Long-Term Improvement in the Patient-Reported Outcomes of Rectal Bleeding, Stool Frequency, and Health-Related Quality of Life with Tofacitinib in the Ulcerative Colitis OCTAVE Clinical Program.

IF 3.4 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Patient-Patient Centered Outcomes Research Pub Date : 2023-03-01 Epub Date: 2022-11-07 DOI:10.1007/s40271-022-00603-w

David P Hudesman, Joana Torres, Leonardo Salese, John C Woolcott, Rajiv Mundayat, Chinyu Su, Mahmoud H Mosli, Jessica R Allegretti

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引用次数: 0

Abstract

Background: Tofacitinib is an oral small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). The tofacitinib OCTAVE clinical program included phase III induction (OCTAVE Induction 1 and 2) and maintenance (OCTAVE Sustain) studies, and an open-label, long-term extension study (OCTAVE Open).

Objective: This post hoc analysis assessed selected long-term, disease-specific patient-reported outcome (PRO) and health-related quality-of-life (HRQoL) measurements in patients with UC receiving tofacitinib in the OCTAVE clinical program.

Methods: Analyses included patients from OCTAVE Open assigned to tofacitinib 5 mg twice daily (subpopulation in remission at Week 52 of OCTAVE Sustain). OCTAVE Open data from the final analyses are shown to Month 48. Endpoints included rectal bleeding subscore (RBS) = 0, stool frequency subscore (SFS) ≤ 1, and HRQoL measure, Inflammatory Bowel Disease Questionnaire (IBDQ) remission (IBDQ total score ≥ 170); with non-responder imputation for missing data at all visits, and last observation carried forward for visits after a patient advanced to the next study (NRI-LOCF). Observed cases were also assessed.

Results: At Month 48, of 175 patients, 95 (54.3%) and 96 (54.9%) achieved/maintained RBS = 0 and SFS ≤ 1, respectively (NRI-LOCF). Additionally, 93 (53.1%) patients achieved/maintained IBDQ remission at Month 48 (NRI-LOCF).

Conclusions: Among patients who entered OCTAVE Open in remission, most maintained normalization of rectal bleeding and improvement in stool frequency for ≤ 4 years of follow-up in OCTAVE Open. IBDQ remission was also generally maintained in OCTAVE Open. These data show robust maintenance of key UC PROs and durability of response with tofacitinib 5 mg twice daily.

Trial registration: http://www.

Clinicaltrials: gov (NCT01465763 [21/10/2011]; NCT01458951 [21/10/2011]; NCT01458574 [21/10/2011]; NCT01470612 [21/10/2011]).

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在溃疡性结肠炎 OCTAVE 临床项目中使用托法替尼长期改善直肠出血、大便次数和与健康相关的生活质量的患者报告结果。

背景介绍托法替尼是一种口服小分子 Janus 激酶抑制剂，用于治疗溃疡性结肠炎（UC）。托法替尼OCTAVE临床项目包括III期诱导（OCTAVE诱导1和2）和维持（OCTAVE维持）研究，以及一项开放标签的长期扩展研究（OCTAVE开放）：这项事后分析评估了在OCTAVE临床项目中接受托法替尼治疗的UC患者的部分长期、疾病特异性患者报告结果（PRO）和健康相关生活质量（HRQoL）测量结果：分析对象包括OCTAVE Open项目中被分配接受每日两次、每次5毫克法替尼治疗的患者（OCTAVE Sustain项目第52周时处于缓解期的亚群）。最终分析中的 OCTAVE Open 数据显示至第 48 个月。终点包括直肠出血子评分（RBS）=0、大便次数子评分（SFS）≤1，以及HRQoL测量指标--炎症性肠病问卷（IBDQ）缓解（IBDQ总分≥170）；所有访视的缺失数据均采用非应答者估算，患者进入下一项研究（NRI-LOCF）后的访视则采用最后一次观察。还对观察病例进行了评估：在第 48 个月，175 名患者中分别有 95 人（54.3%）和 96 人（54.9%）达到/保持 RBS = 0 和 SFS ≤ 1（NRI-LOCF）。此外，93 例（53.1%）患者在第 48 个月达到/维持了 IBDQ 缓解（NRI-LOCF）：结论：在进入 OCTAVE Open 的缓解期患者中，大多数人在 OCTAVE Open 随访的 4 年中保持了直肠出血正常化和大便次数改善。IBDQ 缓解在 OCTAVE Open 中也得到了普遍维持。这些数据表明，托法替尼5毫克，每日两次，能有效维持主要的UC PROs和反应的持久性。试验注册：http://www.Clinicaltrials: gov (NCT01465763 [21/10/2011]; NCT01458951 [21/10/2011]; NCT01458574 [21/10/2011]; NCT01470612 [21/10/2011]).

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来源期刊

Patient-Patient Centered Outcomes Research HEALTH CARE SCIENCES & SERVICES-

CiteScore

6.60

自引率

8.30%

发文量

审稿时长

>12 weeks

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