6.0%. Corresponding effect sizes were -1.35 (caudate), -0.84 (putamen), -0.91 (nucleus accumbens), -1.05 (parietal lobe), and 0.92 (lateral ventricles) leading to N = 36 subjects per study group for detecting a 50% attenuation of atrophy for the best performing structure (caudate). A combined score of volume changes in non-overlapping compartments (striatum, parietal lobes, lateral ventricles) increased the effect size to -1.60 and substantially reduced the required sample sizes by 10 to N = 26 subjects per study group. This combined imaging score correlated significantly both with the CAP score and with the progression of the clinical phenotype.
CONCLUSION: We propose ABV of the striatum together with parietal lobe and lateral ventricle volumes as a combined imaging read-out for progression studies including clinical trials in HD.
METHODS: This study was based on MRI data of 59 patients with HD and 40 controls recruited within the framework of the PADDINGTON study and investigated at baseline and follow-up after 6 and 15 months. Automatic atlas-based volumetry (ABV) of structural T1-weighted scans was used to calculate longitudinal volume changes of brain structures relevant in HD and to assess standardized effect sizes and sample sizes required for potential future studies.
RESULTS: Atrophy rates were largest in the caudate (-3.4%), putamen (-2.8%), nucleus accumbens (-1.6%), and the parietal lobes (-1.7%); the lateral ventricles showed an expansion by 6.0%. Corresponding effect sizes were -1.35 (caudate), -0.84 (putamen), -0.91 (nucleus accumbens), -1.05 (parietal lobe), and 0.92 (lateral ventricles) leading to N = 36 subjects per study group for detecting a 50% attenuation of atrophy for the best performing structure (caudate). A combined score of volume changes in non-overlapping compartments (striatum, parietal lobes, lateral ventricles) increased the effect size to -1.60 and substantially reduced the required sample sizes by 10 to N = 26 subjects per study group. This combined imaging score correlated significantly both with the CAP score and with the progression of the clinical phenotype.
CONCLUSION: We propose ABV of the striatum together with parietal lobe and lateral ventricle volumes as a combined imaging read-out for progression studies including clinical trials in HD.