1 40 1 Dublin Core The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/. Title A name given to the resource Treatment of Symptoms in Children with Q3 Conditions Scoping Review Results Text A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text. URL Address <a href="http://doi.org/10.1016/j.ejpn.2017.09.003" target="_blank" rel="noreferrer noopener">http://doi.org/​10.1016/j.ejpn.2017.09.003</a> Dublin Core The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/. Title A name given to the resource Intrathecal baclofen treatment an option in X-linked adrenoleukodystrophy Publisher An entity responsible for making the resource available European Journal of Paediatric Neurology Date A point or period of time associated with an event in the lifecycle of the resource 2018 Subject The topic of the resource quality of life; pain; limited mobility; priority journal; school child; spasticity; fatigue; clinical examination; human; article; child; male; clinical article; dystonia; case report; 1309378-01-5 (botulinum toxin A); 1638949-86-6 (botulinum toxin A); 1800016-51-6 (botulinum toxin A); 93384-43-1 (botulinum toxin A); 1134-47-0 (baclofen); adrenoleukodystrophy/dt [Drug Therapy]; adrenoleukodystrophy/su [Surgery]; baclofen/dt [Drug Therapy]; baclofen/po [Oral Drug Administration]; baclofen/tl [Intrathecal Drug Administration]; X chromosome linked disorder/dt [Drug Therapy]; X chromosome linked disorder/su [Surgery]; Addison disease; adrenoleukodystrophy/dt [Drug Therapy]; balance disorder; behavior change; bladder dysfunction; botulinum toxin A; clonus; diplopia; drug dose increase; hearing disorder; hyperpigmentation; intrathecal pump; leukodystrophy; range of motion; strabismus; urinary catheter; visual disorder; X chromosome linked disorder/dt [Drug Therapy]; tone and motor problems; X-linked adrenoleukodystrophy; pharmacologic interventions; intrathecal baclofen; baclofen Creator An entity primarily responsible for making the resource Hjartarson H T; Ehrstedt C; Tedroff K Description An account of the resource Background X-linked adrenoleukodystrophy (X-ALD) is a genetic peroxisomal disorder associated with tissue accumulation of very long chain fatty acids (VLCFAs). In approximately one third of affected males, this causes progressive and irreversible damage to the brain white matter. Progress is often rapid with upper motor neuron damage leading to severe spasticity and dystonia. The increased muscle tone is frequently difficult to alleviate with oral drugs. Here, we describe two patients with X-ALD who have received treatment with intrathecal baclofen pumps (ITB). Case study Both boys had a rapidly progressive cerebral form of the disorder resulting, among other things, in escalating spasticity and dystonia causing severe pain, dramatically reducing their quality of life. Both were treated with a variety of oral medications without adequate relief. Both patients tolerated ITB surgery without complications and the positive clinical effects of treatment with ITB became clear in the following weeks and months, with significantly reduced muscle tone, less pain and better sleep. Moreover, general caretaking became easier. Conclusion The treatment of spasticity and dystonia in these patients is difficult partly due to the relentless nature of this progressive disorder. In our two patients, ITB has been effective from both a symptomatic and palliative perspective. We recommend that such treatment be considered as an early option for increased muscle tone in boys with the cerebral form of X-ALD. Identifier An unambiguous reference to the resource within a given context <a href="http://doi.org/10.1016/j.ejpn.2017.09.003" target="_blank" rel="noreferrer noopener">10.1016/j.ejpn.2017.09.003</a> Rights Information about rights held in and over the resource Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s). 1134-47-0 (baclofen) 1309378-01-5 (botulinum toxin A) 1638949-86-6 (botulinum toxin A) 1800016-51-6 (botulinum toxin A) 2018 93384-43-1 (botulinum toxin A) Addison disease adrenoleukodystrophy/dt [Drug Therapy] adrenoleukodystrophy/su [Surgery] Article baclofen Baclofen/dt [drug Therapy] baclofen/po [Oral Drug Administration] baclofen/tl [Intrathecal Drug Administration] balance disorder behavior change bladder dysfunction botulinum toxin A Case Report Child Clinical Article clinical examination clonus diplopia drug dose increase Dystonia Ehrstedt C European Journal of Paediatric Neurology Fatigue hearing disorder Hjartarson H T Human hyperpigmentation intrathecal baclofen intrathecal pump Leukodystrophy limited mobility Male Pain pharmacologic interventions Priority Journal Quality Of Life Range of Motion School Child Spasticity strabismus Tedroff K tone and motor problems urinary catheter visual disorder X chromosome linked disorder/dt [Drug Therapy] X chromosome linked disorder/su [Surgery] X-linked adrenoleukodystrophy