莫菲特癌症中心

Background: Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are an indolent subset of neuroendocrine neoplasms with a propensity to metastasize. In the case of oligoprogression, locoregional therapies are considered but may not be suitable for all patients. Stereotactic body radiotherapy, including magnetic resonance-guided radiotherapy (MRgRT), has emerged as an option to optimize local control and mitigate toxicity. Objective: This study aims to evaluate the efficacy and safety of MRgRT for treating patients with GEP-NETs, focusing on local control and treatment-related toxicity. Methods: A retrospective review was conducted on 25 patients diagnosed with abdominal neuroendocrine neoplasms and treated with MRgRT from 2019 to 2023. After excluding patients with neuroendocrine carcinomas and other noneligible conditions, 20 patients with well-differentiated GEP-NETs were included in the final analysis. Primary endpoints were local control and radiation toxicity; secondary endpoints were progression-free survival and overall survival calculated from the date of MRgRT. Results: The study cohort had a median follow-up of 44.5 months. MRgRT was well-tolerated, with only two (10%) patients experiencing acute grade 3 treatment-related toxicity. Local control was achieved among 95% of patients, with a median progression-free survival of 30 months. No significant variables were associated with progression-free survival or toxicity in the univariate analysis. Conclusion: Magnetic resonance-guided radiotherapy is a well-tolerated treatment that offers high rates of local control for patients with GEP-NETs, including those with metastatic disease. This treatment should be considered a viable alternative for patients who are not candidates for other locoregional therapies.

Endoscopic management offers acceptable oncologic control in select patients with upper tract urothelial carcinoma (UTUC) while preserving renal function. Adjuvant intracavitary treatment with chemotherapy or Bacillus Calmette-Guérin (BCG) has been proposed to reduce recurrence risk. We aimed to evaluate the impact of adjuvant intracavitary treatment on ipsilateral UTUC recurrence following endoscopic management. We queried a multi-institutional cohort of patients who underwent endoscopic management for UTUC. Treatment groups were defined as no instillation, single post-operative instillation, or multiple instillations. Ipsilateral UTUC recurrence-free survival (RFS) was estimated using Kaplan-Meier curves and Cox proportional hazards models evaluated factors associated with recurrence. A total of 599 renal units, of which 43 received single instillation and 86 multiple instillations, in 334 patients treated endoscopically for UTUC were analyzed. The median follow-up time for patients without recurrence was 12 months (IQR 4–33). Multiple adjuvant instillations of any intracavitary treatment were associated with a significantly improved RFS (HR 0.52, 95

Complete thyroid ultrasound reporting allows appropriate risk stratification and management of patients with thyroid nodules. Our objective was to assess the completeness of thyroid ultrasound reports based on documentation of key nodule features recommended by clinical guidelines. We conducted a retrospective review of patients who underwent thyroid ultrasound between 2013 and 2020, including those with at least one nodule larger than 1 cm. Using a rule-based natural language processing (NLP) pipeline, key thyroid nodule features were extracted from the reports. At the nodule level reports were classified as complete if all guideline-recommended characteristics were documented. Out of 8,855 thyroid ultrasound reports reviewed, 5,792 (65

PURPOSE:The continuous development of new imaging approaches, molecular phenotyping, genetic subtypes, prognosis assessments, and effective therapies across a range of disease states has created a need to redefine terminology and best practices for clinical trial conduct in patients with advanced prostate cancer. METHODS:We convened an international expert committee of diverse working groups, the Prostate Cancer Working Group 4 (PCWG4), between 2016 and 2025. Our objective was to formulate updated criteria based on emerging evidence and clinical trial data in a biomarker context to provide guidance for clinical trial design, eligibility, and end point assessments for patients with advanced prostate cancer. RESULTS:PCWG4 redefines terminology around the disease state and previous therapies in a patient-centric context and terminology focused on androgen pathway modulation. We consider imaging, with a particular focus on positron emission tomography (PET)-defined disease. New recommendations are provided for disease state terminology, defining eligibility criteria, response and delay/prevent end points, intervals for reassessments including imaging, and patient-reported outcome determination. We provide recommendations in a biomarker-based context of use for the intended indication, reflective of patient benefit for specific interventions. We emphasize the need for development of validated PET imaging and molecular and phenotypic criteria as well as trial designs to appropriately risk stratify patients, predict and assess benefit, and measure post-treatment outcomes reliably in a trial framework. CONCLUSION:PCWG4 updates recommendations on patient and tumor characterization, therapy development, and imaging criteria and extends guidance into earlier androgen pathway modulator-naïve/sensitive disease states to reflect an evolving, heterogeneous, and diverse patient population to optimize treatment benefits for all patients.

BACKGROUND:Methotrexate-based chemoradiotherapy is effective in primary central nervous system lymphoma (PCNSL) but carries a risk of severe neurotoxicity. In a single-arm study, a regimen with methotrexate, procarbazine, vincristine, and cytarabine was combined with rituximab (R-MPV-A) and substantially reduced doses of whole-brain radiotherapy (LD-WBRT), resulting in excellent progression-free survival (PFS) and overall survival (OS). Because R-MPV-A had never been tested without radiation, we sought to evaluate the efficacy of R-MPV-A with and without LD-WBRT, as well as determining if such low radiotherapy doses influenced disease control and/or neurotoxicity. METHODS:Patients were randomized to receive R-MPV-A alone (Chemo arm) or combined with LD-WBRT (ChemoRT arm), given at 2,340 cGy (180cGy X13). Primary endpoint was intent-to-treat (ITT) PFS. A sample size of 89 would provide 80% power to detect a hazard ratio (HR) of 0.63 (one-sided alpha = 0.15). RESULTS:Ninety-one patients were randomized, with 44 analyzed in the ChemoRT and 46 in the Chemo arm. Median age was 66 and 59.5, respectively. R-MPV-A was well tolerated, achieving a complete response rate of 92.3% (ChemoRT) and 76.3% (Chemo). After median follow-up of 4.6 years, median PFS was not reached (ChemoRT) vs 2.1 years (Chemo), HR = 0.47 (P = .007; 95% CI, 0.26-0.87). The 2-year PFS was 78.7% vs 54%, respectively. Differences in OS did not reach statistical significance (HR = 0.71; P = .33). Neuropsychological evaluation showed no differences in cognitive outcomes, with several tests favoring ChemoRT. CONCLUSIONS:R-MPV-A is a highly efficacious and safe regimen with or without LD-WBRT. LD-WBRT contributes to disease control and increases PFS in PCNSL.