在溃疡性结肠炎 OCTAVE 临床项目中，托法替尼可长期改善患者报告的直肠出血、排便频率和健康相关生活质量的结局。
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.
机构信息
Inflammatory Bowel Disease Center, NYU Langone Health, New York, NY, USA.
Surgical Department, Gastroenterology Division, Hospital Beatriz Ângelo, Loures, Portugal.
出版信息
Patient. 2023 Mar;16(2):95-103. doi: 10.1007/s40271-022-00603-w. Epub 2022 Nov 7.
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
CLINICALTRIALS
gov (NCT01465763 [21/10/2011]; NCT01458951 [21/10/2011]; NCT01458574 [21/10/2011]; NCT01470612 [21/10/2011]).
背景
托法替布是一种用于治疗溃疡性结肠炎（UC）的口服小分子 Janus 激酶抑制剂。托法替布 OCTAVE 临床项目包括三期诱导（OCTAVE Induction 1 和 2）和维持（OCTAVE Sustain）研究，以及一项开放标签、长期扩展研究（OCTAVE Open）。
目的
本事后分析评估了接受托法替布治疗的 UC 患者在 OCTAVE 临床项目中的一些长期、疾病特异性的患者报告结局（PRO）和健康相关生活质量（HRQoL）测量。
方法
分析包括来自 OCTAVE Open 的患者，这些患者被分配至托法替布 5mg，每日两次（OCTAVE Sustain第 52 周时处于缓解的亚组）。OCTAVE Open 的最终分析数据显示至第 48 个月。终点包括直肠出血亚评分（RBS）=0、粪便频率亚评分（SFS）≤1，以及 HRQoL 测量工具，炎症性肠病问卷（IBDQ）缓解（IBDQ 总分≥170）；对于所有就诊时缺失的数据，采用非应答者估算法，对于患者进入下一项研究后的就诊时的数据，则采用最后一次观察结转法（NRI-LOCF）。还评估了观察病例。
结果
在第 48 个月，175 例患者中，95（54.3%）例和 96（54.9%）例患者分别维持了 RBS=0 和 SFS≤1（NRI-LOCF）。此外，93（53.1%）例患者在第 48 个月时维持了 IBDQ 缓解（NRI-LOCF）。
结论
在 OCTAVE Open 进入缓解的患者中，大多数患者在 OCTAVE Open 的随访中，在长达 4 年的时间里维持了直肠出血的正常化和粪便频率的改善。OCTAVE Open 中 IBDQ 缓解也普遍维持。这些数据表明，托法替布 5mg，每日两次治疗可维持关键 UC PROs 的稳健缓解和持续应答。
试验注册
临床试验
http://www.clinicaltrials.gov（NCT01465763[2011 年 10 月 21 日]；NCT01458951[2011 年 10 月 21 日]；NCT01458574[2011 年 10 月 21 日]；NCT01470612[2011 年 10 月 21 日]）。
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