Intrathecal baclofen in metachromatic leukodystrophy

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 youn...

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Bibliographic Details
Published in:Developmental medicine and child neurology 2019-02, Vol.61 (2), p.232-235
Main Authors: Veldt, Nikki, Rappard, Diane F, Pol, Laura A, Knaap, Marjo S, Ouwerkerk, Willem J R, Becher, Jules G, Wolf, Nicole I, Buizer, Annemieke I
Format: Article
Language:English
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Case Report
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Summary: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. Resumen Baclofen intratecal en la leucodistrofia metacromática La leucodistrofia metacromática (LDM) es una rara enfermedad neurológica progresiva, habitualmente acompañada de déficit motor de muy difícil abordaje. En esta serie de casos reportamos el curso del tratamiento con baclofen intratecal (BIT), con el fin de mejorar el cuidado diario y el bienestar en niños y adultos jóvenes con esta afectación. Todos los pacientes de nuestro centro portadores de LDM y en tratamiento con BIT fueron incluidos (n=10; 4 varones, 6 mujeres, edad medias 10 años 8 meses [rango 6‐24 años]). Ocho pacientes tenían una LDM con un desorden del movimiento predominantemente espástico (LDMesp) y dos eran predominantemente diskinética. Los pacientes con LDMesp fueron comparados con pacientes portadores de Parálisis Cerebral Infantil (PCI). Las ratios de complicaciones relacionados con el tratamiento con BIT fueron similares en ambos grupos. El curso del tratamiento con BIT en los primeros 6 meses luego de la implantación de la bomba aparentemente muestra un mayor aumento de la dosis en gran parte de los pacientes con LDM, comparado con los p
ISSN:0012-1622
1469-8749
DOI:10.1111/dmcn.13919