Pediatric Blood & Cancer[JOURNAL]
Sun
200 papers
Systemic health impact of cancer-associated extracellular vesicles and particles.
Wang G, Lucotti S, Bojmar L … +5 more , Tobias GC, Piszczatowski RT, Dror S, Zhang H, Lyden D
Nat Rev Cancer · 2026 Jul · PMID 42386999 · Publisher ↗
Cancer is a systemic disease that perturbs the homeostasis of host tissues and organs, exerting manifestations both locally and distantly. Extracellular vesicles and particles (EVPs) have a pivotal role in intercellular...
Cancer is a systemic disease that perturbs the homeostasis of host tissues and organs, exerting manifestations both locally and distantly. Extracellular vesicles and particles (EVPs) have a pivotal role in intercellular communication between tumours and the host by transferring bioactive cargo to recipient cells, contributing to the systemic effects of cancer. Tumour-derived EVPs prepare distant organs for future metastasis, by creating a pre-metastatic niche (PMN). Additionally, tumours manipulate multiple organ systems to support their growth and evade immune detection. This co-option of host systems leads to cascading dysfunction across multiple organs, ultimately compromising host survival. Here we review the diverse systemic impact of cancer-associated EVPs, including immune dysregulation in PMNs, thrombosis and cardiovascular disease, liver metabolic dysfunction, glucose metabolism disorders, cachexia, and paraneoplastic syndromes of the nervous system. Furthermore, we discuss the intricate communication between host-, diet- and microbiota-derived EVPs and cancer cells, highlighting the complex interplay mediated by these EVPs in cancer progression and anti-cancer treatment responses. We also explore the prospects of EVPs as a systemic therapeutic approach for anti-cancer treatment. Overall, this Review highlights the need to address the systemic effects of cancer and to adopt holistic approaches to cancer treatment, by simultaneously targeting the tumour and its multi-organ paraneoplastic effects. These strategies hold great promise in mitigating the broad impact of disease progression and comorbidities and ultimately improving the quality of life and survival of patients.
Pediatric cancer survival in the United States from 2001 to 2021.
Stevenson JM, Lupo PJ, Miller TP … +5 more , Dai S, Henley SJ, Tai E, Boring MA, Siegel DA
Cancer · 2026 Jul · PMID 42360091 · Publisher ↗
BACKGROUND: Approximately 15,000 United States (US) children and adolescents, 0-14 and 15-19 years old, respectively, are diagnosed with cancer annually. Although overall survival now exceeds 80%, cancer remains the seco...
BACKGROUND: Approximately 15,000 United States (US) children and adolescents, 0-14 and 15-19 years old, respectively, are diagnosed with cancer annually. Although overall survival now exceeds 80%, cancer remains the second leading cause of death in this population, and improvements have not been uniform across subgroups. METHODS: Five- and 10-year relative survival (RS) for cancer cases diagnosed during 2001-2021 among individuals <20 years old at diagnosis were calculated using the National Program of Cancer Registries database, covering 87.4% of the US population. Results were stratified by age, sex, race and ethnicity, US Census region, economic status, cancer type, stage, and diagnosis year. All-cause survival was calculated using the Kaplan-Meier method with log-rank testing. RESULTS: Among 272,279 pediatric cancers diagnosed from 2001 to 2021, 5-year RS was 84.7%, and 10-year RS was 82.3%. Five-year RS increased from 83.0% in 2001-2011 to 86.8% in 2012-2021. Ten-year RS was higher for females than males. By age, adolescents 15-19 years old had the highest RS, whereas infants had the lowest. Non-Hispanic Black patients had the lowest 5- and 10-year RS (79.2% and 76.3%, respectively) compared with non-Hispanic White patients (86.3% and 84.0%). RS was highest for patients in the top 25% of counties by economic status and in the Northeastern US Census region. Overall, all-cause 10-year survival was 81.7% and differed by sex, age, race and ethnicity, stage, and cancer subtypes. CONCLUSIONS: Pediatric cancer survival has continued to improve. However, differences persist by age, sex, race and ethnicity, economic status, geography, and cancer type.
Impact of precision oncology research in pediatric poor prognosis cancer: patient, parent and healthcare provider perspectives.
McCloskey N, Mahon P, Rassekh SR … +5 more , Hetherington K, Regier DA, Wakefield CE, Pollard S, Deyell RJ
BMC Cancer · 2026 Jun · PMID 42316091 · Full text
BACKGROUND: Comprehensive genomic analyses are increasingly accessible to children, adolescents and young adults (AYAs) with poor prognosis cancers. Challenges and successes of pediatric precision oncology studies from t...
BACKGROUND: Comprehensive genomic analyses are increasingly accessible to children, adolescents and young adults (AYAs) with poor prognosis cancers. Challenges and successes of pediatric precision oncology studies from the perspectives of AYA patients, parents and healthcare providers (HCPs) are poorly described. METHODS: Between March 2021 and May 2023, we interviewed AYA patients (12-21 years), parents and HCPs who participated in pediatric precision oncology studies for poor prognosis cancers in British Columbia. Interviews followed an investigator-developed semi-structured topic guide. Data were coded inductively and deductively by one qualitative researcher and one trainee, supported by two additional team members. Analytic themes were established using qualitative thematic analysis. RESULTS: We interviewed 9 AYAs, 10 parents, and 17 HCPs. We identified five analytic themes: importance of clear communication of study information between patients, families and multidisciplinary HCPs; a need to support disclosure, understanding and clinical integration of research results; barriers to accessing innovative therapy and mitigation strategies; approaches to managing parent, patient and HCP hopes and expectations; personal challenges and stressors related to participation. CONCLUSIONS: We highlight unmet needs and offer practical considerations for integrating precision oncology into clinical practice. Considerations include educating and supporting oncologists through genomics results disclosure, increasing engagement with multidisciplinary HCPs, streamlining access to study information and results, coordinating efforts to clinically validate results and access therapies, and establishing real-world outcome data to inform clinical decision-making. Implementation of these strategies will optimize care for patients and families who are navigating poor prognosis cancers.
Clinical profiling of AML1::ETO and KIT exon 17 mutation in pediatric AML by high-throughput drug sensitivity.
Hu Z, Tang X, Chen F … +10 more , Li T, Liu Y, Zhou G, Li Q, Liu S, Wang Y, Wen F, Mai H, Wang L, Liu S
BMC Cancer · 2026 Jun · PMID 42298546 · Full text
BACKGROUND: The favorable prognosis of pediatric AML1::ETO acute myeloid leukemia (AML) is well-established, yet the impact of co-occurring KIT mutations-particularly in exon 17-on clinical outcomes and chemosensitivity...
BACKGROUND: The favorable prognosis of pediatric AML1::ETO acute myeloid leukemia (AML) is well-established, yet the impact of co-occurring KIT mutations-particularly in exon 17-on clinical outcomes and chemosensitivity remains incompletely defined. METHODS: We analyzed the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) database comprising 957 pediatric AML patients to assess the clinical characteristics and prognosis of pediatric patients harboring AML1::ETO and KIT mutation using Kaplan-Meier survival analysis and Cox proportional hazards models. Subsequently, we performed experimental validation using bone marrow samples from 16 pediatric AML patients at Shenzhen Children's Hospital by high-throughput drug sensitivity (HDS) screening against therapeutic agents and whole transcriptome sequencing analysis of significant genes. Functional enrichment analysis of these differential expression genes was carried out by Gene Ontology. RESULTS: Analysis from the TARGET database revealed that while AML1::ETO AML generally carries a favorable prognosis, concomitant KIT exon 17 mutations significantly attenuate this survival advantage. Exploratory experimental validation in a limited cohort of AML1::ETO and KIT exon 17 mutation patients suggested a potential trend of broad drug resistance such as cytarabine and daunorubicin. Preliminary transcriptomic analysis identified upregulation of SOCS1, a negative regulator of the JAK-STAT pathway, as a potential feature of this resistant phenotype. Furthermore, elevated SOCS1 expression was associated with poor prognosis. CONCLUSIONS: We conclude that KIT mutations, especially exon 17, confer a high-risk phenotype in otherwise favorable pediatric AML1::ETO AML. Our exploratory data suggest this may be associated with a chemoresistant profile, potentially driven by SOCS1-associated JAK-STAT dysregulation. These findings highlight the necessity of refined risk stratification based on KIT exon profiling and support targeting the SOCS1/JAK-STAT axis to overcome therapy resistance.
Comparing the association between out-of-pocket cost burden and cost-related care avoidance among individuals with and without a history of cancer.
Liang X, Tay D, Ke Y … +8 more , Ng DQ, Patikorn C, Asche C, Chaiyakunapruk N, Kirchhoff AC, Chan RJ, Chan A, Tan CJ
Cancer · 2026 Jun · PMID 42290381 · Full text
INTRODUCTION: Out-of-pocket (OOP) cost is routinely captured by payers and health systems and may help identify individuals at risk of cost-related care avoidance. This study aimed to evaluate the relationship between OO...
INTRODUCTION: Out-of-pocket (OOP) cost is routinely captured by payers and health systems and may help identify individuals at risk of cost-related care avoidance. This study aimed to evaluate the relationship between OOP cost burden relative to household income (OOP:HHI) and cost-related care avoidance and to assess whether this relationship differs by cancer history. METHODS: A retrospective cross-sectional study was conducted leveraging data from multiple survey waves of the Understanding America Study (2015-2024). Adults aged ≥18 years with data on OOP health care expenditure, household income, and cost-related care avoidance were included. Mixed-effects logistic models with random intercepts were used to assess associations between OOP:HHI and care avoidance, adjusting for respondent characteristics. Effect modification by cancer history was examined. Classification performance of OOP:HHI alone versus multivariable models was evaluated using receiver operating characteristic curves. RESULTS: The analysis included 21,299 responses from 10,811 respondents, of which 2180 responses were from 1052 respondents with cancer history. Higher OOP:HHI was independently associated with increased odds of cost-related care avoidance (adjusted odds ratio per 1% increase = 1.03; 95% CI, 1.02-1.04) with similar trends in both cancer and noncancer subgroups. OOP:HHI alone showed modest discrimination, whereas multivariable models demonstrated excellent performance in identifying respondents who indicated care avoidance. CONCLUSION: Higher OOP cost burden was associated with cost-related care avoidance regardless of cancer history, but cost burden alone was insufficient for risk identification. Integrating OOP costs with routinely available patient characteristics may better identify individuals at risk of care avoidance for targeted interventions to mitigate financial toxicity.
Expression patterns of GPX4 and LINC00618 across disease phases in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL): an exploratory study.
Shayanmanesh M, Ahmadi SE, Faranoush M … +4 more , Safa M, Nourbakhsh SM, Jaafarian AS, Amini A
BMC Cancer · 2026 Jun · PMID 42243765 · Full text
BACKGROUND: Acute lymphoblastic leukemia (ALL) remains a significant challenge in pediatric malignancies, particularly in cases of relapse and treatment resistance. This study aimed to investigate the expression patterns...
BACKGROUND: Acute lymphoblastic leukemia (ALL) remains a significant challenge in pediatric malignancies, particularly in cases of relapse and treatment resistance. This study aimed to investigate the expression patterns of ferroptosis-related genes, GPX4 and LINC00618, across different disease phases in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL). METHODS: This case-control study analyzed 82 samples from pediatric BCP-ALL patients comprising 14 new cases, 29 in remission, and 17 relapsed cases alongside 22 healthy controls. Expression levels of GPX4 and LINC00618 were evaluated using quantitative real-time PCR. The relationship between gene expression and clinical parameters was assessed. Statistical analyses were performed employing parametric or nonparametric tests. RESULTS: GPX4 expression was significantly elevated in ALL patients compared to controls (p < 0.01), with notably higher levels in new cases (p < 0.01) and relapsed patients (p < 0.05). Similarly, LINC00618 exhibited significant upregulation in ALL patients (p < 0.05), particularly in new cases (p < 0.01) and relapsed patients (p < 0.05). Both genes demonstrated reduced expression during the remission. External validation using the GSE13159 dataset demonstrated a consistent direction of increased GPX4 expression in ALL samples compared to controls. CONCLUSIONS: Our findings suggest phase-associated expression patterns of GPX4 and LINC00618 in pediatric BCP-ALL. The consistent direction of GPX4 expression observed in external validation supports its potential relevance in leukemic biology. These results provide a basis for further studies to clarify the role of ferroptosis-related pathways in disease progression and therapeutic response.
The landscape of pediatric hepatocellular carcinoma and fibrolamellar carcinoma: A report from the Children's Hepatic tumors International Collaboration (CHIC).
O'Neill AF, Maibach R, Krailo MD … +13 more , Piao J, Lopez-Terrada D, Alaggio R, Tanaka Y, Häberle B, Aronson DC, Hiyama E, Vokuhl C, Schmid I, Malogolowkin MH, Meyers RL, Czauderna P, Rangaswami A
Cancer · 2026 Jun · PMID 42228354 · Publisher ↗
BACKGROUND: Pediatric hepatocellular carcinoma (HCC) and fibrolamellar carcinoma (FLC) have historically been studied on prospective trials in aggregate with hepatoblastoma (HB). It is now recognized that both HCC and FL...
BACKGROUND: Pediatric hepatocellular carcinoma (HCC) and fibrolamellar carcinoma (FLC) have historically been studied on prospective trials in aggregate with hepatoblastoma (HB). It is now recognized that both HCC and FLC have unique histologies, characteristic genomics, and a more aggressive clinical course than HB. It is anticipated that data analyzed from patients treated on previously conducted international trials will inform current and future approaches to the study of these rare diseases. METHODS: The Children's Hepatic tumors International Collaboration houses data from clinical trials conducted by three global consortia as well as international registries. Data from patients with HCC or FLC who were enrolled on historic trials designed for patients with HB were analyzed for patient demographics, disease characteristics, treatment, and outcomes. RESULTS: The authors identified 236 patients with pediatric HCC or FLC who were enrolled on trials conducted in the United States, Europe, and Japan. As anticipated, HCC and FLC predominantly affected adolescent patients, and outcomes were generally related to extent of disease and resectability. Independent of histology, upfront resection (p = .004) predicted superior survival, likely reflecting the inadequacies of systemic agents to control disease. Outcomes were comparable across studies, with a 5-year survival rate of 36%, independent of chemotherapy received. Survival was equivalent for patients with HCC and FLC after 7-year follow-up. CONCLUSIONS: Treatment of pediatric HCC and FLC remains challenging. The current results support risk-stratification methods currently under study prospectively and the independent study of these tumors in the future.
Treatment patterns and survival outcomes of Ewing sarcoma in Ethiopia: a five-year retrospective analysis from a tertiary hospital.
Gizaw T, Fikadu A, Arega G … +4 more , Tesfaye B, Teshome D, Worku S, Hailemariam F
BMC Cancer · 2026 Jun · PMID 42226167 · Full text
BACKGROUND: Ewing sarcoma is a malignant small round-cell sarcoma of bone or soft tissue that is highly responsive to modern multimodal therapy and considered a highly curable disease in developed countries. However, sur...
BACKGROUND: Ewing sarcoma is a malignant small round-cell sarcoma of bone or soft tissue that is highly responsive to modern multimodal therapy and considered a highly curable disease in developed countries. However, survival remains poor in developing countries. This study aims to assess treatment patterns and survival outcomes of Ewing sarcoma at Tikur Anbessa Hospital. METHODS AND MATERIALS: We conducted a retrospective study on all patients diagnosed with Ewing sarcoma at Tikur Anbessa Hospital between November 15, 2018, and November 15, 2023. Descriptive statistics and Kaplan-Meier survival analysis were used to analyze the data. RESULT: The median age at diagnosis was 19 years, with most of the patients (67.2%) in the age range of 8-25 years. The main modality of treatment for localized disease was chemotherapy combined with surgery, whereas chemotherapy alone was used for metastatic disease. The Kaplan-Meier estimated 1-year and 3-year overall survival (OS) rates for the entire cohort were 96% and 68%, respectively, and the corresponding 1-year and 3-year event-free survival (EFS) rates were 70% and 40%, respectively. A sensitivity analysis treating loss to follow-up as events reduced the 3-year overall survival from 68% to 55% and the 3-year event-free survival from 40 to 33%. The 3-year OS and EFS rates were 89% and 76% for localized disease versus 10% and 0% for metastatic disease. This difference was statistically significant (log-rank p < 0.001). Small tumor size (≤ 8 cm), surgical intervention, and localized stage were associated with statistically significant improvements in OS and EFS, whereas pelvic location was associated with a poorer prognosis. CONCLUSION: Ewing sarcoma at our institution mainly affects adolescents and young adults. Survival is poor, especially for metastatic disease, while localized tumors, smaller size, and surgery are associated with better survival. Therefore, early diagnosis and improved multimodal treatment are needed to improve survival.
Scientific highlights and perspectives from the International Inflammatory Breast Cancer Symposium 2025.
Nicolò E, Bertucci F, Boussen H … +20 more , Curigliano G, Fraser V, Gerratana L, Krishnamurthy S, Le-Petross HT, Lim B, Lynce F, Lucci A, Lucotti S, Manai M, Merajver S, Missal N, Nasrazadani A, Soliman A, Valenza C, Van Berckelaer C, Van Laere S, Woodward WA, Cristofanilli M, Devi GR
Cancer · 2026 May · PMID 42133847 · Publisher ↗
Inflammatory breast cancer (IBC) is a rare yet highly aggressive subtype of breast cancer, characterized by distinct clinical features, rapid progression, and complex biology. Despite decades of research, patient outcome...
Inflammatory breast cancer (IBC) is a rare yet highly aggressive subtype of breast cancer, characterized by distinct clinical features, rapid progression, and complex biology. Despite decades of research, patient outcomes remain poor, underscoring the need for innovative approaches. The International IBC Symposium 2025 convened global experts to discuss advances in the field and to outline strategic priorities for research and clinical care. This report summarizes the key themes discussed. A major topic was IBC biology, with particular emphasis on tumor emboli formation and tumor microenvironment interactions, which shape the distinctive characteristics of the disease. Updates in molecular and immune profiling were presented, illustrating how these data may guide personalized therapeutic approaches. From a therapeutic standpoint, the importance of trimodal therapy in patients with nonmetastatic IBC was reinforced, while highlighting the need to investigate novel agents, rational drug combinations, and biologically informed strategies to improve outcomes. Additional sessions addressed optimization of locoregional management, even for selected patients with metastatic disease. Experts also discussed emerging technologies, including spatial transcriptomics, artificial intelligence tools, and liquid biopsy, as promising methods to advance disease characterization, facilitate earlier detection, and refine disease monitoring. Finally, a common theme across presentations was the critical importance of expanding awareness and education, as well as fostering global collaboration through the IBC International Consortium to standardize diagnosis, accelerate translational research, and ensure equitable access to care. Collectively, these efforts aim to redefine the clinical trajectory of IBC and move the field closer to achieving durable disease control and, ultimately, curative outcomes.
ITPP in early pancreatic zebrafish xenografts mildly impacts tumor cell death without interfering with vascular normalization.
Abou Khouzam R, Amorim F, Rifath A … +9 more , Thaliffdeen L, Ragupathi P, Nawafleh H, Povoa V, Cruz-Ribeiro M, Lehn JM, Fior R, Limani P, Chouaib S
BMC Cancer · 2026 May · PMID 42098650 · Full text
BACKGROUND: Myo-inositol trispyrophosphate (ITPP) is an anti-hypoxic small molecule that acts as an allosteric effector of hemoglobin in erythrocytes. To capture the early and dynamic effects of ITPP on tumor vasculature...
BACKGROUND: Myo-inositol trispyrophosphate (ITPP) is an anti-hypoxic small molecule that acts as an allosteric effector of hemoglobin in erythrocytes. To capture the early and dynamic effects of ITPP on tumor vasculature and cell viability, we employed a rapid zebrafish xenograft model of pancreatic ductal adenocarcinoma (PDAC) that enables real-time, single-cell-resolution imaging. METHODS: Two PDAC cell lines were used to establish xenografts in wildtype and transgenic zebrafish lines. A double transgenic that permits the visualization of both vessels and erythrocytes was included to determine the impact of ITPP on vascular function. The effects of ITPP alone and in combination with multimodal chemotherapy (FOLFIRINOX) on PDAC tumor proliferation, apoptosis and size were investigated using whole-mount immunostaining and confocal microscopy. RESULTS: Within a short treatment window, ITPP produced a detectable enhancement in the number of xenografts with erythrocyte-positive tumor-associated vessels, suggesting an early trend toward vessel functionality. This mild effect, however, came with no measurable changes in vessel architecture. While ITPP did not impact the cytotoxic potency of FOLFIRINOX, ITPP-treated xenografts displayed enhanced apoptosis in vivo. This came without any cytotoxicity in vitro, indicating that its mild pro-apoptotic activity is mediated indirectly, presumably through the tumor microenvironment (TME). CONCLUSIONS: Overall, we could not detect major effects of ITPP in the zebrafish xenograft model, possibly due to the lack of a hypoxic TME. This illustrates the model's shortcomings when investigating oxygen-modulating agents. We propose that experiments such as pre-treatment of tumor cells with hypoxic agents could overcome this, potentially expanding the utility and versatility of the zebrafish model.
Drug development trajectory of anticancer drugs after initial pediatric-eligible trials.
Martin SD, Pitafi D, Bourgeois F … +1 more , DuBois SG
Cancer · 2026 May · PMID 42046241 · Publisher ↗
BACKGROUND: Attrition of new therapies is a major concern in oncology drug development. Little is known about subsequent drug development milestones once an oncology agent has entered clinical testing in children and ado...
BACKGROUND: Attrition of new therapies is a major concern in oncology drug development. Little is known about subsequent drug development milestones once an oncology agent has entered clinical testing in children and adolescents. METHODS: This study identified 191 cancer drugs that first entered clinical trials between 2005 and 2020 and for which patients <18 years were eligible. The authors tracked subsequent drug development and regulatory milestones through 2025. They calculated Kaplan-Meier cumulative incidence rates of reaching each milestone, and they calculated Cox hazard rates according to features of the drug and trial characteristics. RESULTS: The majority (62.8%) of the 191 identified drugs were small molecule inhibitors. The majority of trials evaluated single agents (66.5%) and were multicenter trials (77.9%). At 10 years from first pediatric-eligible trials, the cumulative incidence rates of subsequent phase 1, phase 2, and phase 3 trials were 56.1%, 63.0%, and 17.7%, respectively. Of 191 drugs, 71 (37.2%) had no new trials that allowed patients <18 years of age for 5 or more years from first pediatric-eligible trial. For drugs not already approved at time of initial pediatric-eligible trial, the 10-year cumulative incidence rates for subsequent pediatric Food and Drug Administration and European Medicines Agency approval were 12.0% and 5.6%, respectively. Initial trial phase and drug regulatory status at time of initial pediatric-eligible trial were the most consistent determinants of achieving subsequent drug development milestones. CONCLUSIONS: Oncology drugs entering testing in children and adolescents are at high risk of attrition, including low rates of subsequent late phase trials and pediatric regulatory approvals.
IL-8 and sCD40L predict platelet refractoriness and survival of pediatric patients with solid tumors receiving chemotherapy.
Zhou C, Chen X, Xu J … +3 more , Ma J, Lin J, Wang H
BMC Cancer · 2026 Apr · PMID 42032548 · Full text
OBJECTIVE: To assess inflammation, endothelial damage, and platelet activation during platelet transfusion refractoriness (PTR) in pediatric patients with solid tumors. METHODS: A cohort of 36 PTR and 38 Non-PTR pediatri...
OBJECTIVE: To assess inflammation, endothelial damage, and platelet activation during platelet transfusion refractoriness (PTR) in pediatric patients with solid tumors. METHODS: A cohort of 36 PTR and 38 Non-PTR pediatric patients with solid tumor was enrolled. Patients received standard chemotherapy and platelet transfusion according to guidelines. Levels of inflammatory mediators (IL-1β and IL-8, etc.), endothelial damage marker (vWF), and platelet activation markers (sP-selectin, sCD40L and RANTES) in pre-transfusion plasma were quantified by flow cytometry and ELISA. Associations with PTR, bleeding events, and progression-free survival (PFS) were analyzed by multivariable regression and Cox models. RESULTS: The PTR group showed significantly high levels of sP-selectin (14.1 vs. 6.0 ng/mL), sCD40L (293.4 vs. 206.2 pg/mL), vWF (231.7 vs. 162.3 µg/mL), IL-1β (67.4 vs. 38.9 pg/mL), and IL-8 (61.0 vs. 29.2 pg/mL). Multivariable analysis identified vWF (OR = 1.006), IL-1β (OR = 1.013), and IL-8 (OR = 1.021) as independent predictors of PTR. Predictive model combining these three markers with malignancy risk stratification significantly improved the predictive ability of PTR compared to clinical Model alone (AUC: 0.846 vs. 0.692, P = 0.003). Clinically, PTR patients had delayed platelet recovery (10.2 vs.7.3 days), higher bleeding (36.1% vs. 13.2%), and reduced 500-day PFS (41.5% vs. 55.2%). Elevated sP-selectin (≥ 10.1 ng/mL) and sCD40L (≥ 234.5 pg/mL) predicted bleeding. After adjustment for malignancy risk stratification, high sCD40L (HR = 3.853, 95% CI: 1.097–13.525) remained independently associated with lower PFS, whereas IL ‑ 8 showed a trend but was not statistically significant. CONCLUSION: Elevated IL-8 and sCD40L are novel predictors of PTR and survival prognostic in pediatric patients with solid tumors undergoing chemotherapy. IL-1β, IL-8 and vWF are identified as independent risk factors for PTR. sP-selectin and sCD40L are associated with increased bleeding risk, and high sCD40L is independent predictors of lower 500-day PFS.
Cancer risk in children with congenital anomalies.
Lupo PJ, Plon SE
Nat Rev Cancer · 2026 Jun · PMID 42020808 · Publisher ↗
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Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety study.
Ouyang H, Yao H, He C … +8 more , Zhou Y, Dai J, Wang X, Zeng J, Liu C, Zhong K, Xiao J, Liu X
BMC Cancer · 2026 Mar · PMID 41913125 · Full text
BACKGROUND: This study evaluated the efficacy and safety of dabrafenib or trametinib in pediatric Langerhans cell histiocytosis (LCH) patients with skeletal involvement, for whom conventional therapies offer limited bene...
BACKGROUND: This study evaluated the efficacy and safety of dabrafenib or trametinib in pediatric Langerhans cell histiocytosis (LCH) patients with skeletal involvement, for whom conventional therapies offer limited benefit. METHODS: A single-center retrospective analysis was conducted on 30 children with pathologically confirmed, bone-involved LCH who received dabrafenib or trametinib due to severe, refractory, or chemotherapy-intolerant disease. Primary endpoints were imaging-based objective response rate (ORR) and disease control rate (DCR). Progression-free survival (PFS) was defined as time from targeted therapy initiation to disease progression (AD-Mixed/AD-Worse) or last follow-up, with AD-Better/AD-Stable considered disease control, including subgroup analysis by BRAF mutation status (V600E vs non-V600E). RESULTS: The median age at diagnosis was 3.1 years with a male-to-female ratio of 2:1. The BRAF V600E mutation was detected in 27 patients (90.0%), with two patients harboring BRAF non-V600E mutations and one patient carrying a MAP2K1 mutation. The median duration of targeted therapy was 17.7 months. At last follow-up, 23.3% (7/30) achieved active disease-better status, and 60.0% (18/30) had active disease-stable status, resulting in an ORR of 23.3% and a DCR of 83.3%. All patients survived. Notably, patients with non-V600E driver mutations (BRAF class II or MAP2K1, cases 22, 26, 28) received MEK inhibitor therapy (trametinib) and derived clinical benefit, highlighting the importance of precise genomic characterization. Drug-related adverse events occurred in 20.0% (6/30) of patients, consisting of mild cutaneous toxicities (e.g., rash, paronychia). CONCLUSIONS: In pediatric LCH with bone involvement, particularly in refractory or chemotherapy-intolerant cases, short- to intermediate-term targeted therapy with dabrafenib or trametinib achieved a high disease control rate (83.3%) with a favorable safety profile. Notably, patients with non-V600E driver mutations (BRAF class II or MAP2K1) derived clinical benefit from MEK inhibition, underscoring the importance of comprehensive genomic testing. These real-world findings support the use of targeted therapy in this challenging population.
Sun protection and skin cancer screening after childhood cancer-A report from the Swiss Childhood Cancer Survivor Study.
Nigg C, Žarković M, Jörger P … +7 more , Tinner EME, Mazzara C, Brack E, Castle P, Navarini A, Schindera C, Kuehni CE
Cancer · 2026 Apr · PMID 41906227 · Full text
BACKGROUND: Childhood cancer survivors (CCS) face elevated skin cancer risk, especially after radiotherapy or hematopoietic stem cell transplantation (HSCT). The authors evaluated the prevalence and predictors of sun pro...
BACKGROUND: Childhood cancer survivors (CCS) face elevated skin cancer risk, especially after radiotherapy or hematopoietic stem cell transplantation (HSCT). The authors evaluated the prevalence and predictors of sun protection, sunburn, and physician skin examination (PSE) among CCS in Switzerland. METHODS: The authors surveyed CCS diagnosed <21 years and surviving ≥5 years after diagnosis about sun protection, sunburns during last summer, and PSE within the last year. They retrieved cancer-related data from the Swiss Childhood Cancer Registry and used multivariable logistic regression, stratified by age group, to identify predictors. RESULTS: The authors included 1048 children (5-15 years), 572 adolescents (16-19 years), and 1959 adults (≥20 years). Regular sun protection was reported by 89% of children, 65% of adolescents, and 77% of adults, and sunburns by 23%, 49%, and 43%, respectively. PSE prevalence among those treated with radiotherapy was 21%, 18%, and 17%, and among HSCT recipients 36%, 28%, and 28%, respectively. Radiotherapy was unrelated to sun protection and PSE, but associated with fewer sunburns (odds ratio [OR], 0.63-0.77). HSCT recipients were more likely to have attended a PSE (OR, 2.06-3.75), but not radiotherapy recipients. Across age groups, survivors born more recently were less likely to protect from sun (OR, 0.94-0.97) and more likely to report sunburn (OR, 1.04-1.14). CONCLUSION: Sunburn prevalence was high despite good sun protection. Only few at heightened risk for skin cancer due to their treatment history attend PSEs as recommended by the Children's Oncology Group. Health care practitioners should systematically integrate yearly PSE after radiotherapy or HSCT and encourage consistent sun protection, particularly among younger generations and adolescents. TRIAL REGISTRATION: Clinicaltrials.gov (NCT03297034).
Comparison of clinical, pathological and genomic characteristics between Lynch and non-Lynch patients with MMRd endometrial carcinoma.
Mo H, Zhang W, Wei J … +23 more , Chen J, He J, Zeng X, Liu B, Huang J, Ren L, Li R, Huang X, Chen X, Liu Q, Ren D, Pan T, Qing X, Chen Y, Zheng Y, Li X, Deng Y, Li H, Xian D, Ouyang N, Zhang X, Zhong Q, Ruan X
BMC Cancer · 2026 Mar · PMID 41872806 · Full text
BACKGROUND: Lynch syndrome (LS) is a common hereditary cancer predisposition syndrome caused by pathogenic germline mutations in MMR genes. This study aimed to conduct LS screening in a South Chinese Endometrial carcinom...
BACKGROUND: Lynch syndrome (LS) is a common hereditary cancer predisposition syndrome caused by pathogenic germline mutations in MMR genes. This study aimed to conduct LS screening in a South Chinese Endometrial carcinoma (EC) cohort to identify hereditary mutation profiles. METHODS: Molecular classification was performed on 201 EC patients from South China. Sixty cases were identified as MMR deficiency (MMRd) EC. Tumor tissues from these MMRd EC cases were analyzed for MLH1 promoter methylation. Whole-exome sequencing (WES) of blood samples was conducted to detect germline mutations. RESULTS: There are certain differences among classification based solely on next-generation sequencing (NGS), classification based on the combination of NGS and IHC, and classification based solely on IHC. LS was diagnosed in 21.67% of patients with MMRd EC. Among patients showing MLH1 protein loss by IHC, 90.0% exhibited MLH1 promoter methylation. WES revealed that LS-associated pathogenic mutations predominantly involved MSH6 and MSH2, with MSH6 being the most frequent. Patients harboring MSH6 pathogenic mutations had a significantly older average age at EC diagnosis compared to those with MSH2 mutations (P = 0.014). Three newly identified pathogenic MMR gene mutations were identified among 13 LS patients. CONCLUSIONS: Patients with adequate financial resources should undergo combined testing of microsatellite instability (MSI) status and MMR protein IHC. Patients with MLH1 promoter methylation, those with microsatellite stability (MSS), and those with serous carcinoma of the EC may all need to undergo LS screening.
Evaluating functional hyposplenism in pediatric hodgkin lymphoma: the role of marginal zone b cells post-radiotherapy.
Kaplan Kilic S, Kiykim A, Burtecene N … +6 more , Genc DB, Kasim MS, Turkkan E, Emre S, Ergen SA, Ocak S
BMC Cancer · 2026 Mar · PMID 41862843 · Full text
BACKGROUND: Immune deficiencies are common in Hodgkin lymphoma (HL) patients during and after treatment, and splenic irradiation is one of the potential causes. This study investigates long-term radiation-induced functio...
BACKGROUND: Immune deficiencies are common in Hodgkin lymphoma (HL) patients during and after treatment, and splenic irradiation is one of the potential causes. This study investigates long-term radiation-induced functional hyposplenism and immune dysfunction in pediatric patients with HL. METHODS: This cross-sectional study included 20 patients with HL who underwent splenic irradiation, 20 patients with splenectomy, and 20 healthy controls. Demographic and clinical data were collected, and venous blood samples were analyzed for immune dysfunction, including flow cytometry, and B-lymphocyte subsets. RESULTS: HL group had lower non-class switched memory B cells compared to healthy controls (p = 0.0001). No significant differences were found in the class-switched memory B cells, naïve B cells and MZBs (p = 0.07, p = 0.061 and p = 0.618, respectively). MZBs were significantly lower in the splenectomy group than in the HL group (p = 0.034), while no significant differences between the two groups were observed in other B-cell subsets. Non-class switched memory B cells and MZBs were numerically lowest in the splenectomy group, followed by the HL group, and highest in the healthy control group. In the splenectomy group, both the non-class switched memory B cells and the MZBs were lower compared to healthy controls (p = 0.001 and p = 0.0001, respectively). No statistically significant dose-response relationship was observed between the radiation dose and the MZBs (p = 0.325). CONCLUSION: As a novel contribution, our findings highlight the presence of underrecognized functional hyposplenism in pediatric HL patients treated with splenic irradiation and provide a basis for future prospective studies evaluating immune recovery in childhood cancer survivors.
Consensus recommendations in the management of jaw (gnathic) osteosarcoma.
Dietz MS, Davis LE, Prasad PK … +20 more , Campbell SR, Reith JD, Olsen MR, Kilpatrick SE, Ku JA, Silver NL, Park EP, Tirtei E, Meazza C, Murphy ES, Mallory NC, Palmerini E, Kager L, Hol MLF, van Ewijk R, Lopez J, Elliott LA, Shepard DR, Gupta A, Trucco MM
Cancer · 2026 Mar · PMID 41717876 · Full text
Gnathic osteosarcoma (OS), which includes mandibular and maxillary jaw OSs, account for 6%-9% of OS. Single-institutional and multi-institutional retrospective studies, as well as population-level databases, suggest that...
Gnathic osteosarcoma (OS), which includes mandibular and maxillary jaw OSs, account for 6%-9% of OS. Single-institutional and multi-institutional retrospective studies, as well as population-level databases, suggest that clinical differences exist among gnathic OS and OS of other sides, including other craniofacial OS. To date, no specific prospective studies of gnathic OS have been reported, and aspects of clinical management are controversial. Some elements of care are aligned with extragnathic OS, e.g., margin negative (R0) surgery, whereas others, such as chemotherapy and radiation, are not clearly defined. The authors reviewed the available literature for the diagnosis, treatment, and supportive/survivorship care patients with gnathic OS and offer consensus statements for the comprehensive management of this rare disease.
Shifting toward chronicity: The new reality of chronic treatments and health conditions among adolescent and young adult patients with cancer.
Indini A, Toss A, Razzaboni E … +8 more , Bagliacca EP, Zucchetti G, Quarello P, Mascarin M, Cologgi M, Del Vecchio M, Peccatori F, Ferrari A
Cancer · 2026 Mar · PMID 41717840 · Publisher ↗
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Adolescents' perceptions and lived experiences of cancer patient-hood: a qualitative study.
Akambasisa M, Kemigisha E, Carpenter K … +2 more , Rukundo GZ, Atwiine B
BMC Cancer · 2026 Feb · PMID 41709196 · Full text
BACKGROUND: Adolescents’ perceptions and lived experiences regarding their cancer diagnosis significantly influence their quality of life and treatment adherence. To inform the formulation of tailored interventions, we e...
BACKGROUND: Adolescents’ perceptions and lived experiences regarding their cancer diagnosis significantly influence their quality of life and treatment adherence. To inform the formulation of tailored interventions, we explored these factors among adolescents receiving care at a sub-Saharan African health facility in Western Uganda. METHODS: We conducted a qualitative study from July 2022 to December 2022 at Mbarara Regional Referral Hospital in western Uganda. In-depth interviews were held with 30 adolescents aged 10–17 years who had been diagnosed with cancer. NVivo 12 software was used to develop a codebook and coding framework to generate themes inductively emerging from the data that aligned with the study objectives. Ethical approval was obtained from the Research and Ethics Committee of Mbarara University of Science and Technology. RESULTS: Participants had a median age of 13.5 years; 19 were male. The diagnoses included leukemia (n = 13), lymphoma (n = 10), and solid tumors (n = 7). Initial perceptions regarding their diagnosis were predominantly negative but improved over time as they received information and improved on treatment. Perceptions were poorer among those who responded poorly to treatment and those who had had extremely negative experiences. Negative experiences included body disfigurement, social challenges, emotional distress, physical pain, and interrupted education. Positive experiences included improvement in symptoms and support from health workers and their families. CONCLUSION: Adolescents initially exhibit poor perceptions towards their cancer diagnosis, which tend to improve with clinical improvement on treatment and support. Their experiences and perceptions are mixed, highlighting the need for specialized/ tailored education and counselling services to address knowledge gaps obtained from the research findings, and hence improve overall care outcomes among the study population.
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