Intrathecal baclofen in metachromatic leukodystrophy.
Nikki van der Veldt, Diane F van Rappard, Laura A van de Pol and 5 others
PMID 29806077WHAT IT FOUND
Ten non-walking children with metachromatic leukodystrophy received intrathecal baclofen.
Doses increased steadily over six months as the disease progressed. Families reported clear improvements in comfort and daily care, though no standardized outcome measures were used.
Key findings
01All ten patients with metachromatic leukodystrophy were non-walking at the start of therapy, classified as GMFC-MLD level III to VI.
02In the eight patients with spastic metachromatic leukodystrophy, the mean daily dose rose from 68 micrograms at implantation to 210 micrograms at six months.
03Families reported clear improvements in comfort and daily care, but the study did not use standardized questionnaires to measure outcomes.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTsWhat it means for SLPs
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What it does not show
Only ten patients with metachromatic leukodystrophy were studied, and the two with dyskinetic disease were too few to match or analyse. Small numbers prevented meaningful statistical comparison between metachromatic leukodystrophy and cerebral palsy groups. Outcomes were based on interviews and caregiver reports, not standardized questionnaires or objective measures. Metachromatic leukodystrophy is progressive, so decline in function is expected regardless of treatment, making it hard to attribute comfort gains solely to baclofen. The study only followed patients for six months, so long-term dose requirements and complications are unknown.
Declared interests
No funding source or conflict of interest declaration is stated in the provided text.
The easy way to misread this
Do not read the dose increase from 68 to 210 micrograms as a sign of treatment failure or tolerance. The authors state this rise reflects the progressive nature of the disease, not a problem with the pump or the drug.