Abstract Details

Title Clinical Spectrum of Symptoms and Treatment Response Behaviour in Long-term Treated Neurological Wilson Disease Patients: A Retrospective Analysis Over 10 Years

Topic General Neurology

Presentation(s) S12 - General Neurology: Neurotherapeutics and Clinical Trials (4:18 PM-4:30 PM)

Poster/Presentation
Number 005

Objective To longitudinally characterise the clinical neurological spectrum of 116 long-term treated patients with neurological Wilson´s disease (WD) and to analyse the response behaviour of different symptoms to copper elimination therapy.

Background Wilson disease is a potentially treatable, inherited disorder of copper metabolism that is characterised by the pathological accumulation of copper.

Design/Methods 116 WD-patients (mean age, 42 years), being regularly treated for at least three and up to 35 years in the out-patient department of the University of Düsseldorf-Germany, were included. 10 different neurological symptoms (motor symptoms: Dysarthria, dystonia, bradykinesia, tremor, gait, oculomotor symptoms, cerebellar symptoms/non-motor symptoms: reflex abnormalities, sensory symptoms and neuropsychological symptoms) were scored regarding severity into 4 classifications of absent, mild, moderate and severe. The sum of motor(0-21)- and non-motor(0-9) score amounted to the total score (0-30). For 74 patients clinical scoring was repeated after 2 years, and for 31 patients after 10 years.

Results

In the 116 long-term treated WD-patients bradykinesia, gait abnormalities and tremor were the most frequently observed symptoms. The least frequent symptoms were oculomotor symptoms, reflex abnormalities and sensory symptoms. Mean score was compared by paired T-test, and there was a statistically significant improvement in dysarthria(p=.02, t(61)=-2.25) and a worsening in sensory symptoms(p=.01,t(61)=2.41) after 2 years. The scores comparison after 10 years also revealed a statistically significant improvement in dystonia(p<.01)and tremor(p<.001) and a worsening in sensory symptoms(p<.001). Both motor and non-motor scores improved significantly after 2 and 10 years of treatment by mean differences of 95% CI [-1.28 , -0.46] and [-0.81 , -4.03] respectively.

Conclusions

The spectrum of remaining inert symptoms which are resistant to even long-term treatment of Wilson´s disease matches with the initial frequency of symptoms at the onset of therapy and the response behaviour of each individual symptoms to treatment.

Authors/Disclosures
PRESENTER	No disclosure on file
Philipp Albrecht, PhD (Neurologische Universitatsklinik Dusseldorf)	Dr. Albrecht has received personal compensation for serving as an employee of Abbvie. Dr. Albrecht has received personal compensation for serving as an employee of Biogen. Dr. Albrecht has received personal compensation for serving as an employee of Bristol Myers Squibb. Dr. Albrecht has received personal compensation for serving as an employee of Ipsen. Dr. Albrecht has received personal compensation for serving as an employee of Merck. Dr. Albrecht has received personal compensation for serving as an employee of Merz. Dr. Albrecht has received personal compensation for serving as an employee of Novartis. Dr. Albrecht has received personal compensation for serving as an employee of Roche. Dr. Albrecht has received personal compensation for serving as an employee of Sanofi. Dr. Albrecht has received personal compensation for serving as an employee of Lilly.
	No disclosure on file

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