Is Baclofen (Baclofen) effective for the treatment of dystonia?

From the Guidelines

Baclofen is not typically a first-line treatment for dystonia, though it may be considered in certain cases, particularly when spasticity is a component, as supported by the most recent evidence from 1 and 1. Dystonia is better treated with anticholinergic medications like trihexyphenidyl (starting at 1mg daily and gradually increasing to 6-30mg daily divided into three doses) or benzodiazepines such as diazepam (2-10mg three times daily) or clonazepam (0.5-2mg twice daily). Botulinum toxin injections are the treatment of choice for focal dystonias, administered every 3-4 months directly into affected muscles, as recommended by 1. For generalized dystonia, oral medications like trihexyphenidyl or tetrabenazine (12.5-25mg twice daily) are often used. Baclofen, which works as a GABA-B receptor agonist, is more effective for spasticity than dystonia, though it may help in certain types of dystonia, particularly when spasticity is a component. If oral medications are ineffective, intrathecal baclofen delivered via pump may be considered for severe cases, as suggested by 1 and 1. The typical oral baclofen dose ranges from 5mg three times daily initially, gradually increasing to 20-25mg three times daily as needed. Side effects include sedation, weakness, and dizziness, and abrupt discontinuation should be avoided due to risk of withdrawal symptoms. Key considerations in the management of dystonia include:

• The use of botulinum toxin for focal dystonias
• The consideration of oral medications like trihexyphenidyl or tetrabenazine for generalized dystonia
• The potential role of baclofen in certain cases, particularly when spasticity is a component
• The importance of gradual dose titration and monitoring for side effects when using baclofen. Overall, the management of dystonia requires a tailored approach, taking into account the specific characteristics of the patient and the nature of their condition, as emphasized by the guidelines and evidence from 1, 1, and 1.

From the Research

Baclofen for Treatment of Dystonia

• Baclofen, a pre-synaptic acting GABA agonist, has been reported to benefit dystonia in several retrospective studies 2.
• In one retrospective study, dramatic improvement in symptoms, especially in gait, was found in almost 30% of 31 children and adolescents with idiopathic dystonia using doses ranging from 40 to 180 mg daily 2.
• The response to baclofen of adults with focal dystonia is less dramatic, with one series of 60 adults with cranial dystonia finding sustained benefit in 18% 2.
• Baclofen has been used to treat several secondary dystonias, including tardive dystonia, dystonia occurring in Parkinson's disease, and glutaric aciduria, with occasional reports of improvement 2.

Efficacy of Baclofen in Children with Idiopathic Dystonia

• A study of 16 patients less than 21 years old with idiopathic dystonia found that five had substantial improvement in symptoms, two had moderate improvement, and nine failed to benefit from baclofen treatment 3.
• The response to baclofen did not correlate with age at onset of dystonia or age at onset of therapy, but did correlate with duration of symptoms before therapy, with those who improved having a shorter duration of symptoms (3 years vs 7.8 years, p < 0.002) 3.

Current Treatment Options for Dystonia

• Current treatment options for dystonia include oral agents such as benzodiazepines, baclofen, and anticholinergics, as well as botulinum toxin injections and deep brain stimulation 4.
• Baclofen can be an effective treatment for childhood dystonia, with some patients experiencing sustained improvement in symptoms 3.
• Intrathecal baclofen has been used to treat severe segmental or generalized dystonia, but its efficacy and safety are uncertain, and it may be more effective when dystonia is associated with spasticity or pain 5.

Medical Treatment of Dystonia

• Medications such as anticholinergic drugs, dopamine modulators, baclofen, muscle relaxants, and other pharmacologic agents have been used to treat dystonia, but the introduction of botulinum toxin and deep brain stimulation has revolutionized the symptomatic treatment of this neurological movement disorder 6.
• Therapy of dystonia can be divided into physical, supportive, and ancillary therapy; pharmacologic treatment; chemodenervation with botulinum toxin; and peripheral and central surgery (deep brain stimulation) 6.