Real-World Studies of Ocriplasmin Show Different Outcomes Than Trials

Ocriplasmin was significantly more effective in achieving nonsurgical vitreomacular adhesion resolution (nsVMAR) in studies conducted in real-world settings than it was in randomized controlled trials (RCT), researchers found in a systematic literature review and meta-analysis of its use published in Acta Ophthalmologica. Epiretinal membrane (ERM) in real-world settings was the only significant explanatory variable for the difference between study types.

The researchers sought to demonstrate whether careful patient selection based on certain risk factors — such as presence and size of macular hole (MH) — may make ocriplasmin more effective in real-world settings.

The researchers reviewed 30 studies from real-world settings (2402 patients and 2416 eyes) on the effectiveness of single 125 µg doses of ocriplasmin to treat adults with vitreomacular traction (VMT), both with (MH group) and without MH (VMT group). They also looked into 5 double-blind RCTs (737 patients) on single 125 µg doses vs control in the treatment of adults with VMT/symptomatic VMA. Month 6 was selected as the evaluation time-point for comparison with the real-world settings. Studies had to report either primary VMAR (pVMAR) or nsVMAR outcomes, have a mean follow-up of at least 4 weeks after injection, and include at least 12 patients in the series.

The researchers compared the outcomes between the studies from real-world settings and the RCTs at the 26-week evaluation time-point of the RCTs and found that nsVMAR outcomes were significantly better in real-world settings than in RCTs (odds ratio (OR) 1.66; 95% confidence interval (CI): 1.18-2.34; P =.004). NsVMAR rates were inversely associated with ERM prevalence (OR 0.20; 95%CI: 0.08–0.51, P =.001). The crude prevalence of ERM in RCTs was 263 out of 737 eyes (35.7%) while 261 patients had ERM out of 1771 (14.7%) in the real-world settings studies.

The researchers also compared reported adverse drug reactions (ADRs) from the real-world setting studies with the OASIS RCT (ClinicalTrials.gov Identifier: NCT01429441) and found that photophobia and vitreous floaters were reported at a lower rate in the real-world settings (3.9% of patients vs 22.4% of patients, respectively) than they had been in the OASIS trial (13.0% of patients vs 37.7% of patients, respectively). However, new or worsening subretinal fluid (SRF) was reported twice as frequently in real-world settings than in the OASIS trial. This finding was reported in 22.8% of patients in real-world settings and 100 of 104 cases resolved in the studies that reported resolution rates — while it was reported in 11.6% of patients in the OASIS trial.

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