Propensity analysis
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OBJECTIVES: The HeartWare HVAD (HW) and the HeartMate3 (HM3) are presently the most commonly used continuous-flow left ventricular assist devices worldwide. We compared the outcomes of patients supported with either of these 2 devices based on data from the EUROMACS (European Registry for Patients with Mechanical Circulatory Support). METHODS: A retrospective analysis of the survival and complications profile in propensity score-matched adult patients enrolled in the EUROMACS between 01 January 2016 and 01 September 2020 and supported with either an HW or HM3. Matching included demographic parameters, severity of cardiogenic shock and risk-modifying end-organ parameters that impact long-term survival. Survival on device and major postoperative adverse events were analysed. RESULTS: Following 1:1 propensity score matching, each group consisted of 361 patients. Patients were well balanced (<0.1 standardized mean difference). The median follow-up was similar in both groups [396 (interquartile range (IQR) 112-771) days for HW and 376 (IQR 100-816) days for HM3]. The 2-year survival was similar in both groups [HW: 61% 95% confidence interval (CI) (56-67%) vs HM3: 68% 95% CI (63-73%) (stratified hazard ratio for mortality: 1.13 95% CI (0.83-1.54), P = 0.435].The cumulative incidence for combined major adverse events and unexpected readmissions was similar in both groups [subdistribution hazard ratio (SHR) 1.0 (0.84-1.21), P = 0.96]. Patients in the HW group demonstrated a higher risk of device malfunction [SHR 2.44 (1.45-3.71), P < 0.001], neurological dysfunction [SHR 1.29 (1.02-1.61), P = 0.032] and intracranial bleeding [SHR 1.76 (1.13-2.70), P = 0.012]. CONCLUSIONS: Mid-term survival in both groups was similar in a propensity-matched analysis. The risk of device malfunction, neurological dysfunction and intracranial bleeding was significantly higher in HW patients.
BACKGROUND: We aimed to assess the clinical, oncological, and pathological impact of en bloc resection of bladder tumors (ERBT) compared with conventional transurethral resection of bladder tumors (cTURBT) for pT1 high-grade (HG) bladder cancer. PATIENTS AND METHODS: We retrospectively analyzed the record of 326 patients (cTURBT: n = 216, ERBT: n = 110) diagnosed with pT1 HG bladder cancer at multiple institutions. The cohorts were matched by one-to-one propensity scores based on patient and tumor demographics. Recurrence-free survival (RFS), progression-free survival (PFS), cancer-specific survival (CSS), and perioperative and pathologic outcomes were compared. The prognosticators of RFS and PFS were analyzed using the Cox proportional hazard model. RESULTS: After matching, 202 patients (cTURBT: n = 101, ERBT: n = 101) were retained. There were no differences in perioperative outcomes between the two procedures. The 3-year RFS, PFS, and CSS were not different between the two procedures (p = 0.7, 1, and 0.7, respectively). Among patients who underwent repeat transurethral resection (reTUR), the rate of any residue on reTUR was significantly lower in the ERBT group (cTURBT: 36% versus ERBT: 15%, p = 0.029). Adequate sampling of muscularis propria (83% versus 93%, p = 0.029) and diagnostic rates of pT1a/b substaging (90% versus 100%, p < 0.001) were significantly better in ERBT specimen compared with cTURBT specimen. On multivariable analyses, pT1a/b substaging was a prognosticator of disease progression. CONCLUSIONS: In patients with pT1HG bladder cancer, ERBT had similar perioperative and mid-term oncologic outcomes compared with cTURBT. However, ERBT improves the quality of resection and specimen, yielding less residue on reTUR and yielding superior histopathologic information such as substaging.
- MeSH
- cystektomie MeSH
- lidé MeSH
- nádory močového měchýře * chirurgie patologie MeSH
- retrospektivní studie MeSH
- tendenční skóre MeSH
- urologické chirurgické výkony metody MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
BACKGROUND: In situations of markedly different population characteristics and weak population overlap, inverse propensity score (PS) weights suffer from extreme values. The new propensity score weighting method using overlap weights (PSOW) overcomes this limitation by estimating the overlap population at the point of highest mutual overlap, thus may be preferred to other balancing methods (trimming, target, or inverse weights) in some situations. OBJECTIVES: To evaluate the performance of PSOW with regorafenib effectiveness data from previously treated patients with metastatic colorectal cancer based on the Czech national registry data (regorafenib) and a global phase 3 randomized clinical trial (RCT) (placebo). The second goal was to assess the cost-effectiveness of regorafenib versus placebo. METHODS: Individual data on progression-free survival (PFS)/overall survival (OS) were balanced via PSOW for age, sex, Eastern Cooperative Oncology Group performance status, number of treatment lines, metastatic colorectal cancer location, KRAS mutation, and time from metastases estimated using logistic regression. The weighted Kaplan-Meier PFS/OS curves were used in a 3-state partitioned survival model. The R code is provided. RESULTS: In comparison with target or inverse PS weights, PSOW showed remarkable performance measured by effective sample size and PS weight distribution or extreme weights despite the weak overlap between the registry and RCT. In the registry or RCT cohort, regorafenib provided better survival compared with the RCT. The new PSOW hazard ratio for OS was 0.53 (RCT: 0.79), which is conservative compared with inverse or target weights with a hazard ratio of 0.44 and 0.27, respectively. CONCLUSION: This is the first use of PSOW for clinical data and cost-effectiveness analysis. It is promising in cases of weak or small population overlap and makes pharmacoeconomic modeling, in such cases, feasible.
- MeSH
- analýza nákladů a výnosů MeSH
- doba přežití bez progrese choroby MeSH
- fenylmočovinové sloučeniny ekonomika terapeutické užití MeSH
- klinické zkoušky, fáze III jako téma MeSH
- kolorektální nádory farmakoterapie ekonomika mortalita MeSH
- lidé středního věku MeSH
- lidé MeSH
- přežití bez známek nemoci MeSH
- pyridiny ekonomika terapeutické užití MeSH
- randomizované kontrolované studie jako téma MeSH
- registrace MeSH
- tendenční skóre * MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- hodnotící studie MeSH
- práce podpořená grantem MeSH
BACKGROUND: Evidence of widespread disparities in healthcare for racial and ethnic minorities is well documented. This study aims to evaluate differences in surgical outcomes after breast reduction surgery (BRS) according to patients' ethnicities. METHODS: The American College of Surgeons National Surgical Quality Improvement Program (ACS NSQIP) database (2005-2018) was utilized to analyze two propensity score matched patient cohorts-White and non-White-that underwent BRS. Preoperative variables assessed included demographic data and comorbidities such as diabetes mellitus, hypertension, and obesity. Postoperative outcomes assessed were medical complications, minor and major surgical complications, as well as mortality. RESULTS: In total, 23268 patients underwent BRS and met the inclusion criteria. After propensity score matching, the two cohorts were matched with respect to these preoperative variables, and 7187 patients were included in each cohort of White and non-White patients (total 14374). After matching, overall 30-day major complications were not significantly different between White and non-White cohort (2.25% vs 2.14%, p=0.65). After accounting for differences in confounding variables at the patient and socioeconomic level, racial and ethnic minorities who underwent breast reduction were found to experience fewer minor surgical complications. The analysis of temporal trends identified an overall rise in the number of patients seeking BRS, with a higher increase noted in the non-White population. CONCLUSION: Overall, our findings are reassuring exemptions to prevalent racial and ethnic health inequalities and can serve as a positive example for adequate and fair provision of surgical care.
- MeSH
- etnicita MeSH
- kohortové studie MeSH
- lidé MeSH
- mamoplastika * MeSH
- pooperační komplikace epidemiologie MeSH
- retrospektivní studie MeSH
- tendenční skóre MeSH
- zlepšení kvality * MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
INTRODUCTION: Two antitumor necrosis factor therapies (infliximab [IFX] and adalimumab [ADA]) have been approved for the treatment of pediatric Crohn's disease (CD) but have not been compared in head-to-head trials. The aim of this study was to compare the efficacy and safety of ADA and IFX by propensity score matching in a prospective cohort of pediatric patients with luminal CD and at least a 24-month follow-up. METHODS: Among 100 patients, 75 met the inclusion criteria, and 62 were matched by propensity score. We evaluated time to treatment escalation as the primary outcome and primary nonresponse, predictors of treatment escalation and relapse, serious adverse events, pharmacokinetics, and effect of concomitant immunomodulators as secondary outcomes. RESULTS: There was no difference between ADA and IFX in time to treatment escalation (HR = 0.63 [95% CI 0.31-1.28] P = 0.20), primary nonresponse (P = 0.95), or serious adverse events. The median (interquartile range) trough levels at the primary outcome were 14.05 (10.88-15.40) and 6.15 (2.08-6.58) μg/mL in the ADA and IFX groups, respectively. On a multivariate analysis, the combination of anti-Saccharomyces cerevisiae antibody negativity and antineutrophil cytoplasmic antibody positivity was a strong independent predictor of treatment escalation (HR 5.19, [95% CI 2.41-11.18], P < 0.0001). The simple endoscopic score for CD, L3 disease phenotype, and use of concomitant immunomodulators for at least the first 6 months revealed a trend toward significance on a univariate analysis. DISCUSSION: Propensity score matching did not reveal substantial differences in efficacy or safety between ADA and IFX. The anti-S. cerevisiae antibody negativity and antineutrophil cytoplasmic antibody positivity combination is a strong predictor of treatment escalation.
- MeSH
- adalimumab škodlivé účinky MeSH
- Crohnova nemoc * diagnóza farmakoterapie MeSH
- dítě MeSH
- imunologické faktory MeSH
- infliximab terapeutické užití MeSH
- lidé MeSH
- prospektivní studie MeSH
- protilátky proti cytoplazmě neutrofilů MeSH
- tendenční skóre MeSH
- výsledek terapie MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- MeSH
- cystektomie MeSH
- lidé MeSH
- nádory močového měchýře * chirurgie MeSH
- tendenční skóre MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
BACKGROUND: Despite recent advances in management of patients with advanced heart failure, mortality remains high. Aim of this study was to compare impact of different aetiology of ischaemic and idiopathic cardiomyopathy on early outcomes and long-term survival of patients after left ventricular assist device implantation. METHODS: European Registry for Patients with Mechanical Circulatory Support (EUROMACS) gathers clinical data and follow-up parameters of LVAD recipients. Patients enrolled in the EUROMACS registry with primary diagnosis of either ischaemic (n = 1190) or idiopathic (n = 812) cardiomyopathy were included. Primary Endpoints were early mortality as well as long-term survival. Secondary endpoint were major postoperative adverse events, such as need for rethoracotomy. Additionally, a propensity-score matching analysis was performed for patients with ischaemic (n = 509) and idiopathic (n = 509) cardiomyopathy. RESULTS: In terms of basic demographics and baseline parameters the two groups significantly differed as expected before propensity-score matching due to different aetiology of cardiomyopathy. Seven-day (52 (4.4%) versus 18 (2.2%); p = 0.009), 30-day (153 (12.9%) versus 73 (9.0%); p = 0.008) and in-hospital mortality (253 (19.7%) versus 123 (15.1%); p = 0.009) were significantly lower in the idiopathic cardiomyopathy group compared to the ischaemic cardiomyopathy group, whereas after propensity-score matching 30-day (p = 0.169) was comparable and in-hospital mortality (p = 0.051) was almost significant. Kaplan-Meier survival analysis revealed no significant difference in regard of long-term survival after propensity-score matching (Breslow-test p = 0.161 and LogRank-test p = 0.113). CONCLUSION: Though patients with ischaemic and idiopathic cardiomyopathy suffer from different cardiomyopathy aetiologies, 30-day-mortality and long-term survival of both groups were similar leading to the conclusion that covariates predominately influence mortality and survival of ischaemic and idiopathic cardiomyopathies.
BACKGROUND: The current ACC/AHA guidelines on hypertrophic cardiomyopathy (HCM) caution that alcohol septal ablation (ASA) might be less effective in patients with left ventricular outflow tract obstruction (LVOTO) ≥ 100 mm Hg. METHODS: We used a multinational registry to evaluate the outcome of ASA patients according to baseline LVOTO. RESULTS: A total of 1346 ASA patients were enrolled and followed for 5.8 ± 4.7 years (7764 patient-years). The patients with baseline LVOTO ≥ 100 mm Hg were significantly older (61 ± 14 years vs 57 ± 13 years; P < 0.01), more often women (60% vs 45%; P < 0.01), and had a more pronounced HCM phenotype than those with baseline LVOTO < 100 mm Hg. There were no significant differences in the occurrences of 30-day major cardiovascular adverse events in the 2 groups. After propensity score matching (2 groups, 257 pairs of patients), the long-term survival was similar in both groups (P = 0.10), the relative reduction of LVOTO was higher in the group with baseline LVOTO ≥ 100 mm Hg (82 ± 21% vs 73 ± 26%; P < 0.01), but the residual resting LVOTO remained higher in this group (23 ± 29 mm Hg vs 13 ± 13 mm Hg; P < 0.01). Dyspnoea (NYHA functional class) at the most recent clinical check-up was similar in the 2 groups (1.7 ± 0.7 vs 1.7 ± 0.7; P = 0.85), and patients with baseline LVOTO ≥ 100 mm Hg underwent more reinterventions (P = 0.02). CONCLUSIONS: After propensity matching, ASA patients with baseline LVOTO ≥ 100 mm Hg had similar survival and dyspnoea as patients with baseline LVOTO < 100 mm Hg, but their residual LVOTO and risk of repeated procedures were higher.
- MeSH
- dyspnoe etiologie MeSH
- hypertrofická kardiomyopatie * komplikace diagnóza chirurgie MeSH
- kardiochirurgické výkony * MeSH
- lidé MeSH
- obstrukce výtokového traktu levé komory srdeční * MeSH
- obstrukce výtoku ze srdeční komory * chirurgie MeSH
- retrospektivní studie MeSH
- tendenční skóre MeSH
- výsledek terapie MeSH
- Check Tag
- lidé MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
BACKGROUND: The recent introduction of leadless pacemakers (PMs) was aimed to eliminate transvenous lead- and pocket-related complications. While the initial results with the leadless PMs seem promising, the nonrandomized nature, limited implant experience of operators, and short follow-up period of these studies preclude a simple comparison to transvenous PMs. OBJECTIVES: The objective of this study was to provide a balanced comparison of leadless and transvenous single-chamber PM therapies through a propensity score-matched analysis. METHODS: Leadless patients from 3 experienced leadless implant centers were propensity score-matched to VVI-R patients from a contemporary prospective multicenter transvenous PM registry. The primary outcome was device-related complications that required invasive intervention during mid-term follow-up. Separate analyses including and excluding PM advisory-related complications were performed. RESULTS: A total of 635 patients were match-eligible (leadless: n = 254; transvenous: n = 381), of whom 440 patients (median age 78 years; interquartile range 70-84 years; 61% men) were successfully matched (leadless: n = 220 vs transvenous: n = 220). The complication rate at 800 days of follow-up was 0.9% (95% confidence interval [CI] 0%-2.2%) in the leadless group vs 4.7% (95% CI 1.8%-7.6%) in the transvenous group when excluding PM advisory-related complications (P = .02). When including these PM advisory-related complications, the complication rate at 800 days increased to 10.9% (95% CI 4.8%-16.5%) in the leadless group vs 4.7% (95% CI 1.8%-7.6%) in the transvenous group (P = .063). CONCLUSION: This study reveals favorable complication rates for leadless compared to transvenous single-chamber pacing therapy at mid-term follow-up in a propensity score-matched cohort. When including PM advisory-related complications, this advantage is no longer observed.
- MeSH
- časové faktory MeSH
- design vybavení MeSH
- elektrokardiografie MeSH
- kardiostimulátor * MeSH
- katetrizace centrálních vén metody MeSH
- lidé MeSH
- následné studie MeSH
- retrospektivní studie MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- srdeční arytmie patofyziologie terapie MeSH
- tendenční skóre * MeSH
- výsledek terapie MeSH
- Check Tag
- lidé MeSH
- mužské pohlaví MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- klinické zkoušky MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- srovnávací studie MeSH
Randomizované klinické studie jsou považovány za zlatý standard pro medicínu založenou na důkazech, avšak tyto studie nemusejí být vždy proveditelné nebo etické a vědci musejí spoléhat na observační studie nebo výzkumné databáze. Nicméně získání spolehlivých výsledků z těchto studií vyžaduje odstranění potenciálního vlivu dalších faktorů. Naštěstí existuje několik statistických metod schopných identifikovat a snížit vliv dalších faktorů. Jednou z nich je metoda propensity skóre, která je v současné době často používána pro odhad relevantních klinických účinků, jež jsou adjustovány pro dané zkreslení. Tato práce si klade za cíl poskytnout stručný a praktický návod na použití propensity skóre prostřednictvím snadno srozumitelné případové studie. Případová studie je zaměřena na genderové rozdíly v úmrtnosti u pacientů s akutním srdečním selháním v české výzkumné databázi AHEAD (Acute Heart Failure Database).
Randomized clinical trials represent the gold standard of the evidence based medicine research; nevertheless they may not always be feasible or ethical and the researchers have to rely on observational studies or research databases. However, obtaining reliable results from these studies requires the elimination of potential influence of confounding factors. Fortunately, several statistical methods capable of identifying and reducing the impact of confounding factors exist. One of them is the propensity score which has been frequently used in recent times to estimate relevant clinical effects adjusted for given confounders. This work aims to provide a concise and practical guide to propensity scores by means of an easily understandable case study. The case study is focused on gender differences in mortality rates of patients with acute heart failure in the Czech research database AHEAD (Acute Heart Failure Database).
- Klíčová slova
- propensity skóre,
- MeSH
- akutní koronární syndrom * mortalita MeSH
- databáze jako téma * MeSH
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- pozorování * MeSH
- randomizované kontrolované studie jako téma MeSH
- statistika jako téma * MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- práce podpořená grantem MeSH