Real-world data of enzyme replacement therapy with pabinafusp alfa for neuronopathic MPS-II: Updated clinical data from Japan

To assess anatomic and visual outcomes of ocriplasmin use for symptomatic vitreomacular adhesion (VMA).Retrospective chart review.Macula Society members were surveyed online to collect data on ocriplasmin for symptomatic VMA. Clinical and optical coherence tomography data were collected using standardized data entry forms.There were 208 patients (208 eyes) with symptomatic VMA followed at least 3 weeks after receiving ocriplasmin. At baseline, VMA was focal (<1500 μm) in 179 eyes (86%), broad in 9 eyes (4%), and not reported in 20 eyes (10%). A full-thickness macular hole (MH) was present in 75 eyes (36%); size was <400 μm in 62 eyes (82%). Baseline mean visual acuity was approximately 20/63. Of the 204 eyes with ≥12 weeks follow-up, pars plana vitrectomy (PPVx) was performed in 12 (6%) by 4 weeks, 31 (15%) by 12 weeks, and 64 (31%) by the last visit. VMA had resolved by 12 weeks with ocriplasmin alone in 83 of 191 eyes (43%) by week 12 and in 148 of 200 eyes (74%) by the last visit, including eyes undergoing PPVx. Among eyes with a baseline MH, closure was achieved with ocriplasmin alone in 10 of 65 (15%) by 1 week, 26 of 74 (35%) by 4 weeks, and 30 of 75 (40%) at the last visit. Mean change in visual acuity at the last visit compared with baseline was −0.06±0.40 logarithm of the minimum angle of resolution (logMAR) (modest vision improvement) (P = 0.03). At the last visit, visual acuity improved by ≥2 lines in 69 eyes (35%) and by ≥3 lines in 54 eyes (27%). Visual acuity decreased ≥2 lines in 35 eyes (18%) and by 3 lines in 27 eyes (14%) at the final visit. Complications included photopsias (15%), dimness of vision (14%), decreased color vision (10%), MH development (5%), macular retinal pigment epithelium atrophy (2.7%), retinal detachment (1.9%), and retinal tear (1.4%). No endophthalmitis cases were reported.Physician-reported outcomes on ocriplasmin use confirmed VMA release in 45% and closure of MH in 40% of eyes without PPVx. Visual acuity decreased in approximately 20% of eyes. Adverse events were not infrequent and suggest caution when considering ocriplasmin use.