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INTRODUCTION We investigated real‐world efficacy, safety, and plasma biomarker dynamics of Lecanemab in Chinese patients with Alzheimer's disease (AD). METHODS A multi‐center prospective cohort study enrolled 68 AD patients. Cognitive scales and plasma biomarkers were assessed at baseline (V0), 2.5 months (V1), and 7 months (V2). RESULTS Alzheimer's Disease Assessment Scale‐Cognitive Subscale 14‐item version (ADAS‐cog14) scores improved significantly at both follow‐ups, and plasma p‐tau181 consistently declined. Both p‐tau181 and p‐tau217 correlated with cognition and partially predicted treatment response (area under the curve [AUC] = 0.734 and 0.713). Mixed‐effects modeling confirmed their dynamic association with ADAS‐cog14 scores. Subgroup analyses indicated benefits across sex and apolipoprotein E4 status, while moderate‐to‐severe cases showed limited response. Lecanemab was well tolerated, with asymptomatic amyloid‐related imaging abnormalities in 17.65% and mild infusion reactions in 5.88%. DISCUSSION These findings support the short‐term efficacy and safety of Lecanemab in early AD and highlight plasma biomarkers as a treatment‐responsive biomarker. Highlights Lecanemab improved cognitive function in Chinese patients with mild cognitive impairment due to Alzheimer's disease (AD‐MCI) and mild AD over a short period. Plasma p‐tau181 and p‐tau217 showed significant correlation with cognitive scores, and their baseline level could partially predict the efficacy of lecanemab. Lecanemab showed a favorable safety profile with low, manageable rates of amyloid‐related imaging abnormalities (ARIA) and infusion reactions.

Background Moyamoya disease (MMD) and moyamoya syndrome (MMS) are rare cerebrovascular conditions with unclear distinctions in clinical presentation and prognosis. Aim This study assessed potential differences between MMD and MMS patients using real‐world data on clinical manifestations, surgical outcomes, and stroke risk factors. Methods This multicenter, retrospective cohort study examined patients with MMD or MMS treated at three tertiary academic hospitals in China, with a mean follow‐up of 11.2 ± 3.1 years. Clinical differences were compared between MMD and MMS, and postoperative cerebrovascular events were compared between patients who underwent surgery and those with conservative management. Primary outcomes were postoperative ischemic and hemorrhagic strokes. Risk factors were evaluated via multivariate Cox regression analysis. Results Of the 2565 patients, 2349 had MMD and 216 had MMS. After 1:1 propensity‐score matching, no significant differences were observed between these two cohorts. Surgical patients had fewer cerebrovascular events than those who received conservative treatment (HR, 0.487; 95% CI, 0.334–0.711; p < 0.001). Preadmission modified Rankin scale scores > 2 (HR, 3.139; 95% CI, 1.254–7.857; p = 0.015) and periprocedural complications (HR, 8.666; 95% CI, 3.476–21.604; p < 0.001) were independent stroke risk factors in patients with MMD. Periprocedural complications (HR, 31.807; 95% CI, 10.916–92.684; p < 0.001) increased stroke risk in patients with MMS. Conclusions This real‐world study revealed substantial clinical overlap between MMD and MMS. Both groups derived significant benefits from surgical revascularization, suggesting distinction may not be necessary to guide surgical management decisions. Optimizing preoperative status and preventing periprocedural complications may improve outcomes in these rare cerebrovascular conditions. Trial Registration This study has been registered in the Chinese Clinical trial registry (registration number: ChiCTR2200064160) This real‐world study revealed substantial clinical overlap between MMD and MMS. Both groups derived significant benefits from surgical revascularization, suggesting distinction may not be necessary to guide surgical management decisions. Optimizing preoperative status and preventing periprocedural complications may improve outcomes in these rare cerebrovascular conditions.

Introduction Previous RC48 (Disitamab Vedotin) studies established that the safety and efficacy of RC48‐antibody–drug conjugate (ADC), either alone or combined with toripalimab, for metastatic urothelial carcinoma (mUC) patients exhibiting human epidermal growth factor receptor 2 (HER2)‐positive or even HER2‐negative status after standard chemotherapy failure. Methods With locally advanced or metastatic urothelial carcinoma (la/mUC), patients who received RC48‐ADC monotherapy or a combination with programmed cell death protein 1 (PD‐1) inhibitors between August 2021 and October 2022 were enrolled in this retrospective observational study to evaluate the real‐world antitumor effectiveness and safety. Results Among the 38 enrolled patients (29 males; median age 67.5 years [38–93]), 8 received RC48‐ADC monotherapy, while 30 received combination therapy. Initially, 63.2% (24/38) of the patients had received ≥1 line of prior treatment, and 63.2% (24/38) had visceral metastasis. UC of the bladder represented the majority type in 68.4% (26/38) of cases. By the data cutoff in March 2023, the overall objective response rate (ORR) was 63.2% (95% CI, 47.1%–79.2%), with a disease control rate (DCR) of 89.5% (95% CI, 79.3%–99.7%). Median follow‐up time was 10.6 months. The median progression‐free survival (PFS) was 8.2 months (95% CI, 5.9–10.5), with a 6‐month PFS rate of 63.2% and a 12‐month PFS rate of 34.1%. Median overall survival (OS) was not reached, with a 12‐month OS rate of 76.7%. The median duration of response was 7.3 months (95% CI, 4.6–10.0) among 24 patients evaluated as partial response (PR). The most common treatment‐related adverse events (TRAEs) included anemia (71.1%), anorexia (57.9%), asthenia (52.6%), hypoesthesia (52.6%), bone marrow suppression (47.4%), alopecia (47.4%), nausea (44.7%), proteinuria (36.8%), vomiting (34.2%), and hypoalbuminemia (31.6%). No patient experienced TRAEs of Grade ≥3. One patient had an immune‐related adverse event (irAE) of rash related to toripalimab. Conclusions Both as monotherapy and in combination with PD‐1 inhibitors, RC48‐ADC exhibits promising effectiveness and manageable safety profile for mUC patients in real‐world settings.

We conducted a large‐scale surveillance study as a post–marketing commitment to investigate the safety and effectiveness of alectinib in patients with ALK‐positive non–small‐cell lung cancer (NSCLC) in Japan. Patients receiving 300 mg twice‐daily alectinib (September 2014 to June 2015) were monitored until termination of alectinib or completion of 18 months of treatment at 519 Japanese study sites. The primary endpoint was the incidence of adverse drug reactions (ADR), which are important identified risks for alectinib in Japanese patients. Overall survival (OS), a key secondary endpoint, was assessed according to information on outcome. Overall, 1251 patients were enrolled. The median patient age was 62.0 years; 12.9% of patients were aged ≥75 years. At baseline, 63.0% of patients had received crizotinib and 40.6% had brain metastases. Altogether, 1512 ADR occurred in 654 patients (53.6%), with 164 grade ≥3 ADR in 123 patients (10.1%). Commonly occurring ADR were hepatic disorders (all grades, 19.8%; grade ≥3, 2.0%), decreased neutrophil and/or white blood cell count (all grades, 7.6%; grade ≥3, 1.1%), and interstitial lung disease (all grades, 3.8%; grade ≥3, .7%). Median OS was not estimable. The 18‐month cumulative OS rate was longer in patients with ECOG performance status ≤1 (vs 2 or ≥3; 83.7% vs 44.5% or 27.2%), without prior crizotinib (vs with; 81.1% vs 73.4%), receiving first‐line alectinib (vs second and third or later line; 83.0% vs 79.2% or 71.9%), without brain metastases (vs with; 79.5% vs 71.5%). These data confirm the favorable safety and effectiveness of alectinib in patients with ALK‐positive NSCLC in Japan. The highly selective ALK inhibitor alectinib is approved as the standard of care for advanced anaplastic lymphoma kinase (ALK)‐positive non–small‐cell lung cancer (NSCLC). This large‐scale surveillance study was implemented as part of a post–marketing commitment to investigate the safety and effectiveness of alectinib in patients with ALK‐positive NSCLC in Japan. Data from this real‐world study confirm the favorable safety and effectiveness profile of alectinib in Japanese patients with ALK‐positive NSCLC.

The previous studies had demonstrated the promising effectiveness and acceptable safety of pyrotinib in patients with HER2‐positive metastatic breast cancer. We aimed to investigate the real‐world data of pyrotinib in complex clinical practice and complement the findings of clinical trials. Two hundred and eighteen patients were included for effectiveness analysis. A total of 62.0% had received two or more lines of systematic therapy, and 95.4% had been exposed to prior anti‐HER2 therapy, with 95.4% receiving trastuzumab, 5.0% receiving pertuzumab, and 40.8% receiving lapatinib. The median progression‐free survival (PFS) was 9.3 months and the objective response rate (ORR) was 44.0%. Patients treated with pyrotinib‐based therapy as first, second, or later line had a median PFS of 15.0, 10.3, and 6.8 months, respectively. Patients treated with pyrotinib and trastuzumab received significant benefit in terms of median PFS compared with pyrotinib alone (10.7 (9.1–12.3) vs. 8.8 (8.1–9.5), p = 0.016). Patients pretreated with lapatinib had a median PFS of 6.9 months. The median PFS time was 7.0 months in patients with brain metastasis. Multivariate Cox regression analyses showed that lines of pyrotinib‐based therapy (1 vs. 2 vs. ≥3), prior treatment with lapatinib, and combination treatments with trastuzumab proved to be independent predictors of PFS. Two hundred and forty‐eight patients were included in the safety analysis, and the results showed that the toxicity of pyrotinib was tolerable, with the most common grade 3/4 adverse event being diarrhea (19.8%). Pyrotinib‐based therapy demonstrated promising efficacy and tolerable toxicity in first‐, second‐, and later‐line treatments and in lapatinib‐treated patients. The combination of pyrotinib and trastuzumab showed advantages in PFS, even for patients resisting trastuzumab. Pyrotinib‐based therapy could be the preferred choice for brain metastasis patients, especially when combined with brain radiotherapy. Pyrotinib‐based therapy demonstrated promising efficacy and tolerable toxicity in first‐, second‐, and later‐line treatments and in lapatinib‐treated patients. Dual anti‐HER2 therapy with pyrotinib and trastuzumab showed advantages in PFS, even for patients resisting trastuzumab. Pyrotinib‐based therapy could be the preferred choice for brain metastasis patients, especially when combined with brain radiotherapy.

Aims This study aimed to investigate the efficacy of early intensive statin therapy following intravenous thrombolysis (IVT) in patients with acute ischemic stroke (AIS). Methods AIS patients who received IVT and statin therapy were included from multicenter registry databases. The primary endpoint was functional independence, defined by a modified Rankin Scale (mRS) score of 0–2 at 90 days. Propensity score matching (PSM) analyses were employed. Results A total of 21,349 patients were included in this study, with a mean age of 68.5 ± 12.6 years, of whom 13,578 (63.6%) were male. The baseline NIHSS score was 4 (IQR 2–8). A total of 9532 patients (44.6%) received intensive statin therapy. In the PSM analysis, the proportion of patients with mRS scores of 0–2 was significantly higher in the intensive statin therapy group (OR = 1.095, 95% CI 1.022–1.173, p = 0.010). Statin type modified the effect of intensive statin therapy on functional independence (p‐value for interaction = 0.030). Treatment effects favoring the intensive approach were observed in patients receiving atorvastatin (OR = 1.134, 95% CI 1.051–1.224, p = 0.001). Conclusion Early intensive statin therapy following IVT leads to a significant but modest improvement in neurological outcomes, particularly in patients treated with atorvastatin as part of the intensive regimen. Early intensive statin therapy after IVT results in a significant but modest improvement in neurological outcomes, particularly in patients using atorvastatin as the intensive treatment regimen.

Background The treatment of extensive stage small‐cell lung cancer (ES‐SCLC) has only made modest progress in the past decade, with two immune checkpoint inhibitors (ICIs), atezolizumab and durvalumab, approved for the treatment of SCLC by January 2022. However, currently, there is limited real‐world data on ES‐SCLC patients received immunotherapy. Methods We retrospectively collected and analyzed the demographic and treatment data of ES‐SCLC patients at the First Affiliated Hospital of Guangzhou Medical University from January 2017 to January 2022. Survival and prognosis information was obtained through follow‐up. Results A total of 353 ES‐SCLC patients were included, of which 165 received immunotherapy combined with chemotherapy as the first‐line (FL) treatment (chemo‐immune group), and 188 received chemotherapy (chemotherapy group). The objective response rate (ORR) and disease control rate (DCR) of patients receiving immunotherapy as the FL treatment were better than the chemotherapy group (76.97% vs. 48.40%, p < 0.001, and 83.03% vs. 68.09%, p < 0.001). Moreover, the progression‐free survival (PFS) and overall survival (OS) of ES‐SCLC patients receiving immunotherapy as the FL treatment were better than the chemotherapy group (6.7 months vs. 5.1 months, p < 0.001, and 12.5 months vs. 11.2 months, p < 0.001). Furthermore, the OS of ES‐SCLC patients who received immunotherapy as second‐line treatment was better than that in the chemotherapy group (15.9 months vs. 12.9 months, p = 0.036). Conclusion ICIs combined with chemotherapy as the FL treatment could be beneficial to the ORR, DCR, PFS, and OS of ES‐SCLC patients. Furthermore, ES‐SCLC patients can benefit from ICIs in the second‐line treatment, even if they had not received ICIs in the FL treatment. Small‐cell lung cancer is a subtype with a poor prognosis in lung cancer. Only two target PD‐L1 inhibitors have recently been approved in ES‐SCLC with moderate survival improvements based on clinical trials' results before 2022. Currently, there is relatively little data about the treatment patterns, efficacy, and safety of ES‐SCLC patients who received immunotherapy in the real world. This manuscript retrospectively collected and analyzed the demographic and treatment data of ES‐SCLC patients treated in the First Affiliated Hospital of Guangzhou Medical University from January 2017 to January 2022. We found that ICI combined with etoposide plus platinum (EP/C) as the FL treatment would prolong the PFS and OS in the ES‐SCLC. Chest radiotherapy with ICI administered may benefit the ES‐SCLC. ICI‐combined treatment in second/further‐line strategies might prolong the prognosis of the refractory SCLC even if patients did not receive ICI in the FL treatment. For the choice of ICIs in FL treatment for ES‐SCLC patients, PD‐1 inhibitor or PD‐L1 inhibitor cannot bring about significant differences in efficacy and safety.

To explore the efficacy and safety of pyrotinib in a real-world setting in a population with HER2-positive advanced breast cancer, subgroup analysis was conducted based on different clinicopathological features to further explore the general characteristics of patients, tumor nature, and the effect of various lines of treatment before patients started pyrotinib on the efficacy of pyrotinib in the real-world study. The clinical pathological characteristics, drug efficacy and related adverse reactions of HER2-positive MBC patients treated with pyrotinib in six hospitals in Southeast Zhejiang Province from February 2018 to December 2023 were collected and analyzed retrospectively. A total of 342 patients with HER2-positive MBC were enrolled. The median follow-up time of 42.0 months. The median age of the overall population was 52 years (range from 25-90 year old). Median progression-free survival in the total population was 10.0 months, the median overall survival was 29.0 months. The (objective response rate, ORR) was 40.35% and the (disease control rate, DCR) was 83.92%. The median progression-free survival (PFS) in the total population was 10.0 months, the median overall survival was 29.0 months. And pyrotinib had better mPFS for advanced first-line treatment than for second-third-line and beyond(14.0 months vs.10.0 months vs.6.0 months, P<0.001). Multivariate Cox regression analysis showed that ECOG, HER2 status, brain metastasis, liver metastasis, number of pyrotinib treatment lines, previous lapatinib treatment, combined capecitabine therapy and trastuzumab resistance were independent prognostic factors for PFS. Diarrhea was the most common adverse reaction (ADR) in 205 patients (59.94%), which could be controlled by antidiarrheal drugs. This multicenter study suggested that the use of pyrotinib for HER2 positive MBC had a relatively good efficacy, especially for those who received first-line pyrotinib treatment and those who were sensitive to previous trastuzumab treatment. Patients with brain metastasis and liver metastases also benefit from pyrotinib treatment, especially for patients treated with brain radiotherapy and/or surgery. ECOG, HER2 status, brain metastasis, liver metastasis, number of pyrotinib treatment lines, previous lapatinib treatment, combined capecitabine therapy and trastuzumab resistance were independent prognostic factors for PFS in HER2 Positive MBC patients treated with pyrotinib. The most common adverse reaction associated with pyrotinib is diarrhea, which can be well controlled through antidiarrheal treatment. Pyrotinib combined with vinorelbine has similar efficacy to pyrotinib combined with capecitabine and has fewer side effects, and can be used as an alternative to capecitabine.

Aims HbA1c and body weight were assessed across selected subgroups of adults with type 2 diabetes receiving oral semaglutide in clinical practice. Methods In this non‐interventional study, changes in HbA1c and body weight to end of study (EoS) and safety were assessed by subgroup: baseline age, body mass index (BMI), type 2 diabetes duration, participants switching from dipeptidyl peptidase‐4 inhibitors, and semaglutide dose at EoS. Results All subgroups experienced reductions in HbA1c and body weight. Younger participants had greater reductions in HbA1c than older participants (−0.9, −0.7, −0.7, and −0.5 percentage points for participants aged <55, ≥55–<65, ≥65–<75, and ≥75 years, respectively [P = 0.0467]). Shorter type 2 diabetes duration and lower EoS semaglutide dose were associated with greater HbA1c reductions (−0.8, −0.7, and −0.6 percentage points with ≤5, >5–≤10, and >10 years' duration, respectively [P < 0.0001]; −1.2, −0.7, and −0.4 percentage points with 3, 7, and 14 mg, respectively [P < 0.0001]). Changes in HbA1c were not significantly different across other subgroups. Lower EoS semaglutide dose was associated with greater body weight reductions (−3.8, −2.9, and −2.8 kg with 3, 7, and 14 mg, respectively [P < 0.0001]); body weight reductions were not significantly different across other subgroups. Adverse events were similar between subgroups, except that older subgroups experienced more events. Conclusions HbA1c and body weight decreased across all subgroups, providing insights into oral semaglutide use in clinical practice for individuals with different characteristics in the real‐world setting. This secondary analysis of the PIONEER REAL Japan non‐interventional study investigated the effect of oral semaglutide in people with type 2 diabetes across subgroups based on HbA1c, BMI, type 2 diabetes duration, and dose of oral semaglutide at the end of the study. Reduction in HbA1c and body weight was seen across all subgroups, with no new safety concerns identified. The results of this study indicate that oral semaglutide is effective for people with type 2 diabetes across a range of clinical characteristics.

Background The use of an insertable cardiac monitor (ICM) is recommended to help diagnose patients with unexplained syncope. Compared with conventional testing (CONV), ICM provides continuous long‐term electrocardiogram (ECG) monitoring and has the ability to establish symptom‐rhythm correlations, which is critical for patients with infrequent and randomized syncope. In China, the upfront cost of ICM is relatively high. However, the subsequent costs and outcomes of additional diagnostics and resulting treatments remain unclear. This study sought to evaluate the economic value of ICM versus CONV in patients with unexplained syncope and suspected of arrhythmia from the perspective of the China's healthcare system, aiming at informing clinical and policy decisions. Methods This study used a Markov model to assess the lifetime costs and benefits of ICM versus CONV for diagnosing syncope. In this model, the costs and effectiveness of all relevant diagnostic and follow‐up arrhythmia‐related treatments were considered. The study cohort characteristics and costs were obtained from a retrospective real‐world study conducted in Sichuan Provincial People's Hospital. The mortality rate, syncope recurrence rate, syncope‐related injury rate, and quality of life estimates were obtained from published literature. Total costs and quality‐adjusted life years (QALYs) were modeled for two strategies, and the incremental cost‐effectiveness ratio (ICER) was calculated. Three times China's GDP per capita in 2023 was used as a willingness‐to‐pay (WTP) threshold. Results ICM was predicted to be more expensive but more effective than CONV. The base‐case results showed that the average cost of the ICM group was 111 733 CNY with QALYs of 10.28, while the average cost of the CONV group was 88 386 CNY with QALYs of 10.08. The ICER was calculated to be 115 596 CNY/QALY. With China's GDP per capita in 2023 being 89 358 CNY, the ICER was < 1.5 times the GDP per capita, and similar results were shown in the scenario analysis. Conclusion This analysis suggests that ICM is a cost‐effective strategy, versus conventional testing, for the diagnosis of unexplained syncope in China, and supports wider uptake of the technology. This analysis suggests that ICM is a cost‐effective strategy, versus conventional testing, for the diagnosis of unexplained syncope in China, and supports wider uptake of the technology.