Compassionate access anti-tumour necrosis factor-α therapy for ulcerative colitis in Australia: the benefits to patients.
BACKGROUND
The 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-α (TNF-α) therapy in UC.


METHODS
Case notes review of patients with UC who had received compassionate access (CA) anti-TNF-α therapy from prospectively maintained inflammatory bowel disease databases of six Australian adult teaching hospitals.


RESULTS
Patients 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-α therapy was successful in 20/29 patients (69%); in these cases, anti-TNF-α 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-α therapy for ≥4 months, whereas, 27/29 (93%) of ASUC patients had CA for ≤3 months.


CONCLUSIONS
These data show an excellent overall benefit for anti-TNF-α therapy in both ASUC and MRUC. In particular, only short-duration anti-TNF-α 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.
Inflammatory Bowel Disease Service, Department of Gastroenterology and School of Medicine, University of Adelaide at Royal Adelaide Hospital, Adelaide, South Australia, Australia.
S P
SP
Costello
Department of Gastroenterology, St Vincent's Hospital, Sydney, New South Wales, Australia.
S
S
Ghaly
Department of Gastroenterology, The Alfred Hospital, Melbourne, Victoria, Australia.
L
L
Beswick
Department of Gastroenterology, St Vincent's Hospital, Sydney, New South Wales, Australia.
A
A
Pudipeddi
Inflammatory Bowel Disease Service, Department of Gastroenterology and School of Medicine, University of Adelaide at Royal Adelaide Hospital, Adelaide, South Australia, Australia.
A
A
Agarwal
Department of Gastroenterology, Liverpool Hospital, Sydney, New South Wales, Australia.
A
A
Sechi
Department of Gastroenterology, Princess Alexandra Hospital, Brisbane, Queensland, Australia.
S
S
O'Connor
Department of Gastroenterology, Liverpool Hospital, Sydney, New South Wales, Australia.
S J
SJ
Connor
Department of Gastroenterology, The Alfred Hospital, Melbourne, Victoria, Australia.
M P
MP
Sparrow
Department of Gastroenterology, Flinders Medical Centre, Adelaide, South Australia, Australia.
P
P
Bampton
Department of Gastroenterology, St Vincent's Hospital, Sydney, New South Wales, Australia.
A J
AJ
Walsh
Inflammatory Bowel Disease Service, Department of Gastroenterology and School of Medicine, University of Adelaide at Royal Adelaide Hospital, Adelaide, South Australia, Australia.
J M
JM
Andrews
D000328:Adult; D000368:Aged; D001315:Australia / Q000453:epidemiology; D003093:Colitis, Ulcerative / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D057176:Compassionate Use Trials / Q000379:methods*; D005260:Female; D006801:Humans; D000069285:Infliximab / Q000494:pharmacology / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D012189:Retrospective Studies; D016896:Treatment Outcome; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors*
C439524:TNF protein, human; D014409:Tumor Necrosis Factor-alpha; D000069285:Infliximab
colectomy; inflammatory bowel disease; monoclonal antibody; tumour necrosis factor-α; ulcerative colitis