Pediatric Blood & Cancer最新文献

Lynch Syndrome (LS) is a hereditary cancer predisposition syndrome caused by germline pathogenic/likely pathogenic variants in mismatch repair (MMR) genes. While LS is not traditionally thought to increase childhood cancer risk, some studies have shown an increased prevalence of germline MMR variants in the pediatric oncology population. Herein, we discuss 55 individuals with pediatric cancer and LS. Of these, MSH2 was the most common germline change (36%). Of those tested for loss of heterozygosity (LOH) or microsatellite instability (MSI), the majority had LOH present (78%) and were MSI high (71%), suggesting the contribution of LS to oncogenesis in these individuals.

Background: Families of children with cancer often experience financial toxicity resulting from cancer care. Pediatric patients living in geographically remote locations face unique barriers to access centralized subspecialty medical and support services, potentially worsening financial toxicity.

Methods: We conducted in-depth semi-structured interviews, in English and Spanish, with a purposive sample of adult caregivers of children treated for cancer. We characterized caregivers as geographically distant or proximal based on travel time from their permanent residence, using a cutoff of 60 min. We performed a framework analysis of interview transcripts focused on exploring how geographic distance to care shaped lived experiences with financial toxicity and its subsequent effects on patients and families.

Results: We interviewed 21 caregivers, 14 (67%) of whom were geographically distant. Distance to care was reported as provoking or exacerbating financial toxicity by most geographically distant families (13/14). Three primary themes emerged that were particularly salient for geographically distant versus proximal families: (i) difficulty maintaining income driven by challenging logistics and temporary relocation for care; (ii) increased household spending related to travel and housing; (iii) altered or diminished capacity to respond to financial toxicity.

Conclusion: Families of children with cancer perceived geographic distance to care as an important factor that intensified financial toxicity. Interventions are needed to mitigate the development and effects of financial toxicity, and geographically distant families may require supplementary solutions tailored to their specific support needs.

Background: The Improving Population Outcomes for Renal Tumours of childhood (IMPORT) is a prospective clinical observational study capturing detailed demographic and outcome data on children and young people diagnosed with renal tumours in the United Kingdom and the Republic of Ireland.

Methods: Data were extracted from the IMPORT database for all newly diagnosed cases of Wilms tumour (WT) registered between November 2012 and December 2022. Kaplan-Meier survival was estimated at one, three and five years. For children aged 0-14 years with unilateral WT, Cox proportional hazards models were used to examine patient/tumour characteristics in relation to the excess hazard of death.

Results: Among 620 patients, 570 had unilateral and 50 (8%) bilateral disease; 582 (94%) patients received pre-operative chemotherapy and 38 (6%) immediate surgery. Median age was 41 months (IQR: 41-65). Median tumour volume at diagnosis was 595 cm³ (IQR: 348-914) and 228 cm³ (IQR: 83-535) after pre-operative chemotherapy, with 123/443 (28%) ≥500 cm³. Of 570 patients with unilateral WT, 419 (73.5%) had localised and 151 (26.5%) metastatic disease. Five-year survival was highest for children aged 0-4 years (94.8%). Five-year survival by Toronto stage (assessed at nephrectomy) was 97.0% (stage I/yI), 93.8% (stage II/yII), 85.2% (stage III/yIII) and 88.9% (stage IV), and by histology 74.6% (high risk), 95.2% (intermediate risk) and 100% (low risk). In univariate analysis, histological risk group, age (3 after pre-operative chemotherapy were significantly associated with excess risk of death. In multivariate modelling, only high-risk histology (HR 10.8, 95% CI: 4.0-29.1) retained significance.

Conclusions: Five-year survival for children with WT in the United Kingdom and Ireland is generally above 90%, with high-risk histology as the most important adverse prognostic factor. However, median tumour volume at diagnosis and after pre-operative chemotherapy remains large.

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.

Background/objectives: Osteosarcoma is a radioresistant tumor that may benefit from stereotactic body radiation therapy (SBRT) for locoregional control in metastatic/recurrent disease. We report institutional practice patterns, outcomes, toxicity, and failures in osteosarcoma patients treated with SBRT.

Design/methods: We reviewed cases of recurrent/metastatic osteosarcoma treated with SBRT from 2015 to 2023. Outcomes included local control, overall survival (OS), and toxicity. Local failures (LFs) were categorized as in-field (≥80% of recurrence within the 95% isodose line), marginal (20%-80%), or out-of-field (<20%).

Results: Twenty-four patients (71 lesions) met eligibility. Mean imaging follow-up was 5.9 months (range, 0.6-57.1), and overall mean follow-up was 10.7 months (2.8-57.6). Lesions were osseous (66%), parenchymal (17%), or soft tissue (17%). Median SBRT dose was 40 Gy/5 fractions (range, 16-60 Gy in 1-5 fractions); 32 lesions received concurrent chemotherapy. Eleven LFs occurred: 4 in-field, 3 marginal, and 4 out-of-field. The 6- and 12-month cumulative LF incidences were 10.3% (95% CI 4.5-18.9) and 13.6% (95% CI 6.6-23.2). On multivariable analysis, spine SBRT (HR 3.39, 95% CI 1.03-11.14) and larger planning target volume (HR 1.003, 95% CI 1.001-1.006) were associated with LF. The 6- and 12-month OS rates from first treatment were 87.5% (95% CI 66.1-95.8) and 64.1% (95% CI 40.6-80.3). Grade 2 acute toxicity occurred in 5.6% of cases with no acute grade ≥3 events.

Conclusions: SBRT for recurrent/metastatic osteosarcoma is safe and effective, achieving a 13.6% 1-year LF rate. LFs were evenly distributed amongst in-field, marginal, and out-of-field. Spine SBRT had the highest LF risk, suggesting the need for tailored strategies near critical structures.

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.