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Case Reports
. 2019 Feb;61(2):232-235.
doi: 10.1111/dmcn.13919. Epub 2018 May 27.

Intrathecal baclofen in metachromatic leukodystrophy

Nikki van der Veldt  1 , Diane F van Rappard  2 , Laura A van de Pol  2 , Marjo S van der Knaap  2 , Willem J R van Ouwerkerk  3 , Jules G Becher  1 , Nicole I Wolf  2 , Annemieke I Buizer  1
Affiliations
Case Reports

Intrathecal baclofen in metachromatic leukodystrophy

Nikki van der Veldt et al. Dev Med Child Neurol. 2019 Feb.

Abstract
in English, Spanish, Portuguese

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.

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 pacientes con PCI espástica. Esto podría deberse a las características progresivas de la LDM. El BIT es una terapia factible y mejora el bienestar y cuidado diario de los pacientes con LDM, y, debería por ello, ser considerada tempranamente como tratamiento.

Baclofeno intratecal em leucodistrofia metacromática: A leucodistrofia metacromática (LDM) é uma desordem neurológica rara e progressiva, frequentemente acompanhada por deficiências motoras que são difíceis de tratar. Nesta série de casos, reportamos o curso do tratamento com baclofeno intratecal (BIT), com o objetivo de melhorar o cuidado diário e conforto em crianças e jovens com LDM. Todos os pacientes com LDM em nosso centro para tratamento com BIT por no mínimo 6 meses foram incluídos (n=10; 4 do sexo masculino, 6 do sexo feminino; média de idade 10a 8m [variação 6–24a]). Oito pacientes tinham LDM com desordem do movimento predominantemente espástica (eLDM) e dois eram predominantemente discinéticos. Pacientes com eLDM foram pareados com pacientes com paralisia cerebra (PC) espástica. As taxas de complicação relacionadas ao tratamento com BIT foram similares em ambos os grupos. O curso do tratamento com BIT nos primeiros 6 meses após implantação da bomba parece mostrar maior aumento de dose nos pacientes com LDM, comparados aos com PC espástica. Isso pode ser devido à doença progressiva em LDM. BIT é uma terapia viável para melhorar o cuidado diário e conforto em pacientes com LDM, e deve portanto ser considerado precocemente.

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Figures

Figure 1
Figure 1
Course per patient in sMLD, spastic CP, and dMLD group: 6 months follow‐up. aMean of the group. bCatheter disconnection: one patient with sMLD at 20 days after pump implantation; one patient with spastic CP at 1 month after pump implantation. sMLD, metachromatic leukodystrophy with predominant spastic movement disorder; CP, cerebral palsy; dMLD, metachromatic leukodystrophy with predominant dyskinetic movement disorder.
See this image and copyright information in PMC

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