Randomised Clinical Trial to Evaluate the Efficacy of Acetazolamide for the Treatment of Cystoid Fluid Collections in X-Linked Retinoschisis: The AXIS Trial
In brief
Oral acetazolamide fails to thin central retina in X-linked retinoschisis
In a randomized trial of 19 patients (10 treated, 9 controls), 16-week acetazolamide did not change central subfield thickness compared with observation (average difference +2.7 µm). Visual acuity and fluid volume were unchanged, although microperimetry showed a modest functional gain. A few individuals may benefit, suggesting a need for personalized treatment strategies.
- Journal
- Clinical & experimental ophthalmology (Q1)
- Published
- 11 August 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Jonathan Hensman, Hind Almushattat, Roselie M H Diederen, Tien-En Tan, Peilun Dai, Coen de Vente, et al.
- PMID
- 42578811
- DOI
- 10.1111/ceo.70146
Why clinicians should know about it
- Picked for Ophthalmology (top studies of the week, 16 August 2026): Acetazolamide trial in X‑linked retinoschisis, retinal dystrophy
Abstract
BACKGROUND: This trial aimed to evaluate the efficacy of oral acetazolamide in reducing cystoid fluid collections (CFC) and improving visual function in patients with X-linked retinoschisis (XLRS). METHODS: In this investigator-initiated, single centre, open-label, randomised controlled trial, XLRS patients aged ≥ 12 years with fovea-involving CFC at baseline were eligible. Participants were randomised 1:1 to receive either oral acetazolamide (500 mg/day for 16 weeks, followed by tapering or discontinuation based on response) or no treatment (control) for 32 weeks. Seven study visits were completed over 32 weeks. The primary outcome was central subfield thickness (CST). Secondary outcomes included functional and structural measures, as well as patient-reported visual disability. RESULTS: A total of 19 patients were enrolled: 10 received acetazolamide (17 eyes), and nine were monitored without treatment (16 eyes). The difference in CST from baseline up to week 32 between the treatment and control groups was on average +2.7 μm (p = 0.9; 95% CI, -68.1 to 73.5 μm). Retinal sensitivity on microperimetry showed a modest statistically significant improvement favouring acetazolamide. However, no significant improvements were observed for best-corrected visual acuity, low-luminance visual acuity, cystoid fluid collection volume or patient-reported visual disability in the treatment group compared to the control group. CONCLUSION: This study found no clinically meaningful benefit of oral acetazolamide for reducing CFC or improving visual function in the overall XLRS population. Although structural and functional outcomes were comparable between groups overall, a small subset of patients may experience benefit, suggesting a personalised treatment approach may be warranted.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.