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tovorafenib

✓ Approved

Day One Biopharmaceuticals, Inc. · 辅助诊断 · 辅助诊断

什么是 tovorafenib？

tovorafenib 是一种辅助诊断，由Day One Biopharmaceuticals, Inc.研发。该药已获批，用于治疗相关适应症，给药途径：Unknown。

药物档案

公司Day One Biopharmaceuticals, Inc.
药物类别辅助诊断
给药途径Unknown
状态Approved
来源： ClinicalTrials.gov, Day One Biopharmaceuticals, Inc.

治疗适应症

tovorafenib 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Uterine cancer✓ Approved

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Individualized pharmacotherapy: background and development.

Jørgensen Jan Trøst JT, Westergaard Niels N

Most drug prescriptions are still based on empiricism and not on solid biological data, which often results in considerable patient variability and, sometimes, low patient benefits. Although variability in patient response to pharmacotherapy has long been recognized, only in recent decades have new molecule analytical methods provided insight into some of the causes, which are often related to somatic or germline genetic variations. Based on this insight, different predictive biomarker tests have been developed to optimize and individualize pharmacotherapy. These biomarker tests are classified as companion diagnostic (CDx) or pharmacogenetic (PGx) tests. In both the United States and Europe, CDx and PGx information is part of the regulatory drug labeling and is included in the Prescribing Information for the individual drugs and biologics. In the United States, this type of information is found in the labeling of more than 400 regulatory-approved drugs and biological products. Despite these measures and the documented clinical utility of CDx and PGx testing, clinical implementation is lagging, especially with regard to PGx. There are various reasons for the lack of testing, such as insufficient education and awareness among healthcare professionals, inadequate access to biomarker testing, regulatory hurdles, and insufficient reimbursements. Although progress has been made in recent years, further efforts are needed to fully realize the potential of individualized pharmacotherapy by integrating the use of predictive biomarkers into routine clinical practice.

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Diagnostic Dilemmas and The Benefit of Robotic-Assisted Surgery in the Management of Neurogenic Thoracic Outlet Syndrome in Complex Patients.

Federico Emma M EM, Pohlman Alexander A, Lozanoski Madison M, Abdelsattar Zaid M ZM et al.

A 64-year-old male patient presented with progressive, debilitating bilateral arm pain for 2 years following traumatic cervical spine injuries requiring multiple operations. Despite extensive diagnostic workup by several specialists, he was left without a diagnosis, treatment plan, or ability to perform his job. He was diagnosed with bilateral neurogenic thoracic outlet syndrome, based on clinical diagnostic criteria. A 2-staged bilateral robotic first rib resection resulted in complete resolution of his symptoms.

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PubMedEmergency radiology2026-09-10

Diagnostic yield of repeat CT pulmonary angiography after a negative examination in the emergency department.

Gorelik Natalia N, Fung Tiffany T, Sacher Eliana E, de Albuquerque Lorenna Mendonça LM et al.

Although repeat CT pulmonary angiography (CTPA) after a negative index examination in the emergency department (ED) is common, its diagnostic utility remains uncertain. This study aimed to determine the incidence and diagnostic yield of repeat CTPA after a negative index examination in the ED. This retrospective cohort study included adults undergoing CTPA across three EDs at our center from 2016 to 2017. CTPA results were classified as positive, negative, or other (equivocal or negative with reported limitations) based on radiology reports. Patients with a negative index CTPA were followed for two years to identify repeat examinations. Diagnostic yield of repeat CTPA at 1 month, 1 year, and 2 years was compared with that of the index examination using a likelihood ratio test for nested proportions. Among 3,112 patients (median age, 60.1 years; 59.1% women), 331 (10.6%) had a positive index CTPA. Of 2,412 patients with a negative index examination, 297 (12.3%) underwent repeat CTPA within 2 years, including 202 (8.4%) performed in our ED. The diagnostic yield of repeat CTPA performed in our ED was lower than that of the index examination: 3.8% (1/26; p = 0.20) at 1 month, 6.0% (9/150; p = 0.051) at 1 year, and 6.4% (13/202; p = 0.04) at 2 years. Repeat CTPA after a negative examination demonstrated a lower diagnostic yield than initial imaging, highlighting an opportunity for more selective use of repeat CTPA guided by clinical pretest probability.

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Guidelines for Genetic Testing of Peripheral Nerve Disorders.

Saporta Mario A MA

Inherited peripheral neuropathies (IPNs) comprise a clinically and genetically heterogeneous group of disorders affecting approximately 1 in 2500 individuals and represent one of the most common inherited neurologic diseases. The rapidly expanding identification of disease-causing genes and the widespread implementation of next-generation sequencing (NGS) have fundamentally transformed the diagnostic evaluation of these disorders. Contemporary molecular testing has substantially increased diagnostic yield, shortened the diagnostic delay, refined disease classification, and strengthened genotype-phenotype correlations. In the United States, NGS-based multigene panels have become the most cost-effective first-line molecular diagnostic approach for most patients with suspected inherited neuropathies, whereas phenotype-directed single-gene testing remains appropriate in selected clinical circumstances and in healthcare systems in which access to comprehensive sequencing is limited. Despite these advances, challenges continue to affect diagnostic accuracy, including interpretation of variants of uncertain significance, detection of copy number variants and repeat expansions, technical limitations associated with highly homologous genomic regions such as SORD, and variability in gene content and analytic performance among commercially available testing platforms. Accurate diagnosis therefore requires integration of clinical phenotype, electrodiagnostic findings, family history, and molecular data. Establishing a precise genetic diagnosis has become increasingly important because it improves prognostic accuracy, guides genetic counseling and cascade testing, identifies patients with treatable hereditary neuropathies such as transthyretin amyloidosis, and facilitates enrollment in gene-specific clinical trials and emerging precision therapies. An evidence-based, phenotype-driven approach that incorporates contemporary molecular technologies is essential to maximize diagnostic efficiency while recognizing the strengths and limitations of currently available genetic testing strategies.

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Anatomical level-dependent variation in ultrasonographic skin-to-epiglottis depth and its diagnostic performance for difficult laryngoscopy: A prospective observational study.

Savran Çağdaş Ç, Köner Özge Ö, Temür Sibel S, Türe Hatice H

Ultrasonographic skin-to-epiglottis depth (SED) has been proposed as a predictor of difficult laryngoscopy (DL). Most studies have evaluated transverse measurements at a single anatomical level, and the influence of parasagittal imaging and measurement level on diagnostic performance remains unclear. This study aimed to assess anatomical level-dependent variation in parasagittal SED and to compare its diagnostic accuracy for predicting DL. In this prospective observational study, 150 adult patients undergoing elective surgery under general anaesthesia were evaluated preoperatively. Parasagittal SED was measured at three anatomical landmarks: the upper hyoid border (HUB), lower hyoid border (HLB), and thyrohyoid membrane (THM). Difficult laryngoscopy was defined as Cormack-Lehane grade III-IV. Diagnostic performance was analysed using receiver operating characteristic analysis. DL occurred in 32 patients (21.3%). Parasagittal SED differed significantly across anatomical levels. HUB measurements demonstrated superior predictive performance [area under the curve (AUC) 0.86; 95% confidence interval 0.79-0.92] compared with HLB (AUC 0.75) and THM (AUC 0.65). The HUB/THM ratio showed similarly high discrimination (AUC 0.86). Conventional airway tests showed modest predictive values (AUC 0.62-0.72). Multivariable analysis identified HUB SED >24 mm, inter-incisor distance ≤4 cm, and upper lip bite test class II-III as independent predictors of DL. Parasagittal SED demonstrates anatomical level-dependent variability that affects diagnostic performance. Assessment at the upper hyoid border provides the highest discrimination and may improve ultrasound-based airway risk stratification.

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Diagnostic Accuracy of Simplified Prehospital Sepsis Screening Tools for Identifying Sepsis and Predicting 30-Day Mortality: A Retrospective Diagnostic Accuracy Study.

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Early recognition of sepsis in prehospital settings remains challenging because emergency medical services (EMS) clinicians must make rapid triage decisions using limited physiological information before hospital arrival. Several sepsis screening tools have been validated in emergency department settings; however, comparative evidence in prehospital populations remains limited. This study evaluated the diagnostic and prognostic performance of five simplified screening tools-mNEWS, qSOFA, mSIRS, mSOS, and MEWS-for identifying sepsis and predicting 30-day mortality in prehospital patients. This retrospective diagnostic accuracy study included 268 adult patients (≥18 years) transported by Vajira Emergency Medical Service, Bangkok, Thailand, to Vajira Hospital between January 2018 and October 2024. Prehospital clinical parameters recorded by paramedics and emergency nurse practitioners were analyzed against the final hospital diagnosis of sepsis and 30-day in-hospital mortality. Predefined thresholds (mSOS ≥4, MEWS ≥5, mNEWS ≥5, qSOFA ≥2, and mSIRS ≥2) were used for the primary analyses. Diagnostic performance was evaluated using the area under the receiver operating characteristic curve (AuROC), sensitivity, and specificity. Among 268 patients, MEWS demonstrated the highest discrimination for identifying sepsis (AuROC 0.84; sensitivity 64.9%; specificity 79.1%). mSOS (AuROC 0.83) and mSIRS (AuROC 0.82) demonstrated high sensitivity (>96%) but lower specificity (~60%), while mNEWS showed the highest sensitivity (99.3%) but poor specificity (47.0%). qSOFA demonstrated the lowest discrimination (AuROC 0.72). For predicting 30-day mortality, MEWS demonstrated the highest performance (AuROC 0.70; sensitivity 72.2%; specificity 64.5%), followed by mNEWS and mSOS (AuROC 0.69 each), whereas qSOFA (AuROC 0.64) and mSIRS (AuROC 0.60) demonstrated lower discrimination. MEWS demonstrated the most balanced diagnostic performance for identifying sepsis and predicting 30-day mortality in prehospital patients. Highly sensitive tools may be useful for early screening, whereas MEWS may better support risk stratification. Further external validation is required before broader implementation.

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