Oncologist最新文献

Background: Sacituzumab govitecan (SG), an antibody-drug conjugate targeting TROP-2, demonstrated superior efficacy over standard chemotherapy in heavily pretreated metastatic triple-negative breast cancer (mTNBC) in the ASCENT trial. However, real-world data remain limited. This study evaluated the effectiveness and safety of SG in an unselected multinational cohort of patients with mTNBC.

Methods: This retrospective analysis included 303 women who initiated SG treatment between August 2021 and April 2025 across 18 oncology centers in Poland, the Czech Republic and Slovakia, within the Central European Breast Cancer Collaboration (CEBCC-102). Primary endpoints were median progression-free survival (mPFS) and overall survival (mOS). Secondary objectives included response pattern, safety and identification of factors influencing outcomes. Adverse events (AEs) were graded using Common Terminology Criteria for AE, version 5.0 and treatment response was assessed according to Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1.

Results: The median follow-up was 8.8 months (IQR 4.5-14.4). The mPFS was 4.4 months, and mOS was 11.3 months. The overall response rate was 30.7%. The most frequent AEs were hematologic: neutropenia (69.0%) and anemia (39.6%). In multivariate Cox analyses, poor ECOG performance status and liver metastases were independently associated with worse outcomes. Diarrhea and hypersensitivity reactions showed favorable prognostic associations.

Conclusions: In this largest real-world cohort, the clinical benefit of SG observed in the ASCENT trial was confirmed under routine practice conditions. Poor performance status and liver metastases predicted inferior outcomes, while certain treatment-related AEs warrant further investigation.

Background: Abemaciclib is approved for hormone-receptor positive (HR+), human epidermal growth factor receptor-2 negative (HER2-) locally advanced/metastatic breast cancer (ABC) and high-risk early BC (EBC) in China. This prospective observational study describes real-world abemaciclib safety and effectiveness among Chinese patients with HR+/HER2- EBC/ABC.

Methods: Adults with HR+/HER2- EBC/ABC who received abemaciclib between Mar-2022-Jan-2024 across 32 Chinese centers were enrolled. Primary objective was to evaluate treatment-emergent adverse event (TEAE) and serious AE (SAE) incidence within 24 weeks of treatment. Secondary objective was to describe Week 24 event-free survival (EFS).

Results: Among 387 patients with EBC and 539 with ABC (median age: 50.0 and 55.0 years, respectively), 89.1% and 82.4% received 150 mg twice daily initially. Abemaciclib was combined with aromatase inhibitors in 95.6% (EBC) and 60.5% (ABC) patients, and fulvestrant in 38.0% (ABC). TEAEs occurred in 85.5% (EBC) and 81.8% (ABC) patients (commonly diarrhea, neutrophil count decreased, and white blood cell count decreased), and SAEs in 3.9% and 7.4%. AEs led to discontinuation in 4.4% (EBC) and 7.2% (ABC) patients. Diarrhea was the most common AE leading to discontinuation (2.1% [EBC] and 1.7% [ABC]). Most patients who discontinued treatment due to AEs had no dose modification, while most with dose reduction/interruption remained on abemaciclib. Week 24 EFS rates (95% CI) were 99.7% (97.6-100.0; EBC) and 85.1% (81.3-88.2; ABC).

Conclusions: Real-world safety, tolerability, and effectiveness of abemaciclib in Chinese patients with HR+/HER2- EBC and ABC were consistent with clinical trials, with no new safety signals, supporting its positive real-world benefit-risk profile.

Background: Neuroendocrine neoplasms (NEN) are a heterogeneous disease and chemotherapy (CT) represents the standard first-line treatment for those with a Ki-67 index >20%.

Methods: MAVERIC is a randomized, multicenter, non-comparative phase II study including patients with metastatic gastroenteropancreatic (GEP-NEN) or large-cell neuroendocrine carcinoma (LCNEC) (Ki-67 20%-55%) according to the 2010 WHO grading system and at least stable disease after first-line CT. Patients were randomized (2:1) to everolimus 10 mg/day or observation until progression or treatment intolerance. The primary endpoint was progression-free survival (PFS); secondary endpoints included overall survival (OS) and safety.

Results: Between November 2015 and June 2022, 30 patients were enrolled across five Italian centers, with 20 assigned to everolimus and 10 to observation. The analysis included 29 patients (52% GEP-NEN, 48% LCNEC). Median (m)PFS was 11.8 months in the everolimus arm and 1.8 months in the control arm. MOS was similar between arms (38.3 and 38.2 months). The subgroup of 11 patients with GEP-neuroendocrine tumour (NET) grade 3 treated with everolimus showed a mPFS of 19.9 months and mOS of 48.1 months. Most common adverse events (AEs) were mucositis (80%), dyslipidemia (55%), fatigue (45%), pneumonitis (40%), and peripheral edema (35%). Grade 3 AEs occurred in 70% of patients; no grade 4 AEs were observed.

Conclusions: The MAVERIC trial demonstrated encouraging clinical benefit of everolimus in metastatic GEP-NEN and LCNEC (Ki-67 20%-55%) following first-line CT. Toxicity was consistent with the known safety profile of everolimus. This strategy was particularly effective in GEP-NEN patients and warrants further investigation.

Background: We assessed the clinical relevance of age and sex as risk factors for health-related quality of life (HRQoL) in patients with adult-type diffuse glioma.

Materials and methods: The CODAGLIO 2.0 database contains 16 randomized trials from 5,369 patients with glioma. Patients' HRQoL was assessed using EORTC QLQ-C30 and QLQ-BN20 questionnaires. In 8 HRQoL scales, we compared mean HRQoL at baseline with the general population and evaluated factors associated with HRQoL over time using linear mixed models (LMMs). We used anchor-based minimally important difference to interpret clinically relevant changes.

Results: We included 4,301 patients with baseline HRQoL followed until 3 months. Compared to the general population, patients with glioma at baseline had statistically and clinically relevant worse HRQoL, which was still evident after stratifying by age and sex groups. In LMMs, compared to patients aged ≤60 years, those >60 years had statistically significant associations with worse physical functioning: -2.40 (95% confidence interval (CI) -4.14 to -0.71), better social: 4.88 (2.68 to 7.30) and role: 3.79 (1.39 to 6.16) functioning, and less fatigue: -3.43 (-5.44 to -1.33) and pain: -4.56 (-6.18 to -2.93). Compared to men, women had statistically significant associations with worse physical and social functioning and more fatigue and pain. Associations between age, sex, and HRQoL were not clinically relevant. Performance status had clinically relevant associations in 5/8 scales.

Conclusion: Patients with glioma have clinically relevant worse HRQoL compared to the general population. There are statistically but not clinically significant associations between age, sex, and certain HRQoL scales.

Background: Implementing structured shared decision-making (SDM) requires high-quality, reliable patient information. In radiation oncology, patients often have limited knowledge and misconceptions about therapy and side effects, affecting their decision-making. Large language model-based AI systems (LLMs) may help by providing evidence-based information in accessible language, but successful implementation depends on the willingness of patients and health care professionals (HCPs) to adopt these technologies.

Methods: A survey was conducted among patients undergoing radiation therapy and HCPs between 03/2024 and 02/2025. Data was collected using structured electronic questionnaires (32 items for patients, 35 for HCPs). The survey assessed sociodemographic characteristics, the status of SDM in oncology, sources of information relevant to SDM, and current and anticipated LLM applications. Data were analyzed using descriptive statistics and logistic regression analysis.

Results: The internet was the prime information source for patients (n = 400). Regarding current use of LLMs, a large discrepancy between patients and HCPs (n = 200) was observed (18.2% vs 69.5%). Although 77% of HCPs believed that patients will rely on LLMs in the future, only 29.1% of patients agreed. Most patients (65.8%) stated that even as LLMs improve, they will continue to trust physicians more; 46% of HCPs shared this view. Only 16.5% of patients were convinced that LLMs provide all relevant data for SDM in cancer care. Familiarity with technology was the strongest predictor of LLM use among patients.

Conclusion: Only a minority of radiation oncology patients currently use LLMs, and many remain skeptical about their future role-contrasting with the more optimistic expectations of HCPs.

Importance: Reliable prognostic markers for immune checkpoint inhibitor (ICI) response in metastatic renal cell carcinoma (mRCC) remain limited.

Objective: To examine the impact of splenic volume change after ICI initiation on progression-free survival (PFS) and overall survival (OS) in patients with mRCC.

Design: A retrospective cohort study reviewing data from 2015 to 2023.

Setting: The Emory Kidney Cancer database (single-center academic instution).

Participants: Patients with mRCC who underwent first-line ICI treatment and had available abdominal imaging 30 days before and 60-120 days after ICI initiation. A total of 109 patients met inclusion criteria.

Exposure: Splenic volume change calculated as a percentage difference between baseline and follow-up imaging (median 2.8 months post-initiation) using a standardized formula, grouped into ≥10% increase and <10% increase.

Main outcomes and measures: Differences in OS and PFS assessed using Kaplan-Meier curves and multivariable Cox hazards regression models.

Results: A total of 109 patients met inclusion criteria. Median follow-up time was 25.2 months (IQR 11.2-41.5), during which there were 47 mortality events. Patients with a splenic volume increase ≥ 10% at a median 2.8 months after ICI initiation had worse 2-year PFS (28.5% vs 50.4%, P = .022) but not OS (69.4% vs 77.8%, P = .853) compared to patients with a < 10% increase in splenic volume. On multivariable analysis, a splenic volume increase ≥ 10% was independently associated with worse PFS (2.33 [95% CI 1.37-3.96], P = .002).

Conclusions and relevance: In patients with mRCC, a splenic volume increase ≥ 10% at a median of 2.8 months following ICI initiation is independently associated with worse survival compared to an < 10% increase. Monitoring splenic volume changes may serve as a cost-effective radiographic prognostic marker to guide treatment sequencing.

Bladder cancer (BCa), marked by clinical heterogeneity and late diagnosis, remains a global health challenge. The limitations of conventional diagnostics have spurred the advancement of liquid biopsy approaches, which offer minimally invasive tools for early detection, prognosis, and therapeutic monitoring. This review highlights key components of liquid biopsy in BCa, including circulating tumor cells (CTCs), circulating tumor DNA (ctDNA), exosomes, and metabolomics-especially urinary volatile organic compounds (VOCs). Each modality contributes distinct insights into tumor biology: CTCs and ctDNA provide information on tumor genetics and dynamics; exosomes reflect microenvironmental signaling and lipid metabolism; and urinary VOC profiling enables metabolic characterization and early-stage discrimination. We explore how these dimensions complement each other in tracking disease progression, predicting recurrence, and guiding personalized therapy. Emphasis is placed on recent technological advances, clinical utility, and future integration into practice. This multidimensional perspective underscores the transformative potential of liquid biopsy in improving BCa outcomes.

Context: With new treatment strategies approved in metastatic hormone-sensitive prostate cancer (mHSPC), heterogeneity across trials hinders the physicians' choice for first-line treatment.

Objective: We conducted a systematic review and network meta-analysis to assess the efficacy of currently approved treatments for mHSPC stratifying patients according to their disease burden (high- vs. low-volume as per CHAARTED criteria) and onset of metastatic disease (synchronous vs. metachronous).

Intervention: Eleven randomized controlled trials (RCTs) published until October 30, 2024 were included. Treatment regimens were grouped as triplets for combinations of docetaxel, androgen receptor pathway inhibitors (ARPIs) and androgen-deprivation therapy (ADT), separate doublets for docetaxel plus ADT, ARPI plus ADT, or monotherapy for ADT alone.

Outcome measurements and statistical analysis: Overall survival (OS) and radiographic progression-free survival (rPFS) outcomes were collected. OS as primary endpoint, and rPFS as secondary endpoint, were analyzed separately in high- and low-volume patients. Additional subgroup analyses accounted for timing of metastases categorized as high-volume/synchronous, high-volume/metachronous, low-volume/synchronous, and low-volume/metachronous disease.

Evidence synthesis: Triplet combinations prolonged significantly OS and rPFS in high-volume disease (P-score 0.99), and high-volume/synchronous disease (P-score 0.99). ARPI/ADT doublets performed best in low-volume patients (P-score 0.94), and low-volume/metachronous (P-score 0.99). In the high-volume/metachronous population, triplets, and doublets were equally effective.

Conclusions: The results provide collective evidence for treatment selection based on disease volume and timing of metastasis with strongest survival benefits of triplets for high-volume/synchronous mHSPC patients and of ARPI doublets for low-volume disease.

Ampullary carcinoma (AC) is a rare malignancy. It is often classified within biliary tract cancers (BTC) but lacks dedicated treatment guidelines. This post-hoc analysis evaluated outcomes of patients with advanced AC enrolled in the ABC-01, ABC-02, and ABC-03 clinical trials to provide reference data for future studies. All patients with advanced AC formed the "Descriptive cohort," while those AC treated with cisplatin-gemcitabine (CisGem) comprised the "CisGem-treated cohort." Among 534 trial participants, 28 (5.24%) had AC, and 17 received CisGem. The median age was 63.93 years, and 75.00% were male. Most patients had metastatic disease at baseline (89.29%). Median follow-up for the CisGem-treated cohort was 10.23 months (95% CI 5.98-14.43). The objective response rate was 23.52%, and disease control was achieved in 58.82% of patients. Estimated median progression-free survival (PFS) and overall survival (OS) were 7.98 months (95% CI, 6.86-8.44) and 11.76 months (95% CI, 5.94-14.88), respectively, comparable to outcomes in other BTCs. No reliable prognostic or predictive factors for PFS, OS, or ORR were identified, likely reflecting the small sample size. This analysis underscores the rarity of advanced AC and the challenges in recruiting adequate numbers for dedicated trials. While CisGem remains an appropriate standard-of-care regimen, modest survival outcomes highlight the need for improved therapies. Molecular profiling has revealed potentially actionable alterations, including HER2 amplification and KRAS mutations, supporting precision oncology approaches. This study provides the most comprehensive reference dataset to date for advanced AC treated with CisGem and emphasizes the importance of international collaboration and molecularly guided research to improve outcomes in this rare malignancy.