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Excessive monitoring for Hydroxychloroquine retinal toxicity in a trial to delay clinical type 1 diabetes created burden without benefit

Lord, S. M.; Libman, I. M.; Buckner, J. H.; Dansingani, K. K.; Nischal, K. K.; Bingley, P. J.; Speake, C.; You, L.; Greenbaum, C. J.

2025-01-29 endocrinology
10.1101/2025.01.28.25321283 medRxiv
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Background/AimsType 1 Diabetes TrialNet (TrialNet) conducted a placebo randomized trial in children and adults with pre-clinical type 1 diabetes (T1D) to test whether hydroxychloroquine (HCQ) could delay or prevent progression to clinical T1D. Study regulators required more frequent monitoring for retinal toxicity than is recommended by clinical guidelines and we evaluated the impact of this monitoring. MethodsWe obtained the outcome of all eye exams from TrialNet and focused on exams that were recommended for follow up outside the protocol or for repeat testing within the trial. ResultsNo HCQ related retinal toxicity was found, but increased frequency of monitoring led to additional testing due to incomplete exams or technical difficulties, and incidental findings prompted the need for follow up outside the study. ConclusionOur analysis suggests that increased frequency of retinal monitoring provided no benefit, created additional burdens, and that future trials testing HCQ in a pre-clinical T1D population could safely follow standard clinical guidelines for retinal surveillance.

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