Real-world Cohort Study Research Articles

Reference values for the 1-minute sit-to-stand test to assess functional capacity and short-term mortality in people with idiopathic pulmonary fibrosis and fibrotic connective tissue related interstitial lung diseases: a prospective real-world cohort study.

Early identification of functional decline in fibrotic interstitial lung disease (F-ILD) is crucial for timely treatment and improved survival. While the 6-minute walk test (6MWT) is the standard for functional evaluation, it has practical limitations. The 1-minute sit-to-stand test (1MSTS) offers a simpler alternative; however, its correlation with the 6MWT in F-ILD patients remains unclear. This study aims to establish reference values for the 1MSTS in assessing functional capacity, evaluate its correlation with the 6MWT, and explore its utility in predicting 18-month mortality in F-ILD patients. This prospective study enrolled participants diagnosed with F-ILD based on multidisciplinary team discussions. Assessments included the 1MSTS, 6MWT, pulmonary function test (PFT), GAP score, mMRC scale, and Charlson Comorbidity Index (CCI). The association between 1MSTS repetitions and other variables was calculated using Spearman's rho. Bland-Altman plots assessed the agreement between 1MSTS repetitions and the 6MWT. Predictors of 18-month mortality were evaluated using ROC curve and Kaplan-Meier curve. Of the 150F-ILD patients, 37 (24.6%) had idiopathic pulmonary fibrosis (IPF), and 113 (75.4%) had connective tissue disease-related ILD (CTD-ILD). Using ≤ 23 repetitions as the cutoff for functional impairment in 1MSTS, 74 (47.3%) patients were classified as impaired. The 1MSTS significantly predicted 18-month mortality and demonstrated moderate correlations with GAP score (rs = -0.49), mMRC scale (rs = -0.47), and 6MWT distance (rs = 0.65). Bland-Altman analysis indicated agreement between 1MSTS repetitions and 6MWT distance. Using ≤ 23 repetitions as the cutoff value for the 1MSTS to predict 18-month mortality, the mortality rate was 76.4%, with an AUC of 0.81. The findings suggest that ≤ 23 repetitions in the 1MSTS can serve as an indicator of functional impairment, demonstrate a good correlation with 6MWT distance, and effectively predict 18-month mortality in patients with F-ILD. Not applicable.

Population profile and glycemic control following initiation or switch of injectable therapies in Tianjin, China: A real-world retrospective cohort study of adults with type 2 diabetes.

To evaluate characteristics and glycemic outcomes in individuals with type 2 diabetes using injectable therapies in real-world clinical practice in Tianjin, China. Data from inpatients and outpatients receiving injectable therapies between January 2015 and December 2019 were collected from the Tianjin regional electronic medical records and retrospectively analyzed. Seven cohorts were identified, including individuals initiating injectable therapies (premixed insulin [n = 4,687], basal insulin [4,177], or glucagon-like peptide-1 receptor agonists [541]) or switching injectable therapies (premixed insulin to basal insulin [1,298], basal insulin to premixed insulin [1,457], basal insulin to basal + bolus insulin [1,772], or glucagon-like peptide-1 receptor agonists to basal insulin ± glucagon-like peptide-1 receptor agonists [82]). In participants initiating therapy, glycated hemoglobin and fasting plasma glucose were highest in the basal insulin cohort, while among participants switching therapy, the highest values were in the basal insulin ± glucagon-like peptide-1 receptor agonists cohort. Initiating therapy with premixed or basal insulin and switching from basal insulin to basal + bolus insulin improved glycemic control over 12 months. A mean delay in initiating therapy of up to 13 months after oral glucose-lowering drug failure was observed, with 60% having a delay of >6 months. This delay was associated with a lower proportion achieving glycemic control 3 months after initiation. Effectiveness was not observed at all time points in all cohorts, suggesting some treatments were not used in the appropriate population. Delays in initiating injectable therapies were observed and were associated with poor glycemic control.

Heatwave warnings mitigate long-term cardiovascular diseases risk from heat-related illness: a real-world prospective cohort study

Heatwave warnings mitigate long-term cardiovascular diseases risk from heat-related illness: a real-world prospective cohort study

First line atezolizumab + bevacizumab (atezo/bev) or durvalumab ± tremelimumab (durva±treme) in unresectable hepatocellular carcinoma (HCC): A real world, multi-institutional retrospective cohort study.

532 Background: HCC accounts for 80% of primary liver cancers, and worldwide, is the third common cause of cancer-related death. Two FDA approved 1st line systemic treatment options include atezo/bev and durva ± treme. Herein we report outcomes of these regimens in the 1st line setting via a real-world, multi-institutional retrospective cohort study. Methods: We reviewed clinical outcomes of patients (pts) who initiated 1st line atezo/bev or durva ± treme for unresectable HCC between March 2017 and February 2024, across 5 institutions (Mayo Clinic, University of Alabama, Cedars-Sinai Medical Center, University Hospitals Seidman Cancer Center and University of Michigan). Time to treatment failure (TTF) and overall survival (OS) were analyzed using the Kaplan-Meier method. Best (objective) response was tabulated. Multivariable analyses were performed using Cox models and logistic regression for time-to-event and binary outcomes, respectively, while adjusting for age, sex, race, HCC etiology, Child-Pugh class, and ALBI grade. Results: A total of 433 HCC pts were included; atezo/bev (336), durva ± treme (97). Median age at the start of therapy was 68 years; 77% were male, 82% were Caucasian. Etiologies of HCC were viral hepatitis (38%), MASLD (20%), alcohol (10%), non-viral multifactorial (10%), or unknown (22%). Pts receiving durva ± treme were less likely to have cirrhosis (11% vs. 28%, p<0.01) but similar in terms of macrovascular invasion (63% vs. 58%, p=0.35) compared to pts receiving atezo/bev. Reasons for treatment discontinuation were similar between the two groups, with the most common being disease progression - atezo/bev (56%) and durva ± treme (47%). The differences in clinical outcomes were not statistically significantly and remained consistent after multivariable adjustment. (Table, atezo/bev as the reference group). Outcomes were significantly different between Child-Pugh classes (CP: A,B,C) respectively, OS: 19.0 m, 6.4 m, 1.9 m (p<0.001); TTF: 6.1 m, 2.7 m, 1.3 m (p<0.001). Conclusions: In this real-world retrospective cohort study of unresectable HCC, the differences in overall survival, objective response, and time to treatment failure were not statistically significant between atezo/bev and durva ± treme in the 1st line setting. Outcome Atezo/bev Durva±treme Univariate p-value Multivariable adjusted hazard ratio (95% CI) Multivariable adjusted odds ratio (95% CI) Overall Survival, Median (month) 14.0 14.6 0.77 0.78 (0.54-1.14) --- Time to Treatment Failure, Median (month) 4.9 4.1 0.71 0.95 (0.72-1.26) --- Objective response, % 26.3% 21.1% 0.31 --- 0.78 (0.43-1.39)

Real-world application of CCTA with CT-FFR for coronary assessment pre-TAVI: the CT2TAVI study.

This study aims to evaluate the implementation of concomitant CAD assessment on pre-TAVI (transcatheter aortic valve implantation) planning CTA (CT angiography) aided by CT-FFR (CT-fractional flow reserve) [The CT2TAVI protocol] and investigates the incremental value of CT-FFR to coronary CT angiography (CCTA) alone in the evaluation of patients undergoing CT2TAVI. This is a prospective observational real-world cohort study at an academic health system on consecutive patients who underwent CTA for TAVI planning from 1/2021 to 6/2022. This represented a transition period in our health system, from not formally reporting CAD on pre-TAVI planning CTA (Group A) to routinely reporting CAD on pre-TAVI CTA (Group B; CT2TAVI protocol). All CTAs were retrospective ECG-gated using a dual source 192 slice CT scanner without nitrate or intravenous beta blocker premedication. We assessed downstream ICA and revascularization pre-TAVI and clinical outcomes 30 days and 1 year post-TAVI in both groups. 307 patients were included with 199 patients in Group A and 108 patients in Group B. In Group B, ICA was performed pre-TAVI in only 40.7% of patients. The use of CT-FFR, which was primarily aimed at identifying hemodynamically significant proximal vessel disease, helped avoid downstream invasive testing for 60.5% (23/38) of patients who were deemed to have obstructive proximal vessel disease using CCTA alone or had one or more uninterpretable proximal segments using CCTA. All-cause mortality, cardiovascular mortality, myocardial infarction and need for revascularization at 1-year post-TAVI were comparable between groups with a higher trend toward heart failure hospitalizations in Group A. Routine ICA can safely be deferred pre-TAVI, with the CT2TAVI strategy using modern CT scanners aided by CT-FFR analysis.

Retrospective Cohort Study of Elderly Users of Single- or Multiple-Inhaler Triple Therapy for the Treatment of Asthma in the USA.

Escalation to single- or multiple-inhaler triple therapy (SITT; MITT) is a recommended option for patients with asthma who remain uncontrolled by medium-dose inhaled corticosteroid/long-acting β2-agonist; however, characterization of elderly users of triple therapy is limited. This real-world cohort study describes demographics and clinical characteristics of elderly patients with asthma with and without comorbid chronic obstructive pulmonary disease (COPD) who are new users of triple therapy, and asthma treatment patterns preceding triple therapy initiation. This retrospective cohort study used administrative claims data from the Optum Clinformatics Data Mart database. Eligible patients were ≥ 65years of age with asthma or with asthma and comorbid COPD who initiated either triple therapy with single-inhaler fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI; 100/62.5/25μg) or MITT between September 18, 2017 and September 30, 2020. Demographics, clinical characteristics, healthcare resource utilization, healthcare costs, and asthma treatment patterns were described in the 12-month period before triple therapy initiation (baseline period). In total, 15,557 patients were included. Among FF/UMEC/VI initiators with asthma (N = 635) mean age was 73.3years and 66.6% were female. During the baseline period, > 75% of patients used controller therapy, > 92% used rescue medications, 27.9% experienced ≥ 1 asthma-related exacerbation, with mean annual exacerbation rate of 0.42, and mean all-cause healthcare costs were $23,407. Patients with asthma initiating MITT and patients with asthma and comorbid COPD initiating FF/UMEC/VI or MITT had similar characteristics, healthcare resource utilization, healthcare costs, and asthma treatment patterns to FF/UMEC/VI initiators with asthma. Triple therapy is often initiated following use of other asthma controller medications in real-world practice. Substantial rescue medication use and high disease and economic burden among this elderly patient population suggest that their asthma was not adequately controlled prior to triple therapy initiation. This retrospective study provides an early profile of elderly patients with asthma in the USA.

Gene mutation in diabetic patients with lung adenocarcinoma: a real-world retrospective cohort study

PurposeThe incidence of lung cancer is closely associated with diabetes; however, it remains unclear whether diabetes influences the genetic mutations present in lung cancer. Therefore, we will compare the genetic mutations in patients with lung adenocarcinoma (ADC) who have diabetes against those who do not.MethodsWe included 279 patients diagnosed with lung adenocarcinoma (143 with diabetes and 136 without diabetes) at the Second Affiliated Hospital of Chongqing Medical University between 2016 and 2023, and analyzed the clinical characteristics and genetic mutation profiles of all participants.ResultsIn comparison to ADC patients without diabetes, those with diabetes exhibited a lower overall gene mutation rate (49.7% vs. 65.4%, P = 0.008). Female ADC patients demonstrated a higher total gene mutation rate and EGFR gene mutation rate than their male counterparts (49.3% vs. 66.9%, P = 0.003; 27.6% vs. 58.3%, P < 0.001, respectively), although their TP53 gene mutation rate was lower (8.6% vs. 2.4%, P = 0.027). ADC patients without a smoking history had a higher gene mutation rate and EGFR gene mutation rate than those with a smoking history (62.6% vs. 47.4%, P = 0.014; 51.6% vs. 22.7%, P < 0.001, respectively), but a lower KRAS gene mutation rate (4.4% vs. 14.4%, P = 0.003). Conversely, ADC patients with a drinking history had a lower EGFR gene mutation rate than those without (48% vs. 62.6%, P = 0.018; 31.0% vs. 47.5%, P = 0.007), yet a higher KRAS gene mutation rate (14.0% vs. 4.5%, P = 0.005). Univariate and multivariate linear regression analyses revealed that being female, having no smoking history, and being in phase II or IV of tumor stage were associated with gene mutation. Subgroup analysis indicated that the rate of gene mutation in male smoking lung adenocarcinoma patients with diabetes was significantly lower than in those without diabetes.ConclusionThis retrospective study of real-world data suggests that patients with lung adenocarcinoma and diabetes may have a reduced likelihood of developing genetic mutations, particularly among male smokers. Furthermore, gender, smoking history, and tumor stage may be correlated with the presence of gene mutations.

P0231 Validation and Reformation of the Red Flags Index: Build a Tool of the Early Diagnosis of Crohn's Disease

Abstract Background Early intervention of Crohn's disease (CD) can effectively improve the prognosis. In order to identify CD in the early stage, the World IBD organization proposed the concept of "Red Flags Index (RFI)" in 2015. Various scores were added according to the clinical symptoms of patients, and RFI≥8 was defined as the threshold to distinguish CD from non-CD. Until now, no clinical research in China have verified the effectiveness of RFI in assisting the CD diagnosis. Referring to previous research, this study was designed to evaluate the effectiveness of RFI in identifying early CD, guiding the application of RFI in the early diagnosis of CD. Methods This study is a real-world observational cohort study. The study subjects were people who came to the Department of Gastroenterology of Wuhan Union Hospital, First Hospital of Yangtze University and Taihe Hospital from August 1, 2023 to March 31, 2024 and had lower gastrointestinal symptoms such as chronic diarrhea. They filled in the RFI questionnaire and then were followed up to record disease diagnosis and laboratory test results. Statistical methods were used to analyze the correlation between RFI degree and CD diagnosis, as well as sensitivity, specificity and other indicators. Results A total of 293 valid questionnaires were collected. Most of the subjects were young and middle-aged aged adults. According to the follow-up series, the majority of subjects were diagnosed with CD (54.61%), and other diagnoses included irritable bowel syndrome (IBS), bowel tuberculosis, etc. Data was analyzed and grouped according to diagnosis and RFI degree. Chi-square test showed that RFI was correlated with the incidence of Crohn's disease, with significant differences (sensitivity 72.50%, specificity 68.42%). ROC curve indicated that the model quality was great, RFI≥8 is a suitable threshold for distinguishing CD from non-CD. The diagnostic efficiency, especially the specificity of RFI≥8 combined with Fecal Calprotectin (FC)>60ng/g was significantly improved compared with RFI and FC alone. Conclusion In this real world study, the overall model of RFI was of great quality, the threshold was appropriate, and RFI≥8 was correlated with CD diagnosis, suggesting that RFI could be used as a preliminary screening tool for early CD identification by clinicians, and RFI combined with FC can effectively improve the specificity of CD detection. However, there are differences in the identification of diseases in various items of RFI, suggesting that the weighted score of the original RFI may not fully conform to the CD characteristics in China and even in Asia. RFI items and scores can be improved according to the disease characteristics of China to build diagnostic tools with higher testing efficiency.

P0798 Forty-eight months outcomes of Teduglutide in adult stable patients with short bowel syndrome and home parental nutrition dependence: a real-world italian single center observational cohort study

Abstract Background This real-life study is designed to investigate the short and long-term efficacy and safety of teduglutide (TED) and its effects on quality of life (QoL) in a cohort of adult, stable patients with short bowel syndrome and chronic intestinal failure (SBS-CIF) receiving a long term parenteral support (PS). Methods A prospective, single-center study was conducted for individuals who began using TED between March 2017 and August 2023. Results Ten patients were included in the analysis, among which the median duration of teduglutide administration was 48 (range, 12- 71) months. Data relative to short-term clinical outcomes demonstrate that 4 of 10 (40%) patients were early responders to teduglutide therapy, defined as a ≥20% reduction in PS requirement at 3 months. Six of 10 (60%) were no-responders. Data relative to 48 months long-term clinical outcomes demonstrated the absence of late responder patients and underline that all of 4 early TED responder patients continued to mantein a sustained reductions in PS. Two of the 4 patients (50%) responding to TED discontinued PS while the other 2 patients (50%) reduced the number of weekly PS infusions by approximately 50%. The physical and mental components of QoL improved significantly (p<0.05) in responsive patients while they did not change in non-responsive ones. Predictibility of response to TED therapy in this study seems to be linked to: i) SBS type 3 that is completely absent in the no-responder patients (p<0.0001), ii the residual small bowel length significantly (p<0.02) higher in the responder (102=18 cm) compared to no-responder patients (67=27 cm), and i11) SBS type 1 and 2, enterostomy, surgical complications are significantly predictive of no response. Crohn's disease is equally present in both responder and non-responder patients. The number of side effects observed in our experience is extremely rare both at the 3th (0.01 n°/day/patients) and at the 48th months (0.001 I°/day/patients). No endoscopic neoplastic lesions were observed up to the 48th month of TED therapy. Conclusion This real-world experience allows us to state that, after 48 months of treatment, TED: a) is a safe therapy with minimal side effects: b) produces a rapid reduction in PS volumes in 40% of treated patients; c) consents to weaned off PS in 20% of enrolled patients; d) maintains a stable nutritional state and, finally, e) significantly improves Qol.

P1031 Effectiveness and safety of oral vancomycin in the treatment of non-primary sclerosing cholangitis paediatric Inflammatory Bowel Disease (non-PSC PIBD) patients: a real-world single-center cohort study

Abstract Background Oral vancomycin has had limited use in paediatric Inflammatory Bowel Disease (PIBD), with reported efficacy in primary sclerosing cholangitis (PSC-PIBD)1, acute severe colitis as part of a quadruple antibiotic regimen2, and very early onset IBD3. This study evaluates its effectiveness and safety as single-agent therapy in non-PSC PIBD patients. Methods This single-centre retrospective cohort study included PIBD patients started on oral vancomycin for active IBD or steroid/topical therapy dependency from 01/2017 to 08/2024. Exclusion criteria were PSC diagnosis, positive/equivocal stool test for C. difficile, another induction treatment started within 3 weeks prior and vancomycin use solely in a quadruple regimen2. Disease activity was assessed using PUCAI/wPCDAI, faecal calprotectin (FC), blood parameters at baseline, 1, 3, 6 and 12 months. Treatment regimen, duration, reasons for discontinuation and adverse events (AEs) were recorded. Vancomycin was started at 250mg in patients ≥30kg or 125mg in those <30kg TDS/QDS and tapered in responders after ≥1 month. Results Of 72 patients identified, 31 were included (16 males): 23 (74%) had Ulcerative Colitis (UC), 4 (13%) IBD unclassified (IBDU), 4 (13%) Crohn’s Disease (CD). At baseline, 5/31 were in clinical remission, with 2 also in biochemical remission but drug dependent. Initial vancomycin dosage was 17.5mg/kg/day (IQR 15.3-21.6), with a median duration of 4 (IQR 1-9) months. Concomitant medications are shown in Table 1: 10/31 (32%) were on biologics, with 4 able to discontinue and 2 to reduce biologic doses later. Overall, 17/31 (55%) patients (13 UC, 3 IBDU, 1 CD) achieved/maintained clinical and biochemical remission (Group 1), with 15 reducing/stopping other treatments. Conversely, 14/31 (45%) discontinued vancomycin due to non-response (11) or intolerance (3) (Group 2), with 11 stopping within 1 month. At baseline, Group 1 had lower disease activity score and platelet count than Group 2 (Table 1). A striking difference in response was noticed at 1 month (Figure 1), with FC in Group 1 dropping from 686μg/g (IQR 376-1143) at baseline to 60μg/g (IQR 25-160) at 1 month (p=0.001), while remaining high in Group 2 (p=0.52). In Group 1, 5/17 (29%) stopped vancomycin while in stable remission after 4 (IQR 2-9) months, with 3 restarted due to relapse, recapturing remission in 2/3. No severe AEs were reported. Conclusion Oral vancomycin was rapidly effective and safe, with 55% of non-PSC PIBD patients achieving/maintaining remission and 48% able to reduce/stop concomitant treatments. It was most effective in mildly active disease, especially UC/IBDU, and may offer a medium to long-term treatment option, with promising results in biologic-resistant but antibiotic-responsive PIBD patients.

P0760 Long-term Efficacy and Persistence of Subcutaneous Infliximab after Switching from Intravenous Infliximab in patients with Crohn's disease: A Real-World Multicenter Prospective Cohort Study in Korea

Abstract Background Subcutaneous infliximab (IFX-SC) has shown comparable efficacy and safety to IV infliximab (IFX-IV) in Crohn's disease (CD), since its launch in 2021. We aimed to evaluate the real-world efficacy and durability of IFX-SC within two years of follow-up in a Korean patient cohort. Methods This multicenter, prospective cohort study, conducted from September 2021 to June 2024, included patients with CD receiving IFX maintenance therapy. Patients received IFX-SC 120 mg at 8 weeks after the last IFX-IV administration (Week [W] 0), and every 2 weeks thereafter. Clinical relapse (CREL) at W26, W50, W74, and W98 and drug survival within W98 were assessed. Among CD patients with W0 clinical remission, CREL was defined as Crohn’s disease activity index ≥150 points. Drug survival was evaluated based on the time of drug persistence from W0 to the last date of IFX-SC administration among drug-off cases. Results A total of 372 patients with CD were finally included. Enrolled patients included young adults with a male predominance (Table 1). Most of the patients had ileocolonic disease, and about half had non-stricturing/non-penetrating disease phenotype. At W0, 283 patients with CD showed clinical remission., and the proportion of CREL showed incremental trend (W26 CREL 6.8%, W50 CREL 12.2%, W74 CREL 17.7%, and W98 CREL at 25.3%, Ptrend 0.004) (Figure 1A). On the other hands, drug durability was also investigated as shown on Figure 1B. Drug survival rate at W50 and W98 were 93.4% (95% confidence interval [CI], 0.908–0.961) and 91.3% (95% CI, 0.882–0.945), respectively. During the study, drug re-switch rate was 8.6% (32/372), mainly due to injection site discomfort, and also less than 5% of serious adverse events were reported. Conclusion In real-world settings, IFX-SC administration presents a viable strategy for maintenance therapy subsequent to IFX-IV treatment. Given the favorable drug durability, low relapse rate, and tolerable safety profile, IFX-SC may facilitate more tailored and individualized therapeutic approaches.

DOP102 Comparative effectiveness of risankizumab vs. ustekinumab in bio-naïve Crohn's disease patients: A real-world multicentre retrospective cohort study

Abstract Background The SEQUENCE trial, in bio-exposed Crohn's disease (CD) patients, demonstrated non-inferiority for clinical remission at 24 weeks and superiority for endoscopic remission at week 48 for risankizumab (RZB) compared to ustekinumab (UST). However, this comparison has not been adequately explored in bio-naive CD patients. Our study aimed to compare the clinical effectiveness among bio-naive CD patients treated with RZB vs. UST in a real-world setting, using data from a US-based multicenter cohort. Methods This retrospective cohort study utilized data from the TriNetX US Collaborative Network, comprising sixty-eight tertiary institutions, to examine adult (≥18 years) bio-naive CD patients initiated on RZB or UST from June 17th, 2022 (FDA approval date) to October 31st, 2024. Patients with prior advanced therapy usage and those with approved rheumatologic disease indications for RZB/UST were excluded. A 1:1 propensity score matching (PSM) analysis was conducted to balance cohorts on demographics, baseline comorbidities, and indicators of CD severity, including CD phenotype, extraintestinal manifestations, concurrent corticosteroid use, hemoglobin, and body weight. Cox proportional hazards models estimated hazard ratios (HRs) in matched cohorts for one-year post-treatment composite outcomes (need for corticosteroids, hospitalizations, emergency room visits, surgeries) with a priori significance threshold of p < 0.05. Results The study sample included 1,630 bio-naive CD patients treated with RZB and 3,105 with UST. Among patients on UST, 658 (21%) were dosed every four to six weeks, after the first dose. Baseline characteristics of the PSM RZB and UST cohorts are shown in Table 1 with 1,568 patients in each cohort. After PSM (Figure 1), there were no significant differences in composite outcome (HR 1.077, p = 0.170), corticosteroid use (HR 1.099, p = 0.110), CD-related surgery (HR 1.231, p = 0.284), hospitalization (HR 0.947, p = 0.615) or ER visits (HR 1.041, p = 0.687). Additionally, no significant differences were observed in opioid use (HR 1.062, p = 0.366), switch to different advanced therapy (HR 1.097, p = 0.468) or elevated disease biomarkers, including CRP (>10mg/l, HR 1.186 p = 0.101) or fecal calprotectin (>150 µg/g; HR 1.109, p = 0.407) at 1-year follow-up. Conclusion In this real-world experience allowing for drug optimization, no difference was observed in bio naïve patients between UST and RZB as far as clinical remission and progression of CD.

P0811 Comparative efficacy of vedolizumab and infliximab in patients with moderate-to-severe ulcerative colitis: a multicenter real world cohort study

Abstract Background Vedolizumab (VDZ) and infliximab (IFX) are both recognized as first-line treatment options for moderate to severe ulcerative colitis (UC). However, there is a paucity of comparative studies examining their real-world efficacy. Methods Patients with moderate to severe UC were consecutively enrolled from 11 centers. Efficacy were evaluated after 14 and 52 weeks. Logistic regression was conducted to identify independent risk factors for clinical remission. Results A total of 235 patients were enrolled, of which 154 received VDZ treatment and 81 received IFX treatment. 154 (65.5%) were male, with a median age of 40 (32, 49) years, a median disease duration of 7.0 (2.8, 11.9) years. The median baseline Mayo score was 9 (7, 11), and the median baseline partial Mayo score was 6 (4, 8). 16 patients (6.8%) had perianal lesions, and 41 patients (17.4%) had extraintestinal manifestations. At week 14, patients treated with IFX had higher clinical response rate (84.6% vs 66.2%, p = 0.003) but lower endoscopic remission rate (14.5% vs 34.3%, p = 0.003) compared to those treated with VDZ. There were no statistical difference in clinical remission rate between the two groups (39.7% vs 44.8%, p = 0.462). At week 52, there were no statistically difference in the modified Mayo score, partial Mayo score, clinical response rate, clinical remission rate, and endoscopic remission rate between IFX treatment group and VDZ treatment group (p>0.05). Multivariate regression analysis demonstrated that baseline disease extent was a predictor of clinical remission at week 14 of VDZ treatment (OR = 3.55, 95% CI 1.39–9.06, p = 0.008). Conclusion IFX group received higher clinical response rate but not endoscopic remission rate at week 14. The efficacy of IFX was comparable to VDZ at week 52. Disease extent was a predictor of clinical remission at 14 weeks with VDZ.

P0876 One-year effectiveness and safety of ustekinumab treatment in patients with ulcerative colitis: An Asan-Crohn’s and Colitis Association in Daegu-Gyeongbuk (CCAiD) multicentre real-world cohort study

Abstract Background The real-life data on ustekinumab (UST) for Asian patients with ulcerative colitis (UC) are limited. We aimed to assess one-year effectiveness and safety of UST for Korean patients with UC. Methods This was a multicentre retrospective study on patients with UC who received UST at 4 academic centers in Korea between January 2021 and October 2023. The primary endpoint was clinical remission defined as partial Mayo score (PMS) ≤2 and no subscore >1 at week (W) 8 of therapy. Secondary endpoints included clinical remission (W16–20 and W52–56), corticosteroid-free clinical remission (W8, W16–20, and W52–56), clinical response (W8, W16–20, and W52–56), endoscopic remission defined as Mayo endoscopic subscore (MES) 0–1 (W16–20 and W52–56), interval shortening and durability of UST therapy till W52–56, and adverse events. Results A total of 60 patients (Male, 63.3%; Median age, 44.5 years; Disease duration, 6.5 years; Previous exposure to advanced therapies, 70.0%; Extensive colitis, 65.0%; Median baseline Mayo score, 8; Concomitant use of immunomodulators, 40.0%) were included. Excluding one patient in clinical remission at baseline, 49.2% (29/59) achieved clinical remission at W8. At W16–20, and W52–56, 59.3% (35/59), and 55.9% (33/59) achieved clinical remission. The clinical response rates at W8, W16–20, and W52–56 were 67.9% (40/59), 69.5% (41/59), and 66.1% (39/59), respectively (Figure 1). Endoscopic remission rates at W16–20 and W52–56 were 55.9% (33/59) and 37.3% (22/59), respectively (Figure 1). Multivariable analysis identified factors associated with clinical remission at W52–56 (body mass index, adjusted odds ratio [aOR]: 1.26, 95% confidence interval [CI] 1.00–1.57, p<0.05; previous exposure to advanced therapies, aOR: 0.20, 95% CI 0.04–0.98, p<0.05; concomitant use of immunomodulators, aOR: 4.69, 95% CI 1.04–21.06, p=0.04; endoscopic remission at W16–20, aOR: 13.47 95% CI 2.87–63.30, p<0.01). Till W52–56, the interval shortening rate for subcutaneous UST treatment from every 12 to 8 weeks was 20% (12/60). Nine patients (15.0%) stopped UST, primarily due to a secondary loss of response (8.3%, 5/60), followed by a primary non-response (6.7%, 4/60). Adverse events occurred in 24 patients (40.0%) and serious adverse event in one patient (1.7%) (Table 1). Conclusion UST was effective with an acceptable safety profile for Korean patients with UC. Previous exposure to advanced therapies was associated with a lower probability of clinical remission at one year after UST therapy. Financial support This work was supported by the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) (NRF-2021R1A2C2095096).

P0958 Curcumin-QingDai combination for active ulcerative colitis: A cost-effectiveness analysis

Abstract Background A combination of two herbal compounds, curcumin and QingDai (CurQD), was recently proposed as an effective treatment for patients with active ulcerative colitis (UC) based on randomized controlled and real-world evidence, but no cost-effectiveness analysis for this nutraceutical is currently available Methods A Markov model was generated to simulate the progression of patients with moderate-to-severe UC, by separate analysis for a cohort of advanced therapy-naïve and advanced therapy-experienced patients. Medications costs were derived from published average wholesale price (AWP) in the US. Efficacy data was modeled by synthesis of efficacy reports of randomized placebo-controlled trials and real-world cohort studies of all FDA-approved drugs for UC, and each compared with CurQD. The model used a time horizon of 54 weeks. The threshold of 50,000$/quality adjusted life-year (QALY) was chosen, and a one-way sensitivity analysis was performed for testing analysis robustness. Results The comparison of CurQD with the FDA approved advanced therapies for UC yielded an estimated incremental cost-effectiveness ratio (ICER) of over 200,000$/QALY for all comparisons. Differences ranged from -0.05 to a slight gain of 0.07 QALY in bio-experienced patients and from -0.1 to 0.04 QALY in bio-naïve patients. The one- way sensitivity analysis proved the robustness of the analysis. Conclusion The present analysis suggests that the markedly reduced cost and demonstrated efficacy of CurQD provides a significant cost-effectiveness benefit over advanced therapies for active UC, including over anti-TNF biosimilars.

ASO Visual Abstract: Prognosis of Patients with Breast Cancer Following Delayed Diagnosis During the COVID-19 Pandemic: A Real-World Cohort Study.

ASO Visual Abstract: Prognosis of Patients with Breast Cancer Following Delayed Diagnosis During the COVID-19 Pandemic: A Real-World Cohort Study.

The Effect of Coronary Artery Disease on the Prognosis of Hypertrophic Cardiomyopathy: A Multi-Center Cohort Study.

There is a shortage of patients with hypertrophic cardiomyopathy (HCM) with concurrent coronary artery disease (CAD), and the influence of CAD on the prognosis of patients with HCM is uncertain. This real-world cohort study was conducted to evaluate the prognosis of patients with patients with CAD. This cohort study of patients with HCM was conducted from May 2003 to September 2021. The total number of patients enrolled was 2167, and the mean follow-up period was 6.4 years (interquartile range 2.8-9.5 years). Sudden cardiac death (SCD), cardiovascular death, and all-cause mortality were assessed as outcomes. Using logistic regression, nine indicators were selected for 1:1 propensity score matching (PSM). Additionally, Kaplan-Meier survival curves and Cox proportional hazards regression analyses were used to assess the impact of CAD on the prognosis of patients with HCM. During an average of 6.4 years of follow-up, of the 2167 patients enrolled, 446 (20.6%) died. The patients were classified into two groups: CAD (n = 480) and non-CAD (n = 1,687). After imputation of missing values using the mean and 1:1 propensity score matching, there was no difference in SCD (log-rank χ2 = 0.4, p = 0.540), cardiovascular death (log-rank χ2 = 0.1, p = 0.995) and all-cause mortality (log-rank χ2 = 0.1, p = 0.776) between the CAD and non-CAD groups. After imputation of missing values using the median and 1:1 propensity score matching, patients with and without CAD were not significantly different in terms of SCD (log-rank χ2 = 0.1, p = 0.948), cardiovascular death (log-rank χ2 = 0.1, p = 0.811), and all-cause mortality (log-rank χ2 = 0.5, p = 0.499). In the Cox analysis, CAD was not a significant independent predictor of SCD, cardiovascular death, or all-cause mortality in patients with HCM. In this study, it was observed that there was no statistically significant disparity in mortality rates between patients diagnosed with HCM who concurrently had CAD and those who did not exhibit CAD. This finding underscores the notion that the presence of CAD did not exert a notable influence on the incidence of SCD, cardiovascular death, or all-cause mortality, thereby emphasizing the complexity and multifaceted nature of mortality risk factors in HCM patients.

Age-specific prognosis of acute and steady minor elevations in cardiac troponin among non-acute myocardial infarction inpatients: a large real-world cohort study

BackgroundThe upper reference limit of normal (ULN) of cardiac troponin (cTn) for older adults can be higher than for young adults, while the same ULN is used for both older...

Six-Month Emergent Readmissions Following Hospitalization for Atrial Fibrillation Amid the Syrian Conflict: A Real-World Observational Cohort Study.

Atrial fibrillation (AF) is the most common arrhythmia worldwide. However, data regarding readmissions following index admission for AF in the developing world are not well described. This study assessed the rate, predictors, and trends of 6-month readmission after index admission for AF in Syria. We included adult patients who had an index admission with AF to Latakia's tertiary center between July 2021 and November 2023. Patients were monitored for readmission for 6 months after index discharge. Data were taken from the patient's medical notes. A total of 649 patients were included in the final analysis, of which 320 (49%) were readmitted to the hospital within 6 months following index admission. Cardiac causes were the most common cause of readmission in 76% of patients, of which 70% were AF. Readmitted patients had a higher median age (64 vs. 58; p = 0.001) and fewer males (49% vs. 36%; p = 0.001). In multivariate analysis, factors that independently increased 6-month readmission risk were age ≥ 60 years (hazard ratio [HR]: 1.7, 95% CI: 1.4-2.2), females (HR: 2.2, 95% CI: 1.6-2.7), and congestive heart failure (CCF) (HR: 2.1, 95% CI: 1.4-2.6). Most cardiac readmissions (76%) happened during the first 60 days following index discharge. Almost half the patients were readmitted within 6 months after an index admission for AF. Females, CCF, and advancing age were independently associated with an increased risk of 6-month readmission.

GIMAP1 interacts with TMX1 to improve lung adenocarcinoma prognosis by influencing tumor immune microenvironment.

Recent studies have indicated that the GIMAP family is downregulated in lung cancer and correlates with poor prognosis, although the underlying mechanisms remain unclear. This study aimed to elucidate the mechanism behind GIMAP1 downregulation in lung cancer. Bioinformatics tools were employed to assess the correlation between the GIMAP family and various cancers. Specifically, GIMAP1 was selected for further investigation, and its role in lung adenocarcinoma was confirmed through RNA sequencing analysis, Gene Set Enrichment Analysis (GSEA) of differentially expressed genes, correlation analysis with immune cell infiltration, and assay of the GIMAP1-TMX1 interaction. Based on bioinformatics analysis and real-world cohort studies, it was found that GIMAP1 was underexpressed in lung cancer tissues but exhibited elevated expression following immunotherapy. Overexpression of GIMAP1 was shown to influence several immune signaling pathways. In patients with high GIMAP1 expression, there was a significant increase in the infiltration of CD8+ T cells, activated memory CD4+ T cells, monocytes, and M1 macrophages; conversely, infiltration by M0 macrophages, resting dendritic cells (DCs), and plasma cells was significantly reduced. In vitro experiments showed that high levels of GIMAP1 increased the percentage of Treg, NK, and NKT cells. Additionally, GIMAP1 directly interacted with TMX1 and modulated the expression of downstream immune-related genes including CMTM5, IL17F, TRAV34, and XCR1. Therefore, GIMAP1 may serve as a promising therapeutic target in lung cancer, influencing both disease initiation and progression.