Experts Establish LHON Clinical, Treatment Recommendations
A global panel of leading experts in the field of mitochondrial optic neuropathies has issued a consensus statement to provide clinical and therapeutic guidance for the management of Leber’s hereditary optic neuropathy (LHON).
Focusing on Santhera Pharmaceuticals’ Raxone (idebenone), the only treatment approved by the European Medicines Agency (EMA) for the treatment of LHON, the consensus statement provides recommendations to help establish the optimal target population and the timing, dose, and frequency of treatment.
The consensus statement was published in the Journal of Neuro-Ophthalmology in an article titled, “International Consensus Statement on the Clinical and Therapeutic Management of Leber’s Hereditary Optic Neuropathy.”
“The expert consensus statement provides a much-needed set of recommendations for physicians who are treating patients with LHON, ultimately leading to improved patient care,” Francesco Bandello, MD, professor at the University Vita Salute, Scientific Institute San Raffaele of Milano, and former president of the European Society of Retina Specialists (EURETINA), said in a press release.
The international conference, “Update on Optic Nerve Degeneration—a European network: first international meeting,” held in Milan, Italy, provided the optimal environment to gather international leaders with extensive experience in clinical and genetic management and research on optic neuropathies and LHON.
Sixteen renowned investigators, neurologists, and ophthalmologists from across Europe and North America attended the conference and provided much-needed recommendations for LHON management based on currently available evidence.
The recommendations are expected to help physicians, healthcare providers, and health organizations improve the management of patients with LHON, Bandello said.
A detailed evaluation and confirmation of the mitochondrial genetic defect can help diagnose LHON. After this process, genetic counseling and educational sessions on potential LHON-related lifestyle risk factors and the administration of Raxone can help patients with subacute and dynamic disease manage it.
According to the recommendations, the use of Raxone should be discontinued in patients who don’t respond and should not be administrated to patients in the chronic stages of the disease.
“This is the first time that a consensus for the management of LHON has been developed, and this paper will generate important awareness for the disease and Raxone – the first approved treatment for this heritable form of vision loss,” said Thomas Meier, PhD, CEO of Santhera. “We hope that this publication will allow us to engage in further collaborations with the neuro-ophthalmological and optic nerve specialist community, and that we are able to advance knowledge and continue to improve the clinical outcomes for those suffering from this rare disease.”
Additional research studies and clinical trials are necessary to promote the development of new therapies that can ensure better LHON management, the panel of experts suggested in the report.