Clinical profiles and outcomes in patients with inflammatory bowel disease receiving standard and escalated doses of targeted therapies: findings from a global real-world study

Abstract

Objective

Although dosing regimens of targeted therapies (TT) for ulcerative colitis (UC) and Crohn’s disease (CD) are guided by market authorizations and clinical guidelines, little is known about clinical guideline adherence or outcomes in patients receiving escalated doses of TT due to lack of response. This real-world study explored the prevalence of dose escalation and compared outcomes between patients receiving standard and escalated TT doses.

Methods

Data were from the 2020–2021 Adelphi Disease Specific Programme for inflammatory bowel disease, a cross-sectional survey of gastroenterologists and their UC and CD patients across five European countries and the US. Physicians provided retrospective data collection of patient demographics, clinical characteristics, treatment history, and satisfaction; patients reported quality-of-life and work productivity. Patients were grouped by TT maintenance dose; standard and escalated dose groups were compared. Outcomes were adjusted for time on current TT and severity at current TT initiation using regression analyses.

Results

Of 1,241 UC and 1,477 CD patients, 19.1% and 24.1%, respectively, received escalated TT doses. Despite escalation, a substantial proportion of patients had not achieved remission, had moderate or severe disease activity, or were flaring. Most physicians were not fully satisfied with treatment in the escalated dose group and were more likely to switch patients to another treatment regimen than patients on standard dose.

Conclusion

Dose escalation is not always an effective approach to resolve inadequate or loss of response in UC and CD, highlighting a need for more therapeutic options or alternative treatment strategies in patients unresponsive to TT.

Keywords:

• Inflammatory bowel disease
• ulcerative colitis
• Crohn’s disease
• patient outcome assessments
• administration and dosage
• treatment outcome

Transparency

Declaration of funding

Data collection was undertaken by Adelphi Real World as part of an independent survey, entitled the Adelphi Real World IBD DSP. The analysis described here used data from the Adelphi Real World IBD DSP. The DSP is a wholly owned Adelphi product. Eli Lilly and Company is one of multiple subscribers to the DSP. Eli Lilly and Company did not influence the original survey through either contribution to the design of questionnaires or data collection.

Declaration of financial/other relationships

Axel Dignass has received fees for participation in clinical trials, review activities, such as data monitoring boards, statistical analysis, and end point committees from Falk, Abivax, AbbVie, Janssen, Gilead, Celgene/Bristol Myers Squibb, and Pfizer; consultancy fees from AbbVie, MSD, Ferring, Roche/Genentech, Takeda, Vifor, Pharmacosmos, Boehringer Ingelheim, Gilead, Galapagos, Biogen, Celltrion, Falk, Janssen, Pfizer, Sandoz/Hexal, Fresenius Kabi, Celgene/Bristol Myers Squibb, Tillotts, Lilly, Amgen, and Fresenius Kabi; payment from lectures including service on speakers bureaus from Falk Foundation, Ferring, MSD, Amgen, AbbVie, Vifor, Janssen, Pfizer, Tillotts, Takeda, Biogen, Lilly, and Gilead/Galapagos. Isabel Redondo, Petra Streit, Susanne Hartz, Gamze Gurses, and Theresa Hunter are all employees of Eli Lilly and Company, and may hold stock or stock options. Hannah Knight, Sophie Barlow, and Niamh Harvey are employees of Adelphi Real World. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

Axel Dignass: Investigation (supporting); Supervision (equal); Writing – review and editing (equal). Isabel Redondo: Supervision (equal); Writing – review and editing (equal). Petra Streit: Supervision (equal); Writing – review and editing (equal). Susanne Hartz: Supervision (equal); Writing – review and editing (equal). Gamze Gurses: Supervision (equal); Writing – review and editing (equal). Hannah Knight: Investigation (lead); Methodology (equal); Project administration (equal); Resources (equal); Visualization (equal); Writing – review and editing (equal). Sophie Barlow: Formal analysis (lead); Methodology (equal); Software (lead); Validation (lead); Writing – review and editing (equal). Niamh Harvey: Methodology (supporting); Project administration (equal); Resources (equal); Visualization (equal); Writing – review and editing (equal). Theresa Hunter: Conceptualization (lead); Project administration (equal); Supervision (equal); Writing – review and editing (equal). Theresa Hunter is acting as the submission’s guarantor. All authors have approved the final version of the manuscript.

Acknowledgements

Medical writing support (including development of a draft outline and subsequent drafts in consultation with the authors, assembling tables and figures, collating author comments, copy editing, fact checking, and referencing) was provided by K. Ian Johnson BSc, MBPS, SRPharmS, Harrogate House, Macclesfield, UK and funded by Eli Lilly and Company.

Data availability statement

All data, i.e. methodology, materials, data, and data analysis, that support the findings of this survey are the intellectual property of Adelphi Real World. All requests for access should be addressed directly to Hannah Knight ([email protected]).