Pediatric Blood & Cancer最新文献

Introduction: Rectal adenocarcinoma is exceptionally rare in children and is now recognized as a distinct entity from colon cancer, thereby requiring different treatment approaches. Differences between the pediatric and adult populations have not been previously explored. This study aims to characterize tumor biology, disease presentation, treatment, and survival outcomes in pediatric and adolescent rectal cancer with the goal of improving care in this population.

Methods: A retrospective analysis was conducted using the National Cancer Database (NCDB) for patients aged 0-90 years old diagnosed with rectal adenocarcinoma between 2004 and 2019. Patients were stratified into three age cohorts: pediatric and adolescents (PEDS) (≤21 years old), young adults (YAs) (22-49 years old), and older adults (OAs) (≥50 years old). Chi-squared, multivariate regression, Kaplan-Meier analysis, and Cox regression were performed.

Results: A total of 181,246 patients (127 PEDS, 34,552 YAs, and 146,567 OAs) were included. A greater proportion of PEDS presented with poorly differentiated or undifferentiated tumors. PEDS more commonly presented with Stage III or IV disease. PEDS had equivocal overall survival (OS) compared to OA in all stages and YA in Stage IV disease. PEDS had worse OS compared to YA in Stage I (hazard ratio [HR]: 3.33, confidence interval [CI]: 1.49-7.42, p = 0.003), Stage II (HR: 3.01, CI: 1.62-5.60, p < 0.001), and Stage III (HR: 2.41, CI: 1.69-3.44, p < 0.001) disease.

Conclusion: PEDS with rectal adenocarcinoma present with more aggressive, advanced disease and often worse survival compared to younger adults. The findings suggest distinct biological behavior of pediatric and adolescent rectal cancer, emphasizing the need for further research in this population.

Objectives: To identify predictors of chronic ITP (cITP) and to develop a model based on several machine learning (ML) methods to estimate the individual risk of chronicity at the timepoint of diagnosis.

Methods: We analyzed a longitudinal cohort of 944 children enrolled in the Intercontinental Cooperative immune thrombocytopenia (ITP) Study Group (ICIS) Children's Initiative. cITP was defined as a platelet count <100 × 10⁹/L at 12 months post diagnosis. Thirty-six clinical and laboratory variables collected at diagnosis were evaluated, and key predictors were selected using ML approaches. Model performance was assessed by the area under the receiver operating characteristic (ROC) curve.

Results: cITP developed in 28.7% of patients. Six variables were identified as the most informative predictors of chronicity. The ML model achieved an area under the ROC curve of 73.7% for predicting cITP at diagnosis.

Conclusions: ML-based prediction models can identify children at increased risk for cITP at the time of diagnosis. Integration of such tools into prospective registries may enhance prognostic accuracy and support individualized clinical decision-making.

Background: Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma affecting children and young adults, but few reports describe its presentation and outcomes in Africa.

Procedure: We conducted a retrospective chart review of patients of all ages with RMS presenting to the Butaro Cancer Center of Excellence in Rwanda between July 2012 and June 2022.

Results: Fifty-nine patients with histopathologically confirmed RMS were treated using Intergroup RMS Study Group protocols adapted to the local context. Median age was 9.1 years (interquartile range [IQR] 3.5-16.6); 54.2% were female. Median duration from symptom onset to presentation was 5 months (IQR 4-7). Most had embryonal histology (66.1%), followed by alveolar (20.3%), pleomorphic (6.8%), and anaplastic (1.7%). At presentation, 16 patients (27.1%) had stage I, 3 (5.1%) had stage II, 22 (37.3%) had stage III, and 14 (23.7%) had stage IV disease; 4 (6.8%) were unstaged. Of the 39 patients treated with curative intent, 25 (64.1%) received local control and 16 (41.0%) completed all planned therapy. Two-year event-free survival was 23.3% (95% CI 13.5%-34.8%, n = 55) overall and 61.4% (95% CI 33.3%-80.5%, n = 16) among those who completed treatment.

Conclusions: Treatment of RMS in a rural district hospital is feasible. Although outcomes remain lower than in high-income countries, survival was markedly higher in patients who completed therapy. Future efforts should focus on earlier diagnosis and support for timely transitions between specialties with the goal of optimizing completion of planned care.

Objective: To identify perceived benefits, concerns, and challenges to adolescents with cancer accessing online patient portals.

Study design: Semi-structured, qualitative interviews with 48 dyads of adolescents with cancer (12-17 years) and their parents. Interviews explored parental and adolescent experiences, motivations, and concerns to accessing the portal. Three team members analyzed interview transcripts using thematic analysis.

Results: Most adolescents (41/48, 85%) and parents (42/48, 87.5%) believed that adolescents should have access to their electronic health information (EHI), but that access should depend on certain factors, such as the adolescent's age, maturity level, or ability to understand portal content. Although most parents reported having accessed the portal (42/48, 88%), only 12 adolescents (25%) had previously accessed the portal. We identified seven themes related to both real-life and hypothetical benefits and concerns of adolescent portal access: promoting communication between adolescents, caregivers, and clinicians; providing reassurance to adolescents; supporting adolescent engagement and responsibility; supporting adolescent knowledge and understanding; creating confusion or misunderstanding; creating worry or fear; and potential for misuse.

Conclusion: In our qualitative study, most adolescents with cancer and their parents believed that teens should have access to their EHI but expressed a diversity of opinions on when and under what circumstances teens should have access. Both parents and teens recognized that portals had the potential to both alleviate and contribute to anxiety and worries related to cancer care and prognosis. Our study found novel areas of concern relating to the potential for portal use to negatively impact adolescent mental health.

Introduction: The aim of this study was to evaluate the effect of hydroxyurea on reducing the number of hospitalizations for dengue fever in patients with sickle cell disease (SCD).

Methods: In this nested case-control study, conducted between January 1 and December 31, 2024, we included all pediatric and adult patients with SCD followed up at the Cayenne Sickle Cell Center, in French Guiana.

Results: Our study population consisted of 90 cases and 189 controls. A total of 51 patients (18%) were hospitalized for dengue fever because of the presence of warning signs. Patients receiving hydroxyurea were older (28 years [19-40] vs. 23 years [10-37], p < 0.001) and primarily of the SS phenotype (89% vs. 52 %, p < 0.001). Hospitalizations for dengue fever occurred exclusively in patients younger than 18 years (12 years [6-18], p < 0.001). Among the patients treated with hydroxyurea, 13% have been hospitalized for dengue fever compared to 21% for the controls. However, this difference was not statistically significant. Patients with severe dengue fever had a longer hospital stay, were more often admitted to intensive care, had more dengue hemorrhagic fever and dengue shock syndrome, and were more often transfused. We noted three deaths among the severe dengue patients, all with dengue shock syndrome and multi-organ failure in two SS patients and one SC patient. The fatality rate of dengue fever has been estimated to be 6%.

Discussion: This study showed a lower prevalence of dengue hospitalization in patients with SCD treated with hydroxyurea, suggesting an anti-dengue viral role for hydroxyurea. Further multicenter studies are required to confirm this hypothesis.

Introduction: Survivors of childhood leukemia and lymphoblastic lymphoma are at risk of treatment-related cognitive sequelae and poor educational outcomes. Child self-report could be a resource-efficient screening tool for identifying those in need of further assessment and intervention; however, it has not been empirically evaluated. This study investigated parent- and child-reported school functioning as predictors of performance on objective cognitive and academic measures in school-age Hispanic/Latino survivors.

Methods: One hundred and six Hispanic/Latino survivors of leukemia and lymphoblastic lymphoma, ages 6-12 years, completed neurocognitive assessments. Parent- and child-reported school functioning was measured using the Pediatric Quality of Life Inventory 4.0 Generic Core Scales. Multivariate regression models examined associations between parent-report and child self-report of school functioning and performance in cognitive and academic domains, adjusted for relevant demographic and treatment covariates. The model fit of parent and child as predictors was compared using the Akaike Information Criterion (AIC). Child age was explored as a moderating factor.

Results: Higher child-reported school functioning was significantly associated with better Processing Speed (p < 0.001), Working Memory (p = 0.038), and Reading performance (p = 0.033). In contrast, higher parent-reported school functioning was only significantly associated with better Processing Speed (p = 0.026). Models using child self-report showed stronger associations with all outcomes and demonstrated better fit to the data, as indicated by lower AIC values, compared to models using parent-report.

Conclusion: Results highlight the potential utility of child-reported school functioning in future screening efforts to identify school-age survivors in need of additional evaluation or support.

Background: Cyclophosphamide (CY) is associated with potentially fatal cardiotoxicity, yet no electrocardiographic indices have been established for early detection of CY-induced cardiomyopathy. This study aimed to determine whether corrected QT interval (QTc) prolongation can predict early onset of CY-related cardiac dysfunction in pediatric patients.

Methods: We retrospectively analyzed 62 children who received high-dose CY therapy (≥100 mg/kg) for hematologic or oncologic diseases between 2010 and 2025. CY-induced cardiomyopathy was defined as a ≥10% reduction in the left ventricular ejection fraction to less than 50% or elevated brain natriuretic peptide/N-terminal pro-brain natriuretic peptide levels, according to the 2021 International Cardio-Oncology Society criteria. Resting 12-lead ECGs were obtained before and within 24 h of CY administration. QTc was calculated using the Fridericia and Bazett formulas, and ΔQTc (post-pre) was evaluated using logistic regression and receiver operating characteristic (ROC) analyses.

Results: Seven (11.3%) patients developed CY-induced cardiomyopathy. QTc after CY administration and ΔQTc were significantly greater in the cardiomyopathy group (p < 0.001). In multivariate analysis, ΔQTc was independently associated with cardiomyopathy (odds ratio 2.21, 95% confidence interval [CI]: 1.26-3.90, p = 0.0060). ROC analysis revealed excellent predictive accuracy (area under the curve = 0.96), with a ΔQTc ≥30.5 ms yielding 100% sensitivity and 89.1% specificity. Progressive QTc prolongation was also observed from pre- to post-CY and at diagnosis in the affected patients.

Conclusions: QTc prolongation within 24 h of CY administration suggests subsequent CY-induced cardiomyopathy in children. ΔQTc may serve as an early, noninvasive marker for timely detection and intervention in CY-induced cardiotoxicity.