scholarly journals Greedy caliper propensity score matching can yield variable estimates of the treatment‐outcome association ‐ a simulation study

Author(s):  
Joris J. Komen ◽  
Svetlana V. Belitser ◽  
Richard Wyss ◽  
Sebastian Schneeweiss ◽  
Anne C. Taams ◽  
...  
2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Albee Ling ◽  
Maria Montez-Rath ◽  
Maya Mathur ◽  
Kris Kapphahn ◽  
Manisha Desai

Propensity score matching (PSM) has been widely used to mitigate confounding in observational studies, although complications arise when the covariates used to estimate the PS are only partially observed. Multiple imputation (MI) is a potential solution for handling missing covariates in the estimation of the PS. However, it is not clear how to best apply MI strategies in the context of PSM. We conducted a simulation study to compare the performances of popular non-MI missing data methods and various MI-based strategies under different missing data mechanisms. We found that commonly applied missing data methods resulted in biased and inefficient estimates, and we observed large variation in performance across MI-based strategies. Based on our findings, we recommend 1) estimating the PS after applying MI to impute missing confounders; 2) conducting PSM within each imputed dataset followed by averaging the treatment effects to arrive at one summarized finding; 3) a bootstrapped-based variance to account for uncertainty of PS estimation, matching, and imputation; and 4) inclusion of key auxiliary variables in the imputation model.


Author(s):  
Hee Won Seo ◽  
Ha Na Lee ◽  
Hyun Woong Jun ◽  
Hayoung Byun ◽  
Jae Ho Chung ◽  
...  

Background and Objectives We aimed to compare the treatment outcomes of primary intratympanic steroid (ITS) and the salvage ITS protocol.Subjects and Method We assessed 440 patients with unilateral idiopathic sudden sensorineural hearing loss (ISSNHL) from January 2016 to December 2019. Of the 440 patients, 37 patients received ITS only as a primary treatment while other patients received systemic steroid; of those remaining patients, 276 received systemic steroid as well as ITS as a salvage treatment. We performed a 1:2 propensity score matching analysis for both groups using parameters such as the initial hearing level, presence of vertigo, onset of treatment, age, sex, hypertension and diabetes. The rate of hearing recovery was determinied by comparing the matching propensity score between the primary ITS and the control group according to the American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNS) guideline and Siegel’s criteria.Results Of the 440 ISSNHL patients, 28.9% received systemic steroid only and 62.6% of patients were managed with systemic steroid plus salvage ITS. Thirty-seven patients (8.4%) were managed with ITS only. While 36.8% of patients completely recovered, 35.0% did not recover their hearing according to AAO-HNS guideline. The propensity score matching showed that the initial hearing level, age and onset of treatment were not significantly different between the primary ITS and control group. Regarding the treatment outcome, complete recovery rate for the primary ITS group and control group were 29.7% and 33.8%, respectively. Although the recovery rate of the salvage ITS protocol group was higher than that of the primary ITS group, statistical significance was not identified.Conclusion Treatment strategies of primary ITS and salvage ITS protocol did not significantly affect the clinical outcomes of ISSNHL differently.


2019 ◽  
Vol 28 (6) ◽  
pp. 879-886 ◽  
Author(s):  
Rishi J. Desai ◽  
Richard Wyss ◽  
Younathan Abdia ◽  
Sengwee Toh ◽  
Margaret Johnson ◽  
...  

Cancers ◽  
2020 ◽  
Vol 12 (9) ◽  
pp. 2429
Author(s):  
Maike Trommer ◽  
Jaika Kinsky ◽  
Anne Adams ◽  
Martin Hellmich ◽  
Max Schlaak ◽  
...  

Immune checkpoint inhibition (ICI) has been established as successful modality in cancer treatment. Combination concepts are used to optimize treatment outcome, but may also induce higher toxicity rates than monotherapy. Several rationales support the combination of radiotherapy (RT) with ICI as radioimmunotherapy (RIT), but it is still unknown in which clinical situation RIT would be most beneficial. Therefore, we have conducted a retrospective matched-pair analysis of 201 patients with advanced-stage cancers and formed two groups treated with programmed cell death protein 1 (PD-1) inhibitors only (PD1i) or in combination with local RT (RIT) at our center between 2013 and 2017. We collected baseline characteristics, programmed death ligand 1 (PD-L1) status, mutational status, PD-1 inhibitor and RT treatment details, and side effects according to the Common Terminology Criteria for Adverse Events (CTCAE) v.5.0. Patients received pembrolizumab (n = 93) or nivolumab (n = 108), 153 with additional RT. For overall survival (OS) and progression-free survival (PFS), there was no significant difference between both groups. After propensity score matching (PSM), we analyzed 96 patients, 67 with additional and 29 without RT. We matched for different covariates that could have a possible influence on the treatment outcome. The RIT group displayed a trend towards a longer OS until the PD1i group reached a survival plateau. PD-L1-positive patients, smokers, patients with a BMI ≤ 25, and patients without malignant melanoma showed a longer OS when treated with RIT. Our data show that some subgroups may benefit more from RIT than others. Suitable biomarkers as well as the optimal timing and dosage must be established in order to achieve the best effect on cancer treatment outcome.


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