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. 2024 Nov 20:17:17562848241293938.
doi: 10.1177/17562848241293938. eCollection 2024.

Effectiveness and safety outcomes after long-term (54 weeks) vedolizumab therapy for Crohn's disease: a prospective, real-world observational study including patient-reported outcomes (POLONEZ II)

Ariel Liebert  1 , Maria Kłopocka  1 , Agata Michalak  2 , Halina Cichoz-Lach  2 , Renata Talar-Wojnarowska  3 , Danuta Domz Ał-Magrowska  3 , Łukasz Konecki  4 , Aleksandra Filipiuk  4 , Michał Krogulecki  4 , Maria Kopertowska-Majchrzak  5 , Kamila Stawczyk-Eder  6 , Katarzyna Waszak  6 , Piotr Eder  6 , Edyta Zagórowicz  7   8 , Izabela Smoła  9 , Krzysztof Wojciechowski  10 , Szymon Drygała  11
Affiliations

Effectiveness and safety outcomes after long-term (54 weeks) vedolizumab therapy for Crohn's disease: a prospective, real-world observational study including patient-reported outcomes (POLONEZ II)

Ariel Liebert et al. Therap Adv Gastroenterol. .

Abstract

Background: The Crohn's Disease (CD) Activity Index (CDAI), Inflammatory Bowel Disease (IBD) Questionnaire (IBDQ), and IBD-Fatigue (IBD-F) scale are useful patient-reported outcome (PRO) tools for assessing the treatment benefits of vedolizumab (VDZ) beyond clinical trial endpoints in patients with CD.

Objectives: To assess clinical response, clinical remission, steroid-free remission, changes from baseline for PROs, and safety in a real-world cohort of patients with moderate-to-severe active CD treated with VDZ.

Design: POLONEZ II was a multicenter, observational, prospective study across 10 Polish centers from April 2020 to October 2023 for 54 weeks in patients with CD eligible for reimbursed VDZ.

Methods: Primary endpoints at week 54 (W54) were clinical response (⩾70-point reduction in CDAI and >25% reduction vs baseline), remission (CDAI score ⩽150), and steroid-free remission. Other outcomes were changes in PROs (CDAI score and health-related quality of life) and safety. Kaplan-Meier survival analyses were performed.

Results: Of 98 patients with CD, the mean age was 35.2 years, 57.1% were male, and 72.4% had an ileocolonic disease. At W54 (n = 98), 63.3% of patients achieved clinical response, 48.0% remission, and 36.0% steroid-free remission. The durability of clinical response, remission, and steroid-free remission (W14-W54) were 68.9%, 62.9%, and 57.1%, respectively. By W54, a significant reduction in the PROs, such as the total CDAI score (p < 0.001), stool frequency (p < 0.001), abdominal pain score (p < 0.001), IBDQ (p < 0.001), IBD-F (p < 0.05), and fatigue impact on daily activities (p < 0.001), was observed. During VDZ treatment, arthralgia (23.7%-8.7%) and anemia (22.6%-15.9%) decreased between baseline and W54. Non-serious adverse events (SAEs; 12.2%), SAEs (7.1%), and VDZ-related rash (1.0%) were reported. Mean CD-related hospitalization duration decreased from baseline (10.2 days) to the end of the study (5.3 days).

Conclusion: POLONEZ II demonstrated long-term real-world benefits of VDZ toward effectiveness, safety, and improved PROs and patients' quality of life.

Trial registration: ENCePP (EUPAS32716).

Keywords: Crohn’s disease; Inflammatory Bowel Disease Questionnaire; Inflammatory Bowel Disease-Fatigue self-assessment scale; anti-integrin monoclonal antibody; patient-reported outcomes; quality of life; real-world prospective analysis; response durability; steroid-free remission; vedolizumab.

Plain language summary

Effect of vedolizumab treatment on Crohn’s disease and fatigue The goal of this study was to thoroughly assess the effectiveness and safety of vedolizumab (VDZ), a monoclonal antibody, as a treatment for Crohn’s disease (CD), a weakening inflammatory gut condition. This study used a selection of assessment tools to measure patient-reported outcomes (PROs), which are commonly used to evaluate the benefits of a treatment in terms of features important to patients. These tools included the CD Activity Index (CDAI), which measures disease activity; the Inflammatory Bowel Disease (IBD) Questionnaire (IBDQ), which evaluates patients’ quality of life; and IBD-Fatigue (IBD-F), which assesses fatigue. Medical records of patients treated with VDZ for moderate to severely active CD for 54 weeks were examined. Treatment outcomes showed marked improvements, with 63.3% of patients showing a favorable response and fewer CD symptoms. Approximately 48.0% of patients achieved clinical remission, showing better control of their symptoms and a long period of symptom improvement. Notably, the benefits of the treatment continued during the treatment period, and a total of 57.1% of patients were corticosteroid-free while maintaining good outcomes. From the start of the trial until W54, the CDAI, IBDQ, and IBD-F scores decreased, likely owing to a reduction in disease symptoms. Patients also described decreased abdominal pain and a lower stool frequency. Furthermore, VDZ treatment reduced fatigue, a common problem in CD. Side effects were reported in 7.1% of patients with CD, with only one patient reporting a VDZ-related rash. Although some patients had to stop using VDZ due to the ineffectiveness of treatment, the majority of patients (67.3%) continued their treatment. Overall, VDZ effectively reduced CD symptoms and improved the patients’ quality of life while showing clinical effectiveness and a safety profile consistent with the approved label.

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Conflict of interest statement

A.L. received payment for lectures from Janssen, Takeda, Egis, AbbVie, and Pharmabest, and travel/accommodation/meeting expenses from Janssen, Takeda, Egis, and AbbVie. A.M. received lecture fees from Takeda. M.Kłopocka received payment for lectures from Janssen, Takeda, Ferring, Alfasigma, and Pharmabest, and travel/accommodation/meeting expenses from Ferring, Janssen, Takeda, Alfasigma, and Pharmabest. H.C.-L. received lecture fees from Takeda. R.T.-W. received lecture fees and/or travel grants from AbbVie, Astellas, Ferring, Janssen, and Takeda. A.F. received payment for lectures from Janssen and Pfizer, and travel/accommodation/meeting expenses from Takeda and Janssen. K.S.-E. received travel grants and lecture fees from Janssen, Pfizer, and Takeda. K. Waszak received payment for travel/accommodation/meeting expenses from Janssen-Cilag, Pfizer, PRO.MED.CS, Samsung Bioepis, and Takeda. P.E. received lecture fees and/or travel grants from Takeda, Ferring, Astellas, Pfizer, and Janssen. E.Z. received lecture fees from Janssen, Sandoz, Ferring, and Pfizer; consultancy fees from Pfizer, Janssen, and Takeda; and other compensations from Takeda and Janssen. S.D. is a former employee of Takeda Pharma Sp. z o.o. and is now an employee of AstraZeneca. K. Wojciechowski is a former employee of Takeda Pharma Sp. z o.o. and currently an invited external lecturer for Takeda. He is now an Independent Public Health Care Center employee in Tarczyn, Warszawska 42, Tarczyn, Poland. D.D.-M., Ł.K., M. Krogulecki, M.K.-M., and I.S. declare no conflicts of interest.

Figures

Figure 1.
Figure 1.
The CDAI total score for disease activity and clinical response assessment with CDAI (FAS). (a) Overall. (b) Biologic naive. (c) Biologic exposed. (d) Biologic failure. Boxes correspond to median values and interquartile ranges, and error bars represent minimum–maximum values. W54−W14 difference: The 95% CI was calculated using Wilson’s method. p-Values were calculated using the proportion test and adjusted using the Bonferroni–Holm approach. ***p < 0.001. CDAI, Crohn’s Disease Activity Index; CI, confidence interval; FAS, full analysis set; N, number of patients; NS, not significant; W, week.
Figure 2.
Figure 2.
Primary endpoints of disease activity and assessment at W14 and W54. (a) Clinical response based on the pMayo score. (b) Clinical remission based on the pMayo score. (c) Steroid-free remission assessment using CDAI (FAS). Clinical response was defined as a reduction in the CDAI score ⩾70 points and >25% compared with the baseline value at W14 (or any of the following visits). Clinical remission was defined as achieving a CDAI total score of <150 points. Steroid-free remission was defined as achieving a CDAI total score <150 points in patients not using steroids at the time of assessment but who were using them at baseline. W54−W14 difference: *p < 0.05. **p < 0.01. ***p < 0.001. CDAI, Crohn’s Disease Activity Index; FAS, full analysis set; N, number of patients; NS, not significant; pMayo, partial Mayo; W, week.
Figure 3.
Figure 3.
(a) Corticosteroid intake up to W54 and (b) CS dose reduction at W54 (responders). Bar charts showing corticosteroid intake for up to 54 weeks (responders, n = 66). The numbers indicate the percentage of patients taking different corticosteroids (responders). A ~1.6-fold increase was observed in patients who did not use concomitant corticosteroids. Of the responders who had corticosteroid dose reduction (83.3%, n = 30/36), 73.3% (n = 22/30) had 100% dose reduction (CS-free). CS, corticosteroids; FAS, full analysis set; W, week.
Figure 4.
Figure 4.
Kaplan–Meier analysis of treatment persistence (FAS). The Kaplan–Meier curve illustrates the probability of VDZ persistence over a 54-week period. Initially, a slight decline of approximately 15%–20% in persistence was observed during the early weeks of treatment, specifically up to W15. However, following this initial decrease, the persistence rate stabilized and remained generally consistent until the end of W54. The number of patients at risk of relapse decreased from W0 to W54. FAS, full analysis set; VDZ, vedolizumab.
Figure 5.
Figure 5.
Effect of VDZ on the IBDQ scores (overall FAS). The effect of VDZ induction therapy on IBDQ total score. (a) Overall. (b) Biologic naïve. (c) Biologic exposed. (d) Biologic failure. Boxes correspond to median values and interquartile ranges, and error bars represent minimum−maximum values. *p < 0.05. **p < 0.01. ***p < 0.001. FAS, full analysis set; IBDQ, Inflammatory Bowel Disease Questionnaire; NS, not significant; VDZ, vedolizumab; W, week.
Figure 6.
Figure 6.
Effect of VDZ on the IBDQ scores (overall FAS). The effect of VDZ induction therapy on (a) bowel symptoms, (b) systemic symptoms, (c) emotional function, and (d) social function. Boxes correspond to median values and interquartile ranges, and error bars represent minimum−maximum values. *p < 0.05. **p < 0.01. ***p < 0.001. FAS, full analysis set; IBDQ, Inflammatory Bowel Disease Questionnaire; NS, not significant; VDZ, vedolizumab; W, week.
Figure 7.
Figure 7.
Effect of VDZ on fatigue severity and frequency (IBD-F Questionnaire). (a) Overall. (b) Biologic naïve. (c) Biologic exposed. (d) Biologic failure. Boxes correspond to median values and interquartile ranges, and error bars represent minimum−maximum values. *p < 0.05. ***p < 0.001. FAS, full analysis set; IBD-F, Inflammatory Bowel Disease-Fatigue; NS, not significant; VDZ, vedolizumab; W, week.
Figure 8.
Figure 8.
Effect of VDZ on fatigue impact on daily activities (IBD-F Questionnaire). (a) Overall. (b) Biologic naïve. (c) Biologic exposed. (d) Biologic failure (FAS). Boxes correspond to median values and interquartile ranges, and error bars represent minimum−maximum values. *p < 0.05. **p < 0.01. ***p < 0.001. FAS, full analysis set; IBD-F, Inflammatory Bowel Disease-Fatigue; NS, not significant; VDZ, vedolizumab; W, week.
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