PurposeTo report the long-term clinical outcomes for patients with neovascular age-related macular degeneration (nAMD) who received anti-vascular endothelial growth factor (anti-VEGF) therapy as part of a standardised treatment protocol in a real-world setting.Patients and methodsThis is a retrospective audit of all treatment-naïve patients with nAMD who commenced a pro re nata (PRN) treatment regimen of intravitreal Ranibizumab from January to December 2009 and completed 8 years of follow-up in one single-treatment centre. Electronic medical notes were reviewed to evaluate the outcome measures. Outcome measures included progression of visual acuity (VA), central retinal thickness (CRT) and treatment frequency.Results95 eyes from 86 patients had complete data for 8 years of follow-up. Baseline median CRT was 295µm [IQR 254–349] and improved to 209µm [IQR 182–254] in year 8 (p<0.001); baseline median VA was 61 ETDRS letters which increased to 70 letters post-loading however was reduced to 55 letters by year 8 (mean VA change from baseline was −9.1 letters); 47.4% had stable or improved vision, 10.5% gained ≥15 letters and 33.7% had lost ≥15 letters. The highest visual gain was achieved after the initial loading-phase, with a subsequent steady decline, 26.3% (compared to baseline 33.4%) achieved driving vision standard. Median injection frequency was 6 (range 3–10) in year 1 and 3 injections (range 0–10) in year 8. 51.6% of eyes required at least one injection each year and only 34.7% required no injections in year 8.ConclusionOur real-world nAMD treatment cohort using Ranibizumab PRN regimen achieved an encouraging almost 50% stable or improved VA at year 8 and total injections of 31.6 injections per patient over an 8-year period.
To report long-term efficacy and treatment outcomes of the combination therapy for treating macular oedema (MO) in retinal vein occlusions (RVOs) from a real-world UK practice. Methods: The initial reported 66 RVO patients with MO treated with combination therapy (initial Ranibizumab, later optional addition of Ozurdex and laser) were followed up to Year 3: visual acuity (VA) and central retinal thickness (CRT) were analysed against baseline and previous Year 1 results. Safety and adverse events were also recorded. Results: Baseline LogMAR VA of 0.71 (Snellen 6/30) improved to 0.48 (Snellen 6/18) at Year 3 (p=0.006); 63% experienced VA improvement (40% improved ≥3 lines), 27% had worse vision. Stability of mean VA (6/18) was already achieved at first post-loading phase review and was maintained in each subsequent year. Statistically significant CRT improvement was noted in each year (Year 3 median CRT=264µm) compared to baseline (median CRT=531µm). There was a reduction in the mean number of total injections to 2.5 in Year 3 (vs 5.5 in Year 1). Comparing Year 3 against Year 1, mean Ranibizumab injection frequency was 2.1 vs 4.3; mean Ozurdex injection frequency was 0.2 vs 1.1. In Year 3, 39.6% of patients did not require any form of injections, laser frequency was also reduced to 22.9% (vs 81.8% in Year 1). There was no endophthalmitis in the cohort, one progressed to neovascular glaucoma in Year 2 and mortality rate was recorded as 6%. Conclusion: Our real-world clinical practice for RVO patients using a combined therapy is associated with good long-term VA and anatomical outcomes with less intravitreal retreatment rates.
Retinitis pigmentosa (RP) relates to a heterogeneous group of rod-cone dystrophies of varying genetic aetiology. There is currently great interest in gene replacement therapy. Phenotyping is of particular importance because some RP genes are expressed ubiquitously and it is critically important to understand which retinal layer is primarily affected. RP2 is increasingly diagnosed in patients suffering from X-linked RP, which causes outer retinal degeneration. We present a case of a previously unreported null mutation in RP2 associated with severe RP. Loss of the retinal pigment epithelium (RPE) was noted in the central macula but not around the disc or peripherally. There was therefore no evidence of independent degeneration of the RPE. Hence despite expression in all retinal cells, RP2 deficiency does not appear to be pathogenic to the RPE. This observation may be helpful in considering the promoter and route of delivery of adeno-associated viral gene therapy vectors encoding RP2.
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