SAINT-PREX, Switzerland--(BUSINESS WIRE)--Ferring Pharmaceuticals today announces results from the OPTIMISE study showing the first real-world evidence of the effectiveness of a treat-to-target (T2T) approach based on faecal calprotectin (FC) in patients with mild-to-moderate ulcerative colitis (M2M UC). These results have been published in the Journal of Clinical Medicine.1
UC is a chronic, inflammatory bowel condition which can cause recurring bloody diarrhoea, stomach pain and extreme tiredness.4 Of those living with UC, over 85% have mild-to-moderate disease.5,6,7 Usually 5-ASA compounds are the first-line therapy for people with M2M UC, followed by a stepwise treatment approach in case of non-response or intolerance. There is currently limited guidance on timely escalation and de-escalation of therapies.
OPTIMISE was the first major study to investigate whether a T2T approach based on monitoring of non-invasive parameters, such as clinical symptoms and FC, can provide a significantly higher benefit for patients with M2M UC versus an entirely symptom-based approach. “The OPTIMISE study represents another milestone in the management of M2M UC and demonstrates Ferring’s continued commitment to science and improving the lives of people with inflammatory bowel disease (IBD),” said Pierre-Yves Berclaz, Chief Science & Medical Officer, Ferring Pharmaceuticals.
Results from OPTIMISE, a pragmatic, randomised controlled study, showed that people who had their 5-ASA/mesalazine treatment optimised (with or without steroids) by following the T2T approach had a 17–22% advantage at achieving combined endoscopic and clinical remission over a symptoms-only based approach. For the primary endpoint of Mayo Endoscopic Score (MES)=0 at 12 months, no significant difference was found, however it was noted by the investigators that the COVID-19 pandemic negatively impacted the amount of evaluable data.1
“OPTIMISE has provided the first real-world evidence that a T2T approach can help people living with IBD to achieve long-lasting remission and have a greater quality of life,” said Kristine Paridaens, Senior Medical Director, Gastroenterology, Ferring Pharmaceuticals.
A T2T approach has been advocated by the International Organisation for the Study of IBD (IOIBD). In their Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) Consensus they recommended resolution of clinical signs of disease activity and endoscopic remission as the optimal targets.2 This was further refined in STRIDE-II to include symptomatic relief and normalisation of serum and faecal markers as short-term targets for all therapies.3
The OPTIMISE results coincide with an international expert consensus focusing on the practical management of M2M UC, which was published in Expert Review of Gastroenterology and Hepatology.8 Professor Silvio Danese, a co-author of the Consensus and Principle Investigator of the OPTIMISE study, said, “The OPTIMISE study provides real-world evidence of the effectiveness of a T2T approach based on FC monitoring and how its implementation in clinical practice will allow clinicians to tightly monitor disease activity and promptly adapt treatment, helping to avoid complications and disease progression and enabling patients to achieve better disease control.”
To learn more, register here to attend the free ‘New avenues in mild-to-moderate UC treatment optimisation’ webinar on 8th October, 2024. Principal Investigators of the OPTIMISE study, Prof Silvio Danese and Prof Laurent Peyrin-Biroulet, will provide an expert overview of the results and the implications for clinical practice. The webinar is intended for healthcare professionals only.
About the OPTIMISE study1
OPTIMISE was a European-based, multi-centre, randomised (1:1) controlled study of 250 patients with M2M UC (global Mayo score 2–6) treated with ≤2.4g/day 5-aminosalicylic acid that compared the effectiveness of two management strategies with (T2T arm) and without (reference arm) FC home monitoring over 12 months follow-up. Treatment was optimised (escalated or de-escalated) in the T2T arm using FC values and clinical symptoms (PRO-2), whilst the reference arm used only PRO-2. In both arms, therapy was optimised in line with current ECCO guidelines for UC, including maximal doses of 5-ASA (oral and rectal).
A total of 193 patients completed the study despite the acknowledged impact of the COVID-19 pandemic on all clinical studies conducted during that time. Reduced patient contact during the COVID-19 period also affected the availability of data for analysis. For the primary endpoint of MES=0 at 12 months, there was no significant difference between arms, when patients with missing values were classified as non-responders. A subsequent analysis using Monte Carlo Markov Chain (MCMC) imputation found a numerical advantage for the T2T arm over the reference arm for the primary endpoint (37.0% vs 33.4%, respectively). The secondary endpoints, including clinical symptomatology and quality of life, were similarly impacted by missing data, but again displayed numerical superiority for MES≤1, RB=0 and SF≤1 at 12 months when using MCMC imputation.
A logistic regression analysis pooling results for MES, SF and RB at 12 months, using data derived from MCMC imputation, found a statistically significant advantage for the T2T arm over the reference arm (p<0.001). When these endpoints were combined in a fixed effects meta-analysis, the combined endpoint of MES=0, RB=0 and SF≤1 at 12 months was achieved at a significantly higher rate in the T2T than the reference arm (effect size [ES]: 0.17, 95% CI 0.02, 0.32; p<0.05). A similar result was obtained for MES≤1, RB=0 and SF≤1 at 12 months (ES: 0.22; 95% CI 0.07, 0.37; p<0.05).
References
1 Danese S, Fiorino G, Vicaut E, et al. Pragmatic Randomised Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis – the OPTIMISE study. J Clin Med 2024;13:5147.
2 Peyrin-Biroulet L, Sandborn W, Sands BE, et al. Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE): Determining Therapeutic Goals for Treat-to-Target. Am J Gastroenterol 2015;110:1324–38.
3 Turner D, Ricciuto A, Lewis A, et al. STRIDE-II: An Update on the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) Initiative of the International Organization for the Study of IBD (IOIBD): Determining Therapeutic Goals for Treat-to-Target strategies in IBD. Gastroenterology 2021;160:1570–83.
4 NHS. Ulcerative colitis. Available at: https://www.nhs.uk/conditions/ulcerative-colitis/. Last accessed: September 2024.
5 Raine T, Bonovas S, Kucharzik T, et al. ECCO Guidelines on Therapeutics in Ulcerative Colitis: Medical Treatment. J Crohns Colitis. 2022;16(1):2-17.
6 Fumery M, Singh S, Dulai PS, et al. Natural History of Adult Ulcerative Colitis in Population-based Cohorts: A Systematic Review. 2018;16(3):343-356.e3.
7 CCDS Pentasa All formulations. Version 18. 10 December 2022.
8 D'Amico F, Magro F, Dignass A, et al. Practical management of mild-to-moderate ulcerative colitis: an international expert consensus. Expert Rev Gastroenterol Hepatol 2024;18:421–30.
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