ESPE Abstracts

Background: Hydroxychloroquine sulfate (HCQS) is an immunomodulatory drug used to treat various rheumatological conditions. However, its effect on preserving β-cell function in type 1 diabetes remains unclear.

Objective: To determine the differences in C-peptide (fasting and post mixed meal) and HbA1c in children with new-onset T1D on hydroxychloroquine therapy versus placebo.

Methods: This single centre, double-blind, randomised control pilot trial recruited patients with recent-onset T1D (diagnosed within 100 days) and assigned them to receive either oral HCQS (3 mg/Kg daily QOD) or a matched placebo for 6 months. The primary outcome measure was the change in fasting and stimulated C-peptide levels from baseline to 6 months. Secondary outcome parameters included changes from baseline for HbA1c, daily insulin dose, glycemic variability, and immune markers such as TNF alpha and IL-10.

Results: A total of 45 children were included in the study, with 23 receiving HCQS and 22 receiving placebo, out of which 18 and 19 children completed the 6-month follow-up, respectively. In the per protocol analysis, the change in fasting C-peptide was higher (∆ Fasting C-peptide=-0.18±0.46 ng/ml) in the HCQS arm as compared to the placebo arm (0.11±0.4 ng/ml), however this difference was not statistically significant (P value=0.053). The change in stimulated C-peptide was also higher in the HCQS arm (∆ stimulated C-peptide=-0.38±0.62 ng/ml) as compared to placebo (-0.14±0.65 ng/ml), but this difference also did not attain statistical significance. The mean fasting C-peptide, stimulated C-peptide, HbA1c, change in HbA1c and mean insulin requirement at 6 months were similar in both the arms. The median IDAA1c levels at 6 months showed a significant difference, 9.04 (8.43-9.71) in HCQS arm as against 9.92 (9.24-10.7) in the placebo arm (p value= 0.035). No serious adverse events were observed in both arms.

Conclusion: The six-month treatment with HCQS in newly diagnosed T1D patients suggested the potential for preserving beta-cell function based on median IDAA1c levels. The fasting and stimulated C-peptide increased (delta change) more in the HCQS arm, although this increase was not statistically significant. At six months, no significant differences were observed in other parameters, including mean fasting and stimulated C-peptide, HbA1c, mean blood sugar levels, or insulin requirement.