Ariel Liebert, Maria Kłopocka, Agata Michalak, Halina Cichoz-Lach, Renata Talar-Wojnarowska, Danuta Domz Ał-Magrowska, Łukasz Konecki, Aleksandra Filipiuk, Michał Krogulecki, Maria Kopertowska-Majchrzak, Kamila Stawczyk-Eder, Katarzyna Waszak, Piotr Eder, Edyta Zagórowicz, Izabela Smoła, Krzysztof Wojciechowski, Szymon Drygała
{"title":"长期（54 周）维多利珠单抗治疗克罗恩病的疗效和安全性结果：一项包括患者报告结果在内的前瞻性真实世界观察研究（POLONEZ II）。","authors":"Ariel Liebert, Maria Kłopocka, Agata Michalak, Halina Cichoz-Lach, Renata Talar-Wojnarowska, Danuta Domz Ał-Magrowska, Łukasz Konecki, Aleksandra Filipiuk, Michał Krogulecki, Maria Kopertowska-Majchrzak, Kamila Stawczyk-Eder, Katarzyna Waszak, Piotr Eder, Edyta Zagórowicz, Izabela Smoła, Krzysztof Wojciechowski, Szymon Drygała","doi":"10.1177/17562848241293938","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>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.</p><p><strong>Design: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 (<i>n</i> = 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 (<i>p</i> < 0.001), stool frequency (<i>p</i> < 0.001), abdominal pain score (<i>p</i> < 0.001), IBDQ (<i>p</i> < 0.001), IBD-F (<i>p</i> < 0.05), and fatigue impact on daily activities (<i>p</i> < 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).</p><p><strong>Conclusion: </strong>POLONEZ II demonstrated long-term real-world benefits of VDZ toward effectiveness, safety, and improved PROs and patients' quality of life.</p><p><strong>Trial registration: </strong>ENCePP (EUPAS32716).</p>","PeriodicalId":48770,"journal":{"name":"Therapeutic Advances in Gastroenterology","volume":"17 ","pages":"17562848241293938"},"PeriodicalIF":3.9000,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11580093/pdf/","citationCount":"0","resultStr":"{\"title\":\"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).\",\"authors\":\"Ariel Liebert, Maria Kłopocka, Agata Michalak, Halina Cichoz-Lach, Renata Talar-Wojnarowska, Danuta Domz Ał-Magrowska, Łukasz Konecki, Aleksandra Filipiuk, Michał Krogulecki, Maria Kopertowska-Majchrzak, Kamila Stawczyk-Eder, Katarzyna Waszak, Piotr Eder, Edyta Zagórowicz, Izabela Smoła, Krzysztof Wojciechowski, Szymon Drygała\",\"doi\":\"10.1177/17562848241293938\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>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.</p><p><strong>Design: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 (<i>n</i> = 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 (<i>p</i> < 0.001), stool frequency (<i>p</i> < 0.001), abdominal pain score (<i>p</i> < 0.001), IBDQ (<i>p</i> < 0.001), IBD-F (<i>p</i> < 0.05), and fatigue impact on daily activities (<i>p</i> < 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).</p><p><strong>Conclusion: </strong>POLONEZ II demonstrated long-term real-world benefits of VDZ toward effectiveness, safety, and improved PROs and patients' quality of life.</p><p><strong>Trial registration: </strong>ENCePP (EUPAS32716).</p>\",\"PeriodicalId\":48770,\"journal\":{\"name\":\"Therapeutic Advances in Gastroenterology\",\"volume\":\"17 \",\"pages\":\"17562848241293938\"},\"PeriodicalIF\":3.9000,\"publicationDate\":\"2024-11-20\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11580093/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Gastroenterology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/17562848241293938\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"GASTROENTEROLOGY & HEPATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Gastroenterology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/17562848241293938","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"GASTROENTEROLOGY & HEPATOLOGY","Score":null,"Total":0}
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).
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.
期刊介绍：
Therapeutic Advances in Gastroenterology is an open access journal which delivers the highest quality peer-reviewed original research articles, reviews, and scholarly comment on pioneering efforts and innovative studies in the medical treatment of gastrointestinal and hepatic disorders. The journal has a strong clinical and pharmacological focus and is aimed at an international audience of clinicians and researchers in gastroenterology and related disciplines, providing an online forum for rapid dissemination of recent research and perspectives in this area.
The editors welcome original research articles across all areas of gastroenterology and hepatology.
The journal publishes original research articles and review articles primarily. Original research manuscripts may include laboratory, animal or human/clinical studies – all phases. Letters to the Editor and Case Reports will also be considered.
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