PDF(Pubmed)
Abstract：
Blood pressure (BP) is often inadequately controlled in children treated for hypertension, and personalized (n-of-1) trials show promise for tailoring treatment choices. We assessed whether patients whose treatment choices are informed by an n-of-1 trial have improved BP control compared to usual care.
A randomized clinical trial was conducted in a pediatric hypertension clinic in Houston from April 2018 to September 2020. Hypertensive adolescents and young adults 10-22 years old were randomized 1:1 to a strategy of n-of-1 trial using ambulatory BP monitoring to inform treatment choice or usual care, with treatment selected by physician preference. The primary outcome was the proportion of patients with ambulatory BP control at 6 months in a Bayesian analysis.
Among 49 participants (23 randomized to n-of-1 trials and 26 to usual care), mean age was 15.6 years. Using skeptical priors, we found a 69% probability that n-of-1 trials increased BP control at 6 months (Bayesian odds ratio (OR) 1.24 (95% credible interval (CrI) 0.51, 2.97), and 74% probability using neutral informed priors (OR 1.45 (95% CrI 0.48, 4.53)). Systolic BP was reduced in both groups, with a 93% probability of greater reduction in the n-of-1 trial group (mean difference between groups = -3.6 mm Hg (95% CrI -8.3, 1.28). There was no significant difference in side effect experience or caregiver satisfaction.
Among hypertensive adolescents and young adults, n-of-1 trials with ambulatory BP monitoring likely increased the probability of BP control. A large trial is needed to assess their use in clinical practice.
NCT03461003.
ClinicalTrials.gov; NCT03461003.
A randomized clinical trial was conducted in a pediatric hypertension clinic in Houston from April 2018 to September 2020. Hypertensive adolescents and young adults 10-22 years old were randomized 1:1 to a strategy of n-of-1 trial using ambulatory BP monitoring to inform treatment choice or usual care, with treatment selected by physician preference. The primary outcome was the proportion of patients with ambulatory BP control at 6 months in a Bayesian analysis.
Among 49 participants (23 randomized to n-of-1 trials and 26 to usual care), mean age was 15.6 years. Using skeptical priors, we found a 69% probability that n-of-1 trials increased BP control at 6 months (Bayesian odds ratio (OR) 1.24 (95% credible interval (CrI) 0.51, 2.97), and 74% probability using neutral informed priors (OR 1.45 (95% CrI 0.48, 4.53)). Systolic BP was reduced in both groups, with a 93% probability of greater reduction in the n-of-1 trial group (mean difference between groups = -3.6 mm Hg (95% CrI -8.3, 1.28). There was no significant difference in side effect experience or caregiver satisfaction.
Among hypertensive adolescents and young adults, n-of-1 trials with ambulatory BP monitoring likely increased the probability of BP control. A large trial is needed to assess their use in clinical practice.
NCT03461003.
ClinicalTrials.gov; NCT03461003.
摘要：
在接受高血压治疗的儿童中，血压（BP）通常未得到充分控制。个性化（n-of-1）试验显示出定制治疗选择的希望。与常规治疗相比，我们评估了由n-of-1试验告知治疗选择的患者是否改善了BP控制。
一项随机临床试验于2018年4月至2020年9月在休斯敦的儿科高血压诊所进行。高血压青少年和10-22岁的年轻人以1：1的比例随机分配到n-of-1试验策略中，使用动态BP监测来告知治疗选择或常规护理。根据医生的偏好选择治疗。在贝叶斯分析中，主要结果是6个月时动态血压控制的患者比例。
在49名参与者中（23名随机接受n-of-1试验，26名接受常规治疗），平均年龄为15.6岁。使用怀疑的先验，我们发现，69%的概率，n-of-1试验增加BP控制在6个月（贝叶斯优势比（OR）1.24（95%可信区间（CrI）0.51，2.97），使用中立信息先验的概率为74%(OR1.45(95%CrI0.48，4.53))。两组收缩压均降低，在n-of-1试验组中有93%的概率降低（组间平均差=-3.6mmHg（95%CrI=8.3,1.28）。副作用体验或照顾者满意度没有显着差异。
在高血压青少年和年轻人中，动态BP监测的n-1项试验可能增加BP控制的可能性。需要进行大型试验来评估其在临床实践中的使用。
NCT03461003。
ClinicalTrials.gov；NCT03461003。
一项随机临床试验于2018年4月至2020年9月在休斯敦的儿科高血压诊所进行。
在49名参与者中（23名随机接受n-of-1试验，26名接受常规治疗），
在高血压青少年和年轻人中，
NCT03461003。
ClinicalTrials.gov；NCT03461003。
