Idebenone treatment in Leber's hereditary optic neuropathy

V Carelli, C La Morgia, ML Valentino, G Rizzo… - Brain, 2011 - academic.oup.com
V Carelli, C La Morgia, ML Valentino, G Rizzo, M Carbonelli, AM De Negri, F Sadun, A Carta…
Brain, 2011academic.oup.com
Sir, We have read with great interest the results presented by Klopstock et al.(2011)
concerning the RHODOS study on a clinical trial with idebenone in Leber's hereditary optic
neuropathy (LHON) and we would like to share our own experience of idebenone therapy in
LHON. Idebenone has been an approved drug (MnesisÕ, Takeda Italia Farmaceutici) in Italy
since the early 1990s and, after the initial report by Mashima et al.(1992) on its possible
efficacy in LHON, we offered this therapeutic option to all of our new consecutive patients …
Sir, We have read with great interest the results presented by Klopstock et al.(2011) concerning the RHODOS study on a clinical trial with idebenone in Leber’s hereditary optic neuropathy (LHON) and we would like to share our own experience of idebenone therapy in LHON.
Idebenone has been an approved drug (MnesisÕ, Takeda Italia Farmaceutici) in Italy since the early 1990s and, after the initial report by Mashima et al.(1992) on its possible efficacy in LHON, we offered this therapeutic option to all of our new consecutive patients with LHON, almost all of whom accepted treatment. Idebenone was given after informed consent following the regulation for ‘off-label’drug administration and was provided for free by the National Health Service, under the legislation for certified rare disorders. Patients were initially treated with 270mg/day (Cortelli et al., 1997; Carelli et al., 1998a, b), but following the reports on idebenone treatment in Friedreich ataxia, the dosages were increased to 540–675mg/day (Rustin et al., 1999; Kearney et al., 2009).
Oxford University Press