International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy

Valerio Carelli, Michele Carbonelli, Irenaeus F de Coo, Aki Kawasaki, Thomas Klopstock, Wolf A Lagrèze, Chiara La Morgia, Nancy J Newman, Christophe Orssaud, Jan Willem R Pott, Alfredo A Sadun, Judith van Everdingen, Catherine Vignal-Clermont, Marcela Votruba, Patrick Yu-Wai-Man, Piero Barboni

Research output: Contribution to journalComment/debate

Abstract

Leber hereditary optic neuropathy (LHON) is currently estimated as the most frequent mitochondrial disease (1 in 27,000-45,000). Its molecular pathogenesis and natural history is now fairly well understood. LHON also is the first mitochondrial disease for which a treatment has been approved (idebenone-Raxone, Santhera Pharmaceuticals) by the European Medicine Agency, under exceptional circumstances because of the rarity and severity of the disease. However, what remains unclear includes the optimal target population, timing, dose, and frequency of administration of idebenone in LHON due to lack of accepted definitions, criteria, and general guidelines for the clinical management of LHON. To address these issues, a consensus conference with a panel of experts from Europe and North America was held in Milan, Italy, in 2016. The intent was to provide expert consensus statements for the clinical and therapeutic management of LHON based on the currently available evidence. We report the conclusions of this conference, providing the guidelines for clinical and therapeutic management of LHON.

Original languageEnglish
Pages (from-to)371-381
Number of pages11
JournalJournal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society
Volume37
Issue number4
DOIs
Publication statusPublished - Dec 2017

Keywords

  • Antioxidants/therapeutic use
  • Congresses as Topic
  • Consensus
  • Disease Management
  • Humans
  • International Cooperation
  • Ophthalmology
  • Optic Atrophy, Hereditary, Leber/drug therapy
  • Societies, Medical
  • Ubiquinone/analogs & derivatives

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