Pediatric Blood & Cancer最新文献

Background: Over 89,000 adolescents and young adults (AYAs) are diagnosed with cancer every year in the United States, and despite high survival rates, AYAs continue to experience the late effects of cancer and its treatments into survivorship. Pain, cited as the most distressing, impairing, and undertreated symptom during and after active treatment, is a common concern for AYAs with cancer and a challenge for oncology providers to manage. First-line pain treatments often include prescription opioids, but there is limited evidence to support the efficacy of long-term opioid use to manage pain among AYAs, along with increased risk of opioid-related negative consequences. Given the relative lack of research in this area, there is an opportunity to better understand how oncology providers think about and navigate chronic pain management and mitigate risk for opioid-related negative outcomes.

Method: Therefore, we conducted a semi-structured qualitative study with 14 pediatric oncology providers, assessing current pain management practices, decision-making, attitudes and perspectives, and challenges to providing safe and effective pain management for AYA oncology patients. Interviews were summarized using a Rapid Qualitative Analysis framework.

Results: Results provided support for three themes: perceptions about pain and pain management needs for AYAs with cancer; pharmacological and nonpharmacological treatments for cancer-related pain; and education, standardization, and challenges of opioid pain management.

Conclusions: There are a number of existing barriers to AYA cancer-related pain management in the oncology setting. Oncology providers are providing primary pain management for their patients, but should consider referral to pain specialists when available. Better characterization of cancer-related pain can ideally be leveraged to identify those patients at highest risk for chronic pain and its consequences in survivorship, opening the possibility of developing tailored prevention and intervention strategies.

Background: We describe clinical and biologic characteristics of neuroblastoma in older children, adolescents, and young adults (OCAYA); describe survival outcomes in the post-immunotherapy era; and identify if there is an age cut-off that best discriminates outcomes.

Methods: Patients diagnosed with neuroblastoma at ≥547 days between 2003 and 2022 from the International Neuroblastoma Risk Group Data Commons were compared by age subgroups. Recursive partitioning, dividing younger versus older at all monthly cut-points between 18 months and 15 years, was undertaken using Cox regression models of event-free survival (EFS), overall survival (OS), and OS post-relapse (OSPR). Kaplan-Meier curves of clinical/biologic subgroups were compared with log-rank tests.

Results: 7,835 patients met inclusion criteria: 18 months to <5 years (n = 5841), 5 to <10 years (n = 1488), 10 to <15 years (n = 357), and ≥15 years (n = 149) at diagnosis. Younger patients were more likely to have MYCN amplification (18 months to 5 years: 31%; 5-10 years: 15%) than older (10-15 years: 8%; ≥15 years: 7%) (p < 0.0001), metastatic disease (p < 0.0001), and high mitosis-karyorrhexis index (MKI) (p < 0.0001) and less likely to have diploid tumors (p < 0.001). Repeatedly dichotomizing the cohort, younger patients had superior EFS and OS (p < 0.05) for all cut-offs ≤40 months (hazard ratios: 1.1-1.3). Among high-risk OCAYA (International Neuroblastoma Staging System [INSS] Stage 4; n = 5005 [64% of cohort]), those diagnosed 2010-2022 had superior EFS/OS versus 2003-2009 in each age group (p < 0.0001). OSPR remained poor for all OCAYA (5-year OSPR 14% ± 0.7%).

Conclusions: For patients ≥547 days old, any age cut-off ≤40 months discriminated younger (superior EFS/OS) versus older patients; no cut-off was optimal. OCAYA diagnosed 2010-2022 (post-immunotherapy era) had superior outcomes versus 2003-2009. Stratification by comprehensive molecular biomarkers will likely best inform novel therapeutic strategies for OCAYA.

Background: Psychological safety (PS) is essential for teamwork, communication, and patient safety in complex healthcare environments. In pediatric oncology, interprofessional collaboration occurs under high emotional and organizational demands. Low PS may increase stress, burnout, and adverse events. To assess PS in pediatric oncology teams across Germany, a survey including items on communication, workload, and error management culture was conducted among members of the "Gesellschaft für Paediatrische Onkologie und Haematologie" (GPOH, Society for Pediatric Oncology and Hematology).

Procedure: In October 2025, healthcare professionals from GPOH member institutions completed an online survey on PS. The 38-item questionnaire addressed PS and related aspects like communication, teamwork, leadership, workload, error management, and team climate. Responses were rated on a six-point Likert scale and analyzed descriptively and by subgroups.

Results: A total of 273 professionals completed the survey (Cronbach's α = 0.89). Teams reported a respectful, safety-oriented climate but noted challenges with workload, onboarding, and feedback. Only two-thirds indicated that mistakes in patient care were discussed openly. Physicians reported more open communication and constructive problem handling, while nurses experienced greater stress and more dismissive behavior toward "different" colleagues. Leaders showed higher PS, whereas lower PS across all domains was observed among participants who had seriously considered changing their jobs. Job satisfaction closely correlated with PS, particularly with open communication, trust, and respect.

Conclusions: The findings emphasize that PS is both essential and improvable in pediatric oncology teams; strengthening leadership, feedback, and interprofessional reflection may enhance staff well-being, retention, and patient safety across this high-stakes field.

The improvement of childhood cancer outcomes has relied on clinical trials. The WHO's International Clinical Trials Registry Platform was searched for interventional clinical trials for children and adolescents with cancer in Latin America. Forty-seven trials were identified. Institutions were the primary sponsors for 38 (88.8%) trials, and 31 (65.9%) were conducted at a single site. Supportive care interventions (26, 55.3%) were more common than cancer-directed treatment trials. Thirteen (27.6%) trials had published results. Few trials were registered in Latin America. Strengthening research infrastructure and fostering collaborations are essential for equity in pediatric cancer research in the region.

Introduction: Little is known about psychosocial outcomes in pediatric cancer based on the area of residency. Therefore, we longitudinally examined differences in the adjustment of children with cancer living in rural and Appalachian areas relative to urban and non-Appalachian children.

Methods: Data were from a larger study of coping and communication in families of children with cancer (n = 336; M_age = 10.69). Rural (n = 118) and Appalachian (n = 58) residency were based on federal codes. Mothers, fathers, and children reported on child internalizing problems, externalizing problems, and total competence near diagnosis and 1 year later.

Results: Children with cancer exhibited significant increases in behavioral and emotional difficulties by 1 year post-diagnosis, as reported by all three sources. There were no group differences in behavioral and emotional problems at diagnosis nor in change from diagnosis to 1 year based on rural or Appalachian residency. At 1 year post-diagnosis, children from rural areas had significantly lower social competence based on mother (b = -0.77, p = 0.02) and child reports (b = -1.27, p = 0.02). Additionally, at 1 year post-diagnosis, fathers reported children in Appalachian counties had significantly lower total competence (b = -2.35, p = 0.04) and social competence (b = -1.32, p = 0.03) than non-Appalachian peers.

Conclusions: Survivors in rural and Appalachian areas may experience greater social difficulties 1 year post-diagnosis than those in more urban areas. Clinicians should routinely assess survivors' access to mental health and follow-up care. Future research should examine cultural barriers and beliefs that may contribute to these disparities.

Background: Despite their increased risk for functional impairment resulting from cancer and its treatments, few adolescents and young adults (AYAs) with a hematological malignancy receive the recommended or therapeutic dose of exercise per week during inpatient hospitalizations. Physical therapy (PT) plays a critical role in promoting and supporting exercise in the hospital setting; however, little is known about the factors that influence AYA engagement in PT. This qualitative study aimed to explore barriers and facilitators to inpatient PT through the perspectives of AYAs and healthcare providers.

Procedure: AYAs with cancer (n = 14) and healthcare providers (n = 9) were recruited at a pediatric academic medical center. Participants completed a semi-structured interview assessing barriers and facilitators to inpatient PT adherence. Interviews were video/audio-recorded, transcribed, and coded. Codes and themes were developed iteratively using reflexive thematic analysis.

Results: AYAs (15-29 years, M = 18.95, SD = 3.68) and healthcare providers discussed four primary themes affecting inpatient PT engagement. The themes suggested that social support from family and friends, AYAs' individual goals, and individualized PT programs improved motivation to participate. In contrast, treatment side effects and treatment demands limited AYA inpatient PT engagement.

Conclusions: AYA engagement and motivation for inpatient PT vary with the individual's circumstances, goals, health, and social support. Evidence-based behavior change techniques may support future intervention efforts in addressing barriers specific to inpatient PT adherence in AYAs with hematological malignancies.

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.

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: 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.