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2014 ; 16
(6
): 292
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Treatment of mitochondrial disorders
#MMPMID24700433
Avula S
; Parikh S
; Demarest S
; Kurz J
; Gropman A
Curr Treat Options Neurol
2014[Jun]; 16
(6
): 292
PMID24700433
show ga
While numerous treatments for mitochondrial disorders have been suggested,
relatively few have undergone controlled clinical trials. Treatment of these
disorders is challenging, as only symptomatic therapy is available. In this
review we will focus on newer drugs and treatment trials in mitochondrial
diseases, with a special focus on medications to avoid in treating epilepsy and
ICU patient with mitochondrial disease, which has not been included in such a
review. Readers are also referred to the opinion statement in A Modern Approach
to the Treatment of Mitochondrial Disease published in Current Treatment Options
in Neurology 2009. Many of the supplements used for treatment were reviewed in
the previous abstract, and dosing guidelines were provided. The focus of this
review is on items not previously covered in depth, and our discussion includes
more recently studied compounds as well as any relevant updates on older
compounds . We review a variety of vitamins and xenobiotics, including
dichloroacetate (DCA), arginine, coenzyme Q10, idebenone, EPI-743, and exercise
training. Treatment of epilepsy, which is a common feature in many mitochondrial
phenotypes, warrants special consideration due to the added toxicity of certain
medications, and we provide a discussion of these unique treatment challenges.
Interesting, however, with only a few exceptions, the treatment strategies for
epilepsy in mitochondrial cytopathies are the same as for epilepsy without
mitochondrial dysfunction. We also discuss intensive care management, building
upon similar reviews, adding new dimensions, and demonstrating the complexity of
overall care of these patients.