TY - JOUR
T1 - Real-world clinical experience with idebenone in the treatment of Leber hereditary optic neuropathy
AU - Catarino, Claudia B.
AU - von Livonius, Bettina
AU - Priglinger, Claudia
AU - Banik, Rudrani
AU - Matloob, Selma
AU - Tamhankar, Madhura A.
AU - Castillo, Lorena
AU - Friedburg, Christoph
AU - Halfpenny, Christopher A.
AU - Lincoln, John A.
AU - Traber, Ghislaine L.
AU - Acaroglu, Gölge
AU - Black, Graeme C. M.
AU - Doncel, Carlos
AU - Fraser, Clare L.
AU - Jakubaszko, Joanna
AU - Landau, Klara
AU - Langenegger, Stefan J.
AU - Muñoz-Negrete, Francisco J.
AU - Newman, Nancy J.
AU - Poulton, Joanna
AU - Scoppettuolo, Elisabetta
AU - Subramanian, Prem
AU - Toosy, Ahmed T.
AU - Vidal, Mariona
AU - Vincent, Andrea L.
AU - Votruba, Marcela
AU - Zarowski, Marcin
AU - Zermansky, Adam
AU - Lob, Felice
AU - Rudolph, Günther
AU - Mikazans, Oskars
AU - Silva, Magda
AU - Llòria, Xavier
AU - Metz, Günther
AU - Klopstock, Thomas
N1 - Copyright the Author(s) 2020. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.
PY - 2020/12/1
Y1 - 2020/12/1
N2 - Background: Leber hereditary optic neuropathy (LHON) leads to bilateral central vision loss. In a clinical trial setting, idebenone has been shown to be safe and to provide a trend toward improved visual acuity, but long-term evidence of effectiveness in real-world clinical practice is sparse. Methods: Open-label, multicenter, retrospective, noncontrolled analysis of long-term visual acuity and safety in 111 LHON patients treated with idebenone (900 mg/day) in an expanded access program. Eligible patients had a confirmed mitochondrial DNA mutation and had experienced the onset of symptoms (most recent eye) within 1 year before enrollment. Data on visual acuity and adverse events were collected as per normal clinical practice. Efficacy was assessed as the proportion of patients with either a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS) of visual acuity. In the case of CRR, time to and magnitude of recovery over the course of time were also assessed. Results: At time of analysis, 87 patients had provided longitudinal efficacy data. Average treatment duration was 25.6 months. CRR was observed in 46.0% of patients. Analysis of treatment effect by duration showed that the proportion of patients with recovery and the magnitude of recovery increased with treatment duration. Average gain in best-corrected visual acuity for responders was 0.72 logarithm of the minimal angle of resolution (logMAR), equivalent to more than 7 lines on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Furthermore, 50% of patients who had a visual acuity below 1.0 logMAR in at least one eye at initiation of treatment successfully maintained their vision below this threshold by last observation. Idebenone was well tolerated, with most adverse events classified as minor. Conclusions: These data demonstrate the benefit of idebenone treatment in recovering lost vision and maintaining good residual vision in a real-world setting. Together, these findings indicate that idebenone treatment should be initiated early and be maintained more than 24 months to maximize efficacy. Safety results were consistent with the known safety profile of idebenone.
AB - Background: Leber hereditary optic neuropathy (LHON) leads to bilateral central vision loss. In a clinical trial setting, idebenone has been shown to be safe and to provide a trend toward improved visual acuity, but long-term evidence of effectiveness in real-world clinical practice is sparse. Methods: Open-label, multicenter, retrospective, noncontrolled analysis of long-term visual acuity and safety in 111 LHON patients treated with idebenone (900 mg/day) in an expanded access program. Eligible patients had a confirmed mitochondrial DNA mutation and had experienced the onset of symptoms (most recent eye) within 1 year before enrollment. Data on visual acuity and adverse events were collected as per normal clinical practice. Efficacy was assessed as the proportion of patients with either a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS) of visual acuity. In the case of CRR, time to and magnitude of recovery over the course of time were also assessed. Results: At time of analysis, 87 patients had provided longitudinal efficacy data. Average treatment duration was 25.6 months. CRR was observed in 46.0% of patients. Analysis of treatment effect by duration showed that the proportion of patients with recovery and the magnitude of recovery increased with treatment duration. Average gain in best-corrected visual acuity for responders was 0.72 logarithm of the minimal angle of resolution (logMAR), equivalent to more than 7 lines on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Furthermore, 50% of patients who had a visual acuity below 1.0 logMAR in at least one eye at initiation of treatment successfully maintained their vision below this threshold by last observation. Idebenone was well tolerated, with most adverse events classified as minor. Conclusions: These data demonstrate the benefit of idebenone treatment in recovering lost vision and maintaining good residual vision in a real-world setting. Together, these findings indicate that idebenone treatment should be initiated early and be maintained more than 24 months to maximize efficacy. Safety results were consistent with the known safety profile of idebenone.
UR - http://www.scopus.com/inward/record.url?scp=85095616673&partnerID=8YFLogxK
U2 - 10.1097/WNO.0000000000001023
DO - 10.1097/WNO.0000000000001023
M3 - Article
C2 - 32991388
AN - SCOPUS:85095616673
SN - 1070-8022
VL - 40
SP - 558
EP - 565
JO - Journal of Neuro-Ophthalmology
JF - Journal of Neuro-Ophthalmology
IS - 4
ER -