- Research Article
- 10.1016/j.esmorc.2026.100410
204P Sarcoma representation and outcomes in molecular pan-cancer basket trials: A systematic landscape analysis of clinicaltrials.gov (2010–2025)
- Mar 01, 2026
- ESMO Rare Cancers
- A Sargsyan + 13 more +13
Publications from 2021 to 2026
Showing 10 of 225 papers
204P Sarcoma representation and outcomes in molecular pan-cancer basket trials: A systematic landscape analysis of clinicaltrials.gov (2010–2025)
An Impact of Metacognitive Training and Cognitive-Behavioural Therapy on Quality of Life in Psychosis: A Systematic Review.
Although metacognitive training (MCT) and cognitive-behavioural therapy (CBT) have been proven to be effective interventions for the treatment of symptoms of schizophrenia, still little is known about the effects on quality of life (QoL). This systematic review compares the effectiveness of MCT and CBT-based interventions for QoL in individuals with schizophrenia spectrum disorders (SSD). Three electronic databases were screened up to October 2025 for MCT and CBT studies reporting QoL outcomes for patients diagnosed with SSD. Data from 38 randomized controlled trials (RCTs) met the inclusion criteria, of which eight MCT and 30 studies included CBT-based interventions. When comparing MCT against control interventions, none of the eight MCT studies found significant differences in the overall score of QoL scales at posttest and at 1- or 6-month follow-up. Only one study has assessed the effects of MCT after 3 years, yet it demonstrated significant improvements in QoL. For CBT-based interventions, nine of 26 available between-group comparisons in overall QoL showed a significant improvement in QoL after CBT at the posttest, and five of 19 effects were observed at follow-up. This review provides modest evidence for improvements in QoL from CBT-based interventions and a small number of studies concerning QoL in MCT, emphasizing the need for further research that prioritizes QoL as a primary outcome as well as the need for research with longer follow-ups to capture the delayed effects of MCT and CBT on QoL. It awaits to be tested whether the recent extension of MCT (app, 2 modules on self-esteem and stigma) augments QoL.
Read morePO:03:076 Systemic lupus erythematosus and statins in GLADEL 2.0: are cardiovascular risk prevention guidelines being followed?
Plasma ATN biomarkers across the alzheimer’s disease continuum in a Chilean community- and clinic-based cohort
Plasma biomarkers have emerged as robust indicators of Alzheimer's disease (AD) pathology, offering accessible tools for staging and stratification. However, their expression across globally diverse populations remains poorly characterized. The aim of this study was to evaluate whether the combination of plasma biomarkers could distinguish different stages along the AD continuum and assess their clinical associations in a Latin American cohort. We evaluated plasma amyloid, tau, and neurodegeneration (ATN) biomarkers in 318 older adults from a Chilean community- and clinic-based cohort, including individuals with subjective cognitive complaints (SCC), mild cognitive impairment (MCI), and Alzheimer's disease dementia (ADD), alongside cognitively unimpaired (CU) participants. Plasma ATN biomarkers (Aβ42/Aβ40, p-tau217, NfL, and GFAP) were quantified using Simoa technology. Global cognition was assessed with the Addenbrooke's Cognitive Examination (ACE), memory with the Free and Cued Selective Reminding Test (FCSRT), and functional ability with the Technology-Activities of Daily Living Questionnaire (T-ADLQ). Group differences in plasma biomarkers were examined using ANCOVA models adjusted for age, sex, and education, and associations with cognitive performance were evaluated through linear regression analyses. In addition, supervised machine-learning models were implemented to classify participants across diagnostic categories based on plasma biomarker profiles, using cross-validation to evaluate predictive performance. We observed a progressive decline in the Aβ42/Aβ40 ratio and elevations in p-tau217 and GFAP across the clinical continuum. Additionally, p-tau217 and NfL levels were inversely associated with cognitive, memory, and functional performance. Notably, p-tau217 distinguished ADD from CU with high accuracy (AUC = 0.88), although its performance in earlier stages was limited. These findings support the biological consistency of plasma biomarkers in AD-related neurodegeneration and provide novel evidence from a Latin American population. Further studies are needed to improve early-stage detection and to better understand how genetic, environmental, and health factors shape biomarker expressions in underrepresented regions.
Read moreOne-Stop Transcatheter Pulmonary and Tricuspid Valve Replacement for Carcinoid Heart Disease Treatment.
Carcinoid heart disease is a complex consequence of functional neuroendocrine tumors, characterized by progressive thickening and degeneration of the right-sided valvular apparatus, leading to valvular and heart failure. Management is challenging, requiring multidisciplinary considerations regarding tumor disease management, heart failure pharmacotherapy, and heart valve replacement in individuals with acceptable performance status. We present a case of a middle-aged man with a history of metastatic neuroendocrine tumor receiving biweekly octreotide therapy. He presented with constitutional syndrome and right-sided heart failure signs. Cardiac imaging revealed significant tricuspid and pulmonary regurgitation. The patient was a poor surgical candidate; consequently, after multimodal cardiac imaging assessment, alternative 1-stop transcatheter pulmonary and tricuspid replacement was planned and performed. Patients with symptomatic carcinoid heart disease require comprehensive multidisciplinary evaluation. For high-risk patients with multivalvular disease, transcatheter valve replacements provide a feasible and less invasive alternative to standard surgical replacement. One-stop transcatheter valvular interventions are a feasible therapeutic alternative to surgical replacement in patients with symptomatic carcinoid heart disease and suitable anatomy. Multimodal cardiac imaging is crucial for preprocedural assessment and the performance of transcatheter right-sided heart valve interventions.
Read morePERMEPSY: a multicentre, randomized, double-blind proof-of-concept trial of personalized metacognitive training for adults with psychosis — a study protocol
BackgroundWhile psychological interventions are effective at improving symptoms of psychosis, accessible, cost- and time-efficient treatments remain limited. Personalized medicine has emerged as a promising approach, tailoring interventions to individual needs. Metacognitive Training (MCT), with its established efficacy and adaptable format, is well-suited for personalization. The PERMEPSY project (Towards a Personalized Medicine Approach to Psychological Treatment for Psychosis) aims to deliver tailored MCT intervention for individuals with psychosis.MethodsPERMEPSY is an international study funded by ERAPerMed (JTC2022) involving five clinical partners (Spain, Chile, France, Germany, Poland) and one technological partner (Spain). The project involves a proof-of-concept clinical trial recruiting 51 participants from each center for a total of 255 adult participants with psychosis in a prospective study (Registration: NCT06603922, 19-09-2024). The trial will test the efficacy of a Machine Learning (ML)-derived platform at predicting clinical and functional outcomes from baseline scores and compare a personalized MCT (P-MCT) to a classical MCT based on the platform’s predictions.AimsPERMEPSY seeks to (1) develop and test the predictive power of an algorithm that could support decision-making, and (2) ascertain whether P-MCT is more effective than MCT at improving key symptoms and cognitive impairments associated to psychosis.ResultsA harmonized retrospective database enabled the development of a predictive ML algorithm, integrated into an innovative platform. This platform provides clinicians with the information needed to deliver P-MCT. Predictions include changes in positive symptoms (e.g., delusions), insight, self-esteem, and treatment adherence.DiscussionBy integrating diverse data types and innovative technology, PERMEPSY addresses the need for personalized, effective treatment in psychosis, aiming to reduce individual and systemic burdens while supporting clinicians in their decision-making.
Read moreAssessing Functional Ability in Cognitive Aging: Challenges and Sociocultural Factors
BackgroundFunctional ability is a key indicator of cognitive health, distinguishing cognitively unimpaired (CU) aging, mild cognitive impairment (MCI), and dementia. However, sociocultural factors such as gender roles, education, and age‐related differences influence activity participation, complicating assessments. The Activities of Daily Living Questionnaire‐Technologies (T‐ADLQ) evaluates three domains of functional ability: instrumental activities of daily living (iADL), basic activities of daily living (bADL), and advanced activities of daily living (aADL). It includes a response option, “Never did this activity/Don't know” (ND/DK), which may reflect disparities unrelated to cognitive impairment.MethodThis study analyzed ND/DK responses in the GERO cohort, a prospective study in Santiago, Chile, following 291 dementia‐free older adults with cognitive complaints (≥70 years) for three years to assess cognitive, functional, psychosocial, and medical factors. We examined how gender, education, and age influenced functional ability assessments using a test for equality of proportions to compare ND/DK response distributions.ResultMen were significantly more likely to report never engaging in household tasks such as taking pills (4.1% vs. 0.5%, p = 0.0049), cooking (24.5% vs. 1.0%, p <0.001), housekeeping (10.2% vs. 1.0%, p = 0.0004), and laundry (53.1% vs. 1.9%, p <0.001). Conversely, women had higher ND/DK responses in home repairs (42.0% vs. 19.1%, p = 0.0029), employment (23.2% vs. 6.1%, p = 0.0071), driving (72.5% vs. 20.4%, p <0.001), and ATM usage (58.0% vs. 34.7%, p = 0.0033). Participants with <6 years of education had significantly higher ND/DK responses in financial management (9.4% vs. 3.0%, p = 0.0393), driving (83.0% vs. 56.9%, p = 0.0003), and technology use (computer access: 84.9% vs. 61.4%, p = 0.0013; email access: 96.2% vs. 68.8%, p <0.001). Individuals aged ≥80 showed higher ND/DK rates in home maintenance (7.7% vs. 1.6%, p = 0.0147), mobile phone use (33.9% vs. 13.7%, p = 0.0003), and internet access (78.5% vs. 57.4%, p = 0.0024).ConclusionSociocultural factors strongly influence functional ability assessments, with notable disparities by gender, education, and age. These findings highlight the need to interpret ND/DK responses carefully to prevent misclassification of functional impairment in cognitive aging evaluations. Additionally, improving assessment tools to account for sociocultural variations is essential for more accurate evaluations.
Read moreA machine learning approach for a multidimensional analysis of functional phenotype allows for a better measurement of dementia and its severity
BackgroundFunctional impairment (FI) is a key component in diagnosing and monitoring the severity of neurodegenerative dementia (ND). It manifests in several dimensions, such as everyday cognition, mobility, social functioning, and activities of daily living (ADLs). However, traditional assessments have primarily focused on ADLs, often neglecting the other dimensions of functional phenotype.This study aims to assess the utility of the Chilean multidimensional functional assessment (Ch‐MFA, see Figure 1), including ADLs, social behavior, mobility, and everyday cognition in differentiating Alzheimer's disease (AD) and frontotemporal dementia (FTD), and to predict their severity measured in the Clinical Dementia Rating (CDR).Method308 patients were recruited in Peru, comprising 209 controls, 44 FTD, and 55 AD. Functional assessments include ADL (T‐ADLQ, DAD‐E), social behavior Blessed Dementia Rating Scale (BDR), mobility (WHODAS), everyday cognition (ECog), and CDR for severity. Data was normalized for each assessment, and groups were stratified regarding age and educational level, obtaining 72 controls, 24 FTD, and 45 AD for the predictive analysis. Random Forest (RF) models were trained using 70% of the data, while 30% was reserved for testing the prediction. Cross‐validation (20‐fold) and feature selection were used to reduce overfitting and identify a simpler set of questions.ResultThe Ch‐MFA indicates a 0.81 ± 0.03 average f1‐score when differentiating the group (control, FTD, and AD), which is 0.12 ± 0.10 better than the best‐performing unidimensional assessment (T‐ADLQ) with only 14 questions (4 T‐ADLQ, 2 BDR, 4 ECog and 3 WHODAS). As for the severity, Ch‐MFA shows a 0.65 ± 0.17 average f1‐score when differentiating severities of 0.5, 1, and 2, increasing to 0.88 ± 0.06 when it's only between 1 and 2 with a total of 4 questions (2 T‐ADLQ, 2 ECog). See Figure 2.ConclusionOur results show that the Ch‐MFA achieves better results than a unidimensional assessment. Interestingly, functional dimensions that distinguish types of dementia differed from those that predict their severity. Our results suggest that a multidimensional phenotype may better reflect the specificity of dementia subtypes.
Read moreTranscultural memory markers for Alzheimer's Disease: Evidence from the ReDLat project
BackgroundAlzheimer's disease dementia (ADD) is diagnosed based on evidence of significant cognitive decline from a previous level of performance in one or more cognitive domains (DSM‐5). Jack Jr et al. (2024) acknowledged that clinical judgment remains crucial when biomarkers are discordant with clinical impressions or non‐accessible. However, available assessments lack precision in detecting ADD and are influenced by sociocultural factors, thus calling for cross‐cultural harmonization strategies (Franzen et al., 2020). The Visual Short‐Term Memory Binding Test (VSTMBT) has shown cross‐cultural validity across six European countries (Costa et al., 2017; Parra et al., 2019). We investigated whether this and other promising tests are valid across six Latin American Countries.MethodsADD patients (n = 426) and Healthy Controls (n = 869) were recruited from Argentina, Brazil, Chile, Colombia, México and Perú. They were assessed using a comprehensive protocol (Ibanez et al., 2021), which included the VSTMBT, the Craft Story 21 Test (CS21T) and the Benson Complex Figure Test (CBFT). We used General Linear Models (GLM) to compare data across Groups and Countries controlling for confounders. We explored the classification accuracy of these tests using ROC analysis.ResultsWhole‐sample VSTMBT data entered a mixed GLM revealing the traditionally reported Group (ADD vs HC) by condition interaction (F(1,1287) = 4.62, p = 0.032), driven by disproportional Shape‐Color Binding deficits. When this test, the CS21T and CBFT immediate recall data entered GLMs with Group and Country as fixed factors and age, sex, education, number of languages, and comorbidities as covariates, the main effects and the interaction proved significant. The three tests revealed significant deficits in ADD relative to HC (all p < 0.05). ROC analysis showed a reliable classification accuracy for a memory composite that combined the three tests (AUC: Argentina=1.0, Brazil=0.99, Chile=0.96, Colombia=0.91), México=0.91, and Perú=0.78).ConclusionsThe VSTMBT, CS21T and CBFT proved reliable for assessing ADD across six LAC. Although variability was observed across countries, they revealed significant deficits in ADD, which were unaccounted for by relevant confounding factors. The composite score achieved excellent classification accuracy for most countries. The cross‐cultural validity of the VSTMBT previously reported across EU countries holds for LAC.
Read moreDigits Across Languages: The Role of Lexical and Numerical Characteristics in Digit Span Performance Across Fourteen Languages
BackgroundThe digit span task, a measure of auditory verbal short‐term and working memory, is widely used globally. Emerging research has revealed variations in digit span performance across languages among young adults; however, studies focusing on older populations are scarce and typically involving limited languages. This study investigates digit span performance among older adults (40‐90 year‐old) across fourteen languages and explored the influence of lexical and numerical properties on cognitive assessment.MethodWe examined digit span performance among cognitively normal participants (CN), individuals with mild cognitive impairment (MCI) and Alzheimer's disease (AD) across fourteen language cohorts totalling 3,681 participants: English (n = 446), Mandarin (n = 97), Cantonese (n = 65), Spanish (n = 218), Kannada (n = 69), Hindi (n = 72), Telugu (n = 69), Malayalam (n = 70), Bengali (n = 70), French (n = 299), Korean (n = 1098), Italian (n = 540), Arabic (n = 50), and Turkish (n = 518). First, we analyzed language differences in digit span performance among CN using ANOVA and general linear models. We then conducted Receiver operating characteristic (ROC) analyses to identify the optimal cutoff values for AD. Next, we computed the digit count, syllable count, and numerical magnitude (i.e. the average sum of the digits) of all digit stimuli in the English cohort and analyzed their effects via linear and ridge regression analyses.ResultThe forward (FDS) and backward digit span (BDS) tests revealed significant differences among CN across the fourteen language cohorts even after adjusting for age and education (FDS:F=38.62, p <0.001; BDS:F=19.23, p <0.001). ROC analysis revealed varying optimal cutoff values across languages: English (FDS:6, BDS:4), Italian and Turkish (FDS:5, BDS:4), Mandarin (FDS:7, BDS:5), Cantonese (FDS:7, BDS:4), and French (FDS:6, BDS:3). Further analysis indicated that the interaction between digit and syllable counts significantly impacted FDS accuracy in English speakers (linear:p=0.00035; ridge:p< 0.000001), with no significant effect from digit count alone after adjusting for interaction. Conversely, BDS performance showed a significant negative influence from digit count (p = 0.00858), with numerical magnitude and syllable count nearing significance (p = 0.083 and p = 0.066, respectively).ConclusionVariations in digit span performance across languages illustrate the role of linguistic and numerical factors in cognitive assessments, even with tests targeting non‐language domains using digit stimuli. These findings underscore the critical value of language diversity in cognitive research.
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