Ferring Demonstrates Effectiveness of a Treat-to-Target Approach in Mild-to-Moderate Ulcerative Colitis in First Major Study
October 01 2024 - 3:15AM
Business Wire
- OPTIMISE study found a treat-to-target (T2T) management
strategy in people with mild-to-moderate ulcerative colitis (M2M
UC) being treated with 5-aminosalicylic acid (5-ASA; mesalazine)
utilising faecal calprotectin (FC) home monitoring over 12 months
achieved a significantly higher rate of endoscopic and clinical
remission compared to a symptom-based approach which did not
utilise FC home monitoring.1
- These data support clinical thinking that T2T is the preferred
management strategy for inflammatory bowel disease (IBD)2,3 and
demonstrates that mesalazine therapy is an integral part of this
approach in M2M UC.
- Further details of the study results to be shared by Principal
Investigators of the OPTIMISE study, Prof Silvio Danese and Prof
Laurent Peyrin-Biroulet, in an upcoming webinar on 8th October
2024.
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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trademarks of the Ferring group of companies.
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Matthew Worrall Director, Corporate Communications,
Ferring +44 7442 271 811 Matthew.Worrall@ferring.com