Efficacy of azathioprine (AZA) in refractory ulcerative proctitis (UP) is unknown.
All patients treated with AZA for refractory UP in three referral centers between 2002 and 2012 were included. "Treatment success" in the long-term was defined as the absence of colectomy during follow-up, no need for anti-TNF during follow-up, no ongoing systemic steroids use, no adverse event leading to AZA withdrawal, and clinically quiescent disease at last follow-up.
Of the 1279 adult patients with ulcerative colitis, 25 patients were treated with AZA for refractory UP (median disease duration 4.9 years). Of these, 4 had no short-term clinical assessment. Of the remaining 21, 4 were primary non responders to AZA, 7 discontinued AZA for adverse events and 10 showed clinical improvement. At the long-term assessment at last follow up after a median of 46 months, 5 patients had treatment success and were still on AZA treatment, the remaining 20 were treatment failures. Of these, 5 discontinued AZA for adverse events and 15 were treated with infliximab (clinical response in 11 patients, primary non-response in one patient, and 3 underwent colectomy).
AZA may be efficacious in maintaining clinical response in one-fifth of patients with refractory UP in a real-life setting.