Pediatric Blood & Cancer最新文献

A second allogeneic (allo-)hematopoietic stem cell transplantation (HSCT2) is a potential curative option for pediatric patients with acute lymphoblastic leukemia (ALL) following relapse after first allogeneic transplantation (HSCT1), but its efficacy is limited by high relapse rates and transplant-related toxicity in highly pretreated patients. This retrospective multicenter study analyzed 75 pediatric patients enrolled in the PRSZT registry who underwent HSCT2 between 2011 and 2020. Indications for HSCT2 were relapse in 55 patients (73%) and graft failure in 16 patients (21%). In 4 patients (6%), the indication could not be clearly determined due to incomplete documentation. At 2 and 5 years, overall survival (OS) was 48% and 40%, while relapse-free survival (RFS) was 35.2% and 29.9%. Relapse was the leading cause of mortality, occurring in 36% of patients post-HSCT2, with a median time to relapse of 8.2 months. Univariate analysis identified a longer duration of remission after HSCT1 (>6 months) as a predictor of improved OS (p = 0.013) and chronic graft-versus-host disease (cGVHD) after HSCT2 as beneficial for OS (p = 0.034) and RFS (p = 0.024). In multivariate analysis, older age (>10 years) (HR: 2.778, p < 0.01) and precursor T-cell ALL (HR: 6.62, p = 0.001) were associated with inferior OS, as well as with inferior RFS (HR: 2.2, p = 0.03; HR: 3.89, p < 0.005), while male sex (HR: 0.354, p = 0.02) and ATG administration (HR: 0.35, p = 0.019) predicted improved OS. Despite high relapse rates, HSCT2 remains a potentially curative treatment in a subgroup of patients. These findings highlight the need for better patient selection and the integration of novel immunotherapeutic strategies to further improve outcomes.

Background: Decision-making (DM) dynamics between adolescents and young adults (AYAs) with cancer, parents, and oncologists remain underexplored in diverse populations. We examined cancer treatment DM preferences among an ethnically and socioeconomically diverse group of AYAs and their parents.

Procedure: We surveyed AYAs with cancer (n = 75) and their parents (n = 52) participating in the Texas KidsCanSeq study. Among AYA-parent pairs, we compared preferred and actual treatment DM roles. We explored associations among DM roles, having pediatric-to-adult care transition discussions, and clinical and sociodemographic characteristics. We report AYAs' and parents' preferences for involvement in shared decision-making (SDM) with oncologists about cancer treatment.

Results: AYAs' DM role preferences varied: 44% preferred to equally share DM with parents, 31% preferred AYA-led DM, and 25% preferred parent-led DM. Parents who preferred parent-led DM had younger AYAs than parents who preferred equally shared DM (p = 0.011). For actual DM, 48% of AYAs reported DM was equally shared, 24% reported DM was AYA-led, and 28% reported DM was parent-led. Most AYAs had their preferred role (73%). AYAs' actual role did not significantly differ from their own or their parents' preferences (both p > 0.05). Approximately half of AYAs reported recent pediatric-to-adult care transition discussions. AYAs' DM preferences and participation in transition discussions did not significantly differ along clinical or sociodemographic characteristics (all p > 0.05). AYAs and parents perceived high importance across all aspects of SDM with oncologists.

Conclusion: In this diverse study population, AYAs desired engagement in treatment DM and SDM. Although specific role preferences varied, opportunities existed to increase discussion around pediatric-to-adult care transition.

Background: Hereditary spherocytosis (HS) is an inherited hematologic disorder characterized by spherical erythrocytes that are prematurely destroyed in the spleen. Total splenectomy (TS) and partial splenectomy (PS) are surgical interventions used to manage HS, but long-term outcomes, including recurrence of splenomegaly and splenomegaly-related symptoms, remain poorly understood.

Objective: This study aims to evaluate the long-term recurrence of splenomegaly and splenomegaly-related symptoms in children with HS who underwent PS versus TS at a single institution between 2008 and 2020.

Methods: A retrospective chart review of children with HS who underwent TS or PS was performed. The primary end point was the long-term recurrence of splenomegaly and splenomegaly-related symptoms. Variables collected included age, sex, surgical method (PS vs. TS), postoperative hematologic markers, splenic volume, postoperative complications, length of hospital stay, recurrence of splenomegaly, and need for completion splenectomy. Symptomatic recurrent splenomegaly is defined as splenic volume > 450 mL and the presence of symptoms directly attributable to the spleen and its effects on adjacent organs or hemolysis. Statistical analysis included comparisons of continuous variables using a Kruskal-Wallis test and categorical variables using a Fisher's exact test.

Results: Forty-four patients with HS met the inclusion criteria for the study. Of these, 31 (71%) patients underwent laparoscopic PS and 13 (30%) patients underwent laparoscopic TS. TS was associated with significantly less intraoperative blood loss (p = 0.003), shorter hospital stays (p = 0.01), and greater reduction in hemolysis compared to PS, as evidenced by lower postoperative bilirubin levels and reticulocyte counts (both p < 0.001). Eighteen (58%) PS patients experienced recurrent splenomegaly, and six (19%) PS patients experienced splenomegaly-related symptoms leading to completion splenectomy. Among the six patients who required completion splenectomy, intraoperative hemorrhage requiring conversion to open procedure occurred in half. The median time to need completion splenectomy was 10.5 years (range, 3-15 years).

Conclusion: TS offers more significant long-term hematologic improvements but comes with the loss of immune function and increased risk of overwhelming post-splenectomy infection (OPSI). PS, while preserving some splenic function and theoretically reducing OPSI risk, carries a higher risk of recurrent symptomatic splenomegaly and may require additional surgeries. These findings highlight the importance of long-term follow-up for HS patients who undergo PS and the need to balance the advantages and risks of both surgical approaches.

Introduction: Artificial intelligence (AI) has the potential to enhance oncology diagnostics, treatment planning, and patient monitoring. In pediatric oncology, AI can support both clinical care and research. The roles and awareness of AI in African pediatric oncology units are unknown. We aimed to assess knowledge and utilization of AI in pediatric oncology across Africa.

Methods: A cross-sectional online survey was conducted during July and August 2025, using a structured questionnaire in English, French, and Portuguese distributed via Qualtrics. Eligible respondents included professionals working in pediatric oncology across Africa. Descriptive and comparative analyses were conducted using SPSS, and free-text responses were analyzed thematically.

Results: There were 138 respondents from 33 African countries. Most were pediatric oncologists (55.1%) and completed the survey in English (71.7%). Only 5.8% reported hospital use of AI tools, mainly for imaging and risk stratification. In the previous year, 51.4% used AI for educational content and 44.2% for research, while 68.8% had used an AI platform in the preceding week. More than half (54.3%) reported no impact of AI on their clinical practice. Familiarity with AI tools was low, with machine learning the most recognized (8.5%). Barriers included insufficient training (93.2%), budget constraints (87.3%), and lack of infrastructure (80.4%). Higher World Bank Income Group and greater clinical experience correlated with higher AI familiarity (p < 0.01). Interest in learning AI tools did not differ meaningfully between low-, lower middle-, and upper middle-income settings. Respondents identified key opportunities for AI adoption in diagnostics (32.6%) and clinical management (39.1%), particularly to automate functions in settings with limited human resources.

Conclusion: AI adoption in African pediatric oncology remains limited, but there is strong interest and readiness to learn despite major barriers related to infrastructure, training, and resources. The most immediate opportunities for AI lie in automating clinical and administrative functions where human capacity is constrained.

Background: A routine baseline echocardiogram is often obtained prior to anthracycline administration in children with cancer. The utility of baseline echocardiogram is unclear in patients with standard risk B-cell acute lymphoblastic leukemia (SR B-ALL) as their anthracycline cumulative dose is low. We performed a quality improvement project to understand the clinical utility of routine pre-anthracycline echocardiograms in patients with SR B-ALL.

Methods: We retrospectively reviewed baseline echocardiogram results for all children with SR B-ALL treated at a single center from 2011 to 2024. Echocardiographic findings, including any significant structural or functional abnormalities, and the impact of echocardiogram results on anthracycline administration were analyzed.

Results: A total of 286 patients with a median age at diagnosis of 4.13 years [IQR 2.92-5.52] were included. Abnormal echocardiographic findings were reported in six patients (2.1%), but mainly represented incidental findings with only one patient requiring cardiology follow-up for a bicuspid aortic valve. No echocardiogram identified reduced cardiac function or any finding that altered or delayed anthracycline administration.

Conclusion: A routine baseline echocardiogram in patients with SR B-ALL prior to anthracycline administration is without clear utility. Based on these results, a change in practice was implemented in our institution to discontinue routine pre-anthracycline echocardiograms for this population. Omitting this low-value test may reduce unnecessary discomfort in children, family worry, and health-care-related costs.

Background: Synovial sarcoma (SS) is a rare malignancy, accounting for 5%-10% of all soft tissue sarcomas, and characterized by the SS18::SSX translocation. SS predominantly affects young individuals, with a peak incidence in the fourth decade of life. Children (<15 years), and adolescents and young adults (AYAs; 15-29 years) with SS tend to have better survival rates than older adults; however, the biological basis for these age-related differences remains poorly understood.

Methods: In this study, high-throughput RNA sequencing (RNA-seq) was used to investigate biological differences in SS across age groups. Transcriptomic profiles of primary tumor tissues from 14 patients younger than 29 years were compared with those from 8 adult patients (>29 years), with the aim of identifying age-related molecular features of SS. In addition, enrichment of recently described transcriptional signatures associated with SS tumor components and infiltrating cell populations was evaluated in our dataset.

Results: RNA-seq analysis identified differential expression of RPL39 and GATA3 between children/AYA and adult patients. Immunohistochemical analysis confirmed increased GATA3 expression in adult tumors. Comparative analyses based on SS-related transcriptional signatures further demonstrated enrichment of actively cycling tumor cells in adult samples, as well as a trend toward increased M2 macrophage infiltration in approximately half of the children/AYA samples.

Conclusions: This study reveals age-related molecular and tumor microenvironment differences in SS that may contribute to disparities in clinical outcomes. These findings underscore the need for further studies to validate these results and to support the development of age-tailored therapeutic strategies.