Introduction Current medical options for patients with steroid-dependent, chronic-active ulcerative colitis (UC) with insufficient response or intolerance to immunosuppressants +/- biologicals are limited. This study intended to generate further clinical data on efficacy and identify subpopulations of this difficult-to-treat UC population that would benefit from Granulocyte, Monocyte/Macrophage adsorptive (GMA) apheresis (Adacolumn®).
Method This open-label, multicenter trial was conducted in the UK, France and Germany. 86 consecutive eligible patients (age 18–75) with steroid-dependent active UC (clinical activity index (CAI) ≥6 and Endoscopic Activity Index (EAI) ≥4), and insufficient response or intolerance to immunosuppressants (IS) +/- biologicals were included. Patients received up to 8 GMA aphereses in a single induction series on a weekly basis. Primary endpoint was remission (CAI ≤4) at Week 12; secondary efficacy parameters included clinical response (reduction in CAI ≥3), and steroid-free remission and response rates in the Intention-to-treat (ITT) population. Concomitant medication changes were recorded. 48 weeks results were analysed using the conservative approach. An analysis of sustained remission and sustained response, defined as the respective event observed at Week 12, and at Week 24, and at Week 48, was also performed.
Results In the ITT 85 patients, response rates were 55.95% at week 12, 46.4% at week 24 and 39.3% at week 48. Remission rates were 39.29% at week 12, 34.5% at week 24 and 33.3% at week 48. In 30 patients with prior failure of IS and biologicals, 33.3% were in remission in week 24 and 20% at week 48. Steroid-free remission at week 24 was achieved by 19%, and 15.5% at week 48. Sustained remission or response was seen in 29.8% at 48 weeks. Concomitant IS medication was kept stable, steroid dose equivalents could be reduced, and quality of life improved through to week 48. 30 patients (41.7%) experienced at least 1 AE. 15 subjects experienced SAEs through to Week 48, none considered related to study treatment. Headache was the most prominent AE (21.2% of patients.) No new safety signals were seen.
Conclusion This study describes a cohort of steroid-dependent moderate to severe active UC patients with intolerance to IS and/or biologicals in which treatment with GMA apheresis induction therapy was safe and effective. Benefit was seen in more than 50% of patients at Week 12, in 46.4% at Week 24 and in 39.3% at Week 48.
Disclosure of interest A. Akbar Speaker Bureau of: lecturer fees from Otsuka, A. Dignass Grant/ Research Support from: Investigator fees from Otsuka, Speaker Bureau of: Lecturer fees from Otsuka, B. Bonaz Grant/ Research Support from: Investigator fees from Otsuka, Speaker Bureau of: lecturer fees from Otsuka.