Treatment of Wilson's Disease with Dystonia After Oral Drug Therapy Has Failed

Clinical Scenario

Wilson's disease presenting with dystonia — segmental or generalised — where adequate reduction or control of dystonic symptoms has not been achieved with oral pharmacological treatment.

Previous Treatment Line — Target Not Reached

Oral drug therapy (baclofen, benzodiazepines, levodopa or dopamine agonists, tetrabenazine, or carbamazepine/oxcarbazepine) was used with the goal of reducing and controlling dystonic symptoms. When this goal is not met, escalation to a further treatment line is indicated.

Next-Line Approach (partial overview)

When medical therapy has been exhausted, a neurosurgical intervention targeting specific brain structures is considered as the ultimate treatment option aimed at controlling dystonic symptoms. The full protocol specifies which intervention applies and under what conditions.

Instant Access to Structured Evidence-Based Regimens

References

Focal dystonias are usually effectively treated with BTX injections and segmental or generalized dystonias are treated with oral drugs alone or in combination with BTX injected into the most affected muscles.
Results of exceptional surgical procedures such as DBS or thalamotomy in cases of severe generalized dystonia refractory to long-term anti-copper treatment and symptomatic pharmacological treatment are not convincing.

DOI: 10.1016/j.jhep.2024.11.007

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