Generalized Dystonia - Intrathecal Baclofen

Baclofen (Lioresol®) is a medication approved by the US Food & Drug Administration for the treatment of spasticity, a condition in which damage to the brain or spinal cord cause muses to tighten and stiffen. Baclofen is also used off-label to treat dystonia. Baclofen in the spinal fluid around the brain and spinal cord supplements the body's supply of a chemical neurotransmitter called GABA, which relaxes muscle movement. The drug may be given orally, but very high doses must often be used to ensure that the drug saturates the blood stream and reaches the spinal fluid. High doses of oral baclofen may cause intolerable side effects such as muscle weakness and fatigue. A surgically implanted baclofen pump delivers baclofen directly to the spinal fluid, and only very small doses are needed.

Intrathecal baclofen therapy has been used for children and adults with a combination of dystonia and spasticity, for example as the result of cerebral palsy. It is primarily used to treat dystonia and spasticity affecting the limbs.

In order to determine if an individual is eligible for intrathecal baclofen, they must undergo a screening test to observe the body's response to baclofen.

Starting intrathecal baclofen therapy involves surgically implanting a pacemaker-like device into the abdomen, beneath the skin of the abdomen. The pump is connected to a thin tube that is tunneled around the body and delivers baclofen to the spinal canal. The pump is adjusted by a remote control to deliver the amount of medication appropriate for the individual. Hospital stay may range from four to seven days. Modest improvement of symptoms may be noticeable before the individual is discharged from the hospital, and it make take six months or more to achieve the full extent of benefit.

Regular maintenance is a key component of intrathecal baclofen therapy. Regular exams and physical therapy may be a component of postoperative care. Pumps must be refilled regularly in the physician's office as an outpatient procedure. The frequency of refilling the pump depends on the dose required. The pump battery needs to be replaced about every five to seven years.

Studies have shown that intrathecal baclofen can dramatically improve symptoms and quality of life. Some centers have reported significant improvement in as much as 85% of patients. However, like any surgery, the procedure is not without risks. Pump complications can arise including infection and device breakage and disconnection. In a small percentage of cases, patients may lose effect within the first year of therapy or experience a worsening of symptoms. Common side effects are constipation, decreased muscle control, and drowsiness.

Last updated: November 2021

Thank you to Dystonia Medical Research Foundation (USA) for allowing us to share this information. The DMRF is a 501(c)(3) non-profit organization dedicated to advancing research for improved dystonia treatments and ultimately a cure, promoting awareness, and supporting the well-being of affected individuals and families.

Last update: Nov 2021