Hydroxychloroquine in Stage 1 Type 1 Diabetes.

Document Type

Article

Publication Date

11-1-2023

Identifier

DOI: 10.2337/dc23-1096; PMCID: PMC10620539

Abstract

OBJECTIVE: Innate immune responses may be involved in the earliest phases of type 1 diabetes (T1D).

RESEARCH DESIGN AND METHODS: To test whether blocking innate immaune cells modulated progression of the disease, we randomly assigned 273 individuals with stage 1 T1D to treatment with hydroxychloroquine (n = 183; 5 mg/kg per day to a maximum of 400 mg) or placebo (n = 90) and assessed whether hydroxychloroquine treatment delayed or prevented progression to stage 2 T1D (i.e., two or more islet autoantibodies with abnormal glucose tolerance).

RESULTS: After a median follow-up of 23.3 months, the trial was stopped prematurely by the data safety monitoring board because of futility. There were no safety concerns in the hydroxychloroquine arm, including in annual ophthalmologic examinations. Preplanned secondary analyses showed a transient decrease in the glucose average area under the curve to oral glucose in the hydroxychloroquine-treated arm at month 6 and reduced titers of anti-GAD and anti-insulin autoantibodies and acquisition of positive autoantibodies in the hydroxychloroquine arm (P = 0.032).

CONCLUSIONS: We conclude that hydroxychloroquine does not delay progression to stage 2 T1D in individuals with stage 1 disease. Drug treatment reduces the acquisition of additional autoantibodies and the titers of autoantibodies to GAD and insulin.

Journal Title

Diabetes care

Volume

46

Issue

11

First Page

2035

Last Page

2043

MeSH Keywords

Humans; Hydroxychloroquine; Diabetes Mellitus, Type 1; Autoantibodies; Insulin; Glucose

Keywords

Hydroxychloroquine; Diabetes Mellitus, Type 1; Autoantibodies; Insulin; Glucose

Comments

Grants and funding

Link to Full Text
Library Record

Share

COinS