The debate – when and how should i use idebenone for LHON?

Abstract

The International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy was published in 2017 based on data available in 2016. The intent was to provide expert consensus statements for the clinical and therapeutic management of LHON based on the currently available evidence. It provided the guidelines for clinical and therapeutic management of LHON. It established a number of benchmarks such as clinical staging of LHION into Asymptomatic (mutation carriers), Subacute (<6 months from onset), Dynamic (6–12 months) and Chronic (>12 months) stages. We can all agree on the criteria for the diagnosis of LHON, based on a careful history, evaluation of key structural and functional visual parameters, and on a molecular confirmation of a pathogenic mtDNA mutation. On management it is clearly highly important to include genetic counselling and informing the patient about potentially preventable lifestyle risk factors. The use of idebenone is more debated and much has changed. Further studies and case series have been published and issues around when to start, how long to treat for and whether to treat chronic patients have all emerged. In this debate we shall raise a number of these issues and provide a reflection of the available data, their validity and clinical relevance and debate next steps.