Table 2 Summary of real-world data from a retrospective analysis of cohorts comprising at least four patients.
Authors, Year, Ref | Study design | Patient N, age range | Baseline characteristics | Follow up time | Main findings |
|---|---|---|---|---|---|
Stingl et al. [27] | Single centre | 5; 14–36 years | Patient’s eligibility to treatment in accordance with the German Society of Ophthalmology recommendations Clinical diagnosis: EOSRD; BCVA (decimal) range FC-0.2; FST blue average (dB): −3.39; FST red average (dB); 0.83; DAC blue average (dB): −2.91; DAC red average (dB): −1.01; CPC rods average (relMCA): 0.31; CPC cones average (relMCA): 4.12 | 3 months | VA improved or remained stable in all eyes Age as strong prediction factor with respect to blue light FST gain and the average of the DAC stimuli (cyan) and moderate with respect to the increase of the average macular scotopic CPC response Local retinal volume as a moderate predictor with respect to scotopic CPC response The improvement in the rod function (by CPC) depended on patients’ age and remaining retinal thickness |
Sengillo et al. [16] | Multicentre | 41; 2–44 years | Paediatric eyes mean BCVA 20/150 (20/40-CF); adult eyes mean BCVA 20/260 (20/70-LP); mean CFTs: 210 mm and 176 mm in paediatric and adult patients, respectively Mean white light FST score: 0.6 ± 3.7 dB | Mean 10 months (range 1 week–18.5 months) | 3/4 of eyes remained within one line of baseline BCVA. No significant difference in the mean BCVA changes in adult vs paediatric eyes. Baseline VA did not affect postoperative acuity changes BCVA change from baseline in eyes with and without foveal detachment: no significant difference at follow-ups Mild thinning in mean CFT both in paediatric and adult eyes regardless of intraoperative foveal detachment or age FST mean improvement of 21.1 ± 16.6 dB |
Deng et al.[17] | Single centre | 14; 4–17 years | Mean FST: −2.0 log cd.s/m2 Mean CST: 215 μm (range: 192–247) Mean VA: log- MAR 0.98 (range 0.40–1.70; Snellen equivalent, 20/191) | Median 513 days (167–677 days) | Significant improvements in FST, expansion of III4e isopter, BCVA Significant decrease in mean CST Sub-foveal ONL thickness stable over time |
Testa et al.[39] | Single centre | 6; 7–17 | Mean BCVA: 0.70 ± 0.08 logMAR (Snellen equivalent of 20/100) Reduced central foveal retinal thickness, central foveal ONL thickness and mean ONL thickness of the internal ETDRS ring compared to age-matched healthy eyes | 6 months | Significant change in VA and increase of ONL thickness in the internal ETDRS ring BCVA change positively associated with ONL thickness increase in the internal ETDRS ring BCVA improved irrespective of intra-operative foveal detachment Central foveal ONL thickness decrease associated with intra-operative foveal detachment. ONL thickness of the internal ETDRS ring change not associated with intra-operative foveal detachment |
Gerhardt et al. [38] | Single centre | 4; 3–6 years | Clinical diagnosis: LCA BCVA range: 1.3–0.7 logMAR. Nystagmus Lack of 488 nm autofluorescence in all patients Mobility test failed below 40 lux. III/4 isopter in Goldmann VF testing: not disclosed ERGs and 30 Hz flicker ERGs unrecordable FST reliable in one patient (−9.5 dB) | Mean 18.5 months | Statistically and clinically significant VA improvement at month 6 Damped or amplitude reduced nystagmus Mobility test passed at 4 lux Measurable III4e isopter in three patients Post treatment flicker ERGs recordable in two patients Post-treatment FST measured in two patients and remarkably improved by at least 30 dB in each eye at month 6 |
