Intrathecal baclofen treatment an option in X-linked adrenoleukodystrophy

Hjartarson, Helgi Thor; Ehrstedt, Christoffer; Tedroff, Kristina

doi:10.1016/j.ejpn.2017.09.003

Cited by 10 publications

(6 citation statements)

References 6 publications

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“…1,2 Children with other neurological disorders can also benefit from ITB. [3][4][5] Goals of treatment are typically to improve activities of everyday life. In non-ambulant children, goals are mostly on the level of facilitating personal care 1,2,6 and, in ambulant children, the goal is preservation of walking ability.…”

Section: Introductionmentioning

confidence: 99%

Surgical complications of intrathecal baclofen in children: A single centre, 20-year retrospective cohort study

Bonouvrié

Lagendijk

Beckerman

et al. 2022

European Journal of Paediatric Neurology

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Section: Introductionmentioning

confidence: 99%

Surgical complications of intrathecal baclofen in children: A single centre, 20-year retrospective cohort study

Bonouvrié

Lagendijk

Beckerman

et al. 2022

European Journal of Paediatric Neurology

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“…Aberg et al 80 compared levodopa and selegiline in neuronal ceroid lipofuscinosis types 3 and 6 in an open-label trial. Çak et al 81 investigated how tricyclic antidepressants affect cataplexy in Niemann-Pick type C. In x-linked adrenoleukodystrophy, Hjartarson et al 82 reported the use of intrathecal baclofen for spasticity and dystonia. In ataxia-telangiectasia, Zannolli et al 83 investigated betamethasone in a RCT and Nissenkorn et al 84 investigated amantadine sulfate in a longitudinal open-label study.…”

Section: Resultsmentioning

confidence: 99%

Scoping review of symptoms in children with rare, progressive, life-threatening disorders

Pawliuk

Widger

Dewan

et al. 2019

BMJ Support Palliat Care

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BackgroundQ3 conditions are progressive, metabolic, neurological or chromosomal childhood conditions without a cure. Children with these conditions face an unknown lifespan as well as unstable and uncomfortable symptoms. Clinicians and other healthcare professionals are challenged by a lack of evidence for symptom management for these conditions.AimsIn this scoping review, we systematically identified and mapped the existing literature on symptom management for children with Q3 conditions. We focused on the most common and distressing symptoms, namely alertness, behavioural problems, bowel incontinence, breathing difficulties, constipation, feeding difficulties, sleep disturbance, temperature regulation, tone and motor problems and urinary incontinence. For children with complex health conditions, good symptom management is pertinent to ensure the highest possible quality of life.MethodsScoping review. Electronic database searches in Ovid MEDLINE, Embase and CINAHL and a comprehensive grey literature search.ResultsWe included 292 studies in our final synthesis. The most commonly reported conditions in the studies were Rett syndrome (n=69), followed by Cornelia de Lange syndrome (n=25) and tuberous sclerosis (n=16). Tone and motor problems were the most commonly investigated symptom (n=141), followed by behavioural problems (n=82) and sleep disturbance (n=62).ConclusionThe evidence for symptom management in Q3 conditions is concentrated around a few conditions, and these studies may not be applicable to other conditions. The evidence is dispersed in the literature and difficult to access, which further challenges healthcare providers. More research needs to be done in these conditions to provide high-quality evidence for the care of these children.

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“…Intrathecal baclofen has been widely used for spasticity management in children, mostly with cerebral palsy and brain injury, although some recent studies have shown promising effects of baclofen pumps on tone in patients with lysosomal storage deficiencies such as metachromatic leukodystrophy, 9,10 mucopolysaccharidosis type II, 11 or Gaucher disease. 12 It allows for the treatment of spasticity with fewer side effects such as liver toxicity, sedation, or secretion management.…”

Section: Discussionmentioning

confidence: 99%

“…[6][7][8] A handful of publications have discussed the effect of baclofen pumps in patients with neurodegenerative metabolic disease. [9][10][11][12] There has been no literature about using intrathecal baclofen in cases of Niemann-Pick. An informal survey on the Pediatric Intrathecal Baclofen Pump Network of 200 baclofen pump providers across North America and Europe also yielded no known cases of using intrathecal baclofen for Niemann-Pick.…”

Section: Introductionmentioning

confidence: 99%

The Palliative Use of Intrathecal Baclofen in Niemann-Pick Disease Type C

et al. 2019

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Niemann-Pick disease type C is a rare progressive genetic disorder that leads to the abnormal accumulation of lipids within various tissues of the body, including brain tissue and liver. There is a rapid progression of the disease, resulting in severe disability in only a few years after the first symptoms, and survival is not much longer. Spasticity, dystonia, and chronic pain are common findings that severely impact quality of life in these patients. Analgesic management with traditional pain medications is not always effective, and the risk for secondary effects in medically complex patients is high. Liver function is also a limiting factor in these patients. This is a case report of a boy with advanced Niemann-Pick disease type C with developmental regression, cataplexia, and seizures. His severe spasticity made positioning and care difficult, and intense pain required multiple hospitalizations. He had unsuccessfully trialed multiple drugs. An intrathecal baclofen pump was placed without surgical complications and resulted in positive clinical effects. Baclofen pumps have classically been used for spasticity management in adults and children with nonprogressive diseases such as cerebral palsy or spinal cord injury with relatively long life expectancies. In adults, they have been used in patients with multiple sclerosis; however, use in pediatric neurodegenerative diseases has scarcely been reported. The use of intrathecal baclofen in palliative settings might provide an additional resource to provide comfort and quality of life for children with neurodegenerative diseases not only at end-of-life stages but also earlier on.

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Intrathecal baclofen treatment an option in X-linked adrenoleukodystrophy

Cited by 10 publications

References 6 publications

Surgical complications of intrathecal baclofen in children: A single centre, 20-year retrospective cohort study

Surgical complications of intrathecal baclofen in children: A single centre, 20-year retrospective cohort study

Scoping review of symptoms in children with rare, progressive, life-threatening disorders

The Palliative Use of Intrathecal Baclofen in Niemann-Pick Disease Type C

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