Shire Human Genetic Therapies, Inc. (a member of the Takeda group of companies), Cambridge, Massachusetts, USA.
Analysis Group, Inc., Boston, Massachusetts, USA.
JPEN J Parenter Enteral Nutr. 2020 Jan;44(1):119-128. doi: 10.1002/jpen.1588. Epub 2019 Apr 21.
Teduglutide reduces or eliminates parenteral support (PS) dependency in patients with short bowel syndrome (SBS). Recent post hoc analyses demonstrated that effects are correlated with baseline PS volume. We assessed the SBS-related quality-of-life (QoL) impact of teduglutide, particularly whether improvements are greater among subgroups achieving more PS volume reduction.
Using phase 3 trial data of teduglutide in patients with SBS (NCT00798967), change in Short Bowel Syndrome-Quality of Life (SBS-QoL) scores from baseline were compared between teduglutide vs placebo in the overall population and subgroups classified by baseline PS volume requirement, disease etiology, and bowel anatomy. Generalized estimating equation models were fitted to assess impact of teduglutide on SBS-related QoL using data from all visits, adjusted for baseline characteristics.
Of 86 patients, 43 each were randomized to teduglutide or placebo (mean age: 51 vs 50 years, respectively). In adjusted analyses, teduglutide had a nonsignificant reduction (improvement) of -8.6 points (95% CI: 2.6 to -19.8) in SBS-QoL sum score from baseline to Week-24 vs placebo. The impact of teduglutide varied by subgroup. Patients treated with teduglutide experienced significantly greater reductions in SBS-QoL sum score at Week-24 vs placebo in 2 subgroups, ie, the third (highest) tertile baseline PS volume (-27.3, 95% CI: -50.8 to -3.7) and inflammatory bowel disease (IBD; -29.6, 95% CI: -46.3 to -12.9). Results were similar for SBS-QoL subscale and item scores.
The impact of teduglutide treatment on SBS-related QoL vs placebo varied among subgroups and was significant and most pronounced among patients with highest baseline PS volume requirement or IBD.
特杜古肽可减少或消除短肠综合征（SBS）患者对肠外支持（PS）的依赖。最近的事后分析表明，其效果与 PS 量基线相关。我们评估了特杜古肽对 SBS 相关生活质量（QoL）的影响，特别是在实现 PS 量减少更多的亚组中，改善是否更为明显。
使用特杜古肽治疗 SBS 患者的 III 期临床试验数据（NCT00798967），比较了特杜古肽与安慰剂在总体人群和按 PS 量基线要求、疾病病因和肠道解剖分类的亚组中，从基线到 SBS-QoL 评分的变化。使用所有访视的数据，通过广义估计方程模型，调整基线特征，评估特杜古肽对 SBS 相关 QoL 的影响。
86 例患者中，43 例随机分为特杜古肽组或安慰剂组（平均年龄：51 岁比 50 岁）。调整分析显示，与安慰剂相比，特杜古肽在 24 周时 SBS-QoL 总分的降低（改善）为-8.6 分（95%CI：2.6 至-19.8），无统计学意义。特杜古肽的影响因亚组而异。特杜古肽组在两个亚组中，即 PS 量基线第三（最高）三分位数（-27.3，95%CI：-50.8 至-3.7）和炎症性肠病（IBD；-29.6，95%CI：-46.3 至-12.9），在 24 周时 SBS-QoL 总分较安慰剂组显著降低。SBS-QoL 亚量表和项目评分结果相似。
与安慰剂相比，特杜古肽治疗对 SBS 相关 QoL 的影响在亚组之间存在差异，在 PS 量基线要求最高或 IBD 的患者中最为显著和明显。
