Introduction Anti-tumour necrosis factor (TNF) therapy (infliximab (IFX) or adalimumab (ADA)) has been used as an alternative treatment in patients with Orofacial granulomatosis (OFG) failing conventional therapies, with evidence of promising clinical efficacy. However, concerns regarding safety have been raised and experience is limited. We report on the short and long-term efficacy and safety of anti-TNF therapy for OFG in this case series of 15 patients, the largest reported series to date.

Methods A retrospective study of all patients who received anti-TNF therapy for OFG±Crohn's disease (CD) was performed. Diagnoses of OFG and CD were made on conventional clinical and histological grounds. Demographics, clinical details, oral disease activity scores (ODAS) and laboratory results were obtained from patient records. Patients were considered for induction and maintenance anti-TNF therapy after failing other recognised OFG therapies. Clinical response was assessed at 4 months, 1 and 2 years after anti-TNF initiation. Regular monitoring for adverse effects was performed.

Results Of 207 patients in our OFG clinic database, 15 (9 males) were treated with anti-TNF therapy between 2001 and 2009 (OFG only (n=7), OFG with CD (n=7), OFG with perianal disease (n=1)). Median age at OFG onset and at anti-TNF initiation were 26 years and 36 years. Median time between OFG onset and presentation to clinic was greater for the anti-TNF treated OFG patients than those not requiring anti-TNF (5 vs 2 years, p=0.001). Mean rank ODAS at initial presentation to clinic was higher in anti-TNF treated group than those not requiring anti-TNF (p<0.05).

First line anti-TNF therapy was IFX (n=14) and ADA (n=1). Ten patients received concomitant immunosuppression. Median duration of anti-TNF therapy was 12 months (range; 1 dose to 84 months). Short-term response was achieved in 9/15 (60%) patients. Eight of 15 (53%) and 4/13 (31%) patients remained responders at 1 and 2 years follow-up. Two patients failed IFX therapy and were treated with ADA; one remains responsive at 18 months, the other lost response at 12 months. Oral sulcal involvement was the only predictor for response at 1 and 2 years (p<0.05). Concurrent CD did not predict response. The only adverse event leading to cessation of anti-TNF therapy was a patient with an IFX infusion reaction.

Conclusion Anti TNF therapy has good short and reasonable long-term clinical efficacy (60% and 31%) for patients with OFG failing other therapies. It appears to be safe with a good side effect profile. OFG patients who fail IFX may respond to ADA.