Medicina · 5 authors, 4 centres
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This randomized controlled trial assessed hydroxychloroquine (HCQ) versus placebo in 110 patients with age-related macular degeneration (AMD) over 24 months. The main finding was that HCQ was associated with less visual acuity deterioration and reduced drusen growth and number at two years. A key limitation is the relatively small sample size and short follow-up, with no reported adverse effects.
Age-related macular degeneration (AMD) is a leading cause of blindness, and hydroxychloroquine (HCQ) has been hypothesized to have a protective effect based on observations in rheumatoid arthritis patients. Methods: This was a double-blind, randomized controlled trial with 110 patients (55 per group) assigned to receive 400 mg HCQ or placebo daily for 12 months, with follow-up examinations at 3, 6, 9, 12, and 24 months. Outcomes included visual acuity, central macular thickness, and drusen number, size, and volume. Key Results: Of the 110 randomized patients, 96 completed the study (46 in the HCQ group, 50 in the control group). At two years, the HCQ group showed less visual acuity deterioration (−0.03 ± 0.07 vs. −0.07 ± 0.07, p = 0.027). The mean number of drusen per eye was lower for ARDS2 in the HCQ group (8.1 vs. 12.3, p = 0.045). The proportion of eyes with increased drusen growth was smaller in the HCQ group for both ARDS2 and ARDS3, and total drusen growth was lower (70% vs. 80%, p = 0.051). Drusen volume growth was also more reduced in the HCQ group (0.20 ± 0.15 vs. 0.23 ± 0.16 mm^4^, p = 0.058). No HCQ toxicity or adverse effects were reported. Limitations: The study had a relatively small sample size, a single-center design, and a short treatment and follow-up period. The clinical significance of the small visual acuity difference (0.04 logMAR) is uncertain. Implications: The findings suggest HCQ may be associated with reduced drusen progression and visual deterioration in AMD, but further research is needed to confirm these results and determine long-term safety and optimal dosing.
