Childhood versus early-teenage onset Leber's hereditary optic neuropathy: visual prognosis and capacity for recovery.

Objective: In Leber's hereditary optic neuropathy (LHON) in children and teenagers, the influence of age on visual prognosis has not yet been investigated.
Methods: Patients from the mitoNET registry with LHON onset at age 4-16 years with at least 4 years of follow-up without treatment were included. Visual acuity (VA) at baseline, lowest VA ever recorded (nadir) and VA at end of follow-up were compared between childhood onset (ChO, ≤12 years of age) and early-teenage onset (eTO; 13-16 years).
Results: Out of 231 patients with LHON, 19 met the inclusion criteria (8.2%). There were 11 patients in the ChO and 8 patients in the eTO group. Mean age at onset was 8.6 (SD 2.1) years (ChO) and 15.4 (SD 0.7) years (eTO) (p<0.00001). Follow-up was mean 184 (SD 129) months (ChO) and 119 (SD 78) months (eTO) (p=0.22). Baseline VA was similar between both groups in better (p=0.96) and worse eyes (p=0.54). In worse eyes, both groups deteriorated similarly (p=0.79) until nadir and showed similar recovery until end of follow-up (p=0.38). In better eyes, both groups deteriorated similarly (p=0.16) until nadir. From nadir until end of follow-up, better eyes in the ChO group showed a significantly better recovery (-0.35 (SD 0.36) vs -0.01 (SD 0.06) logMAR; p=0.02) than eTO eyes.
Conclusion: Visual prognosis of LHON in children is much more favourable in cases of childhood onset (≤12 years of age) as compared with teenage onset (13-16 years), mostly due to better recovery from nadir in childhood onset.
Competing Interests: Competing interests: No author reports any financial interest relevant to this publication. JS has received speaker honoraria, consultancy fees and travel expenses from Novartis Pharma GmbH, Bayer AG, Allergan GmbH, Heidelberg Engineering GmbH, Carl Zeiss Meditec AG and Oculentis OSD Medical GmbH, all unrelated to this publication. BS has received previous speaker fees and travel expenses from Novartis Pharma GmbH and Topcon Corporation. TK has received research support, consultancy fees, speaker honoraria and travel funds from GenSight Biologics and Santhera Pharmaceuticals, as well as consultancy fees and speaker honoraria from Chiesi GmbH, all unrelated to this publication. SP has received speaker honoraria, consultancy fees and travel expenses from Novartis Pharma GmbH, Oertli AG, Bayer AG, Alcon Pharma GmbH, Allergan GmbH and Heidelberg Engineering GmbH, all unrelated to this publication. CP has received speaker honoraria from Novartis Pharma GmbH, all unrelated to this publication.
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