Solem Caitlyn T, Kwon Youngmin, Shah Ruchit M, Aly Abdalla, Botteman Marc F
a Real-World Evidence and Data Analytics , Pharmerit International , Bethesda , MD , USA.
Expert Rev Pharmacoecon Outcomes Res. 2018 Jun;18(3):245-253. doi: 10.1080/14737167.2018.1434414. Epub 2018 Feb 5.
The Quality-Adjusted Time Without Symptoms or Toxicity (Q-TWiST) has been used to evaluate the clinical benefits and risks of oncology treatments. However, limited information is available to interpret and contextualize Q-TWiST results.
A systematic review of Q-TWiST literature was conducted to provide contextualizing benchmarks for future studies. 51 articles with 81 unique Q-TWiST comparisons were identified. The mean (95% CI) and median absolute Q-TWiST gains for treatment versus control arms were 2.78 (1.82-3.73) months and 2.20 months across all cancers, respectively. The mean (median) relative Q-TWiST gains were 7.8% (7.2%) across all cancers. Most (88%) studies reported positive gains. The percentage of studies with relative Q-TWiST gains ≥10% (ie, clinically important difference) and ≥15% (ie, clearly clinically important difference) were 40.0% and 22.7%, respectively
The relevance of Q-TWiST in assessing net clinical benefits of cancer therapy has not diminished, despite an arguably low number of published studies. The interest in such assessment is highlighted by the recent emergence of oncology value frameworks. The Q-TWiST should be compelling to clinicians as it integrates clinical information (ie, toxicity, relapse/progression, and survival) and patient preferences for each of these states into a single meaningful index.
质量调整无症状或无毒性时间(Q-TWiST)已被用于评估肿瘤治疗的临床益处和风险。然而,用于解释和背景化Q-TWiST结果的信息有限。
对Q-TWiST文献进行了系统综述,为未来研究提供背景基准。共识别出51篇文章,其中有81个独特的Q-TWiST比较。在所有癌症中,治疗组与对照组的平均(95%CI)和绝对Q-TWiST增益中位数分别为2.78(1.82 - 3.73)个月和2.20个月。在所有癌症中,平均(中位数)相对Q-TWiST增益为7.8%(7.2%)。大多数(88%)研究报告有正向增益。相对Q-TWiST增益≥10%(即临床重要差异)和≥15%(即明显临床重要差异)的研究百分比分别为40.0%和22.7%。
尽管已发表的研究数量可能较少,但Q-TWiST在评估癌症治疗净临床益处方面的相关性并未减弱。肿瘤学价值框架的近期出现凸显了对这种评估的兴趣。Q-TWiST应该对临床医生具有吸引力,因为它将临床信息(即毒性、复发/进展和生存)以及患者对这些状态的偏好整合到一个有意义的单一指标中。