Calibrated meta-analysis to estimate the efficacy of mental health treatments in target populations: an application to paliperidone trials for treatment of schizophrenia.
Calibrated meta-analysis to estimate the efficacy of mental health treatments in target populations: an application to paliperidone trials for treatment of schizophrenia.
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DOI：
10.1186/s12874-023-01958-w
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发表时间：
2023-06-26
影响因子：
4
通讯作者：
Stuart, Elizabeth A.
中科院分区：
文献类型：
--
作者：
Hong, Hwanhee;Liu, Lu;Mojtabai, Ramin;Stuart, Elizabeth A.
关键词：
荟萃分析可以是一个强大的工具，但需要校准纳入试验对目标人群的潜在不代表性。在荟萃分析中估计目标人群平均治疗效果（TATE）对于了解治疗在明确定义的目标人群中的表现非常重要。本研究使用荟萃分析个体患者试验数据和目标人群数据估计棕榈酸帕利哌酮治疗精神分裂症患者的TATE。我们对来自四项随机临床试验的数据和来自临床抗精神病药物干预有效性试验（CATIE）研究的目标人群数据进行了荟萃分析。使用阳性和阴性症状量表（PANSS）测量疗效。通过比较试验和CATIE之间的基线特征，计算等同试验参与者和目标人群的权重。进行随机效应的校准加权荟萃分析，以估计帕利哌酮与安慰剂相比的TATE。共有1,738例患者沿着CATIE中的1,458例患者被纳入荟萃分析。加权后，试验参与者和目标人群的协变量分布相似。在未加权（平均差异9.07 [4.43，13.71]）和校准加权（平均差异6.15 [2.22，10.08]）荟萃分析中，与安慰剂相比，棕榈酸帕利哌酮与PANSS总分显著降低相关。目标人群中棕榈酸帕利哌酮与安慰剂相比的效应略小于直接从未加权荟萃分析中估计的效应。应评估纳入荟萃分析的试验样本对目标人群的代表性，并适当纳入，以获得目标人群治疗效果的最可靠证据。在线版本包含补充材料，可通过10.1186/s12874-023-01958-w获得。
Meta-analyses can be a powerful tool but need to calibrate potential unrepresentativeness of the included trials to a target population. Estimating target population average treatment effects (TATE) in meta-analyses is important to understand how treatments perform in well-defined target populations. This study estimated TATE of paliperidone palmitate in patients with schizophrenia using meta-analysis with individual patient trial data and target population data. We conducted a meta-analysis with data from four randomized clinical trials and target population data from the Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) study. Efficacy was measured using the Positive and Negative Syndrome Scale (PANSS). Weights to equate the trial participants and target population were calculated by comparing baseline characteristics between the trials and CATIE. A calibrated weighted meta-analysis with random effects was performed to estimate the TATE of paliperidone compared to placebo. A total of 1,738 patients were included in the meta-analysis along with 1,458 patients in CATIE. After weighting, the covariate distributions of the trial participants and target population were similar. Compared to placebo, paliperidone palmitate was associated with a significant reduction of the PANSS total score under both unweighted (mean difference 9.07 [4.43, 13.71]) and calibrated weighted (mean difference 6.15 [2.22, 10.08]) meta-analysis. The effect of paliperidone palmitate compared with placebo is slightly smaller in the target population than that estimated directly from the unweighted meta-analysis. Representativeness of samples of trials included in a meta-analysis to a target population should be assessed and incorporated properly to obtain the most reliable evidence of treatment effects in target populations. The online version contains supplementary material available at 10.1186/s12874-023-01958-w.
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影响因子：
25.8
作者：
Furukawa, Toshi A.;Levine, Stephen Z.;Leucht, Stefan
通讯作者：
Leucht, Stefan
影响因子：
2.5
作者：
Kennedy-Martin T;Curtis S;Faries D;Robinson S;Johnston J
通讯作者：
Johnston J
DOI：
10.1016/0197-2456(86)90046-2
发表时间：
1986-09-01
期刊：
CONTROLLED CLINICAL TRIALS
影响因子：
--
作者：
DERSIMONIAN, R;LAIRD, N
通讯作者：
LAIRD, N
影响因子：
5
作者：
Cole, Stephen R.;Stuart, Elizabeth A.
通讯作者：
Stuart, Elizabeth A.
生物医学数据共享和再利用：临床和科学研究人员的态度和实践。
影响因子：
3.7
作者：
Federer LM;Lu YL;Joubert DJ;Welsh J;Brandys B
通讯作者：
Brandys B