Pediatric Blood & Cancer最新文献

Introduction: We developed MedSupport, a multilevel medication adherence intervention designed to address root barriers to medication adherence. This study sought to explore the feasibility and acceptability of the MedSupport intervention strategies to support a future full-scale randomized controlled trial.

Design, setting, participants, and measures: We recruited caregivers of pediatric patients (ages 1-18) with acute lymphoblastic leukemia (ALL) receiving oral home-based chemotherapy at Roswell Park during April and May 2023. Multidisciplinary healthcare providers who were on the care teams of enrolled patients were invited to participate. Feasibility and acceptability among caregivers and healthcare providers were assessed using standardized self-report measures and objective measures of intervention fidelity.

Results: We implemented a pilot study of the MedSupport intervention at Roswell Park, and successfully implemented all study procedures. Feasibility was demonstrated through: (i) Demand: When approached for participation, 100% of eligible caregivers (N = 6) and healthcare providers (N = 8) agreed to participate. (ii) Practicality: 100% of families completed the barriers screening questionnaire on the first prompt. All families endorsed adherence barriers (range: 3-14). (iii) Acceptability: Caregivers rated the intervention as acceptable, appropriate, and feasible (mean: 4.71-5.0). Healthcare providers rated the intervention as acceptable, appropriate, and feasible (mean: 4.41-4.47). Objective data on intervention fidelity were feasible to capture and demonstrated both caregiver and healthcare provider engagement with the intervention.

Conclusions and relevance: MedSupport is an innovative, low-cost, and scalable intervention that warrants additional examination to understand efficacy in enhancing home-based medication adherence in pediatric ALL.

Background: Delayed chemotherapy-induced nausea and vomiting (CINV) in pediatric oncology patients is currently under-recognized. This study aims to develop, validate, and visualize a machine learning-based model to predict delayed CINV risk in children.

Procedure: This prospective cohort study was conducted from November 2021 to December 2022 at a tertiary hospital in southern China. Pediatric delayed CINV data were collected via an electronic diary using the Pediatric Nausea Assessment Tool (PeNAT) and National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) (v4.03), with PeNAT ≥3 or CTCAE grade ≥2 as the primary outcomes. Seven machine learning models, including random forest, support vector machine, and artificial neural network (ANN), were developed and validated using 29 sociodemographic and clinical features. Model performance was assessed using the area under the receiver operating characteristic curve (AUC) and other metrics. Shapley's Additive Explanations (SHAP) enhanced interpretability, and the models were integrated into a web-based calculator for visualization.

Results: Overall, 399 pediatric patients (60.4% male; aged 4-18 years) were included. The AUC of the seven models ranged from 0.782 to 0.815, with the ANN model performing best (AUC 0.815; 95% CI, 0.695-0.903). The ANN model's global SHAP plot revealed that the most influential features were acute CINV, days of chemotherapy, age, number of recreational activities, expectancy of CINV, and control effectiveness of CINV. The ANN model was then deployed as a web-based risk calculator for pediatric delayed CINV.

Conclusion: The ANN model demonstrated good performance in identifying children at high risk of delayed CINV. Our web-based calculator provides a reliable tool for clinical staff to support targeted CINV management.

Objective: Recent studies show evidence of cognitive and psychosocial impairments and reduced quality of life (QoL) in adult survivors of childhood extracranial solid tumors and lymphomas, but limited research has addressed these issues in pediatric populations.

Study design: The French RISK-N prospective study (2014-2021) evaluated 278 survivors of extracranial solid tumors or lymphomas (47% female, mean age at diagnosis and assessment: 6.2 and 11.7 years). Patients with pre-existing neurological conditions were excluded. Sociodemographic, disease-related, and treatment data were collected. Cognitive performance was assessed using Wechsler Intelligence Scales for Children (WISC-IV, WISC-V). Psychosocial outcomes included parent and/or patient-reported executive functions (Behavior Rating Inventory of Executive Function), behavior (Conner's Parent Rating Scale), QoL (Pediatric Quality of Life Inventory), fatigue (Multidimensional Fatigue Scale), and depression (Children's Depression Inventory). Information on schooling and educational/rehabilitative interventions was also recorded.

Results: Diagnoses included lymphoma (25%), nephroblastoma (19%), neuroblastoma (19%), osteosarcoma (7%), other sarcomas (18%), and other tumors (12%). Mean Full Scale Intellectual Quotient [M(SD) = 99.44(15.62)] was as expected in the general population [M(SD) = 100(15)], but the WISC-IV Perceptual Reasoning Index was slightly lower [M(SD) = 95.4(15.0), <1.5 SD 14%]. Parent- and self-reports indicated greater executive dysfunction, inattention, fatigue, and reduced QoL. In multivariable regression models, poorer cognitive outcomes were associated with lower parental education and developmental/learning delays before diagnosis.

Conclusions: Objective cognitive deficits were uncommon among pediatric cancer survivors, contrasting with a relatively high level of subjective cognitive and psychosocial complaints, highlighting the need for systematic screening and tailored clinical interventions.

Background: Therapeutic advances in pediatric oncology have significantly improved overall survival, with a higher incidence of long-term adverse effects and sequelae. Among pediatric cancer survivors, the highest incidence of hepatic lesions has been observed in those previously treated for high-risk neuroblastoma (HRNB). While hepatic nodules in these patients historically raised concern for tumor locations, recent attention has shifted toward non-tumoral, late-onset hepatic sequelae.

Methods: In this study, we retrospectively reviewed long-term liver outcomes from an institutional perspective follow-up protocol, including long-term HRNB survivors (>5 years from diagnosis) treated between 1996 and 2018 at a tertiary pediatric center.

Results: A total of 47 patients were included, with a median follow-up of 11.1 years. Liver parenchyma was normal in 11 (23%) patients throughout the whole follow-up period. Diffuse parenchymal inhomogeneity was observed in 36 patients out of 47 (76%). Out of 36 patients, 16 showed a stable, diffuse, non-nodular alteration of the liver during the whole follow-up period, while in seven patients, the liver involvement completely regressed after a median of 2.8 years. Moreover, 13 out of 36 patients (36%) with diffuse parenchymal inhomogeneity developed nodular lesions after a median of 7.7 years from diagnosis and 1.7 years from the onset of parenchymal alterations. Alpha-fetoprotein levels were normal in all patients.

Conclusions: A structured, integrated, multidisciplinary follow-up program revealed an unexpectedly high prevalence of hepatic abnormalities on imaging. Further studies are needed to define the long-term risk of developing liver complications, both in terms of malignancy and functional impairment.

Background: Children treated for acute lymphoblastic leukemia (ALL) are at risk of neurocognitive deficits in attention-concentration, working memory, executive function, and psychomotor speed.

Objectives: This study evaluated longitudinal trajectories and medical/demographic associations with neurocognitive outcomes during treatment of newly diagnosed ALL.

Methods: Patients ages 3-21 treated on DFCI 16-001 (NCT03020030) across eight North American sites (2017-2022) were evaluated using Cogstate across four timepoints from diagnosis through maintenance phase. Linear mixed models estimated trajectories and interactions with clinical factors over time, incorporating random effects for patients and sites.

Results: Among 298 patients (median age 7.9 years, 53% male), performance changed significantly over time in varying directions for executive functioning, attention, visual learning, and working memory-accuracy (all p < 0.001). There was a significant interaction overall between age and time for psychomotor function (interaction p = 0.01) and working memory-accuracy (interaction p < 0.001). Older age was associated with worse performance on working memory-speed (β = -0.04) and attention (β = -0.05). Female sex was associated with worse performance on psychomotor function (β = -0.27) and working memory-accuracy (β = -0.50), but better on visual learning (β = 0.47) and working memory-speed (β = 0.30). A greater-than-expected proportion of participants performed below -1.5 SD on tests of attention, executive functioning, and psychomotor functioning at multiple timepoints.

Conclusions: While most patients demonstrated normal neurocognitive functioning, including variable trajectories, a subgroup performed poorly on attention, executive functioning, and psychomotor functioning. Risk factors include older age at diagnosis and female sex, which may provide insight into groups warranting early intervention.

Background: Evans syndrome (ES) is a rare immune-mediated disorder involving two or more cytopenias, including immune thrombocytopenia (ITP), autoimmune hemolytic anemia, and/or immune neutropenia. ES may occur secondary to another condition or be idiopathic. While consensus recommendations exist for adults, there is no standardized diagnostic approach for pediatric Evans syndrome (pES). This study aimed to describe typical diagnostic evaluations conducted by clinicians caring for pES patients.

Methods: A cross-sectional survey of the Pediatric ITP Consortium of North America (ICON) assessed typical diagnostic workup for pES, the influence of clinical features on testing, evaluation for underlying disorders, including immune defects and autoimmune disease, subspecialty involvement, and genetic testing practices.

Results: Sixty percent (28/47) of respondents reported performing the same evaluation for all pES patients. There was no consensus on specific diagnostic tests. Providers consistently evaluated for autoimmune conditions, but varied in testing for inborn errors of immunity (IEI). Rheumatology and immunology were most often consulted. Most respondents (85%, n = 40) obtained genetic testing through commercial laboratories, frequently encountering insurance-related barriers.

Conclusions: Even among experts, diagnostic approaches to pES vary widely. Standardized frameworks are needed to guide comprehensive evaluation for this complex disorder.

Surveillance imaging aims to detect tumour relapse before symptoms develop, but it's unclear whether earlier detection of relapse leads to better outcomes in children and young people (CYP) with medulloblastoma and ependymoma. This systematic review aims to identify relevant literature to determine the efficacy of surveillance magnetic resonance imaging (MRI) for CYP with medulloblastoma and ependymoma compared to symptomatic detection. 11 databases and 2 trial registries were searched in March 2025. Studies evaluating MRI surveillance imaging in CYP with medulloblastoma and ependymoma were included. The primary outcome of interest was overall survival (OS) from diagnosis. Studies were screened independently. Data extraction/quality assessment (using QUIPs) were conducted by one reviewer and checked by a second. Narrative synthesis and post-hoc meta-analyses of the proportion of relapses detected by surveillance imaging were conducted. Of 9,575 records screened, seven studies including 196 CYP with medulloblastoma and 309 with ependymoma were eligible. All were deemed moderate/high risk of bias in at least one domain. Single-proportion meta-analysis showed most relapses were detected by surveillance imaging in medulloblastoma (66.7%; 95% CI:60.1-73.2%) and ependymoma (72.6%; 95% CI:67.6-77.7%). Data on OS from diagnosis by method of relapse detection was reported in two studies: neither provide conclusive evidence that earlier detection improves survival. We conclude that while surveillance imaging detects relapses more frequently than symptomatic detection, there is limited high-quality evidence that earlier detection improves survival. Future prospective research should be conducted and should provide more granular reporting of patient characteristics and survival outcomes from diagnosis/end of treatment.

Introduction: Neuroblastoma (NB) with central nervous system (CNS) metastases is rare at diagnosis, but occurs more often during relapse/progression. Patients with CNS metastases face a dismal prognosis, with no standardized curative treatment available. Novel therapeutic approaches, such as intraventricular radio-immunotherapy with ¹³¹I-omburtamab (Omb), have been developed. In this study, we report a retrospective, single-tertiary center analysis of a 23-year cohort of NB patients with CNS metastases, highlighting current treatment strategies.

Patients and methods: Retrospective data analysis of all NB patients with CNS metastases treated at Hospital Sant Joan de Déu, Barcelona, from January 2000 to January 2023. Patient characteristics at diagnosis, first-line treatment, relapse patterns, and CNS metastasis management were analyzed in search of risk variables and survival outcomes.

Results: CNS metastases at relapse were identified in 39/185 (21.1%) patients. Median age at diagnosis was 2.7 years, and 24/39 were male. Stage 4 NB with multisite compartment metastases accounted for most cases (92.2%). CNS events occurred predominantly at first relapse (29/39, 74.4%) and with neurological symptoms (23/38, 60.5%). MCYN amplification and concomitant extra-CNS metastases at CNS relapse were associated with poorer overall survival (OS) (p = 0.018 and p = 0.0059, respectively). Neurological symptoms upon relapse significantly increased the risk for subsequent CNS events (p = 0.028). Curative-intent treatment was attempted in 34/39 (87.2%) patients. After adjusting for immortal time bias, RT plus Omb significantly improved OS (p < 0.0001).

Conclusions: In our experience, MYCN amplification and concomitant extra-CNS metastases at CNS relapse significantly decrease OS. Multimodal treatment, including ¹³¹I-omburtamab radioimmunotherapy, significantly improves survival outcomes.

Central venous access is essential for delivering chemotherapy and supportive care in children with cancer. Yet the practical decisions surrounding device selection, placement, maintenance, and salvage vary widely among institutions. In our center, we use a systematic, multidisciplinary workflow to anticipate the treatment trajectory, prioritize venous preservation, prevent complications, and support structured salvage strategies when device dysfunction or infection occurs. This "How I Approach" article outlines a pragmatic, experience-based model drawn from daily practice in a high-volume pediatric oncology setting. The focus is on applying established concepts to real-world clinical decision-making to maintain continuity of therapy and minimize morbidity.