Neuropediatrics Original Paper

European Journal of Pediatrics

, Volume 154, Issue 11, pp 928-932

First online:

Therapy of complex I deficiency: Peripheral neuropathy during dichloroacetate therapy

  • G. KurlemannAffiliated withChildren's Hospital
  • , I. PaetzkeAffiliated withInstitut für Klinische Chemie, Stadtisches Krankenhaus
  • , H. MöllerAffiliated withInstitut of Physical Chemistry, University of Münster
  • , H. MasurAffiliated withChildren's Hospital
  • , G. SchuiererAffiliated withInstitute of Clinical Radiology, University of Münster
  • , J. WeglageAffiliated withChildren's Hospital
  • , H. G. KochAffiliated withChildren's Hospital

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A therapeutic trial with polyvitamins and dichloroacetate (DCA) in combination with thiamine in a 13-year-old girl with complex I deficiency is reported. The polyvitamin therapy included thiamine, riboflavin, ascorbate, coenzyme Q 10 and carnitine. This therapeutic regine was used over a period of 17 months without any effect. Although DCA lowered the lactate concentration in blood and CNS — measured by magnetic resonance spectroscopy — no clinical benefit was achieved. After 20 weeks of DCA therapy a distal polyneuropathy with areflexia developed although 100 mg thiamine daily as comedication was given from the beginning of DCA therapy. Nerve conduction velocity of the peroneal nerve was not detectable, sensible evoked potentials of the tibialis posterious nerve were normal. This side-effect resolved completely within 6 months after omission of DCA. Our observation suggests a direct toxic effect of DCA only on the peripheral nervous system in our patient since several cerebral MRI and magnetic resonance spectroscopy studies showed no abnormalities.


DCA lowers the lactate concentration in children with complex I deficiency of the respiratory chain in a dose of 100 mg/kg body weight without clinical benefit. Reversible peripheral polyneuropathy may develop under DCA therapy despite thiamine medication.

Key words

Complex I deficiency Polyvitamin therapy Dichloroacetate therapy Polyneuropathy Thiamine administration