We conducted a search of PubMed, Embase, Web of Science, Cochrane Library, LILACS, and LIVIVO to retrieve studies published in English between Jan. 1, 2000, and June 30, 2021. Pooled prevalence with a 95% confidence interval (CI) was estimated using a random-effects meta-analysis based on a generalized linear mixed model with a logit link.
Ten eligible studies were identified (all in females), with a combined sample size of 9.57 million women and 673 Rett syndrome cases. The pooled prevalence estimate (random effects) was 7.1 per 100,000 females (95% CI: 4.8, 10.5, heterogeneity p < 0.001). Despite greatly variable precision of estimation, all estimates were compatible with a prevalence range of approximately 5 to 10 cases per 100,000 females based on their respective 95% CIs.
These findings may facilitate planning of therapeutic trials in this indication in terms of target sample size and accrual times.
方法:我们对PubMed进行了搜索,Embase,WebofScience,科克伦图书馆,LILACS,和LIVIVO检索1月之间以英语发表的研究2000年1月1日和2021年6月30日。使用基于具有logit链接的广义线性混合模型的随机效应荟萃分析来估计具有95%置信区间(CI)的合并患病率。
结果:确定了十项符合条件的研究(均为女性),合并样本量为957万女性和673例Rett综合征病例。合并患病率估计值(随机效应)为7.1/100,000女性(95%CI:4.8,10.5,异质性p<0.001)。尽管估计的精度变化很大,基于她们各自的95%CI,所有估计值与每100,000名女性约5~10例的患病率范围相一致.
结论:这些发现可能有助于在目标样本量和累积时间方面规划本适应症的治疗试验。