Early effectiveness of anifrolumab in systemic lupus erythematosus: real-life data a 3 and 6 months from RALESA registry.

Complement inhibitor pegcetacoplan binds to C3 and its activation product C3b. Pegcetacoplan has been approved for the treatment of paroxysmal nocturnal hemoglobinuria. Because pegcetacoplan exerts broad inhibition of the complement cascade its efficacy is also investigated in numerous other diseases caused by complement dysregulation, such as C3 glomerulopathy. Pegcetacoplan causes a number of counterintuitive changes in laboratory results. In-depth complement analysis in two patients with PNH and three patients with C3 glomerulopathy, all treated with pegcetacoplan. C3 levels increase up to 300 % above reference levels. In vitro testing showed that this is not a turbidimetric artifact in the C3 immunoassay due to pegcetacoplan-C3 complex formation but appears to be caused by increased half-life of C3 bound to pegcetacoplan. Unbiased mass spectrometric plasma proteome analysis confirmed the dramatic pegcetacoplan-induced increase in circulating C3. Surprisingly, also a three-fold increase of properdin was observed during pegcetacoplan treatment. Serum protein electrophoresis showed an additional band in all patients after pegcetacoplan exposure. This C3-band does not migrate at its expected position because of changes in the mass and charge of C3 bound to pegcetacoplan and should therefore not be misinterpreted as an M-protein. Both invitro experiments and real clinical practice laboratory results demonstrated that pegcetacoplan completely blocked the alternative complement pathway while the classical pathway is affected but remains largely intact. Because of the increasing use of pegcetacoplan in routine clinical practice, it is important that both clinicians and laboratory specialists are aware of these unexpected therapy-induced laboratory findings.

The application of artificial intelligence (AI) is increasingly valuable as a tool and assistant in many areas of clinical and academic medicine. Generative AI (GenAI) creates new content used by large language models, which can generate language that strongly resembles or even improves on that of humans. Learners and educators in many areas of education are using GenAI for essays and assessments, raising issues regarding learning and assessment. GenAI is also raising new concerns in health professions education (HPE), an area of health professions training that sometimes has different aims and assessment methods compared to its clinical counterparts. HPE needs to assess levels of knowledge and understanding of pedagogy, and the use of GenAI presents challenges to its current assessments, which are predominantly written. The study aimed to investigate educators' and learners' perspectives on the opportunities and challenges presented by GenAI in postgraduate HPE assessments. It particularly focused on perspectives of how GenAI may influence the future of assessment and essay-based assessments in HPE. Informed by a constructivist paradigm, a qualitative approach was adopted, undertaking 8 semistructured interviews conducted via Microsoft Teams. Purposive sampling ensured a mixture of educators and learners in current HPE courses from a range of health care professions. Data were thematically analyzed. There was no difference between educator and learner perspectives. Four themes were identified: AI is here, students are at a disservice if we do not embrace it; AI as an opportunity to rethink HPE assessments; AI is a "gray area"; and AI is fallible. The findings present AI as an external catalyst, highlighting the current internal desire for assessment change within HPE. It offers opportunities for creative, authentic assessments that reflect real-life academic and clinical practice, aiming to develop competent future HPE educators and keep courses relevant. These findings contribute to the debate around the future potential and development of AI in HPE assessments.

Imatinib has revolutionized the management of chronic-phase chronic myeloid leukaemia (CP-CML). This study aimed to evaluate the long-term efficacy, safety and prognostic determinants of imatinib in a large cohort of CP-CML patients in real-life clinical practice. We conducted a retrospective, monocentric observational study including all adult patients with CP-CML treated with imatinib between 2000 and 2025. Overall survival (OS), event-free survival (EFS) and progression-free survival (PFS) were estimated using Kaplan-Meier analysis. A total of 210 patients were included, of whom 81% received imatinib as first-line therapy. The median follow-up was 12.4 years. At 25 years, OS and PFS were 71% and 88% respectively. The EUTOS long-term survival (ELTS) score independently predicted OS, EFS and PFS (p < 0.001), while lower risk categories were associated with a faster achievement of major molecular response (MMR) and a higher probability of deep molecular response (DMR). Dose reductions were applied in 26.2% of patients, mainly older individuals with higher Charlson comorbidity index (CCI), without affecting molecular or survival outcomes. Higher CCI values correlated with inferior OS. The ELTS score remains a powerful prognostic tool for long-term outcomes. No unexpected safety concerns were observed in the long term. Over 25 years of real-world experience, imatinib has demonstrated sustained efficacy, safety and tolerability in CP-CML.

This post-hoc analysis evaluated how goal attainment evolved over repeated cycles of botulinum toxin A (BoNT-A) treatment in adults with upper-limb spasticity. ULIS-III (NCT02454803) was a 2-year observational study involving adults treated with BoNT-A. The analysis included 538 patients who received ≥ 4 BoNT-A injection cycles and had Goal Attainment Scaling (GAS) assessments for each of the first 4 cycles. GAS T-scores were used to measure treatment response. Multivariate models assessed predictors of achieving a GAS T-score ≥ 50 and being in the top tertile (best) of responders. Patients generally maintained consistent goal domains across cycles. Mean change in GAS T-scores remained above the minimal clinically important difference of 10 throughout. The proportion of patients achieving a GAS T-score ≥ 50 increased from 64.5% in Cycle 1 to 75.8% in Cycle4. Use of injection guidance techniques significantly increased the odds of achieving treatment goals (OR 1.92; 95% CI 1.45-2.55) and being a "best" responder (OR 2.44; 95% CI 1.47-4.06). Higher BoNT-A doses were also associated with better outcomes. Repeated BoNT-A treatment supports sustained goal attainment in upper limb spasticity. Success rates improve with each cycle, particularly when guided injection techniques are used.

Background With increasing endeavours to incorporate teaching material on healthcare for underrepresented groups into medical school curricula, there remains a lack of research exploring the integration of this into assessments. With age and gender as the only demographic information routinely provided in single best answer (SBA) questions, this does not represent the diversity of patients encountered by doctors in clinical practice. This pilot study assessed how representation of patient demographic characteristics influenced medical students answering SBA questions. Methods 200 medical students in their clinical years completed 15 SBA questions in an online simulated exam. Participants were randomised to control and test groups. The control group denoted age and gender, with test groups including neutral stems denoting age only; stems denoting LGBTQ+ identities; stems denoting ethnicity; and a mix of stems. Post-exam Likert scale questions explored their experience of the simulated exam. Results Linear regression modelling demonstrated overall statistically nonsignificant differences between groups, indicating that assignment to control or test group does not explain differences in exam performance. Using a mix of question stems produced statistically significant differences, with participants scoring worse on question stems including LGBT+ identities and ethnicity. Conclusion As a pilot study these results suggest diversification of SBA questions does not inherently disadvantage students. A multimodal approach is needed to integrate diversity into medical school curricula and assessments. SBA questions can form part of this by mirroring diverse patient groups encountered in real-life clinical practice and positioning them as active agents seeking and accessing health care.

Introduction . Effectiveness and safety of satralizumab in the treatment of neuromyelitis optica spectrum disorders (NMOSD) have been demonstrated in randomized clinical trials and continue to be evaluated in the real clinical practice (RCP). Aim . To analyze the experience of satralizumab administration in Russian patients with NMOSD in the RCP. Materials and methods . The study was a multicenter retrospective. Analyzed data were taken from outpatient charts/medical records. Baseline clinical and demographic characteristics of patients with results on efficacy and safety of satralizumab treatment were assessed. Results . Data of 71 patients with ages ranging from 12 to 69 years at the start of satralizumab treatment were analyzed. 100% of patients had NMOSD with antibodies to aquaporin-4. At baseline 18.3% of patients were with highly active NMOSD by exacerbation frequency, 69% had previous relapse preventive treatment, 41% received low-dose glucocorticoids. The annualized relapse rate (ARR) during previous 2 years before treatment was 1.26. The median duration of satralizumab therapy was 15 [12; 24.5] months. 58 (82%) patients were relapse-free and included 9 (16%) persons with highly active NMOSD. The ARR decreased to 0.11 over 2 years of therapy (p &lt; 0.001). In 8 (62%) of 13 patients with relapses first attack developed before 6 months of treatment and in 6 (46.2%) cases – during the “clustered period”, 11 (84.6%) patients did not receive low doses glucocorticoids or they were discontinued before 6 months of satralizumab therapy. Adverse events were reported in 19 (26.8%) patients. The neutropenia was the most common. 27 (84.4%) of 32 patients switched from rituximab to satralizumab were relapse-free with a favorable safety. Conclusions . The analysis of the experience on the satralizumab administration in the RCP demonstrated efficacy and favorable safety profile in Russian patients with NMOSD with antibodies to aquaporin-4 aged 12 years and older.

Chronic bronchitis (CB) and chronic obstructive pulmonary disease (COPD) are among the most common respiratory diseases and are associated with high morbidity and significant disability. Frequent exacerbations of COPD lead to prolonged deterioration in respiratory function and gas exchange, faster disease progression, a significant decrease in the quality of life, and significant treatment costs. The most affordable and effective local therapeutic agent is inhaled aerosol of thiamphenicol glycinate acetylcysteinate (TGA). However, there is insufficient data in the international and domestic literature on using this drug in real clinical practice. The aim of this study was to evaluate the clinical and pharmacoeconomic efficacy of TGA compared with systemic antibacterial drugs in the treatment of exacerbations of CB and COPD. Methods. A retrospective analysis of medical records was carried out, including a total of 1,151 cases of exacerbations of CKD and COPD diagnosed and treated at the Chelyabinsk Regional Pulmonological Center in 2020 – 2024. The clinical and pharmacoeconomic efficacy was evaluated. Results. The use of the topical antibacterial drug TGA (Fluimucil antibiotic IT®) makes it possible to achieve remission in patients with infectious exacerbations of CB and COPD and increase the time interval to the next exacerbation by 28.09 days (95% CI – 16.57 – 39.6 days, p &lt; 0.01) compared with systemic antimicrobial drugs. The use of TGA leads to a rapid decrease in the main symptoms of exacerbation (decreased cough, shortness of breath, and sputum production). The use of TGA reduces the economic cost per patient, thus reducing the economic burden of CB and COPD. Conclusion. The use of TGA in clinical practice helps reduce the frequency of exacerbations, increase the time to the next exacerbation and increase the effectiveness of antibacterial therapy, while reducing the costs.

Cefepime is a key carbapenem-sparing agent due to its stability against AmpC β-lactamases. However, high plasma concentrations are associated with cefepime-induced neurotoxicity (CIN). Following 2019 EUCAST reclassification of 'intermediate' as 'susceptible, increased exposure', higher doses (6 g/day) are often recommended. Pharmacokinetic/pharmacodynamic (PK/PD) simulations suggest that a reduced daily dose of cefepime 4 g/day administered by continuous infusion may achieve adequate target attainment while limiting toxicity, but real-world clinical data are scarce. We conducted a prospective, single-centre observational study including adult inpatients treated with cefepime administered as a 2 g loading dose followed by continuous infusion of 4 g/day. Therapeutic drug monitoring was performed to assess steady-state free cefepime concentrations (ƒCss). The primary endpoint was pharmacodynamic target attainment (100% ƒT > MIC) for EUCAST 'susceptible, increased exposure' breakpoints. Among 46 included patients, median ƒCss was 26.2 mg/L (IQR 18.4-33.2). Pharmacodynamic targets were achieved in 96% of patients for Enterobacterales (MIC 4 mg/L) and 93% for Pseudomonas aeruginosa (MIC 8 mg/L). Clinical efficacy was observed in 96% of cases. Signs consistent with CIN occurred in three patients (6.5%), mainly in the context of renal function deterioration or pre-existing neurological vulnerability. A reduced-dose cefepime regimen consisting of 4 g/day administered by continuous infusion achieves high pharmacodynamic target attainment with a favourable efficacy-toxicity balance in real-life clinical practice. This strategy represents a promising alternative to higher-dose regimens and supports individualized dosing guided by renal function and therapeutic drug monitoring.

The objective: to conduct an analytical study on trends in the impact of clinical trials (CTs) on the public health.Materials and methods. The bibliosemantic and analytical methods were used to study modern (2021–2025) scientific sources on the impact of CTs results on the health of the population to improve the public health system.Results. The theoretical foundations of the impact of CTs are based on the evidence-based medicine as the basis of public health and taking into account translational medicine and health policy. The impact of CTs on prevention and treatment is demonstrated, in particular, by the impact on public health of preventive programs and vaccinations, clinical guidelines and treatment standards. The innovative approaches of CTs are presented on data from real clinical practice, as well as on the use of digital technologies and artificial intelligence. Social and ethical aspects of CTs are considered as a requirement for representativeness, fairness and communication of research results. The current challenges and prospects for CTs are identified, based on financing and sustainability of CTs and their integration into practice.Conclusions. CTs are a critically important tool for improving public health. They shape preventive programs, treatment standards, regulatory decisions, and public trust in medicine. The future impact of CTs will depend on their quality, inclusiveness, ethics, and effective translation of results into policy and practice.

Abstract Background Thyroidectomy has traditionally required inpatient admission secondary to concerns of postoperative complications of bleeding, airway obstruction, and hypocalcaemia. However, increased pressures on healthcare resources have mandated re-evaluation of surgical pathways. British Association of Day Surgery (BADS) considers day case thyroidectomies a safe procedure. In the last BAETs audit only 5.5% of all UK thyroid surgeries were undertaken as day case between 2010–2015. This single-hospital study aimed to review current practice, post-operative complications, and describe a standardised protocol by which day case hemithyroidectomy can be implemented safely. Methods This single-centre retrospective study included all thyroid and parathyroid procedures performed between February 2023 and December 2024 in Portsmouth University Hospitals. Results A total of 456 thyroid and parathyroid operations performed between February 2023 and December 2024, 6 (1.32%) experienced postoperative haematomas with only 4/6 requiring a return to theatres. 2/284 were following hemithyroidectomy (0.85%) and 4/172 after parathyroidectomy (2.33%). There was no mortality reported. Of the remaining patients who had no haematomas, 5% of hemithyroidectomy patients would have met the day-case criteria. Conclusions Our single-centre results suggest that day-case hemithyroidectomy is safe and will contribute to improved bed utilisation, cost reduction, and enhanced patient satisfaction without compromising safety. A two-part day case hemithyroidectomy protocol was developed. An additional prospective evaluation will be carried out to validate the efficacy of the protocol suggested in real clinical practice.

Background: the search for predictors of readmission applicable in real clinical practice is still of interest to schizophrenia researchers. The prognostic value of antipsychotic therapy after discharge from the hospital and its relationship to the duration of the disease remain poorly understood. The aim was to identify risk factors for repeated hospitalization in patients with paranoid schizophrenia, taking into account the duration of the disease and the underlying antipsychotic therapy prescribed at the time of discharge from a psychiatric hospital. Patients and Methods: the study included 163 patients with paranoid schizophrenia (unselected sampling; women — 42.3% (n = 69), average age — 30.26 ± 7.05 years), discharged after inpatient treatment. Antipsychotic therapy was taken into account when the patient was discharged from the hospital, according to medical records for the period from 2018 to 2024. Antipsychotic drugs were divided into the first and second generations (respectively, FGA and SGA). Patients taking clozapine were excluded from the study. The method of taking the medicine (oral or injectable in the form of a depot) was taken into account. All doses of the drugs have been converted to standard daily doses (the equivalent of 5 mg of risperidone according to the WHO Defined daily dose method). The prognostic significance of the duration of the disease was also studied. The risk of re-hospitalization was assessed using the Cox regression method with mixed effects. Results: mean duration of follow-up was 3.87 [2.87; 4.08] years. The average dosage of the antipsychotic was 1.20 [0.99; 1.67] daily doses. Secondgeneration antipsychotics accounted for 76.2% in total structure of drug prescriptions. The share of extended forms of both generations accounted for 18.1% of appointments. With one SGA hospitalization, the risk of repeated hospitalization decreases with an increase in the standard dose (aHR = 0.24 (0.07; 0.83), p = 0.005). In the first episode, the overall risk of rehospitalization was lower relative to that of chronic patients (aHR = 0.49 (0.29, 0.82), p &lt; 0.001). For the remaining combinations of factors “drug dose — antipsychotic generation — number of hospitalizations”, the trends are not unambiguous. Conclusions: the risk of rehospitalization at the first psychotic episode was reduced by 2.04 (1.22; 3.40) times regardless of the therapy received. When patients are prescribed SGA drugs (except clozapine) in a dose exceeding the standard daily dose, the risk of rehospitalization decreases by 4.17 (1.20; 14.29) times regardless of the duration of the disease.

We sought to understand healthcare workers' (HCW) cardiovascular disease (CVD) prevention knowledge, attitudes, and practices to help inform future healthcare policies and optimize preventive cardiology care. Data was collected via an anonymous, online questionnaire which consisted of pre-validated CVD prevention and smoking cessation scales adapted from the Preventive Medicine Attitudes and Activities Questionnaire. Six hundred sixty-eight HCWs (60.5% doctors, 27.8% nurses, 11.7% medical students) from 25 nations responded to the survey. Overall, 74.9% of HCWs routinely assessed patients' cardiovascular risk profiles in clinical practice. About 65.7% of HCWs counselled patients who were asymptomatic for CVD on tangible lifestyle changes to improve their cardiovascular risk profiles, while 68.2% of HCWs did so when patients were overweight. Of note, only 51.3% of HCWs implemented comprehensive smoking cessation interventions for their patients. Practising HCWs demonstrated higher levels of CVD prevention promotion than medical students in all aspects, except for self-reported importance of CVD risk factor counselling (Tukey honestly significant difference diff: 0.31, P-value: .051). Among practising HCWs, there were no significant differences in their CVD prevention practices across varying lengths of clinical practice. HCWs from higher income nations tended to fare worse than their lower income counterparts. A large multi-national survey reveals significant gaps in the promotion of CVD prevention by HCWs. Significant differences between medical students and practising HCWs' CVD prevention behaviours, highlight the role of education for the promotion of long-term positive CVD prevention practices. Further efforts should target the medical education of early-career HCWs, especially in higher income nations. Key message What is already known on this topic: The importance of lifestyle modification for the primordial prevention (risk factor prevention) and primary prevention (risk factor management) of cardiovascular disease (CVD) is indisputable. Studies have shown that physicians and other healthcare workers (HCWs) may be best placed to encourage tangible lifestyle changes and enact meaningful modification in patients' cardiovascular health-related behaviours. What this study adds: However, in practice, the role of HCWs in monitoring and encouraging patients' health behaviours is complicated by the challenges of real-life clinical practice, such as time constraints or lack of manpower. Hence, this large multi-national survey sought to understand HCWs' CVD prevention knowledge, attitudes, and practices to help inform future healthcare policies and optimize preventive cardiology care. How this study might affect research, practice, or policy: This study reveals significant gaps in the promotion of CVD prevention by HCWs, highlighting key differences in CVD prevention practices based on profession, level of training, subspecialty, and national income status. Nurses, cardiology subspecialists, and HCWs from lower middle-income nations were found to be more proficient in promoting CVD prevention compared to their counterparts. Further efforts should target the medical education of undergraduate HCWs, especially in higher income nations, as established clinical practices learned during clinical education typically persist and are resistant to change over time.

Background. Late post-transplant diseases can be latent or present as late graft dysfunction. The objective was to assess the nature of pathological changes in liver transplant recipients in the long-term based on the severity of graft dysfunction. Material and methods. The results of a histological examination of the liver performed no earlier than one year after transplantation in 168 recipients were studied. The median follow-up was 57.8 (26.3; 94.9) months. Graft dysfunction was defined as overt if alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) increased to more than 1.5 times the upper limit of normal (n=73). Borderline dysfunction was defined as an increase in at least one of these parameters to more than 1 but less than 1.5 times the upper limit of normal, or an increase in gamma-glutamyl transferase (GGT) to more than 1.5 times the upper limit of normal (n=37). Graft dysfunction was absent in 58 recipients. Results. In the subgroup of recipients without graft dysfunction, a slight increase in body mass index (BMI) (+1.1 kg/ m 2 ) was noted compared to BMI at transplantation. Recipients with the borderline graft dysfunction had a lower BMI (25.4 kg/m 2 ), and those with the overt dysfunction had an even lower BMI (23.7 kg/m 2 ), than the subgroup without graft dysfunction (26.8 kg/m 2 ; p=0.015). Clinical signs of the graft dysfunction were absent in 34.5% of recipients at the time of examination; however, only 22.4% of these recipients showed no significant abnormalities on histological examination. Among the remaining recipients with normal liver tests, there was evidence of chronic hepatitis (19%), fatty liver disease (31%), or intralobular fibrosis (25.9%), and in one case, graft cirrhosis. Graft fibrosis was observed in 60.3% of recipients without graft dysfunction. Marked fibrosis (classified by the Liver Allograft Fibrosis (LAF) scoring system as LAF &gt; 2) was detected in 31%, and significant portal tract fibrosis (assessed as the meta-analysis of histological data in viral hepatitis (METAVIR) score &gt;2) was found in 20.7% of recipients without signs of graft dysfunction. In the subgroup of recipients with overt graft dysfunction, ductopenia was the only pathological finding in 11.1% of recipients. More than two-thirds of cases of fatty liver disease and intrahepatic fibrosis do not manifest with clinically significant abnormalities in functional liver tests. Histological examination allowed for the clarification of the cause of overt graft dysfunction in 69.4% of cases. Conclusion. Protocol biopsies in long-term liver transplant recipients enable the detection of pathological changes of varying severity, as well as the assessment of hepatitis activity, fibrosis stage, and the cause of graft dysfunction, and the identification of autoimmune disease recurrence.

Background/Objectives: Artificial intelligence (AI) tools, particularly large language models (LLMs) such as ChatGPT-4o, are gaining prominence in medicine. While their diagnostic capabilities have been explored across various oncologic domains, their role in clinical decision-making within multidisciplinary tumor boards (MTBs) remains largely unexamined in urologic oncology. This study evaluates the performance of ChatGPT-4o as a decision-support tool in a real-world MTB setting by comparing its recommendations with those of expert clinicians. Materials and Methods: A retrospective study was conducted using 98 anonymized clinical cases discussed by a urologic MTB between June 2024 and February 2025. An independent urologist entered the same cases into ChatGPT-4o using a standardized prompt replicating real-world presentation. Two certified urologists independently assessed the model's responses. Agreement was analyzed overall and by tumor type, disease stage, clinical context, and treatment strategy. Results: ChatGPT-4o fully agreed with the MTB in 56.1% of cases, was correct but incomplete in 23.5%, and provided partially accurate but flawed recommendations in 18.4%. Overall concordance between ChatGPT-4o and the MTB yielded a Cohen's kappa of 0.61, indicating moderate-to-good agreement. Discrepancies were most common in metastatic prostate cancer, often due to misclassification of tumor burden or errors in treatment sequencing. Highest agreement rates were observed in bladder and renal tumors, and in standardized therapeutic scenarios such as radiotherapy. Conclusions: ChatGPT-4o demonstrated moderate alignment with expert MTB decisions and performed best in well-defined clinical contexts. While it cannot replace multidisciplinary expertise, it may serve as a supportive tool to enhance access to standardized oncologic care.

IntroductionWith health systems facing increasing challenges, it is important to define new care models that may release some of the burden on the workforce, whilst maintaining quality and improving patient convenience. The objective of this study was to create a taxonomy of pharmacist-led interventions aimed at supporting improved health outcomes of people self-managing cancer medication.MethodsThe list was developed following a literature search conducted in Cochrane Library and MEDLINE (PubMed) databases. The Capacity, Motivation and Opportunity (CMO) model developed for people living with HIV was used as theoretical framework to organise the pharmacist-led interventions emerging from literature. A panel of experts (hospital pharmacists with experience in oncology) selected through national hospital pharmacy societies was invited to participate in a consensusseeking Delphi survey, focusing on the most important and feasible interventions.ResultsA total of 28 experts answered and rated 39 interventions. Whilst 35 of the proposed pharmacist-led interventions were considered important (median score above 8, on a scale of 1 to 9, without disagreement among experts), only eight were considered feasible for implementation in practice. The most frequently mentioned reasons for others not to be feasible were understaffing and excessive workload. Nevertheless, there were interventions considered vital for patients’ care and hence kept in the final taxonomy.DiscussionFuture hospital-based studies should incorporate this taxonomy based on the CMO model to measure pharmacists’ interventions in real clinical practice.

Introduction: Tolvaptan is the first drug approved by the FDA for rapidly progressive polycystic kidney disease (PKD). However, there are no studies reporting real-world clinical practice using tolvaptan to treat Chinese patients with autosomal dominant polycystic kidney disease (ADPKD). Methods: This retrospective multicenter cohort study analyzed ADPKD patients diagnosed by genetic or imaging tests, comparing those treated with tolvaptan (15 mg, twice daily) to unmedicated patients. Clinical course, estimated glomerular filtration rate (eGFR), total kidney volume (TKV), Short-Form 36 (SF-36) score, and adverse events (AEs) were recorded for 83 patients, with 40 in the treatment group and 43 in the control group. Results: Compared with the control group, the tolvaptan treatment significantly delayed the increase in height-adjusted total kidney volume growth rate (htTKV%) in the 1st and 2nd years (1 year: difference: −5.58, 95% confidence interval [CI]: −8.39 to −2.78, p < 0.001; 2 years: difference: −9.75, 95% CI: −13.1 to −6.31, p < 0.001). Compared with the control group, the effect of tolvaptan on eGFR was more pronounced in the 2nd year of treatment (2 years: difference: 6.79, 95% CI: 5.10–8.48, p < 0.001). Tolvaptan attenuated the AEs, such as fatigue, back pain, and anxiety, to some extent, while the incidence of water-related AEs, such as thirst, polyuria, and urinary frequency, was significantly higher than that in the control group (p < 0.001, p = 0.006, p = 0.009, respectively). According to the SF-36 health survey, the physical component summary (PCS) score was significantly improved in the treatment group (p < 0.001). Conclusion: This real-world analysis showed that tolvaptan appears to be effective in delaying the clinical course of ADPKD patients in China, extending the results of previous clinical trials in other populations.

Time in Tight Range (TITR) is an emerging continuous glucose monitoring (CGM) metric assessing time spent in the 70-140 mg/dL range. While TITR is increasingly recognized for reflecting optimal glucose control, its psychological impact remains unexplored. This study assessed the relationship between TITR and psychological outcomes in adolescents with type 1 diabetes (T1D). This cross-sectional study included 123 adolescents with T1D. Participants completed two validated questionnaires: the PAID-Teen, which measures diabetes-related distress, and the PERMA model, which evaluates psychological well-being. CGM data were analyzed to determine participants' glucose metrics. Achieving TITR ≥ 50% was associated with higher distress scores (OR = 1.023; 95% CI 1.002-1.044; p = 0.029), while no such association was found with PERMA scores. Conversely, time in range (TIR) ≥ 70% showed no significant relationship with psychological outcomes. These findings suggest TITR may impose additional psychological burden beyond conventional glycemic targets. Further longitudinal studies are needed to evaluate its long-term impact on quality of life and optimize diabetes management strategies for youth with T1D.

Prescribing patterns and clinician preferences for direct oral anticoagulant use in unusual site venous thromboembolism: a cross-sectional analysis from the Direct oral anticoagulants in Unusual Site venous Thromboembolism (DUST) study.

Metastatic colorectal cancer (mCRC) is a difficult-to-treat disease with poor clinical outcomes. Systemic chemotherapy in combination with targeted anti-EGFR therapy has expanded the treatment options for mCRC. However, the therapeutic efficacy of anti-EGFR regimens and relevant prognostic factors vary according to age, performance status and tumor location. The non-interventional ERBITAG study was performed to evaluate the efficacy and safety of cetuximab in first-line therapy in RAS WT mCRC patients under routine clinical practice. ERBITAG is a non-interventional study of wild-type (WT) RAS mCRC patients initiating a first-line therapy with cetuximab from 2010 to 2018. Overall survival (OS) and progression-free survival (PFS) were analyzed using the Kaplan-Maier methods. χ²-test was used to compare selected categorial variables. Of a total of 728 patients included, baseline characteristics were: median 67 years, male sex 69%, ECOG performance status ≤ 1 81.3%, left-sided tumors 64.5%, liver metastasis 73.4% and prior hepatic metastasis resection before cetuximab-based treatment 16.4%. Median PFS was 10.9 months, median OS was 23.6 months and ORR was 58.0%. Resection rate of liver and/or lung metastases under cetuximab-based therapy was 13.9% and 18.9% of liver metastases. The most common treatment-emergent event (TEAE) was acne-like rash (all grades: 46.8%; grade 3–4: 4.7%). Subgroup analysis showed better outcomes in younger patients (ORR, OS), patients with left-sided tumors (ORR, PFS, OS) and lower grade tumors (ORR, PFS, OS), patients with resected liver and/or lung metastases (PFS, OS) and patients with treatment breaks (OS). Skin prophylaxis with systemic antibiotics and/or topical steroids (ORR, OS, PFS) was associated with better outcomes. The ERBITAG study provides insights into the use of cetuximab in a large RAS WT mCRC cohort in real-life clinical practice in Germany. Cetuximab in combination with first-line chemotherapy demonstrates clinical outcomes and safety data similar to results from the other pivotal randomized controlled trials. Study Number: EMR062202-515 (https://www.pei.de/SharedDocs/awb/nis-0101-0200/0114.html) Registered on 04-MAY-2010.