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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 Lecanemab, an anti–amyloid beta (Aβ) protofibril antibody, was introduced in China in 2024, but its real‐world performance remains unknown. METHODS In this prospective, multicenter study across 21 sites, 261 Alzheimer's disease patients (mild cognitive impairment to moderate dementia) received biweekly lecanemab (10 mg/kg). A matched Alzheimer's Disease Neuroimaging Initiative (ADNI) cohort served as comparator. Cognitive tests, plasma biomarkers, and optional amyloid/tau positron emission tomography (PET) were assessed over 6 months. RESULTS Lecanemab significantly attenuated cognitive decline versus ADNI. Plasma Aβ42, Aβ40, phosphorylated tau 217 (p‑tau217), glial fibrillary acidic protein (GFAP), and ratios showed robust changes; a p‑tau217 reduction correlated with amyloid PET clearance (mean −22.1 Centiloid; 29.2% turned amyloid‐negative). Apolipoprotein E (APOE) ε4 non‐carriers showed greater improvements. Infusion reactions occurred in 11.1% and amyloid‐related imaging abnormalities in 9.2% (1.6% symptomatic), with no stage‐related safety differences. CONCLUSION Lecanemab was effective and well tolerated in real‐world Chinese patients. Plasma p‑tau217 may serve as a sensitive, minimally invasive treatment‐response biomarker. Highlights This is the first large‐scale real‐world evaluation of lecanemab in Chinese Alzheimer's disease patients, demonstrating slowed cognitive decline and robust biomarker improvements. Plasma p‐tau217 reduction strongly correlated with amyloid PET clearance, supporting its use as a treatment response marker. Lecanemab was well tolerated across all AD stages, with no increased adverse events in moderate dementia patients.

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.

Microsatellite stable (MSS) metastatic colorectal cancer (mCRC) hardly benefits from immune-monotherapy. Immune checkpoint inhibitor (ICI)-based combinations have been explored with mixed results. To evaluate the treatment patterns and outcomes across lines of immunotherapy in MSS mCRC, a retrospective real-world data analysis was performed in the present study. A total of 202 patients treated at Renmin Hospital of Wuhan University from June 2019 to December 2023 were included in the analysis, encompassing 38, 41 and 123 cases that received first-(1L), second-(2L) and third-line or above (3L+) ICI-based therapies, respectively. ICI combined with chemotherapy, ICI combined with chemotherapy and anti-VEGF(R)/EGFR/HER2 monoclonal antibody, and ICI combined with tyrosine kinase inhibitor (TKI) were the main options for the 1L, 2L and 3L+ cohorts, respectively. The objective response rates of the 1L, 2L and 3L+ treatment cohorts were 63.2, 22.0 and 11.4%, respectively, and the disease control rates of these three cohorts were 94.7, 75.6 and 70.7%, respectively. The median progression-free survival of the 1L, 2L and 3L+ cohorts was 11.1, 6.5 and 4.7 months, respectively, and overall survival (OS) was not reached, 16.0 and 11.9 months, respectively. After propensity score matching, patients who received ICI cross-line therapy at least once in the 3L+ cohort showed directional OS improvement (14.3 vs. 11.3 months; hazard ratio, 0.66; 95% confidence interval, 0.39-1.14; P=0.123) compared with those who did not receive ICI cross-line therapy. Grade ≥3 adverse events (AEs) occurred in 43.6% of patients, with thrombocytopenia, leukopenia and abnormal hepatic function being the most common. The incidence of immune-related AEs (irAEs) was 38.6% and that of grade ≥3 irAEs was 15.3%. These findings suggest that chemotherapy remains the cornerstone of 1L and 2L treatment for MSS mCRC, while the combination of ICI and TKI has developed into a major trend at the 3L+ setting with improved survival and controllable safety.

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.

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.

Objective To evaluate the real‐world effectiveness and safety of adjunctive zonisamide in children with developmental epileptic encephalopathy/epileptic encephalopathy (DEE/EE), focusing on younger children and highly drug‐resistant cases. Methods This open‐label, nonrandomized, self‐controlled, real‐world study included 127 children with DEE/EE at a single center from 2020 to 2025. The primary endpoint was the responder rate (≥ 50% seizure reduction) at 3, 6, 9, and 12 months, analyzed using last observation carried forward. Secondary endpoints included seizure freedom, retention, and adverse events (AEs). Multivariable analysis identified predictors of efficacy. Results Responder rates were 51.2%, 55.3%, 53.7%, and 53.7% at 3, 6, 9, and 12 months, respectively. Seizure‐free rates were 23.6%, 26.8%, 24.4%, and 27.6%. Efficacy was consistent across age and etiology. Female gender (OR = 3.00) and fewer prior anti‐seizure medications (OR = 0.77) independently predicted 12‐month response. Among children failing ≥ 5 prior medications, 44.3% responded, and 20.0% achieved seizure freedom. The 12‐month retention rate was 81.9%. AEs occurred in 14.2%, most commonly reduced appetite (7.9%); most were tolerable. Conclusion Adjunctive zonisamide provides sustained efficacy and favorable tolerability in children with DEE/EE, including young children < 6 years and highly drug‐resistant cases. Gender and prior treatment history predict long‐term efficacy, supporting its reliable risk–benefit profile for refractory epilepsy. Adjunctive zonisamide provides sustained efficacy and favorable tolerability in children with DEE/EE, including young children < 6 years and highly drug‐resistant cases. Gender and prior treatment history predict long‐term efficacy, supporting its reliable risk–benefit profile for refractory epilepsy.

Subject To analyze the treatment response in patients with primary angiitis of the central nervous system (PACNS) in a large vasculitis cohort. Method In this single‐center retrospective observational study, we assessed treatment, relapses, remission, and outcome of patients with PACNS. We pooled the patients' relapses under different treatments as well as various immunotherapies. Multivariate logistic regression analysis was performed to determine factors independently associated with relapse and those associated with good functional status. The time of observation was 96 months. Result The cohort comprised 80 patients, with 38 diagnosed with pathologically confirmed PACNS and 42 with clinically diagnosed PACNS, with a median follow‐up duration of 18 months (range 3–96). Treatment comprised acute‐phase induction therapy with high‐dose corticosteroids, alone or combined with immunosuppressive agents, followed by remission‐phase maintenance immunosuppressive therapy, primarily with cyclophosphamide, rituximab, or mycophenolate mofetil. Following treatment, 49 patients (61.3%) achieved remission and 70 (87.5%) attained favorable functional outcomes. The overall relapse rate was 35%. Group 3 demonstrated significantly higher baseline disease severity (p < 0.05). Multivariate analysis identified seizures and cognitive impairment as predictors of relapse. Conclusion This study demonstrates that a majority of PACNS patients exhibit a favorable response to therapy. For patients presenting with more severe disease at diagnosis, long‐term maintenance therapy following remission induction with glucocorticoids or immunosuppressive agents is required. This study of a large Chinese cohort of PACNS clarifies real‐world treatment strategies, relapse patterns, and functional outcomes. The findings demonstrate most patients respond favorably to therapy. The overall relapse rate was 35%, with seizures and cognitive impairment identified as significant predictors of relapse.

Background Intense pulsed light (IPL) therapy is widely used for facial rejuvenation, targeting vascular, pigmentary, and textural changes. However, comprehensive, real‐world evidence evaluating long‐term, regular IPL treatment across multiple dimensions of skin improvement remains limited. Objectives This study aimed to assess the efficacy and safety of long‐term, regular IPL therapy in improving facial erythema, pigmentation, and wrinkles, and to identify predictors of favorable response. Methods This retrospective real‐world study included 236 patients who underwent six or more IPL sessions between 2020 and 2025. Patients were categorized as acne, rosacea, or cosmetic subjects. VISIA imaging quantified erythema, pigmentation, and wrinkle indices, while the Global Aesthetic Improvement Scale (GAIS) was used to assess aesthetic outcomes. Logistic regression identified predictors of good response. Results Significant improvements were observed in erythema, pigmentation, and wrinkle indices after treatment (all p < 0.05). Regular treatment intervals and total number of sessions were independently associated with better outcomes (OR = 13.62 and 3.80, respectively, both p < 0.05). Patients with Fitzpatrick Type IV skin showed lower response rates (OR = 0.12, p = 0.001). VISIA analyses demonstrated quantifiable reductions in erythema and pigmentation areas, while wrinkles showed notable textural improvement. No severe adverse events occurred. Conclusions Long‐term, regular IPL therapy effectively improves facial erythema, pigmentation, and wrinkles, confirming its value as a comprehensive rejuvenation strategy. Regular treatment intervals optimize cumulative effects, while objective imaging enhances precision in outcome evaluation. These findings support IPL as a safe, evidence‐based, and multidimensional approach to long‐term facial rejuvenation.

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.