PDF(Sci-hub)
Abstract：
OBJECTIVE: To examine pain treatment preferences before and after participation in an N-of-1 trial.
METHODS: In this observational study nested within a randomized trial, we examined chronic pain patients\' preferences before and after treatment in relation to N-of-1 trial results; assessed the influence of different schemes for defining comparative \"superiority\" on potential conclusions; and generated classification trees illustrating the relationship between pre-treatment preferences, N-of-1 trial results, and post-treatment preferences.
RESULTS: Treatment preferences differed pre- and post-trial for 40% of participants. The proportion of patients whose N-of-1 trials demonstrated \"superiority\" of one treatment regimen over the other varied depending on how superiority was defined and ranged from 24% (using criteria that required statistically significant differences between regimens) to 62% (when relying only on differences in point estimates). Regardless of criteria for declaring treatment superiority, nearly three-fourths of patients with equivocal N-of-1 trial results nevertheless expressed definite preferences post-trial.
CONCLUSIONS: A large segment of patients undergoing N-of-1 trials for chronic pain altered their treatment preferences. However, the direction of preference change did not necessarily correspond to the N-of-1 results. More research is needed to understand how patients use N-of-1 trial results, why preferences are \"sticky\" even in the face of personalized data, and how patients and clinicians might be educated to use N-of-1 trial results more informatively.
METHODS: In this observational study nested within a randomized trial, we examined chronic pain patients\' preferences before and after treatment in relation to N-of-1 trial results; assessed the influence of different schemes for defining comparative \"superiority\" on potential conclusions; and generated classification trees illustrating the relationship between pre-treatment preferences, N-of-1 trial results, and post-treatment preferences.
RESULTS: Treatment preferences differed pre- and post-trial for 40% of participants. The proportion of patients whose N-of-1 trials demonstrated \"superiority\" of one treatment regimen over the other varied depending on how superiority was defined and ranged from 24% (using criteria that required statistically significant differences between regimens) to 62% (when relying only on differences in point estimates). Regardless of criteria for declaring treatment superiority, nearly three-fourths of patients with equivocal N-of-1 trial results nevertheless expressed definite preferences post-trial.
CONCLUSIONS: A large segment of patients undergoing N-of-1 trials for chronic pain altered their treatment preferences. However, the direction of preference change did not necessarily correspond to the N-of-1 results. More research is needed to understand how patients use N-of-1 trial results, why preferences are \"sticky\" even in the face of personalized data, and how patients and clinicians might be educated to use N-of-1 trial results more informatively.
摘要：
目的：研究参与N-of-1试验前后疼痛治疗的偏好。
方法：在这项随机试验中嵌套的观察性研究中，我们检查了慢性疼痛患者在治疗前后的偏好与N-of-1试验结果的关系；评估了定义比较“优势”的不同方案对潜在结论的影响；并生成了说明治疗前偏好之间关系的分类树，N-of-1试验结果，和治疗后的偏好。
结果：40%的参与者在试验前后的治疗偏好不同。N-of-1试验证明一种治疗方案优于另一种治疗方案的患者比例根据优势定义的方式而变化，范围从24%(使用要求方案之间有统计学意义差异的标准)到62%(当仅依赖于点估计值的差异时)。无论宣布治疗优势的标准如何，然而,近四分之三的N-of-1试验结果不明确的患者在试验后表达了明确的偏好.
结论：大部分接受N-of-1慢性疼痛试验的患者改变了他们的治疗偏好。然而,偏好变化的方向不一定对应于N-1结果.需要更多的研究来了解患者如何使用N-of-1试验结果，为什么即使面对个性化数据，偏好也是“粘性的”，以及如何教育患者和临床医生更多地使用N-of-1试验结果。
方法：在这项随机试验中嵌套的观察性研究中，我们检查了慢性疼痛患者在治疗前后的偏好与N-of-1试验结果的关系；评估了定义比较“优势”的不同方案对潜在结论的影响；并生成了说明治疗前偏好之间关系的分类树，N-of-1试验结果，和治疗后的偏好。
结果：40%的参与者在试验前后的治疗偏好不同。N-of-1试验证明一种治疗方案优于另一种治疗方案的患者比例根据优势定义的方式而变化，范围从24%(使用要求方案之间有统计学意义差异的标准)到62%(当仅依赖于点估计值的差异时)。无论宣布治疗优势的标准如何，然而,近四分之三的N-of-1试验结果不明确的患者在试验后表达了明确的偏好.
结论：大部分接受N-of-1慢性疼痛试验的患者改变了他们的治疗偏好。然而,偏好变化的方向不一定对应于N-1结果.需要更多的研究来了解患者如何使用N-of-1试验结果，为什么即使面对个性化数据，偏好也是“粘性的”，以及如何教育患者和临床医生更多地使用N-of-1试验结果。
