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Treatment patterns, intensive treatment, and hospitalization-related burden of juvenile generalized myasthenia gravis in Japan: a retrospective cohort study using a hospital-based claims database.2 weeks agoJuvenile generalized myasthenia gravis (MG) management aims to control symptoms while minimizing corticosteroid-related toxicity. However, large-scale evidence on real-world pediatric treatment patterns remains limited.
We conducted a retrospective cohort study using a large Japanese hospital-based claims database (Medical Data Vision). We identified patients aged 0-17 years with juvenile generalized MG between April 2008 and November 2024. We summarized the use of cholinesterase inhibitors, oral corticosteroids, immunosuppressants, and intensive treatments (plasma exchange, intravenous immunoglobulin [IVIg], or high-dose steroid pulse therapy) during the one-year follow-up period after the first confirmed MG diagnosis recorded in the database. We also compared patient characteristics and treatment patterns by age group (children: 0-9 years vs. adolescents: 10-17 years).
We identified 261 patients with juvenile generalized MG, including 125 children and 136 adolescents cases. Cholinesterase inhibitors were the most frequently used medication (69.3% [181/261]). Oral corticosteroids were commonly used (65.9% [172/261]), whereas immunosuppressant use was limited; only 89/261 (34.1%) received tacrolimus. Among the 217 patients with complete one-year follow-up data, 146 received corticosteroid. The mean daily corticosteroid dose, calculated as the annual cumulative corticosteroid dose divided by 365 days, was 8.7 mg/day (SD 7.1) (children: 6.2 mg/day [SD 4.1]; adolescents: 11.4 mg/day [SD 8.6]). Among 261 generalized MG patients, 52 (19.9%) received intensive treatments, with a higher proportion in adolescents than children (25.7% [35/136] vs. 13.6% [17/125]). IVIg was the most common intensive treatment (13.4% [35/261]), followed by steroid pulse therapy (10.3% [27/261]) and plasma exchange (3.8% [10/261]). Adolescent generalized MG had higher intensive care unit hospitalization rates than child generalized MG (8.8% [12/136] vs. 0.8% [1/125]), and all thymectomies occurred in adolescent patients (8.1% [11/136] vs. 0% [0/125]).
In Japanese real-world practice, about one in five children with generalized MG required intensive treatments, particularly among adolescents, while steroid-sparing immunosuppression was used in only one-third. These findings support close monitoring of adolescent generalized MG and highlight opportunities to optimize steroid-sparing management in juvenile generalized MG.CancerAccessCare/ManagementAdvocacy -
Clinical improvement of pemphigus-like mucocutaneous disease in a patient with CLL following initiation of zanubrutinib: a case report.2 weeks agoPemphigus is an autoimmune bullous disease. PNP/PAMS represents a rare subtype of autoimmune bullous disease, and typically associated with an underlying neoplasm, particularly lymphoproliferative disorders. Current management of PNP primarily involves glucocorticoids, immunosuppressants, and rituximab. This article reports a 67-year-old male who presented with generalized erythema, blisters, and oral erosions with pain for one month. Initially diagnosed with pemphigus, he received methylprednisolone treatment, which led to slight improvement in skin lesions but minimal resolution of oral ulcers. Based on the available clinical, histopathological, immunofluorescence, and serological findings, the patient was initially diagnosed with pemphigus vulgaris in the setting of CLL. However, the refractory oral mucositis, dual positivity for Dsg1 and Dsg3, and the underlying B-cell malignancy raised the concern for paraneoplastic pemphigus (PNP). Given that PNP-specific tests-including anti-plakin antibodies, indirect immunofluorescence on rat bladder, and immunoblotting-were not performed, a definitive diagnosis of PNP could not be established. Therefore, the condition was managed as "suspected CLL-associated PNP" Following the initiation of zanubrutinib in combination with ongoing corticosteroid therapy and CLL treatment, significant improvement of oral erosions was observed. In this case, the clinical improvement may be potentially related to zanubrutinib, but the concurrent corticosteroid therapy and the control of CLL likely contributed as well. If zanubrutinib did play a role, the mechanism might involve enhanced disease control or immune modulation. However, the current evidence is insufficient to draw a firm conclusion, and its true efficacy needs to be evaluated in further studies.CancerAccessCare/Management
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Impact of ALC Nadir and EDRIC on survival outcomes in limited-stage small cell lung cancer receiving chemoradiotherapy: a retrospective analysis comparing twice-daily and once-daily radiotherapy.2 weeks agoThis study aimed to evaluate the prognostic value of absolute lymphocyte count (ALC) nadir and estimated dose of radiation to immune cells (EDRIC) in patients with limited-stage small cell lung cancer (LS-SCLC).
This study analyzed 288 patients with LS-SCLC treated with chemoradiotherapy (CRT) between 2019 and 2023. ALC nadir and EDRIC were evaluated for prognostic value across patients receiving twice-daily (BID) and once-daily (QD) radiotherapy. Survival outcomes included overall survival (OS), progression-free survival (PFS), and brain metastasis-free survival (BMFS).
Despite lower lymphocyte counts during early treatment in the BID group, the ALC nadir was significantly higher in the BID group compared with the QD group (median ALC nadir: 0.60 × 109/L vs. 0.49 × 109/L, P < 0.001). In the BID group, patients with ALC nadir > 0.55 × 109/L showed significantly longer OS (median OS: NR vs. 46.7 months, P = 0.011), PFS (P = 0.022), and BMFS (P = 0.013) compared with those with ALC nadir ≤ 0.55 × 109/L. Patients in the high EDRIC group had significantly shorter PFS (P = 0.001) and BMFS (P = 0.003), and a trend toward shorter OS (P = 0.179). In the QD group, no significant differences were observed when stratified by ALC nadir or EDRIC (P > 0.05).
ALC nadir and EDRIC demonstrated prognostic significance in the BID group but not in the QD group, suggesting potential differences in radiation-associated immune effects between fractionation schedules.CancerChronic respiratory diseaseAccessCare/ManagementAdvocacy -
Latent profile analysis and influencing factors of health literacy among family caregivers of cancer patients.2 weeks agoTo understand the current status, categorical characteristics, and influencing factors of health literacy among family caregivers of cancer patients, providing evidence and guidance for enhancing caregivers' health literacy and caregiving capabilities to improve cancer patients' quality of life.
This study employed convenience sampling to select 287 family caregivers of cancer patients admitted to a tertiary-level Class A hospital in Weifang City between February and August 2025. A cross-sectional survey was conducted using a demographic questionnaire, the Chinese version of the Cancer Caregiver Health Literacy Scale, the General Self-Efficacy Scale, the Caregiver Sense of Efficacy Scale, and the Perceived Social Support Scale. Data analysis employed latent profile analysis of health literacy among family caregivers using Mplus 8.0 software, supplemented by descriptive analysis, univariate analysis, and multivariate logistic regression analysis via SPSS 25.0 to explore factors influencing health literacy.
A total of 271 valid questionnaires were retrieved. The mean health literacy score among family caregivers of cancer patients was 134 (120, 153), indicating a moderate level. The dimension "Processing Health Information" had the lowest mean score of 12 (9, 12). The dimension "Understanding Patient Needs and Preferences" scored highest at 18 (18, 22). Based on latent profile analysis, family caregivers of cancer patients were categorized into three groups: low health literacy (49.8%), moderate health literacy (25.4%), and high health literacy (24.7%). Multivariate logistic regression analysis revealed that age (OR = 9.445, 95% CI: 1.646-54.196), educational attainment (OR = 0.50, 95% CI: 0.003-0.777), occupation (OR = 0.116, 95% CI: 0.021-0.641), and other factors were significantly associated with health literacy categories among family caregivers of cancer patients (p < 0.05).
The overall health literacy level among family caregivers of cancer patients was moderate, with nearly half (49.8%) falling into the low health literacy group, though significant heterogeneity existed. Caregivers' health literacy was influenced by multiple factors, including age, educational attainment, economic status, self-efficacy, social support, and caregiving experience.CancerAccessCare/ManagementAdvocacyEducation -
Haematological toxicity associated with antineoplastic drugs: a pharmacovigilance analysis based on the FDA adverse event reporting system database.2 weeks agoAntineoplastic drugs are effective for malignant tumours, but they frequently cause severe adverse drug reactions (ADRs), which seriously affect treatment continuity and patient prognosis. Post - marketing real - world safety monitoring is therefore critical.
We conducted a retrospective pharmacovigilance study using FAERS (FDA Adverse Event Reporting System) data covering the period from 2004 Q1 to 2025 Q4. Safety signals were detected via disproportionate analysis, and associations were identified through LASSO and multivariable logistic regression, followed by an analysis of time to onset (TTO) for haematotoxicity.
A total of 347, 248 reports involving 367 antitumour drugs were analysed. Female patients (46.51%) outnumbered males (38.73%), with a median age of 59 years; patients aged 18 - 64.9 years represented 34.79% of the cohort. Fatal or life - threatening events were recorded in 78, 824 cases (22.70%). The leading drugs by report frequency were lenalidomide (ROR (Reporting Odds Ratio) = 1.51), methotrexate (ROR = 2.98), and rituximab (ROR = 3.69). Most events (61.65%) emerged within the first month, and median TTO differ significantly by sex. Furthermore, WSP analysis revealed that 26 of the top 30 drugs followed an early failure pattern.
Our findings reveal a strong signal of haematological toxicity associated with antitumour agents, supporting enhanced routine monitoring to mitigate clinical risks. Nevertheless, confirmation through pharmacoepidemiological studies with rigorous causality assessment is required, owing to the inherent limitations of the FAERS spontaneous reporting system.CancerAccessCare/ManagementPolicyAdvocacy -
Allogeneic hematopoietic stem cell transplantation is associated with improved survival compared with chemotherapy in patients with acute myeloid leukemia harboring favorable-risk genetic features at initial diagnosis: a single-center retrospective study.2 weeks agoAcute myeloid leukemia (AML) harboring favorable-risk genetic features at initial diagnosis (FR-AML) commonly achieves remission after chemotherapy, but the optimal post-remission strategy remains controversial. This study compared allogeneic hematopoietic stem cell transplantation (allo-HSCT) with chemotherapy and explored prognostic factors.
We retrospectively analyzed 190 patients with FR-AML treated at Fujian Medical University Union Hospital between 2012 and 2022. Patients were stratified into the chemotherapy (n = 118) and allo-HSCT (n = 72) groups according to their post-induction treatment. Survival outcomes, including overall survival (OS), leukemia-free survival (LFS), cumulative incidence of relapse (CIR), and non-relapse mortality (NRM), were assessed using Kaplan-Meier and competing-risk analyses. Multivariate Cox regression identified independent prognostic factors.
At 3 years, the allo-HSCT group had higher OS (86.0% vs. 61.3%, P < 0.0001) and LFS (59.2% vs. 49.3%, P = 0.039) than the chemotherapy group, with lower CIR (3.8% vs. 33.6%, P < 0.0001). Stratified analyses showed that allo-HSCT was associated with improved 3-year OS compared with chemotherapy alone in CR1-AML and R/R AML patients. In CR1-AML, the 3-year OS was 92.8% versus 73.6% (P = 0.0099), whereas in R/R AML, it was 73.5% versus 26.6% (P = 0.00015). By age, outcomes were comparable among patients aged <20 years, whereas allo-HSCT was associated with better survival in those aged 20-60 years. Among 66 transplanted patients with measurable residual disease (MRD) data, 3-year OS did not differ significantly between MRD-positive and MRD-negative groups (86.0% vs. 85.6%, P = 0.35).
Allo-HSCT was associated with improved survival and reduced relapse in FR-AML. This benefit extended beyond patients transplanted in CR1 to those who later develop R/R disease, in whom salvage allo-HSCT confers a marked survival advantage over chemotherapy. Deferred transplantation may remain feasible for selected patients when a suitable donor is available. Prospective studies are warranted to validate these findings and optimize post-remission treatment strategies in this population.CancerAccessCare/ManagementAdvocacy -
Prevalence and clinical relevance of negative histology after transsphenoidal surgery for pituitary neuroendocrine tumors.2 weeks agoNegative histology (NH), defined as normal pituitary tissue without histopathological evidence of tumor despite clinical, biochemical and radiological or functional evidence of a pituitary neuroendocrine tumor (PitNET), is well described in Cushing's disease but poorly characterized in other subtypes. We assessed its prevalence, mechanisms, and clinical implications.
We retrospectively analyzed 651 patients who underwent transsphenoidal surgery for a PitNET (1996-2023). Epidemiological analyses included the entire cohort; clinical and prognostic analyses were restricted to microadenomas of the three main functional subtypes (ACTH, PRL, GH).
NH occurred in 28.1% of ACTH-, 28.1% of PRL-, and 11.4% of GH-secreting tumors, and was strongly associated with microadenomas. Baseline and hormonal characteristics were similar between NH and PH cases, except for lower IGF-1 levels in GH NH tumors. At 6 weeks, remission rates were lower in NH for ACTH (60% vs. 93.2%, p < 0.01) and PRL (50% vs. 79.3%, p = 0.03), but similar in GH. In ACTH tumors, mislocalization accounted for 66.7% of unfavorable NH outcomes and may explain the excess of failures versus PH cases. Mislocalization was rare (6.7%) in PRL tumors and not reported in GH tumors.
NH is heterogeneous, arising from three mechanisms whose contributions vary by subtype. In ACTH tumors, mislocalization predominates and is associated with lower early remission. In PRL tumors, mislocalization is uncommon but may contribute to lower early remission. In GH tumors, NH likely reflects technical or histological limitations and carries a favorable prognosis.CancerAccessCare/ManagementAdvocacy -
Multimedia-assisted narrative intervention for existential distress among patients with advanced cancer: a one-group pre-post study.2 weeks agoPatients with advanced cancer often experience challenges related to life meaning, coping confidence, and death-related concerns, which may impair quality of life. This preliminary one-group quasi-experimental pre-post study examined whether meaning in life, terminal self-efficacy, and death anxiety changed after a multimedia-assisted narrative psychosocial intervention. The uncontrolled design was intended to identify preliminary within-participant signals and cannot establish intervention efficacy.
A one-group quasi-experimental pre-post study was conducted in three hospice care facilities. Of 50 recruited patients, 43 completed the 4-week program and post-intervention assessment. The intervention integrated weekly group storytelling sessions, individual interviews, daily educational dissemination, multimedia-supported reflection, and structured legacy-oriented activities. Outcomes were assessed using validated scales and analyzed with paired-samples t-tests, mean differences, 95% confidence intervals, Cohen's dz, and Holm-adjusted P values.
Meaning in life increased from 2.28 ± 0.65 to 2.70 ± 0.52 (mean difference = 0.42, 95% CI 0.33 to 0.51, dz = 1.49), and terminal self-efficacy increased from 2.38 ± 0.58 to 2.88 ± 0.47 (mean difference = 0.50, 95% CI 0.40 to 0.59, dz = 1.62). After scale-keying the T-DAS scores so that higher scores indicated greater death anxiety, death anxiety decreased from 3.11 ± 0.27 to 2.97 ± 0.29 (mean difference = -0.13, 95% CI -0.22 to -0.05, dz = -0.49). All Holm-adjusted P values for the three primary outcomes were < 0.01.
This preliminary one-group pre-post study observed favorable changes in meaning in life, terminal self-efficacy, and death anxiety following a multimedia-assisted narrative intervention. Because the study lacked a control group and used a small convenience sample, the findings should be interpreted as hypothesis-generating rather than evidence of intervention efficacy.CancerMental HealthAccessCare/ManagementAdvocacyEducation -
Distinct Internalization, Acoustic Behavior and Stress Responses to Nanobubbles in Pancreatic Cancer Cells.2 weeks agoPancreatic ductal adenocarcinoma (PDAC) is one of the most lethal cancers, characterized by an extremely low survival rate once diagnosed. Over 90% of cases harbor activating KRAS mutations, which are associated with metabolic reprogramming and altered endocytic activity. New treatment modalities are urgently needed. Phospholipid-shelled, gas-filled nanobubbles (NBs) are promising ultrasound (US) contrast agents and drug delivery vehicles, with potential applications in pancreatic cancer. However, their mechanisms of cellular internalization and the resulting biological responses need to be better understood before they can be effectively utilized as theranostic agents. Here, we analyzed the internalization kinetics, endocytic pathways, acoustic responses, and cell-stress-related protein expression in perfluorocarbon-filled NBs in two pancreatic cancer cell lines: KRAS-mutant PANC-1 and KRAS wild-type BxPC-3 cells showed distinct NB uptake profiles. Flow cytometry demonstrated approximately 1.5-fold higher TR-NB uptake in PANC-1 cells compared with BxPC-3 cells, suggesting enhanced cellular association and/or intracellular retention. Higher NB signal in PANC-1 cells was accompanied by weaker and less punctate LysoTracker staining, whereas BxPC-3 cells displayed brighter, more granular acidic vesicles, suggesting differences in intracellular trafficking and compartmentalization. Pharmacological inhibition of endocytosis revealed that clathrin-mediated endocytosis contributes predominantly to uptake in BxPC-3, whereas macropinocytosis appears to play a greater role in PANC-1 cells. Upon US exposure, NBs exhibited strong initial acoustic activity that declined over time. Analysis of stress-response-associated protein expression revealed cell-specific differences: PANC-1 cells exhibited higher levels of FABP-1, HSP60, SOD2, Thioredoxin-1, and SIRT2, whereas BxPC-3 cells displayed increased COX-2 and PON-2. Collectively, our findings demonstrate that NB internalization and fate differ between PDAC subtypes, potentially reflecting cell type dependent variations in endocytic, lysosomal and adaptive pathways, underscoring the importance of developing cell-tailored nanocarrier designs to enhance delivery precision and therapeutic efficacy.CancerAccess
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Intersectionality of Race, Rurality on Persistent Poverty Disparities in Cancer Mortality in Alabama.2 weeks agoCounties in persistent poverty (PP) have higher cancer-related mortality rates. Intersectionality with race and rurality is present; individuals identifying as Black residing in rural PP counties have the highest cancer-related mortality rates. However, heterogeneity exists throughout counties (e.g., demographics). To date, few studies evaluated the effect of small-area PP residence on individual-level survival and intersectionality with race and rurality.
This retrospective cohort utilized Alabama Statewide Cancer Registry data of adults with breast, prostate, colorectal, or lung cancer. The exposure was NCI-designated 2010 census tract-level PP (tracts with ≥ 20% population below poverty line for ≥ 30 years) at diagnosis and the outcome was overall survival from date of diagnosis to death or end of follow-up. Associations were estimated via sequential Cox proportional hazards models first with PP as the only predictor then adjusting for demographics (age, race, sex [colorectal, lung]), demographics plus clinical characteristics (stage, subtype [breast], and first course treatment), and finally demographics, clinical characteristics, and social determinants (rurality, insurance status). We also performed a decomposition analysis of the association between PP and survival through race and rurality.
In the fully adjusted model, living in a PP-tract was associated with higher risk of death among those with breast (hazard ratio [95% CI]: 1.18 [1.09-1.27]), prostate (1.21 [1.11-1.31]), and lung cancer (1.09 [1.05-1.14]) compared to living in a non-PP-tract. Race and rurality were independently associated with higher risk of death across cancers. Individuals identifying as Black residing in a rural PP-tract had the highest magnitude of risk for all cancers.
Living in a PP-tract is associated with higher risk of death among patients with breast, prostate, and lung cancer in Alabama. This association is complicated by race and rurality. Multilevel interventions tailored to local context may help overcome adverse effects of social characteristics on cancer outcomes.CancerAccessCare/ManagementAdvocacy