Efficacy and safety of thalidomide in the treatment of recurrent aphthous stomatitis: A systematic review

doi:10.5246/jcps.2025.08.056

Abstract

Abstract:

Thalidomide, an immunomodulatory drug, is widely recommended for the treatment of recurrent aphthous stomatitis (RAS). This review aimed to assess the reliability of thalidomide for managing RAS, oro-genital ulcers associated with Behçet’s disease (BD), and RAS in individuals with HIV infection. A systematic review was conducted following PICOS (Patient, Intervention, Control, Outcome, Study design) principles. Given the heterogeneity across studies, a qualitative analysis was performed in place of a meta-analysis. Eight randomized controlled trials (RCTs) were deemed eligible for inclusion. In three RCTs focused on RAS, a dosage of thalidomide at 100 mg/d demonstrated efficacy, while a lower dose of 25 mg/d helped prolong the recurrence interval of RAS. For oro-genital ulcers of BD, two RCTs indicated that both 300 mg/d for 24 weeks and 200 mg/d for 28 d, preceded by an initial dose of 400 mg/d for 5 d, were effective. In three RCTs investigating RAS in HIV-infected patients, thalidomide at 200 mg/d for either 4 or 7 weeks, with an initial dose of 400 mg/d for the first week, proved effective. However, a regimen of 100 mg three times per week failed to prevent the recurrence of oral ulcers. Adverse reactions to thalidomide were generally tolerable within the dosage ranges used in these studies. Overall, thalidomide showed promising efficacy for treating RAS, oro-genital ulcers in BD, and RAS in HIV-infected individuals. However, the variability in trial designs, dosages, and treatment durations makes it challenging to recommend an optimal dose and course of therapy. Further high-quality RCTs are necessary to establish more definitive guidelines.

Key words: Recurrent aphthous stomatitis, Thalidomide, Behcet’s disease, HIV infection, Systematic review

Supporting: attached/file/20250828/20250828170629_522.pdf

Jiao Yue, Jia Ju, Xinwen Wang, Yao Lin, Jing Huang, Yanfei Ma, Shuibing Liu, Bin Feng, Lifei Cheng. Efficacy and safety of thalidomide in the treatment of recurrent aphthous stomatitis: A systematic review[J]. Journal of Chinese Pharmaceutical Sciences, 2025, 34(8): 755-766.

Figures/Tables 5

Table 1. Summary of studies focused on the treatment effect of thalidomide against RAS.

Table 2. Summary of studies focused on the treatment effect of thalidomide against oro-genital ulcers of BD.

Table 3. Summary of studies focused on the treatment effect of thalidomide against RAS in HIV infection.

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