Prospective Real-world Study Research Articles

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.

Long-term efficacy and safety of dupilumab for moderate-to-severe atopic dermatitis: a prospective real-world cohort study in China

Long-term efficacy and safety of dupilumab for moderate-to-severe atopic dermatitis: a prospective real-world cohort study in China

Treatment strategy for heart failure patients with guideline-directed medical therapy intolerance: a real-world prospective study

Abstract Background Guideline-directed medical therapy (GDMT) reduces the incidence of worsening events in patients with heart failure (HF) with reduced ejection fraction (HFrEF). However, in actual clinical practice, a proportion of patients are unable to tolerate complete GDMT. Purpose This study aimed to assess the medication strategy for patients with HFrEF who are intolerant to GDMT. Methods A total of 110 patients admitted with acute HF were included in the study. The patients were non-randomly assigned to receive complete or incomplete GDMT after assessment by doctors before discharge from the hospital. All patients were followed up for 180 days. The primary composite endpoint was worsening HF-associated events, including death or rehospitalization due to HF, and the need for intravenous diuretic therapy. The secondary endpoints were the New York Heart Association functional class, blood pressure, and cardiac structure and function assessed by echocardiography. Results The main reasons for GDMT intolerance were renal insufficiency (41.8%) and hypotension (26.4%). GDMT-intolerant patients had lower eGFR (50.4 ml/min/1.73 m² vs. 70.9 ml/min/1.73 m²; p=0.002) compared with those who tolerated GDMT, and included more patients with chronic kidney disease (56.4% vs. 23.6%, p&amp;lt;0.001) and higher NT-proBNP levels (5340 pg/ml vs 8080 pg/ml, p=0.039) at baseline. While patient mortality was similar between the two groups, the rate of worsening HF events was lower in patients with GDMT (hazard ratio: 0.736; 95% confidence interval: 0.389−1.393) and vericiguat treatment (p&amp;lt;0.001). In terms of the secondary endpoint, patients who received GDMT showed greater improvements in cardiac structure and function although there was no statistical difference between the groups. In the prespecified subgroup analysis of the effects of vericiguat on the primary endpoint, the incidence of worsening HF events was reduced by vericiguat, regardless of whether the patient was comorbid with GDMT or not, or whether they had a previous history of HF (p-interaction= 0.519 and 0.227, respectively). Conclusions Patients with early in-hospital initiation of complete GDMT and continuous GDMT after discharge had better outcomes compared with patients who were intolerant to GDMT. Co-administration of vericiguat with or without GDMT may reduce the incidence of worsening HF events in HF patients within 180 days.Results of the primary endpoint

S-1-based concurrent chemoradiotherapy plus nimotuzumab in patients with locally advanced esophageal squamous cell carcinoma who failed neoadjuvant therapy: a real-world prospective study

ABSTRACT Purpose This prospective study in a real-world setting investigated the feasibility and safety of S-1 plus nimotuzumab (S-1-Nimo) based concurrent chemoradiotherapy (CCRT) in locally advanced esophageal squamous cell carcinoma (LA-ESCC) patients who failed to neoadjuvant chemotherapy or chemoimmunotherapy. Methods LA-ESCC patients who failed to converse to resectable disease after neoadjuvant chemotherapy or chemoimmunotherapy were enrolled to receive the 4-week S-1-Nimo regimen of radiotherapy (40 Gy in 20 fractions, 5 days per week), S-1 chemotherapy, and nimotuzumab. Then, after surgical assessments, patients evaluated as resectable disease received surgery; patients with unresectable disease continued to receive definitive radiotherapy (50–60 Gy in 25–30 fractions, 5 days per week) concurrently with S-1-Nimo. The primary endpoint was event-free survival (EFS). Results Sixty-four patients were enrolled and evaluated. The median follow-up time was 23.2 months. Median EFS was 9.6 (95% confidence interval [CI], 7.1–14.0) months, with an estimated 2-year EFS rate of 24.2%. The median overall survival (OS) and the estimated OS rate at 2 years were 13.4 (95% CI, 10.3–17.5) months and 31.2%, respectively. Twelve underwent surgery, with a surgical conversion rate of 18.8% and an R0 resection rate of 100.0%. Subgroup analysis identified the significantly prolonged EFS and OS in patients who experienced radical surgery (median EFS, not reached vs. 8.7 months; p = .0117. median OS, 24.9 vs. 10.6 months; p = .0205) as compared to those treated with CCRT. Of 64 patients, grade 3 adverse events mainly included radiation esophagitis (4.7%), anemia (1.6%), and thrombocytopenia (1.6%). Conclusion The study demonstrated the reasonable efficacy and promising safety of the S-1-Nimo-based CCRT in LA-ESCC patients with failure to neoadjuvant chemotherapy or chemoimmunotherapy.

Comparing ultrasound-derived fat fraction and MRI-PDFF for quantifying hepatic steatosis: a real-world prospective study.

To compare the agreement between ultrasound-derived fat fraction (UDFF) with magnetic resonance proton density fat fraction (MRI-PDFF) for quantification of hepatic steatosis and verify its reliability and diagnostic performance by comparing with MRI-PDFF as the reference standard. This prospective study included a primary analysis of 191 patients who underwent MRI-PDFF and UDFF from February 2023 to February 2024. MRI-PDFF were derived from three liver segment measurements with calculation of an overall median PDFF. UDFF was performed by two different sonographers for each of the six measurements, and the median was taken. Intraclass correlation coefficient (ICC) and Bland-Altman analysis were used to assess agreement. Receiver operating characteristics (ROC) curves were used to evaluate the diagnostic performance of UDFF in detecting different degrees of hepatic steatosis. A total of 176 participants were enrolled in the final cohort of this study (median age, 36.0 years; 82 men, 94 women). The median MRI-PDFF value was 11.3% (interquartile range (IQR) 7.5-18.9); 84.7% patients had a median MRI-PDFF value ≥ 6.4%. The median UDFF measured by different sonographers were 9.5% (IQR: 5.0-18.0) and 9.0% (IQR: 5.0-18.0), respectively. The interobserver agreement of UDFF measurement was excellent agreement (ICC = 0.951 [95% CI: 0.934-0.964], p < 0.001). UDFF was positively strongly correlated with MRI-PDFF with ICC of 0.899 (95% CI: 0.852-0.930). The Bland-Altman analysis showed high agreement between UDFF and MRI-PDFF measurements, with a mean bias of 1.7% (95% LOA, -8.7 to 12.2%). The optimal UDFF cutoff values were 5.5%, 15.5% and 17.5% for detecting MRI-PDFF at historic thresholds of 6.4%, 17.4%, and 22.1%, with AUC of 0.851, 0.952, and 0.948, respectively. The sensitivity was 79.2%, 87.5%, 88.9%, and specificity was 81.5%, 90.6%, 90.0%, respectively. UDFF is a reliable and accurate method for quantification and classification of hepatic steatosis, with strong agreement to MRI-PDFF. The UDFF cutoff values of 5.5%, 15.5%, and 17.5% provide high sensitivity and specificity for the detection of mild, moderate, and severe hepatic steatosis, respectively. Question Is ultrasound-derived fat fraction (UDFF) reliable for the quantitative detection of hepatic steatosis compared to MRI proton density fat fraction (MRI-PDFF)? Findings UDFF cutoff values of 5.5%, 15.5%, and 17.5% provided high sensitivity and specificity for the detection of mild, moderate, and severe hepatic steatosis, respectively. Clinical relevance UDFF is a reliable and accurate method for quantification and classification of hepatic steatosis, with strong agreement to MRI-PDFF and high reproducibility of liver fat content by different sonographers.

Efficacy of immune checkpoint inhibitors according to programmed cell death-ligand 1 expression in patients with non-small cell lung cancer and brain metastasis: A real-world prospective observational study.

Studies have shown the antitumor efficacy of immune checkpoint inhibitors (ICI) in patients with non-small cell lung cancer (NSCLC) and brain metastases (BM). However, it is unclear whether the efficacy of ICI is similar between patients with and without BM. It is yet unclear whether the efficacy of ICI in patients with BM increases with higher levels of programmed cell death-ligand 1 (PD-L1) expression, as observed in patients without BM. We compared the outcomes of ICI treatment between patients with and without BM using a cohort containing 1741 prospectively enrolled patients with lung cancer. We investigated whether there were differences in the outcomes of ICI based on PD-L1 expression levels between these patients. We enrolled 240 patients with NSCLC with or without BM who were treated with ICI or both chemotherapy and ICI. There were no significant differences in overall survival (OS) between all patients with or without BM (p = 0.489). However, OS was significantly shorter in patients with BM than in those without in the PD-L1 ≥ 50% group (16.5 M vs. 30.6 M, p = 0.003) but not in the PD-L1 ≥ 1% or negative group. BM was an independent poor prognostic factor for OS (hazard ratio: [95% confidence interval], 2.045; [1.058-3.953], p = 0.033) in the PD-L1 ≥ 50% group. Our study indicated that the outcomes of patients with or without BM treated with ICI were not significantly different. The efficacy of ICI in patients with PD-L1 expression ≥50% would be lower in patients with BM than in those without.

A Texting- and Internet-Based Self-Reporting System for Enhanced Vaccine Safety Surveillance With Insights From a Large Integrated Health Care System in the United States: Prospective Cohort Study.

SMS text messaging- and internet-based self-reporting systems can supplement existing vaccine safety surveillance systems, but real-world participation patterns have not been assessed at scale. This study aimed to describe the participation rates of a new SMS text messaging- and internet-based self-reporting system called the Kaiser Permanente Side Effect Monitor (KPSEM) within a large integrated health care system. We conducted a prospective cohort study of Kaiser Permanente Southern California (KPSC) patients receiving a COVID-19 vaccination from April 23, 2021, to July 31, 2023. Patients received invitations through flyers, SMS text messages, emails, or patient health care portals. After consenting, patients received regular surveys to assess adverse events up to 5 weeks after each dose. Linkage with medical records provided demographic and clinical data. In this study, we describe KPSEM participation rates, defined as providing consent and completing at least 1 survey within 35 days of COVID-19 vaccination. Approximately, 8% (164,636/2,091,975) of all vaccinated patients provided consent and completed at least 1 survey within 35 days. The lowest participation rates were observed for parents of children aged 12-17 years (1349/152,928, 0.9% participation rate), and the highest participation was observed among older adults aged 61-70 years (39,844/329,487, 12.1%). Persons of non-Hispanic White race were more likely to participate compared with other races and ethnicities (13.1% vs 3.9%-7.5%, respectively; P<.001). In addition, patients residing in areas with a higher neighborhood deprivation index were less likely to participate (5.1%, 16,503/323,122 vs 10.8%, 38,084/352,939 in the highest vs lowest deprivation quintiles, respectively; P<.001). Invitations through the individual's Kaiser Permanente health care portal account and by SMS text message were associated with the highest participation rate (19.2%, 70,248/366,377 and 10.5%, 96,169/914,793, respectively), followed by email (19,464/396,912, 4.9%) and then QR codes on flyers (25,882/2,091,975, 1.2%). SMS text messaging-based surveys demonstrated the highest sustained daily response rates compared with internet-based surveys. This real-world prospective study demonstrated that a novel digital vaccine safety self-reporting system implemented through an integrated health care system can achieve high participation rates. Linkage with participants' electronic health records is another unique benefit of this surveillance system. We also identified lower participation among selected vulnerable populations, which may have implications when interpreting data collected from similar digital systems.

The impact of starting dose on overall survival in myelofibrosis patients treated with ruxolitinib: A prospective real-world study on AIFA monitoring registries.

Ruxolitinib is a JAK1/JAK2 inhibitor approved for the treatment of myelofibrosis (MF)-related splenomegaly or symptoms. The recommended starting dose depends on platelet count, regardless of haemoglobin level at baseline. In the recent years, an overall survival (OS) advantage was reported in patients treated with ruxolitinib compared with best available therapy. We analysed a large Italian cohort of 3494 patients identified by Agenzia Italiana del Farmaco (AIFA) monitoring registries. Of them, 2337 (66.9%) started at reduced dose: these patients were older (median age 70 vs. 67), with increased incidence of large splenomegaly (longitudinal diameter 20 vs. 19.1 cm, median volume 1064 cm3 vs. 1016 cm3), with higher IPSS risk (30.9% vs. 26.1%), and worse ECOG score (more than 1 in 14.3% vs. 9.8%). After balancing for baseline characteristics, Kaplan-Meier analysis showed a median OS of 78.2 months (95% CI 65.9-89) for patients who started at full dose and 52.6 (95% CI 49-56.6) months for patients who started with reduced dose (p < 0.001). Group analysis also showed a substantial difference in patients with intermediate-2 and high IPSS risk. The majority of MF patients in real-world analysis started with a reduced dose of ruxolitinib, which is associated with less favourable outcomes.

The status of blood glucose monitoring and its influencing factors in Chinese patients with type 2 diabetes initiating premixed insulin: A prospective real-world study

ObjectiveThis study aimed to assess the current state of self-monitoring of blood glucose (SMBG) in Chinese patients initiating premixed insulin and its influential factors. Research Design and MethodsThis is a single-arm, multi-center, prospective real-world study enrolling a total of 8214 adult patients with type 2 diabetes mellitus (T2DM) newly initiated premixed insulin analogues. Each patient was followed up for 12 weeks, and the data related to SMBG was collected at week 1, week 4, week 8 and week 12, while data related to glycated hemoglobin were collected at week 1 and week 12. The primary outcome was the frequency of SMBG over 12 weeks. ResultsAt week 12, 83.3 % monitored blood glucose at least once, while 20.3 % of participants continued SMBG every week. The average monitoring frequency was 4.78 times/week over the first 4 weeks and 3.33 times/week over 12 weeks. The patients with a higher frequency of SMBG had better control of blood glucose. ConclusionsThis study found that most T2DM patients would take SMBG but the adherence decreased over time. The adherence to SMBG in Chinese T2DM patients was influenced by age, insulin dosage, education level, and diabetes duration. SMBG benefited the improvement of glycemic control.

FODMAP-Specific Enzyme Supplementation Reduces Food Avoidance & Gastrointestinal Symptoms in Patients with Irritable Bowel Syndrome: A Real-World Prospective Study

FODMAP-Specific Enzyme Supplementation Reduces Food Avoidance & Gastrointestinal Symptoms in Patients with Irritable Bowel Syndrome: A Real-World Prospective Study

Intravitreal faricimab for treatment naïve patients with neovascular age-related macular degeneration: a real-world prospective study

BackgroundIntravitreal faricimab, a bispecific antibody targeting both angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A), was recently introduced for the treatment of neovascular age-related macular degeneration (nAMD), diabetic macular oedema and cystoid macular oedema secondary to retinal vein occlusion. The aim of our study was to assess the efficacy, safety and durability of intravitreal faricimab in a real-world cohort of treatment-naïve patients with nAMD.MethodsSingle-centre, prospective cohort study of 21 eyes from 19 treatment-naïve nAMD patients who were treated with intravitreal faricimab from October 2022 to April 2024. Patients underwent a loading dose (LD) of 4 monthly faricimab injections followed by a treat-and-extend regimen. Primary outcomes included best-corrected visual acuity (BCVA) and structural parameters from spectral-domain optical coherence tomography (SD-OCT). Secondary outcomes included the proportion of eyes achieving a dry macula, maximal fluid-free interval and intended interval at last follow-up.ResultsThe study included 21 eyes of 19 patients (mean age 83.1 years). After LD, 93.3% of eyes achieved a dry macular SD-OCT scan within a median time of 8 weeks. At the first extension, 53% of eyes remained dry, while 47% showed fluid recurrence. Long-term analysis (n = 14) revealed significant reductions in macular volume (MV), central subfield thickness (CST), and pigment epithelial detachment (PED) height over a median follow-up of 64.9 weeks, with sustained visual and anatomical improvements. Median BCVA, CST, and MV at the final follow-up were significantly improved from baseline (p < 0.01). The intended interval between injections was ≥ 12 weeks in 42.86% of eyes. No cases of intraocular inflammation were observed, although 10% experienced retinal pigment epithelial tears.ConclusionsIntravitreal faricimab demonstrated favourable efficacy, safety, and durability outcomes in a real-world cohort of treatment-naïve nAMD patients.

Efficacy of galcanezumab in migraine central sensitizatio

Galcanezumab, a monoclonal antibody targeting the calcitonin gene-related peptide pathway (CGRP mAb), acts peripherally due to its large size. However, recent studies have suggested that CGRP mAbs may also have a central mode of action. This study aimed to evaluate the central effects of galcanezumab on migraine central sensitization.This prospective real-world study was conducted at three headache centers in Japan between May 2021 and May 2022. Patients treated with galcanezumab for migraines were included in the study. The primary outcome was the change in the validated Central Sensitization Inventory (CSI) score from baseline to six months of treatment. We also assessed changes in the Allodynia Symptom Checklist (ASC-12) score. Eighty-six patients with migraine (73 female and 13 male) were analyzed. At 6 months, CSI and ASC-12 scores were significantly reduced compared to baseline (CSI: 36.0 vs. 29.3, p < 0.001; and ASC-12: 5.55 vs. 4.26, p < 0.01). Furthermore, these effects were observed as early as three months of treatment. In this study, we demonstrated the real-world efficacy of galcanezumab in improving central sensitization in migraine, with significant effects seen in the early phase of treatment.Please check and confirm whether the corresponding author mail ID is correctly processed We checked it. And, please kindly re-check !!!! !!! To use this Article for commercial purposes should be okay.!!! We asked you to publish this article with the following license; Creative Commons licence CC BY: This licence allows readers to copy, distribute and transmit the Article as long as it is attributed back to the author. Readers are permitted to alter, transform or build upon the Article, and to use the Article for commercial purposes.Please confirm the inserted city name is correct in affiliation 3 and amend if necessary.Checked.Per style, a structured abstract is not allowed so we have changed the structured abstract to an unstructured abstract. Please check and confirm.We Checked. And again, please kindly make sure the followings;!!! Please kindly re-check !!! To use this Article for commercial purposes should be okay.!!! We asked you to publish this article with the following license; Creative Commons licence CC BY: This licence allows readers to copy, distribute and transmit the Article as long as it is attributed back to the author. Readers are permitted to alter, transform or build upon the Article, and to use the Article for commercial purposes.Trial registration: This study was registered with UMIN-CTR on May 2, 2021 (UMIN000044096).

Prospective Evaluation of Artificial Intelligence Triage of Incidental Pulmonary Emboli on Contrast-Enhanced CT Examinations of the Chest or Abdomen.

BACKGROUND. Artificial intelligence (AI) algorithms improved detection of incidental pulmonary embolism (IPE) on contrast-enhanced CT (CECT) examinations in retrospective studies; however, prospective validation studies are lacking. OBJECTIVE. The purpose of this study was to assess the effect on radiologists' real-world diagnostic performance and report turnaround times of a radiology department's clinical implementation of an AI triage system for detecting IPE on CECT examinations of the chest or abdomen. METHODS. This prospective single-center study included consecutive adult patients who underwent CECT of the chest or abdomen for reasons other than pulmonary embolism (PE) detection 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 installed a commercially available AI triage algorithm for IPE detection that automatically processed CT examinations and notified radiologists of positive results through an interactive floating widget. In phase 1, the widget was inactive, and radiologists interpreted examinations without AI assistance. In phase 2, the widget was activated, and radiologists interpreted examinations with AI assistance. A review process involving a panel of radiologists was implemented to establish the reference standard for the presence of IPE. Diagnostic performance and report turnaround times were compared using the Pearson chi-square test and Wilcoxon rank sum test, respectively. RESULTS. Phase 1 included 1467 examinations in 1434 patients (mean age, 53.8 ± 18.5 [SD] years; 753 men, 681 women); phase 2 included 3182 examinations in 2886 patients (mean age, 55.4 ± 18.2 years; 1520 men, 1366 women). The frequency of IPE was 1.4% (20/1467) in phase 1 and 1.6% (52/3182) in phase 2. Radiologists without AI, in comparison to radiologists with AI, showed significantly lower sensitivity (80.0% vs 96.2%, respectively; p = .03), without a significant difference in specificity (99.9% vs 99.9%, p = .58), for the detection of IPE. The mean report turnaround time for IPE-positive examinations was not significantly different between radiologists without AI and radiologists with AI (78.3 vs 74.6 minutes, p = .26). CONCLUSION. An AI triage system improved radiologists' sensitivity for IPE detection on CECT examinations of the chest or abdomen without significant change in report turnaround times. CLINICAL IMPACT. This prospective real-world study supports the use of AI assistance for maximizing IPE detection.

Aflibercept in the clinical routine: the AURIGA study : The 24-month results of the German cohort of treatment-naïve patients with diabetic macular edema receiving intravitreal aflibercept

AURIGA is the largest prospective real-world study to evaluate intravitreal aflibercept (IVT-AFL) treatment of diabetic macular edema (DME) and macular edema secondary to retinal vein occlusion. This article presents 24-month data from the German cohort of treatment-naïve patients with DME. Treatment-naïve patients (≥ 18years) with DME were treated with IVT-AFL at the discretion of the physician in clinical practice. The primary endpoint was mean change in visual acuity (early treatment diabetic retinopathy, ETDRS, letters) at month12 compared to baseline. Statistical analyses were descriptive. The analysis included data from 150 DME patients (54.7% male). At months6, 12 and24, mean (95% confidence interval) visual acuity gains of4.6 (2.6; 6.5), 4.0 (2.1; 6.5) and 5.0 (3.0; 6.9) letters from baseline (mean ±SD: 65.0 ± 15.3 letters) and reductions in retinal thickness of 86µm (109; 64µm), 70µm (94; 43µm) and 75µm (103; 47µm) from baseline (mean ±SD: 391 ± 132 µm), respectively, were achieved. At month24, 54% of patients gained ≥ 5 letters and 22% ≥ 15 letters. Patients received amean number of 5.0 ± 1.6injections until month6, 7.1 ± 3.2 until month12 and9.0 ± 5.3 until month24, 68% of patients received ≥ 5injections until month6 and 56% ≥ 7injections within the first year. The safety profile was consistent with previous studies. In the German AURIGA cohort, treatment-naïve DME patients achieved aclinically relevant gain in visual acuity as well as reduction in central retinal thickness following IVT-AFL treatment in clinical practice. From month6 onwards, improvements were maintained despite alow injection frequency over 24months. In comparison with previous real-world studies, care of DME patients in clinical practice seems to have improved; however, there is still room for further improvement.

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: 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 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 phase 1 and phase 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 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 years; 3773 female, 3598 male) 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 99.7%), 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.

High-Dose Furmonertinib in Patients With EGFR-Mutated NSCLC and Leptomeningeal Metastases: A Prospective Real-World Study

IntroductionLeptomeningeal metastasis (LM) is one of the most severe complications of NSCLC. Furmonertinib is a pan-EGFR tyrosine kinase inhibitor (TKI) with a high rate of brain penetration and a wide therapeutic window. Here, we evaluated the efficacy and safety of high-dose furmonertinib in patients with EGFR-mutated NSCLC and LM. MethodsThis prospective real-world study included patients with EGFR-mutated NSCLC and LM treated with a high-dose furmonertinib (240 mg once daily) as a monotherapy or in combination with other treatments. The primary end point was overall survival, and the secondary end points included time to treatment discontinuation and clinical response rate. Additional efficacy evaluations included changes in brain magnetic resonance imaging by the response assessment in neuro-oncology–LM radiologic criteria. We also introduced next-generation sequencing–based assays to evaluate genomic and epigenomic features of cell-free DNA (cfDNA) in patients’ cerebrospinal fluid (CSF) samples and to analyze their associations with patient outcomes. ResultsWe enrolled 48 patients, of whom 35 (72.9%) had received third-generation EGFR TKIs. The median overall survival was 8.43 months (95% confidence interval: 5.48–11.39 mo), while the median time to treatment discontinuation was 8.27 months (95% confidence interval: 5.40–11.14 mo), and the clinical response rate was 75%. The LM objective response rate and disease control rate assessed with response assessment in neuro-oncology-LM radiologic criteria were 50.0% and 92.1%, respectively. The adverse event profiles were consistent with previous reports of furmonertinib. Briefly, 22 (45.8%) had adverse events possibly related to furmonertinib and 3 (6.3%) had a grade 3-elevated aminotransaminase or nausea or leucopenia, leading to a dose reduction to 160 mg daily. Furthermore, methylation analysis of cfDNA in CSF revealed that there was a considerable correlation between the changes of aberrant methylated fragments from lung cancer cells and the response of the patients. Meanwhile, the copy number burden scores derived from the low-pass whole genome sequencing assay may offer another objective and effective method for the diagnosis and evaluation of treatment efficacy in LM. ConclusionsIn the real world, the high-dose furmonertinib-based treatment may potentially have clinical efficacy and tolerable safety in patients of EGFR-mutated NSCLC with LM, even in patients previously treated with other third-generation EGFR TKIs. Methylation and copy number burden analysis of cfDNA in CSF may be considered objective indicators for the diagnosis of LM and evaluation of treatment response.

1804P Atezolizumab plus chemotherapy followed by atezolizumab plus anlotinib in the first-line treatment for extensive-stage small cell lung cancer: A multicenter, single-arm, prospective real-world study

1804P Atezolizumab plus chemotherapy followed by atezolizumab plus anlotinib in the first-line treatment for extensive-stage small cell lung cancer: A multicenter, single-arm, prospective real-world study

Safety and tolerability of immune checkpoint inhibitors in people with HIV infection and cancer: insights from the national prospective real-world OncoVIHAC ANRS CO24 cohort study

BackgroundImmune checkpoint inhibitors (ICIs) have been a major advance in cancer management. However, we still lack prospective real-world data regarding their usage in people with HIV infection (PWH).MethodsThe ANRS CO24...

An international observational study assessing conservative management in hemorrhoidal disease: results of CHORALIS (aCute HemORrhoidal disease evALuation International Study).

Aim: Real-world evidence on the management of hemorrhoidal disease (HD) is limited. This international study collected clinical practice data on the effectiveness of conservative treatments for acute HD on symptoms and quality of life (QoL), providing perspectives of treatment modalities from different continents. Patients & methods: The 4-week observational prospective CHORALIS study involved adult outpatients consulting for spontaneous complaints of hemorrhoids (graded using Goligher classification) and prescribed conservative treatments according to usual clinical practice. Assessments were: anal pain/discomfort (visual analog scale [VAS]), other signs/symptoms (patient questionnaire), Patient Global Impression of Change (PGI-C) questionnaire and disease-specific QoL (HEMO-FISS-QoL questionnaire). Results: Of 3592 participants, 3505 were analyzed (58.4% male; age 40.5±13.7years; history of HD in 48.4%; 72.1% Goligher grade I and II). Pain and discomfort were the most common symptoms. Most treatments were venoactive drugs (VADs; 90.9%), particularly micronized purified flavonoid fraction (MPFF; 73.7%) and diosmin (14.6%). All VAD-based therapies improved signs/symptoms (number/intensity/frequency of pain, discomfort, bleeding, swelling, itching and soiling) and QoL. MPFF was associated with a significantly greater proportion of patients with no symptoms (48.8 vs diosmin 34.4%, p<0.001), pain disappearance (69.7 vs diosmin 52.8%, p<0.001), treatment impact at 1week rated on PGI-C as 'very much better' (30.5 vs diosmin 17.9%, p<0.001) and shorter times to improvement (mean±SD 3.9±1.5days vs diosmin 4.2±1.7days). Conclusion: In this prospective real-world study of patients with acute HD, conservative therapies consisting mainly of VADs, including MPFF, improved the clinical signs and symptoms of disease, as well as QoL. This study evidence supports clinical advantages associated with VADs, mostly MPFF, for effectively managing acute HD.

Perceived Benefit and Satisfaction With a Tablet Computer and an Emergency Smartwatch by Older Adults and Their Relatives: Prospective Real-World Pilot Study.

Assistive technologies (ATs) have the potential to promote the quality of life and independent living of older adults and, further, to relieve the burden of formal and informal caregivers and relatives. Technological developments over the last decades have led to a boost of available ATs. However, evidence on the benefits and satisfaction with ATs in real-world applications remains scarce. This prospective, real-world, pilot study tested the perceived benefit and satisfaction with different ATs in the real-world environment. Community-dwelling adults aged ≥65 and their relatives tested a tablet computer with a simplified interface or a smartwatch with programmable emergency contacts for 8 weeks in their everyday life. Perceived benefits and satisfaction with ATs were assessed by all older adults and their relatives using different assessment tools before and after the intervention. Outcome measures included the Technology Usage Inventory, Quebec User Evaluation of Satisfaction with Assistive Technology 2.0, and Canadian Occupational Performance Measure. A total of 17 older adults (tablet computer: n=8, 47% and smartwatch: n=9, 53%) and 16 relatives (tablet computer: n=7, 44% and smartwatch: n=9, 56%) were included in the study. The number of participants that were frail (according to the Clinical Frailty Scale) and received care was higher in the smartwatch group than in the tablet computer group. Older adults of the smartwatch group reported higher technology acceptance (Technology Usage Inventory) and satisfaction (Quebec User Evaluation of Satisfaction with Assistive Technology 2.0) scores than those of the tablet computer group, although the differences were not significant (all P>.05). In the tablet computer group, relatives had significantly higher ratings on the item intention to use than older adults (t12.3=3.3, P=.006). Identified everyday issues with the Canadian Occupational Performance Measure included contact/communication and entertainment/information for the tablet computer, safety and getting help in emergency situations for the smartwatch, and the usability of the AT for both devices. While the performance (t8=3.5, P=.008) and satisfaction (t8=3.2, P=.01) in these domains significantly improved in the smartwatch group, changes in the tablet computer group were inconsistent (all P>.05). This study highlights the remaining obstacles for the widespread and effective application of ATs in the everyday life of older adults and their relatives. While the results do not provide evidence for a positive effect regarding communication deficits, perceived benefits could be shown for the area of safety. Future research and technical developments need to consider not only the preferences, problems, and goals of older adults but also their relatives and caregivers to improve the acceptability and effectiveness of ATs.