Multicenter Retrospective Cohort Study Research Articles

Development of a clinical prognostic model for primary plasma cell leukemia patients treated with novel agents: a multicenter retrospective cohort study.

The prognosis of primary plasma cell leukemia (pPCL) is poor, and the relevant prognostic factors are incompletely understood. We aimed to explore the prognostic factors and develop a validated prognostic prediction model for pPCL patients in the new era. This multicenter retrospective study was conducted across 16 hospitals in China. Cox proportional hazards regression analysis was used to develop a prediction model. The predictive performance of the model was assessed using multiple metrics. Internal validation was conducted using bootstrap resampling. A total of 102 pPCL patients were included in this study, and 57 (55.9%) were male. The 12-month, 24-month, and 36-month OS rates for pPCL patients were 75.4%, 58.3%, and 47.6%, respectively. An overall survival prognostic nomogram for pPCL patients was established by integrating independent prognostic factors, including age, B2MG, and del17p. The nomogram exhibited good performance, with a C-index of 0.720 (95% CI 0.642-0.797) and an AUC of 0.653. Bootstrap validation yielded a C-index of 0.721 (95% CI 0.629-0.787) and an AUC of 0.653 (95% CI 0.546-0.759), indicating a relatively good fit of the calibration curve. A nomogram incorporating age, B2MG grade, and del17p were developed and validated to accurately and consistently predict the prognosis of pPCL patients.

Hematocrit and Albumin Levels at Admission Predict in-Hospital Mortality in Pediatric COVID-19 Omicron Variant Patients.

The Omicron variant is the present predominant COVID-19 strain worldwide. Accurate mortality prediction can facilitate risk stratification and targeted therapies. The study aimed to evaluate the feasibility of the difference in hematocrit and albumin (HCT-ALB) levels, alone or combined with the pediatric Sequential Organ Failure Assessment (pSOFA) score and lactate level, to predict the in-hospital mortality of COVID-19 Omicron variant-infected pediatric patients. A multicenter retrospective cohort study was performed for children with COVID-19 Omicron variant infection between December 2021 and January 2022. The demographics, clinical characteristics, hospital admission laboratory test results, and treatments were recorded. The in-hospital mortality was documented. The associations between HCT-ALB levels and mortality, and between HCT-ALB+pSOFA+lactate and mortality were analyzed. A total of 119 children were included. The median age was 1.6 (interquartile range: 0.5-6.2) years old. There were 70 boys and 49 girls. The mortality rate was 14.3% (17/119). The univariate and multivariate Cox regression analysis revealed that HCT-ALB was associated to in-hospital mortality (hazard ratio: 1.500, 95% confidence interval: 1.235-1.822, p<0.001). The receiver operating characteristic curve analysis revealed that HCT-ALB can be used to accurately predict in-hospital mortality at a cut-off value of -0.7 (area under the curve: 0.888, sensitivity: 0.882, specificity: 0.225, Youden index: 0.657, p<0.001). These patients were assigned into three groups based on the HCT-ALB level, pSOFA score, and lactate level (low-, medium-, and high-risk groups). The Kaplan-Meier analysis revealed that the mortality increased in the high-risk group, when compared to the medium-risk group (p<0.01). The latter group had a higher mortality, when compared to the low-risk group (p<0.01). The HCT-ALB level can be applied to predict the in-hospital mortality of children infected with the COVID-19 Omicron variant. Its combination with other variables can improve prediction performance.

The outcomes of component separation technique versus no component separation technique in the repair of large ventral hernias and impact on quality of life: a multicenter retrospective cohort study.

This study aimed to compare the results of patients who underwent anterior component separation techniques (ACST) and those who did not undergo component separation techniques (non-CST) in complicated ventral hernia repairs (VHRs) and to investigate the effect of these techniques on quality of life (QoL). A total of 105 patients who were operated for large ventral hernias were retrospectively analyzed. The patients were divided into the ACST group (n = 48) and the non-CST group (n = 57). Demographic, intraoperative, and postoperative data were recorded. Postoperative follow-up was conducted at 2 and 4 weeks, and 6, 12, and 24 months. The primary and secondary outcomes and QoL were measured. The female ratio was higher in both groups (P = 0.512). There was no significant difference between age and body mass index between the groups (P = 0.705 and P = 0.803). The mean defect size and mesh size were similar between the groups (P = 0.775 and P = 0.245). The mean operation duration and amount of blood loss were similar between the groups (P = 0.801 and P = 0.142). There was no statistically significant difference in the median visual analog scale scores between the groups (P = 0.551). During follow-up, only 3 patients (6.3%) in the ACST group and 4 patients (7.0%) in the non-CST group had recurrence. There was no significant difference in the short- and long-term QoL between the groups. The ACST is a feasible surgical option for patients with complicated VHRs. In addition, by improving QoL, the recurrence rate is similar to patients undergoing standard VHR.

241.3: A multicenter retrospective cohort study on management protocols and clinical outcomes after ABO-incompatible kidney transplantation in India.

241.3: A multicenter retrospective cohort study on management protocols and clinical outcomes after ABO-incompatible kidney transplantation in India.

975P Lenvatinib plus drug-eluting bead transarterial chemoembolization with or without hepatic arterial infusion chemotherapy for hepatocellular carcinoma (> 7 cm) with portal vein tumor thrombosis: A retrospective multicenter cohort study

975P Lenvatinib plus drug-eluting bead transarterial chemoembolization with or without hepatic arterial infusion chemotherapy for hepatocellular carcinoma (> 7 cm) with portal vein tumor thrombosis: A retrospective multicenter cohort study

Timing of relapse as a key indicator of steroid-sparing requirements in childhood idiopathic nephrotic syndrome.

Managing children with frequent relapses or steroid-dependent nephrotic syndrome poses challenges due to recurrent relapses necessitating prolonged steroid exposure, thus increasing susceptibility to long-term complications. Identifying those at risk of poor response to steroid therapy may be helpful to guide timely intervention with steroid-sparing agents. This study aimed to identify factors associated with steroid-sparing agent needs in children with frequent relapses or steroid-dependent nephrotic syndrome. A retrospective multicenter cohort study was conducted by reviewing the medical records of children with idiopathic nephrotic syndrome treated between 2006 and 2023. Cox proportional regression analyzed prognostic factors for steroid-sparing agent requirements in children with frequent relapses or steroid-dependent nephrotic syndrome. The time-to-event analysis utilizing the Kaplan-Meier estimate examined the proportion of children needing steroid-sparing agents after diagnosis. Medical records of 121 children (85 males) diagnosed with idiopathic nephrotic syndrome at a median age of 4.5 years (range 1.3-12.8) were reviewed over a median follow-up of 3.7 years (range 1.0-15.0). Time to subsequent relapse post-frequent relapses or steroid-dependent nephrotic syndrome diagnosis (at 3-month threshold) emerged as the sole significant predictor of steroid-sparing agent requirement, adjustedhazard ratio (aHR) = 2.26, 95% confidence interval (CI) 1.26-4.05. Kaplan-Meier analysis indicated that an earlier first relapse (< 3 months) led to earlier steroid-sparing agent requirement (log-rank p = 0.005). Children who relapsed within 3 months post-frequent relapses or steroid-dependent nephrotic syndrome diagnosis exhibited a higher frequency of relapses, a greater incidence of steroid-related adverse events, and were more likely to develop steroid dependency. Early subsequent relapse following diagnosis of frequent relapses or steroid-dependent nephrotic syndrome was linked to earlier requirement of steroid-sparing agent therapy. Further prospective research is necessary to confirm this observation.

Establishing a nomogram for predicting necrotizing enterocolitis in premature infants: A retrospective multicenter cohort study

BackgroundTo construct a nomogram for predicting necrotizing enterocolitis (NEC) in preterm infants. MethodsA total of 4,724 preterm infants who were admitted into 8 hospitals between April 2019 and September 2020 were initially enrolled this retrospective multicenter cohort study. Finally, 1,092 eligible cases were divided into training set and test set based on a 7:3 ratio. A univariate logistic regression analysis was performed to compare the variables between the two groups. Stepwise backward regression, LASSO regression, and Boruta feature selection were utilized in the multivariate analysis to identify independent risk factors. Then a nomogram model was constructed based on the identified risk factors. ResultsRisk factors for NEC included gestational diabetes mellitus, gestational age, small for gestational age, patent ductus arteriosus, septicemia, red blood cell transfusion, intravenous immunoglobulin, severe feeding intolerance, and absence of breastfeeding. The nomogram model developed based on these factors showed well discriminative ability. Calibration and decision curve analysis curves confirmed the good consistency and clinical utility of the model. ConclusionsWe developed a nomogram model with strong discriminative ability, consistency, and clinical utility for predicting NEC. This model could be valuable for the early prediction of preterm infants at risk of developing NEC.

Association of gastrointestinal symptoms with mortality in patients with severe fever with thrombocytopenia syndrome

BackgroundThe clinical significance of gastrointestinal (GI) symptoms in patients with severe fever and thrombocytopenia syndrome (SFTS) is poorly characterized. This study aimed to determine the prevalence and effect of GI symptoms on the prognosis of patients with SFTS. MethodsThis was a retrospective multi-center cohort study that included hospitalized patients with SFTS from three institutions between October 2010 and August 2022. The risk factors for mortality and intensive care unit (ICU) admission were identified by Cox and logistic regression analyses, respectively. Kaplan-Meier curves were used to analyze the cumulative mortality risk. ResultsAmong 304 patients, the median age was 62.0 years and 51.0 % of the patients were male. A total of 202 patients (66.4 %) had at least one GI symptom on admission. Diarrhea (69.8 %) and nausea (57.4 %) were the most common symptoms. Patients with GI symptoms had lower male proportion (46.0 % vs. 60.8 %, P = 0.015), higher aspartate aminotransferase (177.5 U/L vs. 118.0 U/L, P = 0.010) and lactic dehydrogenase (771.0 U/L vs. 666.5 U/L, P = 0.017) levels than that of patients without GI symptoms. However, there was no significant difference in mortality rates (23.8 % vs. 21.6 %, P = 0.668) and ICU admission (14.4 % vs. 12.7 %, P = 0.701) between SFTS patients with and without GI symptoms. Multivariate analysis suggested that GI symptoms at admission were not associated with mortality and ICU admission. ConclusionsGI symptoms are common in patients with SFTS. However, the presence of GI symptoms was not an independent risk factor for poor prognosis.

379P Safety and efficacy of trastuzumab deruxtecan and concomitant radiation therapy in patients with metastatic breast cancer: A multicentre international retrospective cohort study

379P Safety and efficacy of trastuzumab deruxtecan and concomitant radiation therapy in patients with metastatic breast cancer: A multicentre international retrospective cohort study

405.1: A multicenter retrospective cohort study of Indian experience with the kidney paired donation transplantation.

405.1: A multicenter retrospective cohort study of Indian experience with the kidney paired donation transplantation.

367. IMPACT OF NEOADJUVANT IMMUNOTHERAPY ON POSTOPERATIVE COMPLICATIONS IN RESECTABLE ESOPHAGEAL ADENOCARCINOMA

Abstract Background Several studies are ongoing on the use of immunotherapy in the neoadjuvant setting for esophageal and gastroesophageal junction cancer but it is unclear the effect of an increased activation of the immune system on surgery and the postoperative course. Methods This multicenter retrospective cohort study included data from 3 high-volume centers for upper-GI surgery. Only patients with esophageal adenocarcinoma who underwent neoadjuvant therapy and radical surgery between 2015 and 2022 were included. Clinical characteristics and the postoperative outcomes were compared between patients who received standard neoadjuvant therapy or chemotherapy plus immunotherapy. A 1-to-1 matched comparison was also performed. The difference in overall survival (OS) and disease free survival (DFS) between the two groups was also evaluated. Results Overall, 563 patients were included and 21 received preoperative Pembrolizumab or Trastuzumab combined with at least a couple of chemotherapy drugs. The incidence of complications was 47% in the group with immunotherapy and 40.7% in the other group (p=0.53). The matched analysis confirmed no differences in rate of complications (p&amp;gt;0.99), gravity of complications (p=0.17) and pulmonary complications (p&amp;gt;0.99). Anastomotic leak rate was 4.7% in the immunotherapy group and 19% in the other one (p=0.34). Overall survival and disease free survival were comparable (respectively p=0.4 and p=0.9) also after the matched analysis (respectively p=0.15 and p=0.52). Conclusion Neoadjuvant immunotherapy-based regimen does not affect the postoperative course, in particular it does not appear to aggravate the major complications of esophagectomy.

The recurrence of disease activity after ocrelizumab discontinuation in multiple sclerosis

IntroductionOcrelizumab (OCR) is a highly effective treatment of multiple sclerosis (MS), and B cell repopulation profiles suggest that it might be used as an immune reconstitution therapy. However, data on disease recurrence after stopping treatment with OCR are scarce. Our objective was to evaluate the recurrence of disease activity after OCR discontinuation. MethodsIn this multicenter retrospective cohort study, we included MS patients who discontinued OCR, without switching to another treatment, for twelve months or more, after having received at least one full dosage of 600mg. We defined focal inflammation as the occurrence of a clinical relapse or significant MRI activity (≥3 new T2 lesions or ≥2 contrast-enhancing lesions). ResultsWe included 53 MS patients; 41 relapsing remitting (RRMS), 5 secondary progressive (SPMS) and 7 primary progressive (PPMS) patients. Median follow-up period after OCR discontinuation was 16 months. We only observed focal inflammation after discontinuation in RRMS patients; 2.4 % (1/41) patients presented with significant MRI activity and matching clinical symptoms, and 7.3% (3/41) patients presented with a suspected clinical relapse without radiological activity: a total of 9.8% (4/41) at a median time of 17 months after the last infusion. DiscussionWe found focal inflammation after discontinuation of OCR in 4 (9.8%) of the RRMS patients, of which 1 was radiologically confirmed. Our observations highlight that recurrence of focal inflammation seems low but discontinuation may not be appropriate for everyone. Further larger studies are important to determine the immune reconstitution therapy potential of OCR.

IBCL-236 Similar Safety Profile for Rituximab and Obinutuzumab as Maintenance for Follicular Lymphoma: Evidence From a Real-World Multicenter Retrospective Cohort Study

IBCL-236 Similar Safety Profile for Rituximab and Obinutuzumab as Maintenance for Follicular Lymphoma: Evidence From a Real-World Multicenter Retrospective Cohort Study

Presentation and Outcome in S1P-RM and Natalizumab-Associated Progressive Multifocal Leukoencephalopathy: A Multicenter Cohort Study.

Progressive multifocal leukoencephalopathy (PML) is a severe neurologic disease resulting from JC virus reactivation in immunocompromised patients. Certain multiple sclerosis (MS) disease-modifying therapies (DMTs) are associated with PML risk, such as natalizumab and, more rarely, sphingosine-1-phosphate receptor modulators (S1P-RMs). Although natalizumab-associated PML is well documented, information on S1P-RM-associated PML is limited. The aim of this study is to compare clinical presentations and outcomes between the 2 groups. A retrospective multicenter cohort study included patients with PML from 2009 to 2022 treated with S1P-RMs or natalizumab. Data on clinical and radiologic presentation, outcomes, immune reconstitution inflammatory syndrome (IRIS), survival, disability (using the modified Ranking scale-mRS), and MS relapses post-PML were analyzed. Of 88 patients, 84 were analyzed (20 S1P-RM, 64 natalizumab). S1P-RM-associated PML was diagnosed in older patients (median age 52 vs 44 years, p < 0.001) and after longer treatment duration (median 63.9 vs 40 months, p < 0.001). Similarly, S1P-RM patients were more prone to show symptoms at diagnosis (100 vs 80.6%, p = 0.035), had more disseminated lesions (80% vs 34.9%, p = 0.002), and had higher gadolinium enhancement (65% vs 39.1%, p = 0.042). Natalizumab patients had a higher IRIS development rate (OR: 8.3 [1.92-33.3]). Overall, the outcome (mRS) at 12 months was similar in the 2 groups (OR: 0.81 [0.32-2.0]). Yet, post-treatment MS activity was higher in S1P-RM cases (OR: 5.7 [1.4-22.2]). S1P-RM-associated PML shows reduced IRIS risk but higher post-treatment MS activity. Clinicians should tailor post-PML treatment based on pre-PML medication.

Prognostic Significance of Hemodynamics in Patients With Transposition of the Great Arteries and Systemic Right Ventricle.

Patients with transposition of the great arteries (TGA) and systemic right ventricle often confront significant adverse cardiac events. The prognostic significance of invasive hemodynamic parameters in this context remains uncertain. Our hypothesis is that the aortic pulsatility index and hemodynamic profiling utilizing invasive measures provide prognostic insights for patients with TGA and a systemic right ventricle. This retrospective multicenter cohort study encompasses adults with TGA and a systemic right ventricle who underwent cardiac catheterization. Data collection, spanning from 1994 to 2020, encompasses clinical and hemodynamic parameters, including measured and calculated values such as pulmonary capillary wedge pressure, aortic pulsatility index, and cardiac index. Pulmonary capillary wedge pressure and cardiac index values were used to establish 4 distinct hemodynamic profiles. A pulmonary capillary wedge pressure of ≥15 mm Hg indicated congestion, termed wet, while a cardiac index <2.2 L/min per m2 signified inadequate perfusion, labeled cold. The primary outcome comprised a composite of all-cause death, heart transplantation, or the requirement for mechanical circulatory support. Of 1721 patients with TGA, 242 individuals with available invasive hemodynamic data were included. The median follow-up duration after cardiac catheterization was 11.4 (interquartile range, 7.5-15.9) years, with a mean age of 38.5±10.8 years at the time of cardiac catheterization. Among hemodynamic parameters, an aortic pulsatility index <1.5 emerged as a robust predictor of the primary outcome, with adjusted hazard ratios of 5.90 (95% CI, 3.01-11.62; P<0.001). Among the identified 4 hemodynamic profiles, the cold/wet profile was associated with the highest risk for the primary outcome, with an adjusted hazard ratio of 3.83 (95% CI, 1.63-9.02; P<0.001). A low aortic pulsatility index (<1.5) and the cold/wet hemodynamic profile are linked with an elevated risk of adverse long-term cardiac outcomes in patients with TGA and systemic right ventricle.

The efficacy of diazepam administration during embryo transfer: a retrospective multicenter cohort study on reproductive outcomes.

This retrospective multicenter cohort study aimed to investigate the impact of diazepam administration during embryo transfer on reproductive outcomes, focusing primarily on the live birth rate. Secondary outcomes included the positive beta-hCG rate, clinical pregnancy rate, miscarriage rate, ectopic pregnancy rate, and preterm birth rate. Data from 5607 embryo transfers, encompassing 465 cases with diazepam administration, were retrospectively analyzed. The study included single blastocyst transfers from 12 clinics in Portugal and Spain between January 2015 and December 2022. Comparison of reproductive outcomes between patients receiving diazepam and those who did not showed no statistically significant differences. Positive beta-hCG rates (60.8% non-diazepam vs. 60.4% diazepam, p = 0.92, adjusted p = 0.32) and clinical pregnancy rates (45.6% non-diazepam vs. 46.2% diazepam, p = 0.81, adjusted p = 0.11) were comparable. Miscarriage rates (11.0% diazepam vs. 9.3% non-diazepam, p = 0.25, adjusted p = 0.26) and ectopic pregnancy rates (0.9% diazepam vs. 0.1% non-diazepam, p = 0.1, adjusted p = 0.20) were similar. Live birth rates (36.3% non-diazepam vs. 35.3% diazepam, p = 0.69, adjusted p = 0.82) and prematurity rates (0.3% non-diazepam vs. 0% diazepam, p > 0.99, adjusted p = 0.99) also exhibited no statistically significant differences. Based on the results, diazepam administration during embryo transfer did not show a discernible impact on reproductive outcomes, including live birth rates, suggesting its limited effectiveness in enhancing success.

Does timing of tocilizumab administration affect mortality in COVID-19? A Scottish multicentre retrospective cohort study

BackgroundThe optimal timing of tocilizumab treatment during the disease course of COVID-19 has yet to be adequately defined in the context of randomised controlled trials and the effect of tocilizumab...

Machine-learning model to predict the tacrolimus concentration and suggest optimal dose in liver transplantation recipients: a multicenter retrospective cohort study

Titrating tacrolimus concentration in liver transplantation recipients remains a challenge in the early post-transplant period. This multicenter retrospective cohort study aimed to develop and validate a machine-learning algorithm to predict tacrolimus concentration. Data from 443 patients undergoing liver transplantation between 2017 and 2020 at an academic hospital in South Korea were collected to train machine-learning models. Long short-term memory (LSTM) and gradient-boosted regression tree (GBRT) models were developed using time-series doses and concentrations of tacrolimus with covariates of age, sex, weight, height, liver enzymes, total bilirubin, international normalized ratio, albumin, serum creatinine, and hematocrit. We conducted performance comparisons with linear regression and populational pharmacokinetic models, followed by external validation using the eICU Collaborative Research Database collected in the United States between 2014 and 2015. In the external validation, the LSTM outperformed the GBRT, linear regression, and populational pharmacokinetic models with median performance error (8.8%, 25.3%, 13.9%, and − 11.4%, respectively; P < 0.001) and median absolute performance error (22.3%, 33.1%, 26.8%, and 23.4%, respectively; P < 0.001). Dosing based on the LSTM model’s suggestions achieved therapeutic concentrations more frequently on the chi-square test (P < 0.001). Patients who received doses outside the suggested range were associated with longer ICU stays by an average of 2.5 days (P = 0.042). In conclusion, machine learning models showed excellent performance in predicting tacrolimus concentration in liver transplantation recipients and can be useful for concentration titration in these patients.

Deep venous outflow as a surrogate for collaterals in late-window patients with successful revascularization from the DEFUSE 3 cohort.

Deep venous outflow (VO) may be an important surrogate marker of collateral blood flow in acute ischemic stroke patients with a large vessel occlusion (AIS-LVO). Researchers have yet to determine the relationship between deep VO status in late-window patients and imaging measures of collaterals, which are key in preserving tissue. We performed a multicenter retrospective cohort study on a subset of DEFUSE 3 patients recruited across 38 centers between May 2016 and May 2017 who underwent successful thrombectomy revascularization. Internal cerebral vein opacification was scored on a scale of 0-2. This metric was added to the cortical vein opacification score to derive the comprehensive VO (CVO) score from 0 to 8. Patients were stratified by favorable (ICV+) and unfavorable (ICV-) ICV scores, and similarly CVO+ and CVO-. Analyses comparing outcomes were primarily conducted by Mann-Whitney U and χ2 tests. Forty-five patients from DEFUSE 3 were scored and dichotomized into CVO+, CVO-, ICV+, and ICV- categories, with comparable demographics. Hypoperfusion intensity ratio, a marker of tissue level collaterals, was significantly worse in the ICV- and CVO- groups (p = 0.005). ICV- alone was also associated with a larger perfusion lesion (138 ml vs 87 ml; p = 0.023). No significant differences were noted in functional and safety outcomes. Impaired deep venous drainage alone may be a marker of poor tissue level collaterals and a greater degree of affected tissue in AIS-LVO patients presenting in the late-window who subsequently undergo successful revascularization.

Role of Hematological Markers in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma Treated With Pembrolizumab.

The predictive role of hematological markers in patients with recurrent or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) treated with pembrolizumab remains unclear. We conducted a multicenter retrospective cohort study to investigate the predictive impact of the pre-treatment hematological markers neutrophil-to-lymphocyte ratio (NLR), platelet-to-lymphocyte ratio (PLR), CRP-albumin-lymphocyte (CALLY) index, and the modified Glasgow prognostic score (mGPS) on overall survival (OS) and progression-free survival (PFS) in patients with R/M SCCHN treated with pembrolizumab. From December 2019 to February 2022, 119 and 28 patients were treated with pembrolizumab alone and pembrolizumab plus chemotherapy, respectively. The optimal cut-off point of dichotomized hematological markers was calculated using the area under the receiver operating characteristic curve. Hazard ratios (HRs) and 95% confidence intervals (95%CIs) were estimated using Cox proportional hazard models with adjustment for potential confounders. In the pembrolizumab monotherapy group, patients with higher NLR, PLR, and mGPS and a lower CALLY index showed significantly shorter OS after adjustment for potential confounders. In addition, all hematological markers examined in this study tended to be associated with clinical response, such as overall response rate or disease control rate (DCR); in particular, a lower CALLY index and higher mGPS were significantly associated with poor DCR. In the pembrolizumab with chemotherapy group, these hematological markers had a similar association with OS but not with clinical response. Pre-treatment NLR, PLR, CALLY index, and mGPS might be predictive markers of survival in patients with R/M SCCHN treated with pembrolizumab.