Compassionate access anti-tumour necrosis factor- therapy for ulcerative colitis in Australia: the benefits to patients

BackgroundThe efficacy of infliximab has been demonstrated in patients with both acute severe and moderate-severe ulcerative colitis (UC). However, there is a need for real-life data' to ensure that conclusions from trial settings are applicable in usual care. We therefore examined the national experience of anti-tumour necrosis factor- (TNF-) therapy in UC. MethodsCase notes review of patients with UC who had received compassionate access (CA) anti-TNF- therapy from prospectively maintained inflammatory bowel disease databases of six Australian adult teaching hospitals. ResultsPatients either received drug for acute severe UC (ASUC) failing steroids (n = 29) or for medically refractory UC (MRUC) (n = 35). In ASUC, the treating physicians judged that anti-TNF- therapy was successful in 20/29 patients (69%); in these cases, anti-TNF- was able to be discontinued (after 1-3 infusions in 19/20 responders) as clinical remission was achieved. Consistent with this perceived benefit, only 7/29 (24%) subsequently underwent colectomy during a median follow up of 12 months (interquartile range (IQR) 5-16). Eight of the 35 patients with MRUC (23%) required colectomy during a median follow up of 28 months (IQR 11-43). The majority of these patients (20/35 or 57%) had anti-TNF- therapy for 4 months, whereas, 27/29 (93%) of ASUC patients had CA for 3 months. ConclusionsThese data show an excellent overall benefit for anti-TNF- therapy in both ASUC and MRUC. In particular, only short-duration anti-TNF- was required in ASUC. These real-life data thus support the clinical trial data and should lead to broader use of this therapy in UC.