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Long-term outcome of patients with neurological form of Wilson’s disease compliant to the de-coppering treatment

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Abstract

Background

A substantial proportion of Wilson’s disease (WD) patients exhibit residual neurological symptoms. Data on the prognostic value of initial clinical features and treatment choices in WD patients compliant to the therapy is relatively sparse.

Aim

The aim of the present study was to identify predictors of the long-term outcome of patients with WD with good treatment adherence.

Methods

Forty patients with neurological form of WD were evaluated before the de-coppering treatment initiation (based on the medical records) and after mean 15.25 ± 11.24 years of the stable treatment. Severity of neurological symptoms were assessed with a tier two of Global Assessment Scale (GAS) for Wilson’s Disease.

Results

The most frequent symptoms prior to treatment initiation were dysarthria (90%), tremor (90%), clumsiness (67.5%), depression (67.5%), and gait disturbance (62.5%). Significant decrease in the frequency of dysarthria, clumsiness, tremor, gait disturbance, postural instability and an improvement in school/work performance were observed after the long-term treatment, while frequency of dysphagia, drooling, bradykinesia and rigidity, dystonic and choreatic features did not change. Overall symptom severity decreased over time. Presence of dystonia before treatment initiation was the only identified predictor of worse residual GAS score. Greater severity of residual dystonia was associated with female gender and longer disease duration.

Conclusion

Although patients with neurological form of WD compliant to de-coppering treatment had favorable disease outcome, a significant burden of residual neurological symptoms was observed after the long-term follow-up. Dystonia at disease onset was the only identified predictor of the worse long-term outcome.

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Data Availability

Data used in this study is available upon request.

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Funding

No funding was received for conducting this study. Marina Svetel, Nikola Kresojević, Milica Ječmenica Lukić, Igor Petrović, Nataša Dragašević-Mišković, have received a speaker honorarium from Salveo. Iva Stanković, Čarna Jovanović, Jelena Vitković, Tatjana Pekmezović, Aleksandra Tomić, Vladana Marković and Vladimir Kostić have nothing to disclose.

Author information

Authors and Affiliations

  1. Neurology Clinic, University Clinical Center of Serbia, Dr Subotića 6, 11000, Belgrade, Serbia

    Iva Stanković, Čarna Jovanović, Jelena Vitković, Marina Svetel, Aleksandra Tomić, Nikola Kresojević, Vladana Marković, Milica Ječmenica Lukić, Igor Petrović, Nataša Dragašević-Mišković & Vladimir Kostić

  2. Faculty of Medicine, University of Belgrade, Belgrade, Serbia

    Iva Stanković, Marina Svetel, Aleksandra Tomić, Nikola Kresojević, Vladana Marković, Milica Ječmenica Lukić, Igor Petrović, Nataša Dragašević-Mišković & Vladimir Kostić

  3. Institute of Epidemiology, School of Medicine, University of Belgrade, Belgrade, Serbia

    Tatjana Pekmezović

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  1. Iva Stanković
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  2. Čarna Jovanović
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Contributions

Conceptualization: MS, VK. Methodology: MS, VK, ND, TP. Formal analysis and investigation: IS, MJL, CJ, JV. Writing–original draft preparation: IS, NK, AT, VM, MJ. Writing–review and editing: MS, TP, IP, ND, VK. Funding acquisition: /Resources: /Supervision: MS, IP, ND, VK.

Corresponding author

Correspondence to Marina Svetel.

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Conflicts of interest

Marina Svetel, Nikola Kresojević, Milica Ječmenica Lukić, Igor Petrović, Nataša Dragašević-Mišković, have received a speaker honorarium from Salveo. Iva Stanković, Čarna Jovanović, Jelena Vitković, Tatjana Pekmezović, Aleksandra Tomić, Vladana Marković and Vladimir Kostić have nothing to disclose.

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Stanković, I., Jovanović, Č., Vitković, J. et al. Long-term outcome of patients with neurological form of Wilson’s disease compliant to the de-coppering treatment. J Neurol 270, 3492–3498 (2023). https://doi.org/10.1007/s00415-023-11681-7

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  • DOI: https://doi.org/10.1007/s00415-023-11681-7

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