TY - JOUR
T1 - Intrathecal baclofen in metachromatic leukodystrophy
AU - Van Der Veldt, Nikki
AU - Van Rappard, Diane F
AU - Van De Pol, Laura A
AU - Van Der Knaap, Marjo S
AU - Van Ouwerkerk, Willem Jr
AU - Becher, Jules G
AU - Wolf, Nicole I
AU - Buizer, Annemieke I
PY - 2019/2/1
Y1 - 2019/2/1
N2 - Metachromatic leukodystrophy (MLD) is a rare progressive neurological disorder, often accompanied by motor impairments that are challenging to treat. In this case series, we report the course of treatment with intrathecal baclofen (ITB), aimed at improving daily care and comfort in children and young adults with MLD. All patients with MLD in our centre on ITB treatment for a minimum of 6 months were included (n=10; 4 males, 6 females; mean age 10y 8mo [range 6-24y]). Eight patients had MLD with a predominant spastic movement disorder (sMLD) and two were mainly dyskinetic. Patients with sMLD were compared with matched patients with spastic cerebral palsy (CP). Complication rates related to ITB treatment were similar in both groups. ITB treatment course in the first 6 months after pump implantation appears to show more dose increase in most patients MLD, compared to patients with spastic CP. This may be due to the progressive disease in MLD. ITB is a feasible therapy to improve daily care and comfort in patients with MLD and should therefore be considered early.WHAT THIS PAPER ADDS: Intrathecal baclofen (ITB) is a feasible therapy to improve comfort and daily care in children and young people with metachromatic leukodystrophy (MLD). In the first 6 months of ITB treatment, MLD seems to show more dose increase compared to spastic cerebral palsy.
AB - Metachromatic leukodystrophy (MLD) is a rare progressive neurological disorder, often accompanied by motor impairments that are challenging to treat. In this case series, we report the course of treatment with intrathecal baclofen (ITB), aimed at improving daily care and comfort in children and young adults with MLD. All patients with MLD in our centre on ITB treatment for a minimum of 6 months were included (n=10; 4 males, 6 females; mean age 10y 8mo [range 6-24y]). Eight patients had MLD with a predominant spastic movement disorder (sMLD) and two were mainly dyskinetic. Patients with sMLD were compared with matched patients with spastic cerebral palsy (CP). Complication rates related to ITB treatment were similar in both groups. ITB treatment course in the first 6 months after pump implantation appears to show more dose increase in most patients MLD, compared to patients with spastic CP. This may be due to the progressive disease in MLD. ITB is a feasible therapy to improve daily care and comfort in patients with MLD and should therefore be considered early.WHAT THIS PAPER ADDS: Intrathecal baclofen (ITB) is a feasible therapy to improve comfort and daily care in children and young people with metachromatic leukodystrophy (MLD). In the first 6 months of ITB treatment, MLD seems to show more dose increase compared to spastic cerebral palsy.
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85047658526&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/29806077
U2 - https://doi.org/10.1111/dmcn.13919
DO - https://doi.org/10.1111/dmcn.13919
M3 - Article
C2 - 29806077
SN - 0012-1622
VL - 61
SP - 232
EP - 235
JO - Developmental Medicine and Child Neurology
JF - Developmental Medicine and Child Neurology
IS - 2
ER -