Long-term effectiveness of intralesional triamcinolone acetonide therapy in orofacial granulomatosis: an observational cohort study

SUMMARY: BACKGROUND: It has been suggested that intralesional triamcinolone injections represent a safe and effective therapeutic strategy in controlling the permanent disfiguring swelling of orofacial granulomatosis (OFG). However, robust supporting evidence is lacking, due to the variable and inconsistent design of available studies. OBJECTIVES: To investigate whether a standardized regimen of intralesional triamcinolone has beneficial long-term effects on orofacial swelling of OFG. We also studied potential associations with a number of prognostic factors. METHODS: We designed a retrospective observational study of a homogeneous cohort of 22 well-phenotyped patients with OFG. The primary outcome was defined as a statistically significant decrease in post-treatment disease severity. Statistically significant association with prognostic factors was the secondary outcome. Statistical analysis included Wilcoxon signed-rank tests and logistic regression. RESULTS: Compared with pretreatment, there were statistically significant decreases in disease severity scores at all time points until 48 months post-treatment (P < 0·01). Logistic regression analysis showed there was no independent prognostic variable of statistical significance (P > 0·05). The majority of patients (14/22, 63·6%) received one course of intralesional triamcinolone and did not experience disease recurrence. The mean disease-free period after the first course of intralesional therapy was 28·9 ± 18 months (95% confidence interval 28·7–29·1). No adverse effects were reported. CONCLUSIONS: This is the first study to have employed robust cohort methodology and sound statistics to demonstrate long-term effectiveness of intralesional triamcinolone in controlling the disfiguring swelling of OFG. Because of limitations inherent in observational studies, further research in the form of randomized case-control trials is needed to confirm the present findings. WHAT'S ALREADY KNOWN ABOUT THIS TOPIC? It has been suggested that intralesional corticosteroid therapy is effective in controlling the permanent disfiguring swelling of orofacial granulomatosis (OFG); however, robust supporting evidence is lacking due to the variable and inconsistent design of available studies. . WHAT DOES THIS STUDY ADD? This is the first cohort study on intralesional therapy to employ robust cohort design, consistent methodology and a standardized regimen of triamcinolone injections. It provides reliable evidence of long-term effectiveness in reducing the orofacial swelling of OFG.