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Clinical Efficacy of 5-Aminolevulinic Acid Photodynamic Therapy in the Treatment of Oral Leukoplakia: A Prospective Study with Long-Term Follow-Up.5 days agoTo observe the clinical efficacy and influencing factors of 5-aminolevulinic acid photodynamic therapy (5-ALA-PDT) in treatment oral leukoplakia (OL), so as to provide therapeutic references for clinical application.
After optimizing the 5-ALA-PDT treatment process, 31 OL patients were locally treated with 5- ALA-PDT (wavelength, 635 nm; energy density, 100 J/cm2), with 3 sessions per course of treatment. Efficacy was evaluated one month post-treatment by assessing lesion changes. Additionally, the factors that might influence therapeutic efficacy were analyzed.
An overall response rate of 90.3 % was achieved in this study, including 19 patients with complete remission, 9 with partial remission, and 3 with stable disease.The patients were followed for a median of 15 months (95% CI: 9-20 months; range: 3-25 months), during which recurrence occurred in 3 patients and malignant transformation in 2 patients. The treatment effect appeared better for lesions on the ventral tongue than at other sites. Patients who experienced local symptoms such as pain or ulcers after 5-ALA-PDT showed a more favorable response than those without any postoperative reactions. Patients with heterogeneous oral leukoplakia and moderate epithelial dysplasia had a higher recurrence rate than those with homogeneous lesions, simple hyperplasia, or mild epithelial dysplasia, with the peak of recurrence registered at 1-6 months after treatment.
5-ALA-PDT appears to be a promising treatment for OL. However, recurrence and malignant transformation remain concerns.CancerAccessCare/ManagementAdvocacy -
The SCF/KIT signaling pathway in multisystem diseases: Mechanisms and therapeutic targeting.5 days agoThe stem cell factor (SCF)/KIT signaling axis regulates the survival, proliferation, differentiation, and migration of hematopoietic stem and progenitor cells, mast cells, interstitial cells of Cajal, germ cells, and melanocytes through multiple downstream pathways, including PI3K/AKT and RAS/MAPK. Its biological and pathological effects are determined by cell lineage, ligand presentation, and mutational context. At the pathway level, SCF expression and processing, KIT abundance, and activating mutations determine signal initiation and intensity, whereas CBL-mediated receptor degradation and SHP-1-mediated dephosphorylation limit signal duration. This narrative review critically examines representative multisystem diseases for which SCF/KIT involvement is supported by genetic, functional, or therapeutic evidence and distinguishes established treatments from investigational approaches. Activating KIT mutations drive gastrointestinal stromal tumors (GISTs) and systemic mastocytosis. Clinically established KIT-directed tyrosine kinase inhibitors, including imatinib for mutation-sensitive GIST and midostaurin or avapritinib for advanced systemic mastocytosis, have transformed disease management. By contrast, increased local SCF availability and KIT-dependent mast cell persistence may amplify inflammatory diseases, whereas impaired signaling is associated with gastrointestinal dysmotility, reproductive dysfunction, and pigmentary abnormalities. Emerging anti-KIT antibodies are being investigated as a strategy to deplete mast cells in chronic urticaria, whereas recombinant SCF-based mobilization has largely historical clinical relevance and regenerative applications remain experimental. Mutation-dependent drug sensitivity, polyclonal secondary resistance, multikinase-related toxicity, and on-target effects in normal KIT-dependent tissues remain major challenges. A disease-, genotype-, and cell-specific framework is therefore essential for translating SCF/KIT biology into safe and effective precision therapies.CancerAccessCare/Management
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Clinical, economic, and quality-of-life outcomes of brexucabtagene autoleucel for the treatment of relapsed/refractory mantle cell lymphoma and B-cell acute lymphoblastic leukemia: A systematic literature review and meta-analysis.5 days agoBrexucabtagene autoleucel (brexu-cel), a CD19 chimeric antigen receptor T-cell therapy, has shown efficacy in clinical trials for relapsed/refractory (R/R) mantle cell lymphoma (MCL) and B-cell acute lymphoblastic leukemia (B-ALL). However, although effectiveness and safety evidence is well established, real-world evidence (RWE) on health care resource use (HCRU), costs, and health-related quality of life (HRQoL) remains limited.
To systematically review and synthesize RWE on clinical, HCRU, and cost outcomes, and both RWE and trial evidence on HRQoL, for brexu-cel in adults with R/R MCL and B-ALL.
Systematic searches were conducted to identify studies reporting outcomes for adults with R/R MCL and B-ALL treated with brexu-cel. RWE was included for clinical, HCRU, and costs outcomes, and HRQoL outcomes. Effectiveness and safety were analyzed using meta-analysis, while HCRU, cost, and HRQoL were summarized descriptively.
69 publications representing 31 cohorts were identified (19 MCL, 7 B-ALL, 3 mixed). Pooled overall response and complete response rates in MCL were 88% and 76%, while B-ALL showed complete response and measurable residual disease negativity of 74% and 73%, consistent with pivotal trials ZUMA-2 and ZUMA-3, respectively. Median overall survival was 40.3 months in MCL and 23.3 months in B-ALL. Cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome were the most common adverse events; severe events were less frequent in real-world cohorts compared with trials, potentially reflecting advances in safety management and earlier identification of toxicities. HCRU and cost evidence was limited; 1 MCL study reported reduced post-chimeric antigen receptor T costs. No real-world HRQoL data were identified.
Brexu-cel provides substantial and durable benefit in R/R MCL and B-ALL, with real-world effectiveness and safety consistent with pivotal trials. However, evidence gaps remain for HRQoL and economic outcomes, underscoring priorities for future research.CancerAccessCare/ManagementAdvocacy -
Association between cachexia-associated diagnoses, health care resource utilization, and costs in patients with pancreatic, lung, and colorectal cancers: A US insurance claims database analysis.5 days agoCancer cachexia is a wasting disease characterized by changes in metabolism, muscle and weight loss, inadequate food intake, and functional decline. The disease affects 30%-50% of patients with solid tumors. There is limited literature describing the relationship between cachexia and health care resource utilization (HRU) and costs in the United States.
To compare the HRU and direct health plan costs for patients with colorectal, lung, and pancreatic cancer with and without a cachexia-associated diagnosis.
We conducted a retrospective cohort study in Merative MarketScan commercial and Medicare claims databases. Patients with cachexia-associated diagnoses were identified following a colorectal, lung, or pancreatic cancer diagnosis between October 1, 2016, and December 31, 2022. Patients without a cachexia-associated diagnosis were propensity score-matched 2:1 to patients with a cachexia-associated diagnosis. Average 1-year HRU and direct health plan costs were calculated using a modified Kaplan-Meier sample average approach to account for patients lost to follow-up before the end of the 1-year observation period. The significance of group differences was assessed by nonoverlapping bootstrap 95% CIs.
The matched sample included 10,445 patients with a cachexia-associated diagnosis and 20,890 without across colorectal, lung, and pancreatic cancers. One-year health care costs were significantly higher for patients with a cachexia-associated diagnosis ($123,092; 95% CI = $119,664-$126,557) than for those without ($88,959; 95% CI = $86,964-$91,022). Patients with cachexia-associated diagnoses had significantly greater HRU than those without cachexia-associated diagnoses, including higher average 1-year hospital admissions (1.50; 95% CI = 1.47-1.53 vs 0.99; 95% CI = 0.97-1.01), emergency department visits (1.46; 95% CI = 1.42-1.50 vs 0.96; 95% CI = 0.93-0.98), outpatient visits (15.49; 95% CI = 15.15-15.83 vs 14.24; 95% CI = 14.02-14.46), and prescription fills (26.83; 95% CI = 26.31-27.35 vs 24.46; 95% CI = 24.11-24.80).
Patients with colorectal, lung, and pancreatic cancer and cachexia-associated diagnoses had substantially higher 1-year HRU and cost of care compared with patients with cancer without cachexia-associated diagnoses. Our results highlight evidence of clinical and economic burden associated with cachexia-associated diagnoses in the US health care system.CancerChronic respiratory diseaseAccessCare/ManagementAdvocacy -
Silent threat: A case report of acute fulminant exogenous lipoid pneumonia leading to severe ARDS.5 days agoExogenous lipoid pneumonia (ELP) is a rare pulmonary disorder resulting from the aspiration or inhalation of exogenous lipid substances. While most documented cases follow an indolent, chronic course and are associated with favorable outcomes, fulminant postoperative ELP remains exceedingly uncommon-often presenting with rapid respiratory decompensation that may clinically and radiologically mimic severe community-acquired pneumonia or acute respiratory distress syndrome (ARDS). We report a 61-year-old man with occult fulminant postoperative ELP after repeated oral liquid paraffin use for bowel obstruction caused by a large rectal neoplasm. No definite aspiration event was witnessed. After emergency diverting ileostomy, he developed rapidly progressive refractory hypoxemia with bilateral ground-glass opacities and consolidations on chest CT. BALF pathogen testing was largely negative, although a positive galactomannan result meant that fungal coinfection could not be fully excluded. Oil Red O staining demonstrated lipid-laden macrophages, supporting the diagnosis of ELP. Despite invasive mechanical ventilation, prone positioning, segmented alveolar lavage, systemic corticosteroids, adjunctive IVIG, respiratory failure progressed. The patient died three days after discharge against medical advice, most likely from progressive respiratory failure. This case highlights a rare and fulminant form of postoperative exogenous lipoid pneumonia following liquid paraffin exposure for bowel obstruction. Early recognition is important because ELP may coexist with infection and can progress rapidly despite intensive supportive care.CancerChronic respiratory diseaseAccessAdvocacy
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The Clinical Impact of End-of-Consolidation Measurable Residual Disease in High-Risk Pediatric B-Cell Acute Lymphoblastic Leukemia.5 days agoIntroductionBlinatumomab (BLINA) has improved event-free survival (EFS) and overall survival (OS) in pediatric patients with high-risk B-cell acute lymphoblastic leukemia (ALL) and has become an effective bridging therapy to hematopoietic cell transplantation. However, the prognostic significance of end-of-consolidation (EoC) measurable residual disease (MRD) in the current BLINA-incorporated treatment era remains unclear.MethodsWe retrospectively enrolled pediatric patients diagnosed with high-risk B-cell ALL between February 2022 and December 2024 at Chang Gung Memorial Hospital. Patients were categorized into EoC MRD-negative and EoC MRD-positive groups. EoC MRD was assessed by multiparameter flow cytometry, with real-time quantitative polymerase chain reaction used as a complementary molecular method when applicable. Outcomes of interest included EFS, OS, and BLINA-related adverse events (AEs).ResultsThe cohort consisted of 15 patients. With a median follow-up of 24 months, the observed 2-year EFS and OS rates were 71.5% and 86.7%, respectively. The EoC MRD-positive group demonstrated significantly higher relapse rates (57.1% vs. 0%, P = 0.0256) and numerically higher mortality rates (28.6% vs. 0%, P = 0.2). The 2-year EFS was significantly higher in the EoC MRD-negative group (100% vs. 42.9%, P = 0.02). Although the EoC MRD-negative group demonstrated a numerically higher 2-year OS (100% vs. 71.9%, P = 0.12), the difference did not reach statistical significance. Regarding safety, no high-grade short-term or long-term BLINA-related AEs were observed in either group.ConclusionIn this preliminary retrospective study, EoC MRD negativity was associated with sustained disease control during short-term follow-up and favorable survival outcomes in pediatric patients with high-risk B-cell ALL treated with BLINA-based therapy. These findings suggest that EoC MRD assessment might provide additional prognostic information in the contemporary BLINA treatment era. Further prospective studies with larger cohorts are warranted to validate these observations.CancerAccessCare/ManagementAdvocacy
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Prevalence and Predictors of Awareness of the Kuwait National Mammography Screening Program Among Women in Kuwait: A Cross-Sectional Study.5 days agoBreast cancer is the leading malignancy among women in Kuwait. The Kuwait National Mammography Screening Program (KNMSP), launched in 2014, provides free biennial mammography to asymptomatic women aged 40-69 years; however, participation remains persistently low and awareness is insufficiently characterized. This study aimed to estimate the prevalence of KNMSP awareness and identify its independent predictors across sociodemographic, knowledge, behavioral, and attitudinal domains.
A community-based cross-sectional survey was conducted among 458 women aged ≥20 years residing across all six governorates of Kuwait between January and April 2025, using convenience sampling via social media and community networks. Of 516 women who accessed the questionnaire, 458 met eligibility criteria and completed all items (response rate 88.7%). Standardized validated instruments assessed breast cancer warning-sign knowledge, risk-factor knowledge, screening beliefs, and perceived barriers. Hierarchical multivariable logistic regression identified independent predictors of KNMSP awareness.
Overall, 65.3% of participants (n = 299) reported awareness of the KNMSP. In the final adjusted model (Nagelkerke R² = 0.355; AUC = 0.798), knowledge of breast self-examination (BSE) was the strongest independent predictor (OR = 3.60; 95% CI 1.87-6.93; p < 0.001). Women aged 20-29 years (OR = 0.29), unpartnered women (OR = 0.40), and non-Kuwaiti women (OR = 0.47) had significantly lower adjusted odds of awareness. Perceived barriers and screening beliefs were not independently associated with awareness.
Two-thirds of women in Kuwait are aware of the KNMSP; however, substantial gaps persist among younger, unpartnered, and non-Kuwaiti women. Integrating BSE education into outreach initiatives, strengthening primary-care referral, and developing multilingual campaigns targeting high-gap subgroups are priority actions to improve program visibility and, ultimately, screening uptake.CancerAccessAdvocacy -
Circulating miR-181a-3p, ATR, and HMGB-1 as Integrated Predictors of Induction Blast Clearance in Adults with Acute Myeloid Leukemia.5 days agoThis study included 71 adult patients with newly diagnosed AML who received standard "7+3" induction chemotherapy. Baseline circulating miR-181a-3p and miR-181b-5p levels were quantified using quantitative real-time polymerase chain reaction, while ATM, ATR, and HMGB-1 protein levels were measured using enzyme-linked immunosorbent assay. Induction blast clearance was defined as a post-induction bone marrow blast count of <5%. Receiver operating characteristic (ROC) curve analysis was used to determine optimal cut-off values. Associations were evaluated using Spearman correlation and relative risk (RR) with 95% confidence intervals.
Circulating miR-181a-3p demonstrated limited discriminatory performance (AUC = 0.564), while HMGB-1 showed modest performance (AUC = 0.615). Using ROC-derived cut-off values, low baseline miR-181a-3p expression was associated with a significantly higher likelihood of achieving induction blast clearance in monocytic AML (FAB M4-M5) (RR = 2.33, 95% CI: 1.18-4.63, p = 0.015), but not in non-monocytic subtypes. Higher ATR (RR = 1.53, 95% CI: 0.99-2.38, p = 0.047) and HMGB-1 levels (RR = 1.61, 95% CI: 1.04-2.50, p = 0.031) were also associated with improved induction response. Circulating miR-181a-3p correlated with baseline bone marrow blast percentage, suggesting its potential as a surrogate marker for leukemic burden.
Circulating miR-181a-3p, ATR, and HMGB-1 are associated with induction blast clearance in AML, with lineage-specific effects observed for miR-181a-3p. Although the individual biomarkers demonstrated limited discriminatory performance, their integration may provide clinically relevant insights into early treatment response.CancerAccessCare/ManagementAdvocacy -
Breast Cancer-Related Quality of Life among Women Undergoing Treatment: A Hospital-Based Study in Vietnam.5 days agoDespite the rising incidence of breast cancer (BC), survival rates have improved due to advancements in early detection and treatment. With longer survival, it is increasingly important to evaluate and enhance patients' quality of life (QoL), particularly the functional and symptom-related aspects of BC. This study aims to assess BC-related QoL and its associated factors among women undergoing treatment.
A hospital-based cross-sectional study assessed BC-related QoL in 284 patients using the European Organization for Research and Treatment of Cancer (EORTC) QoL Questionnaire for BC (QLQ-BR45) between March 10 and May 14, 2025. Statistical analyses included multivariable linear regression to identify factors associated with 12 domains of patient QoL.
QoL findings showed high body image scores (72.51) but low sexual functioning (13.50) and sexual enjoyment (26.29), with hair-loss distress (51.32) and systemic therapy side effects (30.92) being the most burdensome symptoms. Older age and higher body mass index (BMI) had both positive and negative associations with QoL, while marriage, higher education, urban residence, and longer breastfeeding were linked to better outcomes. Late-stage disease, older age at first childbirth, and receiving no treatment were linked to poorer outcomes. Treatment had varying effects on QoL domains: surgery plus chemotherapy improved sexual enjoyment, while non-surgical approaches alleviated symptoms across several QoL-related domains.
BC patients showed strong body image but poor sexual functioning and notable symptom burdens, underscoring the need for supportive care that addresses physical and psychosocial issues. Routine QoL monitoring and broader multicenter studies are essential to guide tailored interventions and improve overall patient QoL.CancerAccessAdvocacy -
Prognostic Nutritional Index Is Associated With Gastrointestinal Symptom Severity in Patients Receiving Chemotherapy for Gastrointestinal Cancer: A Cross-Sectional Study.5 days agoThis study aimed to investigate the relationship between the Prognostic Nutritional Index (PNI) and chemotherapy-related gastrointestinal (GI) symptom severity in patients with GI cancer.
This observational cross-sectional study included 262 adult patients with GI cancer receiving chemotherapy at a research hospital in eastern Türkiye. Nutritional status was assessed using the Prognostic Nutritional Index, calculated from serum albumin levels and lymphocyte counts obtained prior to chemotherapy cycles. Gastrointestinal symptom burden was evaluated using the Gastrointestinal Symptom Rating Scale (GSRS). Descriptive statistics, group comparisons, and correlation analyses were performed to evaluate associations between clinical variables, PNI, and GSRS scores.
The mean PNI score was 39.34 ± 12.31, and nearly half of the participants were classified as at risk for malnutrition based on sample-specific cut-off values. The mean total GSRS score was 36.23 ± 12.06, indicating mild-to-moderate symptom severity. GSRS scores were significantly higher among patients with advanced disease stages and lower PNI values. Correlation analysis revealed a statistically significant negative association between PNI and GSRS scores (r = -0.239, p < 0.05), demonstrating that poorer nutritional status was associated with greater gastrointestinal symptom severity. In the multiple linear regression analysis, it was determined that cancer stage and chemotherapy protocol had significant effects on PNI and GSRS scores (p<0.05).
This study demonstrates that lower PNI is linked to greater gastrointestinal symptom severity, underscoring the need to integrate routine nutritional evaluation with gastrointestinal symptom monitoring in GI cancer care.CancerAccessCare/ManagementAdvocacy