Prospective Real-world Study Research Articles

Clinical outcomes of a real-world prospective study using Dexcom ONE continuous glucose monitoring in people with diabetes treated with two or more insulin injections per day.

This study assessed real-world glycaemic outcomes associated with the use of Dexcom ONE in adults with suboptimally controlled diabetes. In this single-site prospective study, adults with type 1 (T1D) or type 2 diabetes (T2D) taking two or more insulin injections per day initiated Dexcom ONE CGM use and attended follow-up data collection visits after 3 and 6 months. During the study, participants received usual diabetes care. Primary outcome was a change in HbA1c at 6 months. Additional outcomes included change in participant-reported outcomes and CGM-derived time in glucose range 3.9-10 mmol/L (TIR), time above range >10 mmol/L (TAR), and time below range <3.9 mmol/L (TBR). There were 110 adults enrolled [T1D (n = 34): mean age 36.6 years, 55.9% female; T2D (n = 76): mean age 54.9 years, 38.2% female]. Mean HbA1c significantly decreased from 90 mmol/mol (10.3%) to 79 mmol/mol (9.4%) at 6 months (∆-12 mmol/mol, p < 0.001) in T1D users and from 86 mmol/mol (10.1%) to 67 mmol/mol (8.3%) in T2D users (∆-18 mmol/mol, p < 0.001). Perception of health and diabetes distress improved at 6 months for both groups. T1D users had modest improvement in TBR. T2D users exhibited a clinically meaningful increase in TIR (∆ + 9.0%). Real-world Dexcom ONE use was associated with clinically significant reductions in mean HbA1c after 6 months, along with meaningful improvements in participant-reported outcomes. CGM-derived outcomes also improved, with the possibility of there being greater improvement than could be captured in this study. These findings support expanding access to this real-time CGM system.

Impact of reporting rapid susceptibility results in Gram negative bloodstream infections: a real world prospective study.

Mortality and morbidity of patients with bloodstream infection (BSI) remain high despite advances in diagnostic methods and efforts to speed up reporting. This study investigated the impact of reporting rapid Minimum Inhibitory Concentration (MIC)-results in Gram negative BSIs with the ASTar system (Q-linea, Uppsala, Sweden) on the adaptation of empirically started antimicrobial therapy. We performed a real-world study during which antimicrobial susceptibility testing (AST) results were instantly reported to the treating physician in an established multidisciplinary antimicrobial stewardship setting. Consecutive patients with Gram negative bacteremia were included in the study (monomicrobial Gram stain, life expectancy of at least 48h and flagging positive before 2 PM). Rapid AST (RAST) reporting with ASTar was added on top of the standard workflow. Technical performance of the system was evaluated as well as the impact on antimicrobial treatment and timelines of achieving effective and optimal antimicrobial therapy. A total of 79 analyses were performed in 77 patients, of which 68 episodes were eligible for analysis. A categorical agreement was observed in 97,5% of 1160 MIC results without false susceptible results. All patients on ineffective empirical therapy (12/68) were switched after a median time of approximately one hour (5min - 15h) after communication of the result. Furthermore, 20/55 non-optimal therapies were adapted within a median period of 3h after communication. The implementation of rapid MICs, measured by the ASTar system, in our low antimicrobial resistance setting with elaborate antimicrobial guidelines, was easy and led to early adaptation of empirical treatment in 32/55 instances (12 ineffective and 20 non-optimal therapy). NCT06218277 (date of registration: 18-12-2023).

P0727 Effectiveness and Safety of ustekinumab in the Treatment of Penetrating Crohn’s Disease: A Prospective Real-World Study

Abstract Background Ustekinumab (UST) has been proved to be effective in the treatment of Crohn's disease (CD). However, data on the efficacy of UST in treating CD patients with penetrating complications are limited. The objective of this study was to evaluate the efficacy and safety of UST in the treatment of penetrating CD. Methods In this single-center, prospective, observational real-world study, we enrolled patients with penetrating CD who were undergoing UST treatment. The primary endpoint was the rate of transmural healing at week 26 and 52 evaluated by computerized tomography enterography or magnetic resonance enterography. Secondary endpoints included radiologic response rates and surgery-free survival rates at week 26 and 52, as well as surgery-free survival duration and safety. Statistical analysis was conducted using the Kaplan-Meier method. Results The study included 134 patients, among whom 26.8% had complex intestinal fistula. Transmural healing rates at week 26 and 52 were 11.9% and 17.8%, respectively. Radiologic response rates were 60% and 50.6% at week 26 and 52. Surgery-free survival rates were 90.5% and 87.1% at week 26 and 52, respectively. After a median follow-up period of 55.57 weeks, 17 patients required abdominal surgery. The median surgery-free survival duration was 164.5 weeks (95% CI 145.6-183.5 weeks). During the follow-up period, only one patient developed erythema, with no serious adverse events reported. Conclusion UST demonstrates promising efficacy and safety profiles for treating penetrating CD.

N11 Switching from intravenous to subcutaneous infliximab enhances trough levels and can facilitate de-escalation of concomitant immunomodulation, irrespective of the HLA DQA1*05 status: a prospective real-world study

Abstract Background There is emerging data on the continued efficacy of infliximab when switched from intravenous (IV) to subcutaneous (SC) route in Inflammatory bowel disease (IBD). The impact of cessation of concomitant immunomodulators (IMMs) following switch on trough levels or development of anti-drug antibodies (ADAs) is not evaluated in real world practice. We aimed to study the impact of cessation of IMMs following switch of infliximab from IV to SC route and determine relationship with HLA- DQA1 *05 status. Methods Consecutive patients who participated in a planned switch programme were prospectively included. Clinical, biomarker and pharmacokinetic data were collected pre-switch, and at 8 weekly intervals post switch for 6 months. Trough levels and remission status in patients who discontinued or reduced the dose of IMMs were compared with those who remained or continued same dose on IMMs. All patients had HLA-DQA1*05 status determined at initiation of Infliximab and we evaluated the impact of HLA status on development of anti-Infliximab antibodies post switch. Results One hundred and fifteen patients (M: F=1.4:1, Median age 44 years (13-76), UC/CD (48/67) who completed 6 month follow up were included. All patients were in clinical and biomarker remission prior to the switch. 72 patients (62.6%) were on concomitant IMMs and 46 (40.86%) was HLADQA1*05 positive. The mean infliximab trough level prior to switch was 5.54 µg/mL which increased 8 weeks post switch to 12.52 µg/mL (p=0.006). The enhancement of levels persisted at 16 weeks (Mean 16.28) and at week 24 (17.25). 36 patients (32%) were positive for ADAs before switch; in 8 patients (22%) the ADAs disappeared at 8 weeks and in further 4 (total 33%) by 6 months. In total antibody titre reduced or become negative in 17 patients (48.6%), static in 14 patients (40%) but increased in 5 patients (14%) without significant lowering of trough levels. 18 of the 72 patients (25%) had IMMs discontinued, and dose reduced in further 21 patients. The total de-escalation rate was 54% and none of these patients developed ADAs at 6 months irrespective of the HLADQA1*05 status (HR = 0.76, 95% = 0.64 to 1.73, p 0.12). One patient switched back to IV due to personal preference. At 6 months none of the patients discontinued SC infliximab or were switched within or out of class. Conclusion Switch from IV to SC infliximab is effective in maintaining and enhancing trough levels with positive impact on reducing ADAs. It is feasible to de-escalate concomitant IMMs in a considerable proportion of patients following switch. Economic analysis of switch should be incorporated in the feasibility of IMMs withdrawal.

Safety of dupilumab in Chinese pediatric patients aged 6 months and older: a prospective real-world study

Safety of dupilumab in Chinese pediatric patients aged 6 months and older: a prospective real-world study

Change in fatigue in patients with ulcerative colitis or Crohn's disease initiating biologic therapy.

Fatigue is common among patients with inflammatory bowel diseases (IBDs) and is associated with decreased quality of life (QoL). Describe fatigue evolution and identify factors associated with fatigue outcomes in patients with ulcerative colitis (UC) or Crohn's disease (CD) initiating biologic treatment. Data from adult Belgian patients with UC or CD enrolled in a prospective real-world study were utilized. Fatigue and QoL were assessed using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and the Short Inflammatory Bowel Disease Questionnaire, respectively. Factors associated with fatigue outcomes were assessed using multivariate regression. 465 patients were included: 174 with UC and 291 with CD. Average FACIT-F scores indicated improvements in fatigue after 6 months, before stabilizing. A higher probability of fatigue disappearance was associated with clinical remission and was more likely in patients with UC than CD. Patients achieving clinical remission had lower probability of fatigue. Patients with fatigue improvements experienced greater QoL improvements than patients with fatigue persistence. Real-world findings suggest fatigue partly improves in the first 6 months of biologic treatment. Clinical remission was associated with greater probability of fatigue disappearance and lower likelihood of fatigue persistence. Further research into factors associated with fatigue in patients with IBD is warranted.

Prospective Validation of CAR-HEMATOTOX and a simplified version predict Survival in Patients with Large B-Cell Lymphoma treated with anti-CD19 CAR T-cells: data from CART-SIE study.

Anti-CD19 CAR T-cells have revolutionized outcomes in relapsed/refractory large B-cell lymphomas. Long-term follow-up underscored the role of hematological toxicity in non-relapse mortality, largely driven by infections, leading to the development of the CAR-HEMATOTOX (HT) score for predicting neutropenia. The European scientific community (EHA/EBMT) later reached a consensus, defining a new entity: immune effector cell-associated hematotoxicity (ICAHT). To validate the ability of the HT score to predict ICAHT and survival. The CART-SIE is an ongoing multicenter prospective observational study collecting data on patients affected by B-cell lymphoma treated with commercial anti-CD19 CAR T-cells (ClinicalTrials.gov ID: NCT06339255). Since 2019 to 2024, 1002 consecutive patients were enrolled. Out of 746 patients infused, the HT score at infusion was evaluable in 389. Median age was 59 years (48-66). Patients with high HT score had greater disease burden and a greater need for bridge therapy. Patients with a HTHIGH score had a 4-fold higher risk of experiencing late ICAHT of grade≥3 (OR=3.99, 95%CI=1.16-13.77, p=0.03). Patients with a HTHIGH score also showed lower overall response rates (ORR) and complete response rates (CRR) at 90 days (CRR at 90 days: 59% HTLOW vs 38% HTHIGH, OR=0.42, 95%CI=0.27-0.66, p=0.0002; ORR at 90 days: 67% HTLOW vs 49% HTHIGH, OR=0.47, 95%CI=0.29-0.74, p=0.001). Adjusted logistic models confirmed that the effect of HT score was independent from baseline characteristics. With a median follow-up of 18 months, patients with a HTHIGH score have lower OS and PFS (1-year OS: 78% HTLOW versus 62% HTHIGH, p=0.0002; 1-year PFS: 49% versus 39%, p=0.003). Adjusted Cox models confirmed that HT was an independent prognostic factor for OS. A high HT-score was found to be associated with higher risk of secondary primary malignancy (HR=2.8, 95%CI=1.03-7.8, p=0.04). A simplified version of HT (simpleHT), based solely on the platelet count and C-reactive protein at infusion, was calculated for 560 patients and proved significant in predicting both OS and PFS (1-year was 72% simpleHTLOW vs 37% simpleHTHIGH, p<0.0001, 1-year PFS was 48% simpleHTLOW vs 22% simpleHTHIGH, p<0.0001). In our prospective real-world study, we validated the ability of the HT score to predict ICAHT and survival. SimpleHT identified a population at very high risk with an impaired progression free and overall survival.

PlGF and sFlt-1, reduced fetal movements and adverse delivery outcome of a likely placental cause: A real world prospective observational study

PlGF and sFlt-1, reduced fetal movements and adverse delivery outcome of a likely placental cause: A real world prospective observational study

Safety and effectiveness of an herbal decoction (modified Saengmaeksan) in hypertensive patients: Protocol for a real-world prospective observational study.

Hypertension, a common chronic disease, often leads to serious complications. While conventional management relies on antihypertensive drugs, which can cause side effects and adherence issues, alternative treatments like herbal medicine are gaining attention. This study examines the efficacy and safety of modified Saengmaeksan, an East Asian herbal remedy, in treating hypertension. This single-arm, prospective, observational study will be conducted at Kyunghee Bichedam Korean Medicine Clinic from October 23, 2023 to August 30, 2024, enrolling 30 hypertensive patients. Over 12 weeks, participants will undergo 4 visits, receiving modified Saengmaeksan twice daily for 8 weeks, with a subsequent 4-week follow-up. Primary outcome is the change in systolic blood pressure from the baseline to week 8. Secondary outcomes include diastolic blood pressure changes, radial artery tonometry, and quality of life evaluations. Safety assessments will include monitoring hematologic parameters and adverse events. Data will be analyzed using an ANCOVA model for adjusting confounders. Modified Saengmaeksan has shown potential for lowering blood pressure in clinical settings, supported by animal and cell studies. However, human studies are scarce. This research will employ radial artery tonometry to analyze blood pressure comprehensively, exploring Saengmaeksan's hemodynamic effects. The study's goal is to support the approval of modified Saengmaeksan as a hypertension treatment by the South Korean Food and Drug Administration and to promote the industrialization of traditional herbal medicine in managing hypertension. The findings will provide essential data for future clinical research, aiding in feasibility assessments and sample size determinations for randomized controlled trials.

Application of efgartigimod in Chinese patients with myasthenia gravis: a single-center real-world prospective study.

China has a large number of myasthenia gravis (MG) patients, creating an urgent need for rapid and tolerable treatment options. As the first-approved Fc receptor antagonist, efgartigimod has bright prospects for treating MG. However, real-world evidence on its application within the Chinese MG population are limited. This study aims to evaluate the rapid efficacy and safety of efgartigimod in Chinese MG population. This single-center prospective study enrolled Chinese MG patients aged 18 and older who were treated with efgartigimod, classified as Myasthenia Gravis Foundation of America I-IV, with a baseline Myasthenia Gravis Activities of Daily Living (MG-ADL) score of at least 4. Patients received efgartigimod at a dose of 10 mg/kg infused once weekly for 4 weeks. During the treatment, the corticosteroids dosage could be adjusted as appropriate or the non-steroidal immunosuppressive therapies (NSISTs) added. Prior to each infusion, patients' MG-ADL scores, IgG levels, and routine laboratory tests were evaluated, while also recording the prednisone tapering and any adverse events occurring during the treatment. Twenty five Chinese MG patients were enrolled between November 2023 and June 2024, including 3 with ocular MG (OMG) and 22 with generalized MG (GMG). During the 8-week follow-up, in GMG patients, whether positive for acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK) antibodies, the overall efficacy was significant. Within one treatment cycle, 18 (82%) patients showed a reduction of at least 2 points in MG-ADL scores and sustained for at least 4 weeks, and 6 (27%) attained minimal symptom expression (MSE) and sustained for at least 4 weeks. Only 1 patient experienced exacerbation. Among OMG patients, 1 achieved MSE within the treatment cycle, while 2 showed minor improvements. Patients who added tacrolimus concurrently with efgartigimod did not achieve better improvement in MG-ADL scores compared to others. The average reduction in prednisone dosage was 27.4%. Only one patient experienced transient vomiting and diarrhea, with no serious adverse reactions reported. This study confirmed the short-term efficacy and safety of efgartigimod in Chinese MG patients. However, in clinical practice, careful consideration is needed regarding its application in OMG and whether to add NSISTs regimen during the treatment. Efgartigimod could potentially serve as an alternative to long-term corticosteroids therapy.

Predictive model for HBsAg clearance rate in chronic hepatitis B patients treated with pegylated interferon α-2b for 48 weeks.

Chronic hepatitis B (CHB) is a major global health concern. This study aims to investigate the factors influencing hepatitis B surface antigen (HBsAg) clearance in CHB patients treated with pegylated interferon α-2b (Peg-IFNα-2b) for 48weeks and to establish a predictive model. This analysis is based on the "OASIS" project, a prospective real-world multicenter study in China. We included CHB patients who completed 48weeks of Peg-IFNα-2b treatment. Patients were randomly assigned to a training set and a validation set in a ratio of approximately 4:1 by spss 26.0, and were divided into clearance and non-clearance groups based on HBsAg status at 48weeks. Clinical data were analyzed using SPSS 26.0, employing chi-square tests for categorical data and Mann-Whitney U tests for continuous variables. Significant factors (p < 0.05) were incorporated into a binary logistic regression model to identify independent predictors of HBsAg clearance. The predictive model's performance was evaluated using ROC curve analysis. We included 868 subjects, divided into the clearance group (187 cases) and the non-clearance group (681 cases). They were randomly assigned to a training set (702 cases) and a validation set (166 cases). Key predictors included female gender (OR = 1.879), lower baseline HBsAg levels (OR = 0.371), and cirrhosis (OR = 0.438). The final predictive model was: Logit(P) = 0.92 + Gender (Female) * 0.66-HBsAg (log) * 0.96-Cirrhosis * 0.88. ROC analysis showed an AUC of 0.80 for the training set and 0.82 for the validation set, indicating good predictive performance. Gender, baseline HBsAg levels, and cirrhosis are significant predictors of HBsAg clearance in CHB patients after 48weeks of Peg-IFNα-2b therapy. The developed predictive model demonstrates high accuracy and potential clinical utility.

Low vs. conventional intra-abdominal pressure in laparoscopic colorectal surgery: a prospective cohort study.

Low intraabdominal pressure (IAP) during laparoscopy is associated with improved post-operative outcomes across a variety of surgical specialties. A prospective cohort study was undertaken to assess post-operative outcomes in patients undergoing laparoscopic colorectal surgery (LCRS) with low (8mmHg) versus conventional (15mmHg) IAP. A prospective real-world observational study of patients undergoing LCRS in a single-centre, between June 2020 and June 2023 was performed. Operative procedures for diverse colonic pathology such as diverticular disease, inflammatory bowel disease (IBD), and colorectal cancers (CRC) were included. The evaluated primary outcomes were post-operative pain, return of gastrointestinal motility, and length of hospital stay. Secondary outcomes were the overall safety profile including intra- and post-operative complications and morbidity. Outcomes of interest were investigated using multivariate analysis. A total of 120 patients were included of which 69 (57.5%) were male. Median age and BMI of the cohort was 67 years (51-75 years) and 27kg/m2 (24-32kg/m2), respectively. 61 (50.8%) patients were categorised as an ASA grade 3. Two (1.7%) patients had diverticular disease; 31 (25.9%) had IBD, and 87 (72.4%) were operated on for colonic malignancy. Low IAP (8mmHg) was used in 53 (44.2%) cases, whilst the remainder (55.8%) had IAP set at 15mmHg (conventional). Low-pressure surgery was associated with improved intraoperative lung compliance (p < 0.001) and peak inspiratory pressures up to 6h (p < 0.001); reduced analgesic requirement (p ≤ 0.028), and decreased postoperative pain both at rest (p = 0.001) and on exertion (p < 0.001). Moreover, low IAP was associated with an earlier time to pass flatus postoperatively (p = 0.047) with no significant difference in length of hospital stay (p = 0.574). Additionally, no significant difference was observed between the groups for outcomes including median operating time (p = 0.089), conversion to open surgery (p = 0.056), overall complication rate (p = 0.102), and 90-day mortality (p = 0.381). Low IAP use during LCRS is feasible with a comparable safety profile to conventional laparoscopy. Intra-operative respiratory physiology is improved with reduced postoperative pain and analgesic requirement, and earlier time to pass flatus. Future rationally designed; well-powered, randomised trials are needed to understand the benefits of low intra-peritoneal pressure during laparoscopic colorectal resections.

Case management strategy enhances virological response in people living with human immunodeficiency virus in a resource-limited region in Southwest China: a real-world prospective observational study

This study assessed the effectiveness of case management compared to that of standard care in improving treatment outcomes for first-time antiretroviral therapy (ART) patients in Liangshan Prefecture, China. First-time ART patients (total n = 828) were divided into the Standard Care Group (SCG, n = 419) and Case Management Group (CMG, n = 409). At week 48, the CMG showed higher retention rates (97.7% vs. 93.6%), better adherence, and more complete virological responses (84.2% vs. 64.0%) compared to the SCG. These findings suggest that case management improves adherence and virological suppression in resource-limited areas, though further research is needed to confirm its broader applicability.

Real-life study on the effectiveness and safety of sofosbuvir/velpatasvir-based antiviral agents for HCV eradication in Chinese patients

BackgroundHepatitis C virus (HCV) eradication with Sofosbuvir/velpatasvir (SOF/VEL) represents a significant advancement, offering hope for eliminating the virus in diverse patient populations. But real-world data on its effectiveness and safety remains scarce for patients with chronic hepatitis C (CHC) in China, especially those with HCV GT3b, cirrhosis, HCC, or HCV/HBV, HCV/HIV, or HCV/HBV/HIV coinfection. MethodsIn this real-world prospective observational study, we recruited patients from West China Hospital and Public Health Clinical Center of Chengdu in China. Patients included adults chronically infected with any HCV genotype, either with or without cirrhosis, HCC, HCV/HBV, HCV/HIV, or HCV/HBV/HIV coinfection. Patients were administered with SOF/VEL (400/100 mg) ± ribavirin (RBV) once daily for 12 weeks. The primary effectiveness endpoint was sustained virological response at post-treatment Week 12 (SVR12). Adverse events (AEs) were evaluated during treatment. ResultsThe study included 483 patients, with HCV genotypes 1, 2, 3, 6 and uncertain. Among them, 35.4% (171/483, ITT) and 36.7% (166/452, mITT) received SOF/VEL + RBV. At EOT, 99.2% (ITT, 479/483) and 99.1% (mITT, 448/452) of patients had undetectable HCV RNA. The SVR12 rates were 92.8% (ITT, 448/483) and 99.1% (mITT, 448/452). In mITT analysis, the SVR12 for patients infected with HCV GT3b, patients with cirrhosis or HCC, and patients coinfected with HBV/HIV was 99.2% (130/131), 99.4% (168/169), and 97.6% (40/41), respectively. ALBI (-3.01 vs. -3.18 P<0.001), FIB4 (2.53 vs. 1.88, P=0.004) and APRI (0.99 vs. 0.44, P<0.001) showed a significant decrease from baseline to SVR12. None of the involved patients experienced grade 3-5 AEs. ConclusionsAlthough included a high proportion of patients with HCV GT3b, cirrhosis, HCC, or HCV/HBV, HCV/HIV, or HCV/HBV/HIV coinfection, SOF/VEL ± RBV was highly effective and well tolerated in patients with CHC in China.

Prospective Evaluation of Artificial Intelligence Triage of Intracranial Hemorrhage on Noncontrast Head CT Examinations.

BACKGROUND. Retrospective studies evaluating artificial intelligence (AI) algorithms for intracranial hemorrhage (ICH) detection on noncontrast CT (NCCT) have shown promising results but lack prospective validation. OBJECTIVE. The purpose of this article was to evaluate the impact on radiologists' real-world aggregate performance for ICH detection and report turnaround times for ICH-positive examinations of a radiology department's implementation of an AI triage and notification system for ICH detection on head NCCT examinations. METHODS. This prospective single-center study included adult patients who underwent head NCCT examinations from May 12, 2021, to June 30, 2021 (phase 1), or from September 30, 2021, to December 4, 2021 (phase 2). Before phase 1, the radiology department implemented a commercial AI triage system for ICH detection that processed head NCCT examinations and notified radiologists of positive results through a widget with a floating pop-up display. Examinations were interpreted by neuroradiologists or emergency radiologists, who evaluated examinations without and with AI assistance in phases 1 and 2, respectively. A panel of radiologists conducted a review process for all examinations with discordance between the radiology report and AI and a subset of remaining examinations to establish the reference standard. Diagnostic performance and report turnaround times were compared using the Pearson chi-square test and Wilcoxon rank sum test, respectively. Bonferroni correction was used to account for five diagnostic performance metrics (adjusted significance threshold, .01 [α = .05/5]). RESULTS. A total of 9954 examinations from 7371 patients (mean age, 54.8 ± 19.8 [SD] years; 3773 women, 3598 men) were included. In phases 1 and 2, 19.8% (735/3716) and 21.9% (1368/6238) of examinations, respectively, were positive for ICH (p = .01). Radiologists without versus with AI showed no significant difference in accuracy (99.5% vs 99.2%), sensitivity (98.6% vs 98.9%), PPV (99.0% vs 97.5%), or NPV (99.7% vs 99.7%) (all p > .01); specificity was higher for radiologists without than with AI (99.8% vs 99.3%, respectively, p = .004). Mean report turnaround time for ICH-positive examinations was 147.1 minutes without AI versus 149.9 minutes with AI (p = .11). CONCLUSION. An AI triage system for ICH detection did not improve radiologists' diagnostic performance or report turnaround times. CLINICAL IMPACT. This large prospective real-world study does not support use of AI assistance for ICH detection.

Association between the systemic immune-inflammation index and the outcome of liver fibrosis in patients with chronic hepatitis C.

Risk factors that influence the outcome of patients with chronic hepatitis C (CHC) are not fully understood. The systemic immune-inflammatory index (SII) is an independent prognostic factor for multiple diseases. However, the impact of the SII on the outcome of liver fibrosis is unclear. This prospective real-world study enrolled patients with CHC treated with sofosbuvir/velpatasvir. Logistic regression models were used to investigate the relationship between the SII and the outcome of liver fibrosis in treatment-naive patients. Liver fibrosis was assessed using aspartate aminotransferase-to-platelet ratio index (APRI) and fibrosis-4 index (FIB-4). Of the 288 participants, the SII was 238.2 (153.0-358.2). The non-improved outcomes of liver fibrosis assessed with APRI (non-improved APRI) and FIB-4 (non-improved FIB-4) were 83.0 and 87.5%, respectively. Adjusted models showed that the SII was positively associated with non-improved APRI (adjusted OR (95% CI): 1.013 (1.009-1.017), p < 0.001) and FIB-4 (adjusted OR (95% CI): 1.004 (1.001-1.007), p = 0.012). Similarly, a higher SII was associated with a higher risk of non-improved APRI (adjusted OR (95% CI): 13.53 (5.60-32.68), p < 0.001) and FIB-4 (adjusted OR (95% CI): 5.69 (2.17-14.90), p < 0.001). The association with non-improved APRI was much more remarkable in patients with alanine aminotransferase <2 ULN, and the association with non-improved FIB-4 was remarkable in patients aged <50 years. Multiple imputation analyses confirmed the robustness of these results. Our findings suggested that the SII was positively associated with non-improved outcomes of liver fibrosis in patients with CHC. These results need to be validated in large-scale prospective cohorts.

Treatment appropriateness of direct oral anticoagulants in patients with atrial fibrillation for stroke prevention: A real-world prospective study.

Inappropriate use of direct oral anticoagulants (DOACs) is common, affecting up to 30% of atrial fibrillation (AF) population receiving treatment for stroke prevention. This study assessed appropriateness of anticoagulation in anticoagulation-naive AF patients treated with DOACs during a 12-month prospective follow-up. This prospective cohort study included all anticoagulation-naive AF patients referred for anticoagulation for stroke prevention at a tertiary cardiovascular center. Participants were prospectively followed through phone call interviews by a dedicated nurse at 1, 3, 6, 9, and 12 months after enrollment. Of 403 anticoagulation-naive AF patients, rivaroxaban was prescribed for 325 patients (80.7%) and apixaban for 78 (19.3%). Inappropriate therapy was observed in 23% (76/325) and 46% (36/78) of patients treated with rivaroxaban and apixaban, respectively. Undertreatment was predominant scenario for both drugs, detected in 78.5% (78/112) of patients treated inappropriately, and was more frequently observed with apixaban versus rivaroxaban (44.8% vs 16.3%). During 12 months, inappropriate treatment was corrected in only 13% of all patients. The multivariate regression model identified creatinine clearance ≤ 49 mL/min (odds ratio [OR], 2.17; 95% confidence interval [CI], 1.12 to 4.21), female sex (OR, 1.81; 95% CI, 1.11 to 2.97), and age ≥ 80 years (OR, 1.85; 95% CI, 1.22 to 2.83) as independent covariates associated with inappropriate dosing. Inappropriate therapy with DOACs for stroke prevention in patients with AF is common, and the dosage were corrected in few patients during the 12-month follow-up. Our findings highlight the persistent lack of knowledge regarding appropriate DOAC dosage and need for continuous education.

Centralized Investigator Review of Radiological and Functional Imaging Reports in Real-World Oncology Studies: The SACHA-France Experience.

SACHA-France (NCT04477681) is a prospective real-world study that collects clinical safety and efficacy data of novel anticancer therapies prescribed off-label or on compassionate use to patients <25 years. From March 2020 until February 2024, 640 patients with solid tumors or lymphomas were included, with 176 (28%) reported objective tumor responses. Centralized medical monitoring of local radiological/functional imaging reports by the SACHA coordinating investigator led to response modification in 45 out of 176 cases (26%), highlighting the relevance of the medical review of study data. We suggest this pragmatic approach for improving clinical trial data when centralized radiological review is not performed.

Evaluating the Impact of Cyclofert Male™ Supplement on Sperm Parameters and Male Fertility: A Prospective Real-World Study in Lebanese Men

This prospective observational study assesses the impact of Cyclofert Male™, a supplement containing antioxidants and micronutrients, on sperm parameters among forty-nine Lebanese men with sub-fertility. Over six months, participants showed significant improvements in sperm density, progressive motility, morphology although sperm volume remained unchanged. Pregnancy rates were 18% at three month and 29% at month six. This observational real world data study highlights the benefits of antioxidants like L-carnitine, CoQ10, zinc and N-acetyl-cysteine in improving male fertility, with Cyclofert Male™ showing promising results. Limitations include the small sample size, localized setting, and lack of stratification by socioeconomic factors. Larger studies are recommended for further validation.

Impact of Siponimod on Clinical and Radiological Parameters of Secondary Progressive Multiple Sclerosis: A Real-World Prospective Study.

Secondary progressive multiple sclerosis (SPMS) presents with a challenging clinical phenotype, and siponimod has a potential to treat the active clinical phenotype of this disease. This single-center longitudinal study aimed to determine the therapeutic effects of siponimod in patients with active SPMS over 12 months. The clinical and radiological parameters of 50 patients with active SPMS treated using siponimod were assessed at baseline and after a 1-year follow-up period using the annual relapse rate (ARR), the Expanded Disability Status Scale (EDSS), the occurrence of gadolinium-enhanced lesion (GdE+), the Modified Fatigue Impact Scale (MFIS), and the Symbol Digit Modalities Test. The urine bladder postvoid residual (PVR) volume was also measured in a subcohort of 39 participants. Participants with an EDSS score ≥5.0 at baseline were finally assessed separately in prespecified subgroup analyses. There were significant reductions in ARR (p<0.001), GdE+ (p<0.001), and MFIS score (p=0.001) during the follow-up period. The progression of physical and cognitive disabilities remained stable (p>0.05). The PVR-volume analysis revealed a significant decrease in urine bladder PVR volume (p<0.001). These observations were consistent for the subgroup with EDSS score ≥5.0. Siponimod demonstrated efficacy in reducing ARR, GdE+, fatigue levels, and PVR volume, while maintaining stability in the cognitive and physical disability statuses of patients with SPMS. Similar findings were documented in the subgroup with EDSS score ≥5.0.