Mix of Treatments Best for DCP Due to Bilirubin Encephalopathy, Caregivers Say

A multidisciplinary approach consisting of different treatment strategies for dyskinetic cerebral palsy (DCP) due to bilirubin encephalopathy — a condition in which damage arises due to very high levels of bilirubin, a substance produced by the breakdown of red blood cells — is necessary to improve daily life for children with the disease, according to a survey of caregivers in Japan.

The study, “A questionnaire survey on the efficacy of various treatments for dyskinetic cerebral palsy due to preterm bilirubin encephalopathy,” was published in the journal Brain & Development.

Bilirubin encephalopathy is known to cause DCP in preterm babies. When high levels of bilirubin— a yellow substance produced as a consequence of red blood cells’ degradation — accumulate in the brain, neurological damage leading to DCP may occur.

One of the main features of DCP is dyskinesia, or involuntary muscle movements. Children with the disease may also have variations in muscle tone and abnormal posture.

These symptoms can interfere with children’s daily activities, such as moving, playing, communicating, eating, and sleeping. The increased muscle tone also tends to cause pain in various parts of the body.

Currently, there is no established treatment regimen to manage DCP due to bilirubin encephalopathy, with clinical strategies varying from patient to patient.

Researchers in Japan mailed a questionnaire to caregivers of 67 children older than 4 with DCP due to preterm bilurubin encephalopathy. Forty-one — 39 mothers and two fathers — provided complete answers.

The researchers asked what treatments the children were receiving, or had received in the past. Choices included rehabilitation, oral medicines, botulinum toxin administration, orthopedic surgery, intrathecal baclofen pump (ITB)  and deep brain stimulation (DBS).

ITB enables the continuous delivery of baclofen, a muscle relaxant, to the cerebrospinal fluid, which is the fluid that surrounds the brain and spinal cord. Using a pump surgically implanted under the skin of the abdomen, the therapy employs a catheter — a thin, flexible tube — to bring the medication from the pump into the spinal fluid.

DBS is a non-destructive surgical treatment, often used for Parkinson’s disease, that involves implanting a device to stimulate targeted regions of the brain with electrical impulses generated by a battery-operated neurostimulator.

Caregivers were also asked to evaluate the efficacy of each treatment according to five categories — motor function, postural stability, sleep, pain, and care burden — and rate them on a five-point scale.

All 41 of the children had undergone rehabilitation. Thirty of them (73%) took oral medicines, 22 (54%) used botulinum toxin, 12 (29%) underwent orthopedic surgery, and three were implanted with an intrathecal baclofen pump. No patient had tried DBS.

Twenty-four percent of the patients underwent one treatment in addition to rehabilitation, 34% reported two additional treatments, 17% had three additional treatments, and 5% took four additional treatments. Twenty percent did not receive any treatment other than rehabilitation.

Most families reported that rehabilitation had positive outcomes in all categories, especially motor function, postural stability and care burden.

Oral therapies also had a positive effect across all five categories, especially in sleep.

There was a positive association between Gross Motor Function Classification System (GMFCS) level and the number of oral medicines that had been used.

Chlordiazepoxide (sold under brand names including Librium) was the most frequently used oral treatment (28 children), followed by triclofos sodium (14 children), phenobarbital (brand names Luminal, Solfoton) (13 children) and cyprotadine (brand names including Periactin) (11 children).

Twelve caregivers considered chlordiazepoxide to be the most effective oral therapy, while gabapentin (brand names including Neurontin) and triclofos were considered the most effective by three families.

Botulinum toxin injection, one of the most effective treatments for spasticity currently available, and orthopedic surgery had positive outcomes for motor function, postural stability and care burden. Intrathecal baclofen pump also showed positive outcomes for all five categories.

“Each treatment showed some beneficial effects, but the optimal treatment was difficult to determine,” the researchers wrote, adding that a “multidisciplinary approach is important to control muscle tone and improve the [quality of life] of preterm children with [bilirubin encephalopathy] and their families.”

“The appropriate combination of treatments, including rehabilitation, oral drugs, botulinum toxin, orthopaedic surgery, and ITB therapy is critical,” the researchers said.