One‐sample aggregate data meta‐analysis of medians

McGrath, Seán; Zhao, XiaoFei; Qin, Zhi Zhen; Steele, Russell; Benedetti, Andrea

doi:10.1002/sim.8013

Cited by 120 publications

(151 citation statements)

References 33 publications

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“…They include, for example, extending the data transformation methods from normal data to non-normal data, [13][14][15] or developing new meta-analytical methods to directly synthesize the data with the five-number summary. 16,17 We note that the proposed method in this article can also be readily extended to non-normal data, yet further work is needed to assess the effectiveness of these new estimators when included in a meta-analysis.…”

Section: Discussionmentioning

confidence: 99%

Optimally estimating the sample standard deviation from the five‐number summary

Shi

Luo

Weng

et al. 2020

Research Synthesis Methods

320

255

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When reporting the results of clinical studies, some researchers may choose the five‐number summary (including the sample median, the first and third quartiles, and the minimum and maximum values) rather than the sample mean and standard deviation (SD), particularly for skewed data. For these studies, when included in a meta‐analysis, it is often desired to convert the five‐number summary back to the sample mean and SD. For this purpose, several methods have been proposed in the recent literature and they are increasingly used nowadays. In this article, we propose to further advance the literature by developing a smoothly weighted estimator for the sample SD that fully utilizes the sample size information. For ease of implementation, we also derive an approximation formula for the optimal weight, as well as a shortcut formula for the sample SD. Numerical results show that our new estimator provides a more accurate estimate for normal data and also performs favorably for non‐normal data. Together with the optimal sample mean estimator in Luo et al., our new methods have dramatically improved the existing methods for data transformation, and they are capable to serve as “rules of thumb” in meta‐analysis for studies reported with the five‐number summary. Finally for practical use, an Excel spreadsheet and an online calculator are also provided for implementing our optimal estimators.

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Section: Discussionmentioning

confidence: 99%

Optimally estimating the sample standard deviation from the five‐number summary

Shi

Luo

Weng

et al. 2020

Research Synthesis Methods

320

255

View full text Add to dashboard Cite

show abstract

“…These methods have been shown to perform better than transformation-based approaches, where the sample mean and its sampling variance are estimated from median data [17]. We constructed approximate 95% confidence intervals (CIs) for the pooled median using the 1 [17]. We performed stratified analysis by ventilatory support (i.e., mechanical non-invasive ventilation or tracheotomy, as reported by the identified publications) and low versus high risk of bias.…”

Section: Screening Data Extraction and Synthesismentioning

confidence: 99%

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

et al. 2020

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Several studies indicate that prognosis for survival in Duchenne muscular dystrophy (DMD) has improved in recent decades. However, published evidence is inconclusive and some estimates may be obsolete due to improvements in standards of care, in particular the routine use of mechanical ventilatory support in advanced stages of the disease. In this systematic review and meta-analysis (PROSPERO identifier: CRD42019121800), we searched MEDLINE (through PubMed), CINAHL, Embase, PsycINFO, and Web of Science for studies published from inception up until December 31, 2018, reporting results of life expectancy in DMD. We pooled median survival estimates from individual studies using the median of medians, and weighted median of medians, methods. Risk of bias was established with the Newcastle-Ottawa Scale. Results were stratified by ventilatory support and risk of bias. We identified 15 publications involving 2662 patients from 12 countries from all inhabited continents except Africa. Median life expectancy without ventilatory support ranged between 14.4 and 27.0 years (pooled median: 19.0 years, 95% CI 18.0-20.9; weighted pooled median: 19.4 years, 18.2-20.1). Median life expectancy with ventilatory support, introduced in most settings in the 1990s, ranged between 21.0 and 39.6 years (pooled median: 29.9 years, 26.5-30.8; weighted pooled median: 31.8 years, 29.3-36.2). Risk of bias had little impact on pooled results. In conclusion, median life expectancy at birth in DMD seems to have improved considerably during the last decades. With current standards of care, many patients with DMD can now expect to live into their fourth decade of life.

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“…In this scenario, the PFS and OS curves are usually summarised by medians and accompanied by their 95% confidence interval (95% CI), as is the case of the present review. Following the method used by McGrath et al, pooled estimates were obtained as the median of the study-specific PFS and OS medians (22), whereas the corresponding 95%CIs were obtained as the 1/2 ± min 1/2, z 0.975 /(2 √ K) quantiles of the k observed study medians, with z α the α quantile of the standard normal distribution.…”

Section: Discussionmentioning

confidence: 99%

Phase-II Trials of Pazopanib in Metastatic Neuroendocrine Neoplasia (mNEN): A Systematic Review and Meta-Analysis

et al. 2020

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Background: Several phase-II trials have been designed to evaluate tyrosine kinase inhibitors (TKIs), in particular, pazopanib in neuroendocrine neoplasia (NEN), but its efficacy has not yet been demonstrated in a randomised-controlled Phase III trial. A systematic review of the published clinical trials of metastatic NEN patients could reduce the possible bias of single phase II studies. The present systematic review focuses on the efficacy and safety of pazopanib in patients with metastatic and locally advanced NEN. Methods: A systematic search in the major databases Medline/PubMed, Cochrane and Embase and in supplementary material from important international Meetings was performed to identify publications on pazopanib for the treatment of neuroendocrine neoplasia. English language was defined as a restriction. Four authors of the present review independently performed the study selection, assessed the risk of bias and extracted study data. Four published clinical trials and 2 abstracts were identified. One trial was excluded because the topic was Von-Hippel Landau disease and one abstract was eliminated because of the lack of information on meeting proceedings. Results: In all of the trials pazopanib was orally administered at a dose of 800 mg daily continuously with a 28-day cycle. The intention-to-treat population for efficacy was composed of 230 patients with a median age of 62 years. The partial response rate was 10.7% (95% confidence interval 2.6-20.5). The rate for stable disease was 79.6% (range: 61.7-92.1%) with a disease control rate (DCR) of 90.3%. Progressive disease was reported in 9.7% (range 5.2-17.6) of patients. No complete responses were observed. Median progression-free survival was 11.6 months (95% CI: 9.2-13.9). Overall survival from all the trials was 24.6 (95% CI: 18.7-40.8) months. Severe adverse events (grade III-IV) included hypertension 31%, 16% increase in AST/ALT, diarrhoea 10% and fatigue 10%. Bongiovanni et al. Systematic Review on Pazopanib in mNEN Conclusions: Pazopanib monotherapy achieved a DCR of 90.3% in patients with locally advanced and/or metastatic neuroendocrine neoplasia, with an overall response rate comparable to other TKIs and mTOR inhibitors and a safety profile similar to that of drugs of the same class.

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One‐sample aggregate data meta‐analysis of medians

Cited by 120 publications

References 33 publications

Optimally estimating the sample standard deviation from the five‐number summary

Optimally estimating the sample standard deviation from the five‐number summary

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

Phase-II Trials of Pazopanib in Metastatic Neuroendocrine Neoplasia (mNEN): A Systematic Review and Meta-Analysis

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