Pediatric Blood & Cancer最新文献

Background: B-cell lymphoblastic lymphoma (B-LBL) represents a rare variety of non-Hodgkin lymphoma, with limited research on its biology, progression, and management.

Methods: A retrospective analysis was performed on the clinical characteristics of 256 patients aged ≤18 years who received treatment under the China Net Childhood Lymphoma (CNCL)-NHL-2017-lymphoblastic lymphoma regimen from April 2017 to March 2023.

Results: Among the 256 patients, the median age at diagnosis was 5.0 years, with a slight male predominance. Subcutaneous tissues, skin, and osteolytic bone were the most common sites of the disease. More than 90% of patients exhibited disseminated disease (Stage III or IV). Approximately 19.9% of the diagnosed patients exhibited central nervous system involvement. Adverse events were observed in 33 patients (12.9%), with disease progression or relapse occurring in 10.2% of cases, particularly linked to unfavorable outcomes in instances of early relapse or progression. Salvage chemotherapy combined with immunotherapies, followed by bridging hematopoietic stem cell transplantation, significantly improved the prognosis of relapse and disease progression in children. Overall, the follow-up time was 37.6 (Q1-Q3, 28.9-38.0) months, and 3-year event-free survival rate and overall survivals were 86.3% ± 2.5% and 95.2% ± 1.5%, respectively, with a treatment-related mortality of 1.6%. Multivariate analysis showed that poor prednisone response and no complete remission on Day 33 of induction were risk factors for poor prognosis.

Conclusion: The CNCL-NHL-2017-lymphoblastic lymphoma regimen was effective in children with B-LBL. The response to initial treatment is vital for improving prognosis in patients with B-LBL.

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.

Objective: This study aimed to explore the impact of different parenting styles on the physical activity of childhood cancer survivors based on the parent-based expansion of the theory of planned behavior.

Methods: This cross-sectional study investigated 271 childhood cancer survivors and their parents in China.

Results: The path model was well fitted with χ²/df = 1.740, RMSEA = 0.052, GFI = 0.964, NFI = 0.958, IFI = 0.982, TLI = 0.969, CFI = 0.981. Positive parenting styles (β = 0.181) directly affected the physical activity of childhood cancer survivors. Positive parenting styles indirectly influenced behavioral intention through both attitude (β = 0.022) and perceived behavioral control (β = 0.027), ultimately affecting physical activity in childhood cancer survivors. Parents who frequently encouraged or co-participated in physical activity with children had children with significantly higher physical activity levels.

Conclusions: These findings highlight the critical role of positive parental style influence in fostering physical activity among childhood cancer survivors and suggest that interventions promoting positive parenting styles could enhance physical activity and overall well-being in this vulnerable population.

Neurocognitive deficits in adult survivors of childhood cancer are well established, but less is known about developmental disorders (DD) arising shortly after cancer diagnosis. Using 2016-2019 linked Ohio cancer registry and Medicaid data, we compared DD among 324 children with cancer and 606,913 cancer-free controls. Pre-diagnosis, DD prevalence was similar (16% vs. 16.9%). However, post-diagnosis, children with cancer had over twice the risk of DD (ARR 2.09, 95% CI 1.55-2.83) across developmental domains. These findings reveal that DD emerges soon after cancer diagnosis, potentially related to treatment and/or secondary toxicities and highlight the need for early screening and rehabilitative intervention.

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.

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.

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.

Current pediatric allogeneic hematopoietic stem cell transplantation (HSCT) services in South Africa do not meet the substantial demand in the country. The factors leading to this paucity are multifactorial, including a limited number of appropriate donors on our local registries, inadequate identification and referral of appropriate patients, long distances to travel to health facilities, socioeconomic inequality, and inadequate infrastructure and clinical expertise for the number of transplants required. We describe a model for a large HSCT unit that caters to insured and uninsured patients in order to ensure equitable access, and which is in line with the WHO health system building blocks. The scale at which transplantation will be achieved will allow for the development of local skills and expertise, which can be decentralized in the future to further improve HSCT access.