Propensity score methods and regression adjustment for analysis of nonrandomized studies with health‐related quality of life outcomes

Cottone, Francesco; Anota, Amélie; Bonnetain, Franck; Collins, Gary S.; Efficace, Fabio

doi:10.1002/pds.4756

Cited by 11 publications

(5 citation statements)

References 44 publications

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“…Alternatively, we tried to perform the PS regression adjustment method to preserve the total number of the study population for analyzing the effect of EOR. 30 The concordance of results was observed from both PS approaches. For other limitations, fluorescence-guided resections with 5-ALA was not performed in the present study because it is unavailable in our institute.…”

Section: Discussionsupporting

confidence: 53%

Effect of the Extent of Resection on Survival Outcome in Glioblastoma: Propensity Score Approach

Tunthanathip

Madteng

2020

Arquivos Brasileiros de Neurocirurgia: Brazilian Neurosurgery

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Objective To evaluate the effectiveness of the extent of resection (EOR) on survival outcome using propensity score-based approaches. Materials and Methods A retrospective cohort study was performed in patients with newly diagnosed glioblastoma. Propensity score matching (PSM) and propensity score regression adjustment were used in the matched and unmatched dataset, respectively. Therefore, the Kaplan-Meier survival curve and Cox's regression analyses were performed to determine the effect of the EOR on survival outcomes. Results One hundred and sixty-eight patients were included for analyzes. The total tumor resection in the unmatched dataset was 22.6% of all cases. Using PSM, incomplete tumor resection had an unfavorable survival outcome when compared with total tumor resection (hazard ratio (HR) 2.92, 95% confidence interval [CI] 1.72–4.94). Additionally, biopsy and partial tumor resection were significantly associated with poor prognosis when compared with total tumor resection using propensity score regression adjustment (HR of biopsy 1.89, 95%CI 1.13–3.16 and HR of partial resection 1.89, 95%CI 1.28–2.80). Conclusions Patients with total tumor resection tend to have a more favorable prognosis than patients with partial tumor resection. The propensity score-based analysis is an alternative approach to evaluate the effect of an intervention that has limitations to perform a randomized controlled trial.

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Section: Discussionsupporting

confidence: 53%

Effect of the Extent of Resection on Survival Outcome in Glioblastoma: Propensity Score Approach

Tunthanathip

Madteng

2020

Arquivos Brasileiros de Neurocirurgia: Brazilian Neurosurgery

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“…This method has been used previously in observational studies [16,17] and has the advantage of summarising a long list of covariates as a single score and obviating the need to adjust for a long list of confounders in regression modelling; which is especially problematic for the binary safety outcome due to sparse data in this context. Although this method has been previously criticised for being biased [18,19], in our context intervention assignment was independent of the patient baseline variables and therefore we expect any bias to be negative (towards the null) [18]. Further research may be needed however relating to the most appropriate way to model propensity score, and the consistency and unbiasedness of this method when used in the context of stepped wedge trials and/ or before-after designs.…”

Section: Regression Adjustment For Propensity Scorementioning

confidence: 94%

Divergent confidence intervals among pre-specified analyses in the HiSTORIC stepped wedge trial: An exploratory post-hoc investigation

et al. 2022

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Background The high-sensitivity cardiac troponin on presentation to rule out myocardial infarction (HiSTORIC) study was a stepped-wedge cluster randomised trial with long before-and-after periods, involving seven hospitals across Scotland. Results were divergent for the binary safety endpoint (type 1 or type 4b myocardial infarction or cardiac death) across certain pre-specified analyses, which warranted further investigation. In particular, the calendar-matched analysis produced an odds ratio in the opposite direction to the primary logistic mixed-effects model analysis. Methods Several post-hoc statistical models were fitted to each of the co-primary outcomes of length of hospital stay and safety events, which included adjusting for exposure time, incorporating splines, and fitting a random time effect. We improved control of patient characteristics over time by adjusting for multiple additional covariates using different methods: direct inclusion, regression adjustment for propensity score, and weighting. A data augmentation approach was also conducted aiming to reduce the effect of sparse data bias. Finally, the raw data was examined. Results The new statistical models confirmed the results of the pre-specified trial analysis. In particular, the observed divergence between the calendar-matched and other analyses remained, even after performing the covariate adjustment methods, and after using data augmentation. Divergence was particularly acute for the safety endpoint, which had an event rate of 0.36% overall. Examining the raw data was particularly helpful to assess the sensitivity of the results to small changes in event rates and identify patterns in the data. Conclusions Our experience reveals the importance of conducting multiple pre-specified sensitivity analyses and examining the raw data, particularly for stepped wedge trials with low event rates or with a small number of sites. Before-and-after analytical approaches that adjust for differences in patient populations but avoid direct modelling of the time trend should be considered in future stepped wedge trials with similar designs.

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“…17,18 Third, the inverse probability of treatment weighting (IPTW) via the average treatment effect in the treated (ATT) method was also used for the above comparison. 19 Each patient was assigned an inverse weighting of these 2 groups of patients using calculated propensity scores and the following formula: patients with TEVAR=1; patients without TEVAR=propensity score/1−propensity score. Therefore, the weight of patients without TEVAR was reduced, and the weight of patients with TEVAR was increased.…”

Section: Study Design and Statistical Analysismentioning

confidence: 99%

“…Inverse probability of treatment weighting with ATT was used to avoid the exclusion of several hundred patients from PSM. 19 Before matching, the standardized mean difference (SMD) was used to confirm a balanced matching result. The matching result was considered balanced when the SMD was less than 0.1.…”

Section: Study Design and Statistical Analysismentioning

confidence: 99%

Does Thoracic Endovascular Aortic Repair for Blunt Thoracic Aortic Injury Increase the Probability of Delayed Intra-abdominal Hemorrhage in Patients With Concomitant Blunt Abdominal Trauma?

et al. 2022

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Purpose: Most blunt thoracic aortic injuries (BTAIs) may be treated using thoracic endovascular aortic repair (TEVAR), and most blunt abdominal traumas (BATs) are managed conservatively. We hypothesized that severe trauma that needs TEVAR may increase the risk of delayed intra-abdominal hemorrhage in patients with concomitant BTAI and BAT because of the potential hemorrhagic shock, disseminated intravascular coagulopathy, blood loss, consequent need for blood transfusion, and procedure-associated heparinization. Materials and Methods: From 2015 to 2019, blunt trauma patients with concomitant severe chest trauma and abdominal trauma who could be managed conservatively were studied. The probability of delayed intra-abdominal hemorrhage was compared between patients with concomitant BTAI who received or did not undergo TEVAR. Propensity score matching (PSM), inverse probability of treatment weighting (IPTW), and multivariate logistic regression (MLR) were used to eliminate discrepancies between these 2 groups. Results: Among the 341 studied patients, there were 26 patients with BTAI, and 19 of them underwent TEVAR. Delayed intra-abdominal hemorrhage was observed in 4 patients (21.1%, 4/19) who underwent TEVAR. Both PSM and IPTW showed that patients who underwent TEVAR for concomitant BTAI had a greater delayed need for blood transfusions and a larger proportion of delayed intra-abdominal hemorrhage than patients who did not undergo the procedure. The MLR analysis showed that TEVAR for BTAI was an independent risk factor for delayed intra-abdominal hemorrhage (odds ratio: 10.534, 95%, p<0.001). Conclusion: An increased probability of delayed intra-abdominal hemorrhage in patients with BAT (who could be managed conservatively) was observed in patients who underwent TEVAR for concomitant BTAI. Clinical Impact More attention should be give in patients with high grade aortic injuries and concomitant abdominal trauma.

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Propensity score methods and regression adjustment for analysis of nonrandomized studies with health‐related quality of life outcomes

Cited by 11 publications

References 44 publications

Effect of the Extent of Resection on Survival Outcome in Glioblastoma: Propensity Score Approach

Effect of the Extent of Resection on Survival Outcome in Glioblastoma: Propensity Score Approach

Divergent confidence intervals among pre-specified analyses in the HiSTORIC stepped wedge trial: An exploratory post-hoc investigation

Does Thoracic Endovascular Aortic Repair for Blunt Thoracic Aortic Injury Increase the Probability of Delayed Intra-abdominal Hemorrhage in Patients With Concomitant Blunt Abdominal Trauma?

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