This multicenter retrospective study compared clinical outcomes and sequential therapy patterns between upper urinary tract urothelial carcinoma (UTUC) and bladder cancer (BC). Data from 517 patients with locally advanced/metastatic urothelial carcinoma diagnosed between January 2020 and December 2021 at 54 Japanese institutions were analysed (232 UTUC, 285 BC). UTUC patients were more likely to have poor renal function and metastases to the lung, liver. Multivariate analysis identified UTUC as an independent risk factor for overall survival (OS). UTUC showed significantly shorter progression-free survival (PFS) and lower response rates to first-line chemotherapy than BC (28.9% vs. 38.2%). These findings persisted after propensity score matching. OS following second-line therapy was also inferior in UTUC, although the rate of transition to second-line therapy was similar between groups. However, a higher proportion of UTUC patients (47.9%) could not proceed to third-line therapy, mainly due to disease progression or adverse events. Overall, UTUC was associated with a worse prognosis than BC. These results highlight the urgent need for more effective first-line treatments, such as immune-combination therapies, particularly in synchronous UTUC cases. ・Keywords: bladder cancer, chemotherapy, prognosis, sequential therapy, upper urinary tract urothelial cancer.Supplementary InformationThe online version contains supplementary material available at 10.1038/s41598-025-25906-4.

Psoriasis is an immune-mediated chronic inflammatory skin disease with a prevalence in Spain of between 2.3% and 2.7%. One-third of patients present with moderate to severe psoriasis (Pso). This article aims to retrospectively describe the characteristics of patients with Pso, as well as severity, patterns of treatment, quality of life (QoL), and associated direct healthcare resources utilized in routine clinical practice in Spain. The SUMMER project is an ambispective, non-interventional, multicenter study including adult patients with a diagnosis of Pso. In the retrospective phase, data were extracted from patients' electronic medical records. Data on disease severity scores (PASI and BSA) and impact on quality-of-life impact (DLQI) were captured by natural language recognition processors. Of 10,874 patients with a diagnosis of psoriasis identified from five participating sites, 2734 did not meet inclusion criteria; a total of 8140 patients were included. Mean age (SD) was 57.7 (16.1) years and 51.3% were male. Most patients had plaque psoriasis (91.5%) and lesions in visible areas (70.8%). The most common comorbidities were dyslipidemia (32%), hypertension (25.6%), and anxiety (18.5%). On the basis of thresholds of PASI (5%) and BSA (3%), psoriasis was not controlled in 17.1% and 37.2% of the patients, respectively, and 25.1% of patients were receiving biological treatments. Between 2017 and 2022, ustekinumab showed the highest persistence rate, especially when used as first-line treatment. There was a tendency to prescribe guselkumab and risankizumab most commonly as second- and third-line therapies. DLQI scores showed that Pso had a moderate or higher impact on QoL for 38.0% of patients. The results show how patients with moderate-severe psoriasis are managed in routine clinical practice in Spain. Between 17% and 37% of patients with Pso are not on the appropriate therapeutic target. Almost a quarter of patients required biological treatments to control the disease.

Neuroendocrine neoplasms (NENs) are rare tumors with increasing incidence. Data on the prognosis of NEN patients in the real-world setting are limited. We aim to explore survival predictors and the impact of novel treatments in a cohort of patients with NENs from various primary sites. This was a retrospective study of subjects diagnosed with gastroenteropancreatic (GEP) or thoracic NENs and referred to a single institution over more than a decade (2010-2023). The primary objective was to describe the overall survival (OS) and progression-free survival (PFS) of patients with advanced-stage disease undergoing first, second, and third-line therapy. The secondary objectives were to identify predictors of OS and PFS. We included 239 NEN patients. Systemic antineoplastic therapies were administered to 149 subjects (62.3%). In patients treated only with first-line therapy, the OS rate was 75% and 74% at 12 and 18 months, respectively. We observed significant univariable associations between Ki-67 index, primary tumor site, morphology, clinical stage, and the OS. The multivariable analysis confirmed a significant association between Ki-67 index, clinical stage, and OS. In patients undergoing second-line therapy, the OS rate was 72% and 61% at 12 and 18 months, respectively, and the Ki-67 index was again associated with OS in the multivariable analysis. GEP NEN patients who received 177Lutetium-Dotatate showed a numerically higher OS rate at 24 months compared to those who did not receive radioligand therapy. In this real-world study of patients with NENs, we confirmed Ki-67 as a strong prognostic parameter but also suggested that 177Lutetium-Dotatate has the potential to prolong OS.

Although patients with extensive-stage small cell lung cancer (ES-SCLC) typically respond well to first-line (1L) platinum-based chemotherapy (PBC)-containing regimens, disease recurrence is common, and survival is short. Treatment options beyond 1L are limited, leaving an urgent need for more effective treatment options. Understanding patient characteristics, treatment patterns, and clinical outcomes in this setting may inform clinical development of novel therapies for ES-SCLC. This study is a retrospective, observational analysis of real-world data from a US nationwide electronic health record-derived de-identified database. Patients with ES-SCLC who received 1L PBC between January 1, 2018, and June 30, 2023, were included. Treatment patterns were analyzed in all patients, and clinical outcomes from third-line (3L) therapy initiation were assessed in those who also received 3L treatment. The study period (January 1, 2018, through December 31, 2023) allowed for≥6months' potential follow-up. Of 2573 patients (50.5% female; 49.5% male) included in the overall population, 992 (38.6%), 344 (13.4%), and 114 (4.4%) received≥1,≥2, and≥3 subsequent treatment lines, respectively. Treatment patterns beyond 1L were fragmented: the most common second-line treatments were lurbinectedin-containing regimens (26.5%), and in 3L were lurbinectedin-containing regimens (21.8%) or topoisomerase inhibitors (21.8%). From 3L therapy initiation, median real-world overall survival (rwOS) was 4.53months (95% confidence interval [CI] 3.71-5.39), median real-world time to treatment discontinuation or death (rwTTD/D) was 2.56months (95% CI, 2.27-2.79), median real-world time to next treatment or death (rwTTNT/D) was 2.92months (95% CI, 2.69-3.12), and real-world response rate among 77 evaluable patients was 11.7% (95% CI, 5.5-21.0). This study demonstrated the heterogeneity of treatments after 1L PBC-containing therapy for patients with ES-SCLC, with no clear standard of care identified. In 3L, rwTTD/D, rwTTNT/D, and rwOS were short, demonstrating the substantial unmet need for novel treatments in this setting.

Abstract PURPOSE This phase 2 non-randomized prospective study was conducted to evaluate the efficacy and safety of regorafenib monotherapy in bevacizumab-refractory recurrent glioblastoma (GBM). METHODS Patients were enrolled based on documented radiographic progression of histologically confirmed GBM following second- or third-line bevacizumab therapy. Patients received oral regorafenib monotherapy at a target dose of 160 mg once daily, administered for 3 weeks on/1 week off in 28-day cycles. Patients underwent brain magnetic resonance imaging (MRI) every 8 weeks and were assessed via modified Response Assessment in Neuro-Oncology (RANO) criteria. Patients continued treatment with regorafenib therapy until they experienced progression or dose-limiting toxicity. RESULTS Based on the analysis of 13 patients (median age 66 years, KPS ≥70), 12 of 13 (92%) had progressive disease (PD) on brain MRI; 1 patient went off study due to Grade 3 weakness prior to first assessment, 3-month progression-free survival (PFS-3) was 7.7% with median (95% CI) PFS of 1.0 (0.4 - 1.8) months. Overall survival (OS) was 38% at 6 months and 23% at 12 months, with median (95% CI) OS 4.1 (2.0 - 7.3) months. Grade 3 or 4 adverse events were fatigue (31%), muscle weakness (23%), and 7% (n=1) each for pancreatitis, palmar-plantar erythrodysesthesia syndrome, and thromboembolic event. There were no Grade 5 adverse events. CONCLUSION Regorafenib did not improve overall or progression-free survival in bevacizumab-refractory recurrent glioblastoma.

Objectives: The rapid evolution of the treatment landscape for non-small cell lung cancer (NSCLC) indicates a need to assess real-world treatment patterns in clinical practice in the US. This study aims to describe treatment patterns and patient characteristics, taking into account new drug entities and newly emerging biomarkers for NSCLC. Methods: A descriptive cohort study was conducted using a specialized electronic health record data for NSCLC. Patients who initiated NSCLC treatment following diagnosis of advanced NSCLC (stage IIIB/IIIC/IV) from 01/01/2015–12/31/2021 were identified. Baseline characteristics and treatment patterns were described; continuous variables were described as mean and standard deviation (SD) and categorical variables as proportions. Results: Of 8,811 patients identified with advanced NSCLC, 67.9% had non-squamous cell carcinoma, 22.5% had squamous cell carcinoma, and 9.6% had an unknown histology. The majority of patients (78.7%) were treated at community hospitals, while 13.4% were treated at academic centers, and 7.9% had an unknown treatment setting. Most patients (59.0%) were aged 65 years or older, and 47.3% were female. There were 22 patients (0.2%) with documentation of NSCLC but no identified guideline-recommended line of therapy (LoT). Among all patients, 51.6% received a second line of therapy (LoT2), and 26.4% received third-line therapy (LoT3). For stage IIIB/IIIC patients, the five most common first-line therapies (LoT1), accounting for 83.2% of all LoT1, included traditional platinum-based chemotherapy or pembrolizumab. In stage IV patients, the five most common LoT1, comprising 64.4% of all LoT1, were pembrolizumab either as monotherapy or combined with other platinum-based chemotherapeutic agents. Conclusions: Traditional platinum-based chemotherapy was the most common LoT1 for stage IIIB/IIIC patients, while the extensive use of immunotherapy (IO) was found in stage IV patients and in later lines of treatment in stage IIIB/IIIC patients. Results indicate that new evidence regarding treatment patterns is rapidly adopted into practice and medication therapy management.

Predicting CAR T-cell therapy outcomes in large B-cell lymphoma using pre-infusion clinical and inflammatory markers: A machine learning approach with explainable artificial intelligence

Background: Upadacitinib (UPA), a selective anti-JAK1 agent, obtained refundability from the Italian National Health System in July 2023 for its use in patients with ulcerative colitis (UC) refractory to other therapies, including anti-TNF-α, anti-integrins, and ustekinumab. At present, no Italian data are available about its effectiveness and safety in the real world. Methods: A retrospective assessment of clinical and endoscopic activity was performed according to the Mayo score. The primary endpoints were to evaluate the effectiveness and safety of UPA. Results: We included 202 consecutive UC patients (M/F 119/83, median age 42). The clinical remission and clinical response rates were 45.5% (92/202) and 63.5% (128/202), respectively, at 8 weeks and 60.2% and 81.7%, respectively, at the end of the follow-up. Clinical remission was achieved more frequently when UPA was used as a first-line rather than a second-/third-line treatment (p = 0.609). Mucosal healing was reported in 84.6% of patients at the median follow-up time. Adverse events occurred in six patients (2.5%), whereas four patients (2%) underwent colectomy. Conclusions: This large real-world study shows that UPA is an effective and safe treatment for UC patients.

Hairy cell leukemia treatment strategies: A real-world multicenter experience

Real-world survival outcomes of Japanese adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia treated with ponatinib

Cytokine signatures predict response, progression, and immune-related toxicity in Sézary syndrome patients treated with mogamulizumab

Vedolizumab for treating steroid-refractory lower gastrointestinal acute graft-versus-host disease: a single-center real-world study

Real-world cardiovascular risk of ponatinib in chronic Phase CML: Insights from a nationwide Korean claims-based cohort study

First-line salvage therapies in relapsed/refractory large B-cell lymphoma after second- or third-line CD19-directed CAR T-cell therapy

Real-world utilization of bispecific antibodies for treatment of diffuse large B-cell lymphoma (DLBCL) in the US community oncology setting

Comparison of axicabtagene ciloleucel to standard regimens as second line treatment for large B cell lymphoma in real life: A lysa study from descar-T and realysa registries

Impact of pulmonary and cardiac comorbidities on cytokine release syndrome incidence in chimeric antigen receptor (CAR) t-treated diffuse large B-cell lymphoma (DLBCL) patients: A real-world data analysis

Effects of general anesthetics on seizure termination, mortality, and neurological prognosis in a rat model of pediatric refractory status epilepticus: A comparative randomized controlled study.

Effectiveness and safety of third-line advanced therapies in patients with ulcerative colitis: A multicentre retrospective cohort study: Third-line treatment in UC.

Evolocumab before percutaneous coronary intervention for acute myocardial infarction: Design of the AMUNDSEN trial.