Oncologist [JOURNAL]
Sun
200 papers
The association between financial toxicity and mortality in hematologic malignancies: a systematic review and meta-analysis.
Liao F, Xu QX, Lin P … +2 more , Feng JQ, Chen D
Oncologist · 2026 Jun · PMID 42097298 · Full text
BACKGROUND: Hematologic malignancies impose substantial economic burdens, but the quantitative association between financial toxicity and overall survival remains poorly defined across diverse clinical settings and disea...
BACKGROUND: Hematologic malignancies impose substantial economic burdens, but the quantitative association between financial toxicity and overall survival remains poorly defined across diverse clinical settings and disease lineages. METHODS: Adhering to PRISMA and MOOSE guidelines, we searched electronic databases through January 2026 for studies reporting multivariable-adjusted hazard ratios (aHRs) for the association between financial toxicity and mortality in adults diagnosed with hematologic malignancies. A random-effects model with Hartung-Knapp adjustment was used for data synthesis. Small-study effects were addressed via the Duval and Tweedie trim-and-fill method. RESULTS: Eleven cohorts involving 280 826 individuals were included. Meta-analysis identified a significant association between financial toxicity and inferior survival (pooled aHR: 1.57; 95% CI, 1.29-1.91; P < .001). High heterogeneity (I2 = 85.1%) and a wide 95% prediction interval (0.89-2.77) indicated substantial variability across settings. Subgroup analysis revealed that mortality risk was primarily linked to structural barriers, specifically lack of insurance (aHR: 1.66; 95% CI, 1.39-1.99), whereas this association was weaker in specialized transplant settings with integrated psychosocial support. Findings remained statistically significant after trim-and-fill adjustment for potential publication bias (adjusted aHR: 1.33; 95% CI, 1.05-1.69; P = .023). CONCLUSIONS: Financial toxicity is a significant factor independently associated with mortality in hematologic malignancies. The strength of this association varies depending upon the care environment, with structural access barriers representing the highest risk. Integrating proactive financial navigation into standard clinical pathways is essential to alleviate socioeconomic disparities and may help improve survival outcomes.
The albumin-bilirubin score predicts outcomes in advanced biliary tract cancer treated with durvalumab immunochemotherapy.
Chen MY, Kou HW, Wu TH … +5 more , Lan CC, Chen JS, Hsieh CH, Hung YS, Chou WC
Oncologist · 2026 May · PMID 42095607 · Full text
BACKGROUND: The albumin-bilirubin (ALBI) score objectively reflects hepatic functional reserve and is widely used as a prognostic biomarker in hepatocellular carcinoma. However, its prognostic value in patients with bili...
BACKGROUND: The albumin-bilirubin (ALBI) score objectively reflects hepatic functional reserve and is widely used as a prognostic biomarker in hepatocellular carcinoma. However, its prognostic value in patients with biliary tract carcinoma (BTC) treated with durvalumab plus gemcitabine-cisplatin (GCD) remains unclear. MATERIAL AND METHODS: We retrospectively analyzed 172 patients with unresectable or metastatic BTC who received first-line GCD between 2021 and 2025. Baseline ALBI scores were calculated and patients were stratified into cohort-specific tertiles (Q1-Q3). Overall survival (OS) was estimated using Kaplan-Meier method and Cox regression hazards models. Predictive performance for OS was compared between ALBI and the Eastern Cooperative Oncology Group performance status (ECOG PS) using time-dependent area under the receiver operating characteristic curves (AUC) analysis. RESULTS: The median OS was 13.8 months (95% CI, 9.9-17.7). Survival differed significantly across ALBI tertiles: median OS was 24.0 months (95% CI, 10.3-37.8) for Q1, 14.4 months (95% CI, 9.2-19.6) for Q2, and 5.9 months (95% CI, 2.5-9.2) for Q3 (P < .001). In multivariate analysis, ALBI remained an independent prognostic factor for OS (Q3 vs Q1, adjusted hazard ratio 2.16; 95% CI, 1.16-4.04; P = .016). The ALBI score demonstrated improved prognostic discrimination (AUC 0.714 at 6 months; 0.656 at 12 months) compared with ECOG PS (AUC 0.591 and 0.551, respectively). Tumor response rates were similar across tertiles, but higher ALBI scores were associated with an increased incidence of grade ≥3 toxicities. CONCLUSION: The pretreatment ALBI score is a simple, objective, and independent prognostic biomarker for patients with advanced BTC receiving durvalumab-based immunochemotherapy. Incorporating ALBI into baseline assessment may help identify high-risk patients who warrant closer monitoring or individualized therapeutic strategies.
Real-world treatment durations, subsequent treatments, and switching of CDK4/6 inhibitors among patients with HR+/HER2- metastatic breast cancer.
Brufsky A, Layman RM, Liu X … +8 more , Li B, McRoy L, Cohen A, Estevez M, Cottu P, Thill M, Curigliano G, Rugo HS
Oncologist · 2026 May · PMID 42095602 · Full text
BACKGROUND: Cyclin-dependent kinase 4/6 inhibitors (CDK4/6is), palbociclib, ribociclib, and abemaciclib, are approved for HR+/HER2- metastatic breast cancer (mBC). This real-world study evaluated treatment durations and...
BACKGROUND: Cyclin-dependent kinase 4/6 inhibitors (CDK4/6is), palbociclib, ribociclib, and abemaciclib, are approved for HR+/HER2- metastatic breast cancer (mBC). This real-world study evaluated treatment durations and subsequent treatments of patients with HR+/HER2- mBC who received CDK4/6is plus aromatase inhibitor (AI) in the United States. METHODS: Adult patients with HR+/HER2- mBC who initiated first-line (1L) CDK4/6is plus AI (February 2015-July 2024) were selected from the US-based Flatiron Health Research database. Stabilized inverse probability of treatment weighting (sIPTW) was conducted. Kaplan-Meier analyses estimated treatment durations. RESULTS: Of 11 557 patients, 8109, 2006, and 1442 received 1L palbociclib, ribociclib, and abemaciclib, respectively. After sIPTW, treatment duration was longer for the palbociclib group (median: 20.7 months) than the ribociclib (18.3 months) and abemaciclib (17.1 months) groups (ribociclib vs palbociclib: hazard ratio [HR] = 1.12 [95% confidence interval (CI), 1.05-1.20], P = .0008; abemaciclib vs palbociclib: HR = 1.13 [95% CI, 1.05-1.22], P = .0012). Treatment durations were similar between the ribociclib and abemaciclib groups (HR = 1.01 [95% CI, 0.92-1.11], P = .8280). Twelve-month treatment discontinuation rates were higher among patients initiating ribociclib or abemaciclib, at 39.4% and 41.1%, respectively, than among patients initiating palbociclib (33.3%). In the analysis of patients who initiated palbociclib, ribociclib, or abemaciclib from 2017 onward, 49.9%, 37.3%, and 39.4%, respectively, received subsequent treatments; CDK4/6i-containing regimens accounted for 42.3%, 54.1%, and 55.7%, respectively; notably, more patients initially treated with ribociclib or abemaciclib transitioned to palbociclib than those who switched oppositely. CONCLUSIONS: Treatment durations, discontinuation rates, and subsequent treatments differ between CDK4/6is for HR+/HER2- mBC in US routine clinical practice. CLINICAL TRIAL REGISTRATION NUMBER: NCT06495164.
Knowledge and awareness of colorectal cancer in central Uganda: a community cross-sectional survey.
Matovu N, Coleman HG, Mutungi G … +5 more , Donnelly M, Johnston BT, Loughrey MB, Jatho A, McShane CM
Oncologist · 2026 May · PMID 42095601 · Full text
BACKGROUND: Improving public knowledge and awareness of cancer aids prevention and early detection, yet limited data exist on this subject in low- and middle-income African countries. This study aimed to assess knowledge...
BACKGROUND: Improving public knowledge and awareness of cancer aids prevention and early detection, yet limited data exist on this subject in low- and middle-income African countries. This study aimed to assess knowledge and awareness of colorectal cancer (CRC) in the general population of central Uganda. METHODS: A community-based, cross-sectional survey was conducted among a random sample of 428 individuals from the general population in 2 districts of central Uganda. Data were collected using a semi-structured questionnaire and a modified version of the validated bowel cancer awareness measure. Multiple linear regression analysis was performed to identify factors associated with CRC awareness. RESULTS: Just over half of respondents had good awareness of CRC warning signs (57.9%, ≥ 6/9 correct responses), and risk factors (55.8%, ≥ 7/10 correct responses). Participants scored higher for awareness (responses to prompted questions), than knowledge (responses to unprompted questions) in relation to CRC warning signs (mean: 5.60 vs 0.46, out of 9) and risk factors (mean: 6.46 vs 0.22, out of 10). Residing in an urban area, lacking awareness of screenable cancers and not having private health insurance were associated with lower awareness of CRC warning signs. Being female and having non-degree tertiary education were associated with lower awareness of CRC risk factors. CONCLUSION: This study found very low CRC knowledge in central Uganda, despite good recognition of warning signs and risk factors, with notable rural-urban disparities. These findings highlight the need of reinforcing CRC prevention messages within ongoing cancer sensitization efforts to improve public understanding of CRC.
Prolonged response to pembrolizumab in BRAFV600E microsatellite stable metastatic colorectal cancer following an increase in tumor mutational burden.
Yang YH, Starling N, Cartwright E
Oncologist · 2026 May · PMID 42093086 · Full text
BRAFV600E-mutant metastatic colorectal cancer (mCRC) is an aggressive molecular subtype associated with poor prognosis and relative chemoresistance. Outcomes following progression on chemotherapy and MAPK-targeted therap...
BRAFV600E-mutant metastatic colorectal cancer (mCRC) is an aggressive molecular subtype associated with poor prognosis and relative chemoresistance. Outcomes following progression on chemotherapy and MAPK-targeted therapy with encorafenib plus cetuximab remain poor, highlighting an unmet need for effective later-line treatments. Immune checkpoint inhibitors have limited activity in microsatellite-stable (MSS) mCRC, and predictive biomarkers beyond mismatch repair deficiency remain poorly defined. We report a patient with BRAFV600E-mutant MSS mCRC who achieved a prolonged response to PD-1 blockade with pembrolizumab following a dynamic increase in tumor mutational burden (TMB). A 58-year-old woman initially responded to first-line chemotherapy but subsequently progressed on multiple treatment lines, including encorafenib plus cetuximab. Longitudinal genomic profiling using tumor tissue and circulating tumor DNA (ctDNA) revealed persistent BRAFV600E signaling with molecular evolution, including acquisition of a PIK3R1 variant associated with resistance to MAPK inhibition and emergence of TMB-high status. Fifth-line pembrolizumab produced a dramatic radiological response, with ongoing disease control more than 2 years later. We discuss the mechanistic framework by which a subset of BRAFV600E-mutant MSS mCRC may respond to immune checkpoint inhibition through an inflamed immune microenvironment driven by constitutive MAPK signaling. This case illustrates the interplay between tumor-agnostic biomarkers such as TMB-high and tumor-specific context, and highlights the value of longitudinal genomic profiling, including ctDNA, to identify resistance mechanisms and guide treatment selection.
Clinical implications of variant allele frequencies of genes in patients with acute myeloid leukemia.
Zhao L, Cheng F, Wei J … +4 more , Liu R, Zhao Q, Zhang Y, Li Y
Oncologist · 2026 May · PMID 42085510 · Full text
PURPOSES: Although next-generation sequencing (NGS) has allowed for the detection of mutations in acute myeloid leukemia (AML), the clinical relevance of variant allele frequency (VAF) for the majority of mutations is un...
PURPOSES: Although next-generation sequencing (NGS) has allowed for the detection of mutations in acute myeloid leukemia (AML), the clinical relevance of variant allele frequency (VAF) for the majority of mutations is unknown. This study aimed to investigate whether the VAFs of AML-associated mutations could improve the prognostic classification in AML. METHODS: Bone marrow samples from 254 AML patients were included for targeted sequencing. Univariate Cox regression analyses identified mutations affecting survival, and optimal VAF cutoffs were determined by X-tile software. Multivariate Cox regression analyses were used to validate the independent prognostic value of the variables. RESULTS: The presence of ASXL1, SF3B1, DNMT3A, and TP53 mutations had a significantly adverse effect on survival outcomes. High FLT3-ITD allelic ratio (≥35%) and high mutation VAFs of ASXL1 (≥2.8%), DNMT3A (≥45%), DNMT3A R882 (≥45%), NPM1 (≥38%), NPM1 type A (≥39%), SF3B1 (≥10%), and TP53 (≥10%) genes were the significant risk factors of overall survival (OS). Patients with high VAFs of bZIP in-frame mutated CEBPA (≥2%) had favorable OS. Notably, the prognostic utility of VAF for ASXL1 and CEBPA was limited compared to their binary mutation status due to the relatively low cutoff values. Based on optimal VAF cutoff-based classifications of five genes (DNMT3A, FLT3-ITD, NPM1, SF3B1, and TP53), mutation status-based classifications of two genes (ASXL1 and CEBPA) and cytogenetic stratification according to European LeukemiaNet (ELN) 2022 guidelines, we developed a prognostic model. This model effectively stratified non-transplanted AML patients into low-, intermediate-, and high-risk groups in both the internal and external cohorts (all pairwise comparisons, P <.05). However, the model showed limited utility for prognostic stratification in transplant patients (P >.05). CONCLUSIONS: Our study revealed that beyond binary mutation status, the relative amount of mutations exerts a significant prognostic impact in AML patients, suggesting its potential integration into risk stratification systems to guide clinical management in AML.
Shifting a paradigm: highlights of model informed drug development in dosage selection and optimization for oncology products.
Xiong Y, Konicki R, Zirkelbach JF … +8 more , Bi Y, Okusanya O, Shord S, Rahman NA, Booth B, Li Y, Liu J, Zhu H
Oncologist · 2026 May · PMID 42082405 · Full text
Applying model-based approaches to leverage clinical and non-clinical data can support the identification of optimized dosage(s) for oncology products and allow for a better benefit-risk balance. In oncology, conventiona...
Applying model-based approaches to leverage clinical and non-clinical data can support the identification of optimized dosage(s) for oncology products and allow for a better benefit-risk balance. In oncology, conventional dose-finding studies may lead to the selection of a poorly optimized dosage into subsequent studies, including those designed to demonstrate safety and efficacy in support of a marketing application. As a result, the clinical data obtained may be insufficient to optimize the benefit-risk profile of the drug without postmarketing studies. Model-informed drug development (MIDD), which includes modeling and simulation (eg, quantitative system pharmacology, empirical or mechanistic pharmacokinetic/pharmacodynamic models), and integrative exposure-response analysis, can significantly enhance dosage selection and optimization. This review provides an update on the recent utilization and unique challenges of MIDD for dosage optimization in oncology drug development.
Cost, Context, and Confounding: Rethinking Real-World data Generation and Interpretation in Advanced NSCLC.
Çatlı MM, Önder AH
Oncologist · 2026 May · PMID 42082389 · Publisher ↗
BACKGROUND: As immunotherapy becomes entrenched in the frontline management of advanced NSCLC, post-progression treatment decisions face increasing uncertainty. A recent JAMA Network Open study using Flatiron Health data...
BACKGROUND: As immunotherapy becomes entrenched in the frontline management of advanced NSCLC, post-progression treatment decisions face increasing uncertainty. A recent JAMA Network Open study using Flatiron Health data suggested that rechallenging with pembrolizumab may offer survival benefit after chemo immunotherapy progression. However, these findings raise fundamental questions about how real-world data (RWD) should be interpreted and operationalized in health economics. MAIN ARGUMENT: In this Viewpoint, we critically analyze the methodological and interpretive assumptions behind retrospective RWD-based cost-effectiveness modeling. We argue that unadjusted survival metrics absent stratification for PD-L1 status, tumor burden, or clinical performance may artificially elevate ICER/QALY estimates and misguide reimbursement decisions. Using the Velcheti et al. study as a case anchor, we highlight how structural biases in RWD can lead to premature or inflated conclusions about the value of immunotherapy rechallenge. We also propose a visual framework to distinguish between plausible clinical signals and policy-grade evidence. IMPLICATIONS: Health systems and stakeholders must apply greater rigor when translating RWD into economic models. We advocate for analytic transparency, pre-registered sensitivity analyses, and adjustment for patient-level variables before using RWD to inform value-based oncology. CONCLUSION: Real-world data are powerful hypothesis generators, not substitutes for controlled evidence. Without methodological discipline, their policy application risks becoming speculative rather than strategic.
Prevalence of adverse events following T-cell redirecting therapies in patients with metastatic castration-resistant prostate cancer: a pooled analysis.
Saxena A, Yorker M, Zorko NA … +10 more , Narayan V, Siddiqui BA, Saraiya B, Tester WJ, Mille PJ, Shaver AL, Nikita N, Lu-Yao GL, Kelly WK, Zarrabi KK
Oncologist · 2026 May · PMID 42081291 · Full text
BACKGROUND: T-cell redirecting therapies, including T-cell engagers (TCEs) and Chimeric Antigen Receptor T-cell (CAR-T), are under investigation in metastatic castration-resistant prostate cancer (mCRPC). Despite their p...
BACKGROUND: T-cell redirecting therapies, including T-cell engagers (TCEs) and Chimeric Antigen Receptor T-cell (CAR-T), are under investigation in metastatic castration-resistant prostate cancer (mCRPC). Despite their promising therapeutic potential, these therapies remain in early stages of development due to treatment-related adverse events (TRAEs). This study aims to quantify the frequency and severity of adverse events in clinical trials with a pooled analysis. MATERIALS AND METHODS: A systematic search of PubMed, CINHAL, Scopus, and Ovid was conducted to identify clinical trials evaluating TCEs or CAR-T in patients with mCRPC. Types and frequencies of TRAEs were collected and analyzed using a random-effects model. RESULTS: Thirteen TCEs trials involving 861 patients and 5 CAR-T trials involving 55 patients were identified. Cytokine release syndrome occurred in 59% (95% confidence interval: 25, 86) and 43% (28, 59) with TCEs and CAR-T, respectively. Neurologic TRAEs occurred in 39% (22, 60) with CAR-T compared to 9% (4, 17) with TCEs (P < 0.0001). Hematologic and hepatic TRAEs occurred in 34% (8, 75) and 25% (12, 44) with CAR-T, compared to 23% (7, 56) and 33% (12, 64) with TCEs, respectively. Musculoskeletal/dermatologic TRAEs occurred in 33% (24, 42) and 29% (8, 58) with TCEs and CAR-T, respectively. Renal and gastrointestinal TRAEs occurred in 21% (5, 51) and 14% (2, 43) with CAR-T, compared to 15% (8, 25) and 21% (12, 36) with TCEs, respectively. CONCLUSIONS: These data underscore the TRAE profiles associated with these therapies and emphasize the importance of differentiating off-target toxicities from immune effector cell-toxicities to inform the development of effective TRAE mitigation strategies.
Female sex is associated with worse survival in laryngeal head and neck squamous cell carcinoma.
Yao AG, Arif A, Paolucci CA … +7 more , Jain S, Modi JN, Mallick S, Mezghani N, Henick BS, Momen-Heravi F, Taylor AM
Oncologist · 2026 May · PMID 42063229 · Full text
BACKGROUND: Despite treatment, approximately one-third of patients with head and neck squamous cell carcinoma (HNSCC) die within 5 years of diagnosis. Here, we assessed molecular and clinical features that correlate with...
BACKGROUND: Despite treatment, approximately one-third of patients with head and neck squamous cell carcinoma (HNSCC) die within 5 years of diagnosis. Here, we assessed molecular and clinical features that correlate with biological sex and overall survival in HNSCC. METHODS: We analyzed HNSCC cases in The Cancer Genome Atlas, which includes tumors from 382 males and 141 females. When possible, findings were validated with the National Cancer Institute's Surveillance, Epidemiology, and End Results dataset (November 2023 Submission, 1975-2021). RESULTS: Tumors from females had a significantly lower fraction of genome altered, along with fewer aneuploidy events. HNSCCs in females had increased expression of immune genes and higher overall immune infiltrate. Females showed worse overall survival in HNSCC compared to males (hazard ratio [HR]: 1.39). This disparity was statistically attributable to age at diagnosis as well as HPV status, as HNSCCs in females were less likely to be HPV-positive than in males. For laryngeal squamous cell carcinoma (L-HNSCC), females also had significantly worse outcomes (HR: 3.42), but here the disparity could not be attributed to available clinicogenomic features such as HPV or smoking status. The association of biological sex with outcome in L-HNSCC was also observed in the SEER database and was not present among patients diagnosed before the age of 50. CONCLUSION: Biological sex is associated with differences in survival and tumor genomic features in HNSCC. HPV infection status, diagnosis age, and tumor location contribute to a worse prognosis for females with HNSCC, particularly strong in L-HNSCC.
Acupressure for anxiety: A pilot study of a nurse-led acupressure intervention for patients receiving chemotherapy.
Rhee L, Cutshall S, Metzger A … +10 more , Kruger M, Burckhard J, Mallory M, Whalen F, D'Andre S, Ruddy K, Loprinzi CL, Fischer KM, Manggaard JM, Cathcart-Rake E
Oncologist · 2026 Apr · PMID 42063228 · Publisher ↗
BACKGROUND: Anxiety is a common symptom experienced by patients with cancer, who are increasingly interested in non-pharmacologic symptom management. Acupressure, a noninvasive intervention thought to impact the neurohor...
BACKGROUND: Anxiety is a common symptom experienced by patients with cancer, who are increasingly interested in non-pharmacologic symptom management. Acupressure, a noninvasive intervention thought to impact the neurohormonal axis, has shown promise in reducing anxiety. However, research on acupressure for anxiety in patients with cancer has been limited. METHODS: Individuals receiving chemotherapy, who reported baseline anxiety, were eligible for this study. Consenting participants received one 15-minute nurse-led acupressure session and completed pre- and post-intervention surveys. The primary endpoint was a change in anxiety. Participants were also asked about symptoms of nausea, pain, wellbeing, relaxation, worry, and distress. Participants were invited to learn self-acupressure techniques. These participants were asked to complete a final survey 1-week post-intervention. RESULTS: Thirty adult patients participated in this study. Mean baseline anxiety was 5on a 0 to 10 Likertscale. Immediate post-intervention anxiety scores significantly improved for all thirty patients to an average of 2.0 (P < 0.001). Mean nausea scores improved from 1.5 at baseline to 0 post-intervention (P < 0.001), pain scores improved from 2.0 at baseline to 1.0 post-intervention (P = 0.001). Participants reported significant improvement in sense of wellbeing (P = 0.0004), distress (P < 0.001), ability to achieve relaxation (p < 0.001) and ability to control their worries (P < 0.001). The improvement in anxiety, wellbeing and relaxation was sustained at one week. 100% of participants stated that they would recommend acupressure for symptom management. CONCLUSION: Acupressure is a promising non-pharmacologic intervention for the management of cancer-associated anxiety.
Trust in researchers and willingness to engage in cancer research.
Sonawane K, Alekseyenko A, Magwood GS … +3 more , Mehrotra S, Devi GR, Ford ME
Oncologist · 2026 May · PMID 42063223 · Full text
BACKGROUND: Trust in medical scientists shapes public engagement in health and biomedical research, yet its influence on cancer research participation is not well understood. This study assessed trust in researchers and...
BACKGROUND: Trust in medical scientists shapes public engagement in health and biomedical research, yet its influence on cancer research participation is not well understood. This study assessed trust in researchers and examined its relationship with willingness to engage in diverse cancer research activities. METHODS: Cross-sectional analyses of a 2023 statewide survey of US adults evaluated Trust in Medical Researcher Scale (TMRS) scores (range 0-48) and willingness to participate in cancer research activities (research studies, biobanking, and data-sharing). Associations between trust and participation were examined using descriptive statistics and adjusted logistic regression models. RESULTS: Among 1780 respondents, the mean TMRS score was 27.3 ± 9.3, with most reporting moderate trust. Willingness to participate in cancer research varied across activity types. Low trust was consistently associated with reduced willingness across all activity types. CONCLUSIONS: Limited trust in researchers represents a significant barrier to participation in cancer research.
Adherence to oral antineoplastic agents, an overview and meta-analysis.
Mourath D, Fohlin H
Oncologist · 2026 May · PMID 42063222 · Full text
BACKGROUND: The use of oral antineoplastic therapies has accelerated in recent years. Non-supervised administration of oncological therapies introduces uncertainty in regard to adherence. This review and meta-analysis re...
BACKGROUND: The use of oral antineoplastic therapies has accelerated in recent years. Non-supervised administration of oncological therapies introduces uncertainty in regard to adherence. This review and meta-analysis reports on available literature relating to adherence, adherence-measuring methods, interventions to improve adherence, and estimates patient adherence to common oral antineoplastic therapies. METHODS: A literature search was conducted in the PubMed database up to January 29, 2024 using relevant terminology for oral antineoplastic agents. Titles and abstracts of retrieved articles were screened, and full-text articles deemed of interest were reviewed. Studies suitable for meta-analysis and consistent with the analytical approach were selected based on adherence measurement methods and definitions. Random effects meta-analysis was conducted using the R package metafor and visualized with forest plots for cumulative adherence and lower-bound adherence separately. RESULTS: We identified eight reviews and 75 original studies on adherence to non-endocrine oral antineoplastic agents, spanning the years from 1987 to 2023, with the vast majority published from 2010 onward. This review estimates the cumulative adherence to oral antineoplastic agents to be 84% (95% CI, 78-89) and lower-bound adherence as 71% (95% CI, 63-78). CONCLUSION: The definition of non-adherence and the measurement methods used across studies vary, rendering the comprehensive understanding of the subject challenging. This review identifies an adherence rate that deviates from full compliance across a wide variety of treatment regimens and tumor types, which could provide valuable insights for healthcare professionals in optimizing patient management.
Durable response to chemoimmunotherapy in a patient with refractory metastatic ovarian clear cell carcinoma: a case report and literature review.
Wang L, Chen X, Li Y … +2 more , Chang H, Meng X
Oncologist · 2026 May · PMID 42057611 · Full text
OBJECTIVE: Ovarian clear cell carcinoma (OCCC) frequently exhibits resistance to conventional chemotherapy with limited treatment options. This study reports a heavily pretreated advanced case with liver metastases to in...
OBJECTIVE: Ovarian clear cell carcinoma (OCCC) frequently exhibits resistance to conventional chemotherapy with limited treatment options. This study reports a heavily pretreated advanced case with liver metastases to investigate the efficacy of gemcitabine combined with PD-1 inhibitor in such refractory patients. METHODS: We present a case of stage IIIC OCCC that progressed after first-line platinum-based chemotherapy. The patient was treated with gemcitabine in combination with the PD-1 inhibitor toripalimab. Genetic alterations were analyzed using next-generation sequencing, and PD-L1 expression was assessed by immunohistochemistry (combined positive score [CPS]). Treatment response and safety profiles were regularly monitored. RESULTS: The combination therapy induced a complete remission lasting over 24 months, with a manageable safety profile. Molecular profiling revealed a loss-of-function mutation in ARID1A, which may enhance tumor sensitivity to gemcitabine, while elevated PD-L1 expression (CPS = 30) may serve as a predictive biomarker for immunotherapy response in OCCC. Notably, the deep and durable clinical response observed suggests a potential synergistic effect between gemcitabine and immunotherapy, wherein ARID1A deficiency-induced chemosensitivity may prime the tumor microenvironment for enhanced immune checkpoint inhibition, offering a compelling rationale for this combination strategy in platinum-resistant OCCC. CONCLUSION: This study proposes a promising therapeutic strategy for advanced OCCC and supports the clinical value of molecular profiling-guided treatment. Both ARID1A status and PD-L1 expression warrant further validation as potential biomarkers in prospective clinical trials.
Watchful Waiting in Children with Unifocal Osseous Langerhans Cell Histiocytosis.
Zhou C, Yang H, Liu S … +19 more , Shi W, Sun B, Zhao Y, Liu W, Tang L, Liu R, Ge J, Wang D, Yao J, Fang Z, Wang W, Tang X, Luo H, Liu L, Cao J, Wang T, Gao J, Zhang R, Chinese Children Histiocytosis Group
Oncologist · 2026 Apr · PMID 42057312 · Publisher ↗
BACKGROUND: Langerhans cell histiocytosis (LCH) is a rare inflammatory neoplasm most commonly involving bone. However, data on single-system, single-site LCH (SS-s-LCH), particularly with unifocal bone disease, remain li...
BACKGROUND: Langerhans cell histiocytosis (LCH) is a rare inflammatory neoplasm most commonly involving bone. However, data on single-system, single-site LCH (SS-s-LCH), particularly with unifocal bone disease, remain limited. Management strategies for unifocal osseous LCH vary globally, and the safety of a watchful-waiting approach is not well-defined. This study aimed to evaluate whether patients with osseous SS-s-LCH can be managed with observation alone without compromising long-term outcomes. METHODS: In this retrospective, multicenter study, we enrolled children with biopsy-proven osseous SS-s-LCH from seven centers in the Chinese Children's Histiocytosis Group, excluding those with central nervous system (CNS)-risk bone lesions. The primary endpoint was 3-year event-free survival (EFS). Events were defined as disease progression or relapse. RESULTS: The cohort included 134 patients with a median follow-up of 36.0 months. Involved sites were cranial bones (n = 37), vertebrae (n = 33), and other bones (n = 64). At final follow-up, 16 patients had experienced an event, yielding a 3-year EFS of 88.1% (95% CI: 81.5-92.5%). Events included new lesions (n = 14) and primary lesion expansion (n = 2), with 93.8% (15/16) occurring within the first 12 months after diagnosis. EFS did not differ significantly by involved site or mutation status. However, patients staged by PET-CT at diagnosis had a significantly higher 3-year EFS (97.6%) than those staged by conventional imaging (83.7%) (p < 0.05). Long-term quality of life was satisfactory in all assessed patients. CONCLUSION: Watchful waiting is a safe and effective management strategy for children with unifocal osseous SS-s-LCH. Initial staging with PET-CT may be associated with superior event-free survival, highlighting its potential value in refining risk assessment.
Proactive identification of candidates for newly approved targeted therapies using a clinical decision support program.
Feldman R, Abdulla F, Hinton A … +14 more , Hamrick J, Hill B, Fernandez J, Walton A, Shah N, Appleberry G, El Shawa H, Sheridan RL, Geist BM, Coleman J, Duncavage E, Arnal S, Vacirca J, Holloway J
Oncologist · 2026 May · PMID 42057310 · Full text
BACKGROUND: Comprehensive molecular profiling (CMP) enables identification of multiple biomarkers from a single sample to inform targeted therapy selection. However, the static nature of test reports limits their clinica...
BACKGROUND: Comprehensive molecular profiling (CMP) enables identification of multiple biomarkers from a single sample to inform targeted therapy selection. However, the static nature of test reports limits their clinical relevance as new therapies emerge. Based on the presence of molecular alterations, the population of patients qualifying for FDA-approved therapies nearly doubled since 2017, while FDA-recognized biomarkers of drug response quadrupled. Lookback programs are clinical decision support services designed to notify clinicians when previously profiled patients become candidates for new targeted therapies based on original test results. This study describes biomarkers assessed in a lookback program and summarizes identification of patients who become candidates for novel treatment options following new FDA approvals. MATERIALS AND METHODS: Between 2018 and 2025, tumor samples were submitted to Caris Life Sciences for whole-exome sequencing, whole-transcriptome sequencing, and immunohistochemistry. With new biomarker-directed approvals, the support tool is prompted to evaluate indications as candidates for lookbacks. For approved candidates, molecular profiles from the database are retrospectively assessed for relevant biomarker status within disease-site context. After evaluation, a molecular scientist sends standardized communication informing oncologists of potential candidates for the new therapy. RESULTS: Since 2018, 87 biomarker-directed FDA approvals were evaluated for the lookback program. Among 483 000 molecular profiles, 13 293 patients were identified as candidates for newly approved agents in ten distinct tumor types and several tumor-agnostic indications. CONCLUSIONS: By coupling CMP with continuous evidence surveillance and proactive provider clinician engagement, lookback programs potentially close the gap between therapeutic innovation and clinical application by providing timely information about emerging targeted therapies.
Exploring biologically-based complementary and alternative medicine use among Irish cancer survivors: findings from a national survey.
Scannell C, Sullivan ES, Matvienko-Sikar K … +3 more , Grimes DR, Power D, Ryan A
Oncologist · 2026 May · PMID 42050866 · Full text
BACKGROUND: Biologically based complementary and alternative medicine (BBCAM) includes special diets, dietary supplement and herbal remedies, not prescribed by a doctor or dietitian. The use of BBCAM is common among canc...
BACKGROUND: Biologically based complementary and alternative medicine (BBCAM) includes special diets, dietary supplement and herbal remedies, not prescribed by a doctor or dietitian. The use of BBCAM is common among cancer survivors. BBCAM can interact with conventional treatments and unregulated products may cause harm. This study aimed to determine the prevalence, types, and motivations for BBCAM use among cancer survivors. METHODS: A survey assessed clinical characteristics and BBCAM use in participants >18 yrs who had received cancer treatment in Ireland from 2018 to 2022. RESULTS: Amongst 295 respondents (77% female, mean age 53 yrs), BBCAM use increased from 28% pre-diagnosis to 34% post-diagnosis (p < 0.001). For BBCAM users (n = 97, 33%), "daily-use" increased from 38% to 72% (p < 0.001) post-diagnosis. Common types included: mineral/vitamin supplements (84%), dietary supplements (e.g. turmeric, coenzyme-Q10) (78%), herbal remedies/botanicals (e.g. mistletoe, St. John's Wort, echinacea, ginseng) (50%), cannabis (21%), and other natural products (laetrile, shark cartilage, apricot kernels) (19%). Biological medicines (GcMAF, immuno-augmentative therapy) were used by 12% of BBCAM users. Special diets including dairy free (32%), gluten-free (19%), intermittent fasting (17%), ketogenic diet (15%), juicing/detox (10%) were also common. Perceived benefits included: improved well-being (63%) and reduced psychological stress (59%). CONCLUSION: BBCAM use increases after a cancer diagnosis. Patient perceived benefits highlight potential gaps in the current healthcare model, indicating a need for greater emphasis on safe survivorship care.
Effect of patient factors and quality of life on ECOG assignment and inter-rater agreement in hospitalized cancer patients.
Blaya Boluda N, García-Torralba E, García-Romero A … +7 more , Rodríguez García P, Montenegro S, Sánchez-Canovas M, Ivars Rubio A, Sánchez-Saura A, Vicente MÁ, Ayala de la Peña F
Oncologist · 2026 May · PMID 42050183 · Full text
BACKGROUND: Assigning the ECOG performance status is critical for decision-making in oncology, but it poses challenges in hospitalized patients due to lower concordance and potential alteration by factors related to admi...
BACKGROUND: Assigning the ECOG performance status is critical for decision-making in oncology, but it poses challenges in hospitalized patients due to lower concordance and potential alteration by factors related to admission. This study aimed to evaluate the concordance of ECOG assessments among observers, identify associated factors with assignment, and explore its relationship with quality of life (QoL) in hospitalized oncology patients. METHODS: A prospective, observational study was conducted on patients admitted to a Medical Oncology Department. Three oncologists independently assessed the ECOG at admission, with a consensus value (ECOGa) being established. The EQ-5D-5L scale was administered within the first 48 hours. Linearly weighted Fleiss' Kappa (wFK) was used to assess concordance, and ordinal generalized linear mixed regression models were applied to analyze associated factors and to calculate values of model-based Kappa for agreement. RESULTS: A total of 167 patients were included, most of them (76%) with stage IV disease. Overall concordance was wFK 0.773 (95% CI 0.711-0.823). ECOGa was significantly associated with cancer stage (P = .03), reason for admission (P = .01), age (P = .04), and absence of active treatment (P = .01). The pain (P < .001) and self-care (P = .01) subscales of the EQ-5D-5L enhanced the prediction of ECOG performance status, outperforming models based on clinical model or QoL variables. Interobserver agreement, as determined by model-based kappa, was lower in the subgroup of patients with ECOGa = 2 and in patients admitted due to toxicity. CONCLUSIONS: Although ECOG assignment showed substantial concordance, both patient- and admission-related factors significantly influence its evaluation in hospitalized oncology patients. QoL measures may provide valuable complementary information for functional assessment in this population. A better understanding of the factors and clinical settings associated with lower interobserver agreement could help improve the functional assessment of cancer patients admitted to the hospital.
Evaluation of Dronabinol to Decrease Opioid Use for Cancer-Induced Bone Pain.
Segar J, Farr K, Junak M … +5 more , Roe D, Ehsani S, Ibrahim M, Vanderah TW, Chalasani P
Oncologist · 2026 Apr · PMID 42050177 · Publisher ↗
BACKGROUND: Bone metastases (BM) from breast cancer cause significant cancer-induced bone pain (CIBP). Management of CIBP is primarily with opioids, which have notable side effects. In preclinical models, cannabinoid rec...
BACKGROUND: Bone metastases (BM) from breast cancer cause significant cancer-induced bone pain (CIBP). Management of CIBP is primarily with opioids, which have notable side effects. In preclinical models, cannabinoid receptor (CB)2 and CB1 agonists were shown to decrease CIBP and bone degradation. We hypothesized that the addition of CB2/CB1 agonists would decrease opioid requirements in patients with BM. METHODS: We conducted a single-arm study among breast cancer patients with BM on opioid therapy. Patients were treated with 10 mg dronabinol BID for 8 weeks. Our primary objective was to determine the proportion who decreased their opioid use by ≥ 20%. Participants completed the Brief Pain Inventory and the European Organization for Research and Treatment of Cancer quality of life questionnaires before and after treatment. Pre- and post-treatment blood and urine were collected for analysis of biomarkers of bone remodeling. RESULTS: We enrolled 14 evaluable patients, and 4 decreased opioid use by ≥ 20%, meeting the primary endpoint. Patients reported significant improvements in pain severity, interference scores, quality of life, and insomnia. There was one grade 3 adverse event (dizziness) related to the study drug. A significant decrease was noted in serum C-terminal telopeptide levels with therapy. CONCLUSION: Our pilot study shows that the addition of dronabinol resulted in decreased opioid requirements for CIBP. Patient-reported outcomes also demonstrated improved pain and QOL with addition of dronabinol. Our results are promising and warrant further investigation into novel analgesics for CIBP to decrease opioid use.
Multicenter phase II study of neoadjuvant FOLFOXIRI followed by concurrent chemoradiotherapy in Chinese patients with high-risk rectal cancer.
Lui RN, Chu S, Ng DCK … +16 more , Li L, Cho CCM, Hung EHY, Wong KH, Mo FKF, Wong ECH, Hui CWC, Lam DCM, Suen J, Kang W, Ho WM, Futaba K, Hon SSF, Yan K, Ng SSM, Ma BBY
Oncologist · 2026 May · PMID 42050144 · Full text
BACKGROUND: We investigated the feasibility of adding neoadjuvant FOLFOXIRI to chemoradiotherapy (CRT) in Chinese patients with high-risk, locally advanced rectal adenocarcinoma (LARC) and examined the prognostic signifi...
BACKGROUND: We investigated the feasibility of adding neoadjuvant FOLFOXIRI to chemoradiotherapy (CRT) in Chinese patients with high-risk, locally advanced rectal adenocarcinoma (LARC) and examined the prognostic significance of IGF2 and L1CAM expression. METHODS: Eligible patients had non-metastatic, T3/T4 disease with or without nodal involvement, threatened circumferential resection margin (CRM) and/or sphincter involvement, received 4 cycles of a modified FOLFOXIRI regimen, followed by CRT, surgery, then adjuvant chemotherapy. Co-primary endpoints were objective response rate (ORR) and pathologic complete response rate (pCR). Secondary endpoints included overall survival (OS), relapse-free survival (RFS) and safety. Archival biopsies were analyzed for IGF2 and L1CAM expression using immunostaining. RESULTS: Forty patients were enrolled with median age of 60 years and median follow up of 72.7 months. The ORR of FOLFOXIRI and CRT was 30.8% and 64.1%, respectively in 39 patients evaluated, 71.8% of them exhibiting TNM downstaging. For the entire cohort, the pCR rate was 20.5% and CRM was negative in 30 patients. The 3-year and 5-year OS were 79.5% and 59.5%, respectively. The 3-year RFS was 72.4%. Grade 3-4 toxicities to FOLFOXIRI were diarrhea (13%), neutropenia (8%), and vomiting (5%). Grade 3-4 toxicities to CRT were diarrhea (3%), rectal hemorrhage (3%), and sexual dysfunction (3%). High IGF2 expression was negatively correlated with disease-free survival (P = .0176) but not OS. CONCLUSIONS: Neoadjuvant modified FOLFOXIRI followed by concurrent capecitabine-RT and surgery was effective with manageable toxicities in Chinese patients with high risk LARC. Exploratory analyses showed that IGF2 expression may be a negative prognostic factor in LARC. (NCT01941641).
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