Verlauf des Morbus Huntington in frühen Stadien und Selektion sensitiver Verlaufsparameter: Analyse klinischer Daten von 379 Patienten über 3 Jahre

Abstract

BACKGROUND: Sensitive outcome measures for patients with Huntington´s disease are required for future clinical trials. METHODS: Longitudinal data were collected from a three-year study of 379 patients suffering from early Huntington´s disease who were not treated by antipsychotics. Progression of UHDRS item scores was evaluated by linear regression and slope, correlation coefficient, standard error and p-values were estimated on the basis of the data of eight evaluations from screening to study end (36 months). For the functional assessment dimension, the proportion of ‘no’ responses at baseline and at study end was determined. RESULTS: Linear progression was observed for the motor score and for all three functional measures (functional assessment score, independence assessment score and the total functional capacity score). In contrast, there was little evidence for progression of the behavioural assessment score over the study period, whereas the cognitive assessment score was intermediate. Twenty-two motor score items showed linear progression with a slope of > 0.003. These included all chorea items, finger tapping and pronation/supination (left and right), gait, tongue protrusion and tandem walking. CONCLUSIONS: Different symptom domains and individual items evolve at different rates in this group of patients suffering from early Huntington’s disease. It may be possible to select sensitive items to create a simplified version of the UHDRS which will be more efficient and more sensitive for the assessment of disease progression in clinical trials and natural history studies.