CLINICAL AND BRAIN BIOENERGETICS IMPROVEMENT WITH IDEBENONE IN A PATIENT WITH LEBERS HEREDITARY OPTIC NEUROPATHY - A CLINICAL AND P-31-MRS STUDY

We used phosphorus magnetic resonance spectroscopy (P-31-MRS) to study in vivo brain and muscle bioenergetics in a male patient with Leber's hereditary optic neuropathy (LHON) and mtDNA mutation at 11778 bp who developed spastic paraparesis with white matter lesions on brain MR i maging. The study was performed before and during treatment with idebe none (135 mg t.i.d.) and after withdrawal. Clinical amelioration and w orsening were associated with parallel changes in brain and skeletal m uscle bioenergetics following the administration or withdrawal of ideb enone. Reversal of paraparesis by idebenone was paralleled by normaliz ation of P-31-MRS, serum lactate and central motor conduction. Extra-o cular neurological dysfunction in LHON may be amenable to treatment by appropriate quinones. (C) 1997 Elsevier Science B.V.