Coenzyme Q10 treatment in Leber's hereditary optic neuropathy

The clinical manifestations and molecular genetic analysis of two patients with Leber's hereditary optic neuropathy (LHON) in a Taiwanese family are reported. A point mutation from G to A at the 11778th nucleotide position in the ND4 gene of mitochondrial DNA was found in the two probands. They were characterized by acute visual loss in the second or third decade of life. The clinical course was progressive in nature. Except for optic atrophy, there were no other neurological abnormalities. Intriguingly, a trial with coenzyme Q10 therapy for four months resulted in the rapid improvement of visual acuity in these two patients. To our knowledge, the prognosis of visual function is poor in LHON patients with the G11778A mutation. We conclude that, although spontaneous improvement may exist, coenzyme Q10 treatment may either be effective or speed up spontaneous improvement, particularly in the acute stage in the treatment of LHON patients.