Pediatric Hematology and Oncology最新文献

Neuroblastoma (NBL) is a tumor that develops from sympathetic ganglia and most frequently occurs in young children. In mediastinal and cervical cases, Horner syndrome (HS) may occur as a symptom or a surgical complication. The study aimed to evaluate the factors that influence the occurrence of postsurgical HS. The data concerning the clinical presentation, imaging examinations, performed therapies and treatment outcomes of 454 children with NBL were collected, including 117 cases of thoracic and cervical tumors. The statistical analyses of correlations between postsurgical HS occurrence and different features was performed. HS appeared in 57.89% of patients with tumors adjacent to oculosympathetic pathway. Statistical analyses demonstrated a significant impact of tumor location, patients' age and the duration between the onset of symptoms and the diagnosis on HS incidence as a complication of NBL resection. Neoadjuvant chemotherapy was also associated with a lower incidence of postsurgical HS. The radicality of surgery, the time from diagnosis to the procedure and the used technique did not present any statistically significant association with the occurrence of HS. In conclusion, parents of patients should be informed about the risk of HS in every case of a tumor localized near the oculosympathetic tract. Neoadjuvant chemotherapy might help reduce the risk of postsurgical complications.

Hematopoietic stem cell transplantation (HSCT) is a therapeutic modality for pediatric malignancies and inherited and acquired hematological, immunological, and metabolic disorders. The efficacy of HSCT depends on many factors, including conditioning regimens, which are critical for eradicating malignant cells, suppressing the immune system to prevent graft rejection, and establishing an optimal microenvironment for engraftment.Conditioning methodologies have evolved from high-dose chemotherapy and total body irradiation to refined approaches that mitigate toxicity while maintaining their efficacy.The relationship between the conditioning regimen and donor selection influences short- and long-term transplant outcomes and minimizes adverse sequelae.With advances in molecular medicine, there is growing interest in chemotherapy-free conditioning protocols that leverage biological mechanisms to facilitate stem cell acceptance and minimize risks.The concept of "the art in the science" captures the intricate balance and decision-making essential for applying scientific principles to pediatric HSCT conditioning.Personalized medicine, expertise in regimen selection, and ethical considerations are key aspects of this approach.Examples include the customization of conditioning protocols, reduced-intensity conditioning, pharmacogenomics in personalized treatment, precision in drug sequencing and dosing, and innovations in serotherapy and chemo-free conditioning.We discuss historical perspectives, scientific foundations of conditioning, the role of radiation and serotherapy, and the potential shift toward chemo-light and chemo-free approaches.This review explores HSCT conditioning in pediatric patients, examining the scientific underpinnings and expertise required for protocol adaptation, challenges in resource-constrained environments, and evolving clinical demands.

This study aims to investigate sexual activity, focusing on partner sex, in young adult survivors of childhood cancer in comparison to the general population. Furthermore, the study seeks to identify factors among the survivors associated with never having had partner sex. A population-based observational survey study was conducted with adult (aged 19-40) survivors of childhood cancer (n = 2545) and a comparison group from the general population (n = 819). Multivariable logistic regression analyses, stratified by gender, were performed to examine sociodemographic, clinical and psychological factors associated with never having had sex with another person. In comparison to the general population, survivors exhibited lower levels of sexual activity with a partner during the past month (women: 78% vs. 65%, p < 0.001; men: 62% vs. 56%, p = 0.031). A small proportion of survivors reported never having engaged in partner sex, with men reporting this to a greater extent than women (14% vs. 7%, p < 0.001). Factors associated with never having had partner sex included younger age at study, no current occupation and prior cranial irradiation. Additionally, men who self-reported depressive symptoms and women who had undergone hematopoietic stem cell transplantation were more likely to have never engaged in sexual activity with a partner. In conclusion, young adult survivors of childhood cancer have partner sex to a lesser extent than peers. Intensive cancer treatment was related to never having engaged in partner sex. These findings highlight the need for health care professionals to actively address sexuality during surveillance to support sexual health and well-being.

Febrile neutropenia (FN) is a common complication in pediatric oncology patients that typically necessitates hospitalization for intravenous antibiotics, resulting in significant use of hospital resources and decreased patient and family quality of life. Recently several centers have published early discharge guidelines for patients with low-risk FN, but this has not yet been reported at large tertiary centers with high acuity. Here we describe implementation of a low-risk early discharge FN guideline at our tertiary cancer center with resulting decrease in median length of stay (2.39 days saved per early discharge encounter) and no adverse events, including sepsis or infection-related mortality.

The 2014 National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering hydroxyurea to all patients with sickle cell disease SS/Sβ0 (SCD) aged ≥ 9 months. The relationship between early hydroxyurea initiation and the development of cerebrovascular disease (CVD) is unclear. This retrospective study describes trends in HU prescriptions and CVD outcomes in patients with SCD SS/Sβ⁰ followed at a single center before and after guideline publication. CVD was defined as the first of abnormal transcranial Doppler ultrasound, silent cerebral infarct, steno-occlusive vasculopathy, or arterial ischemic stroke. Among 530 patients with SCD, hydroxyurea prescriptions rose post-guideline, with lower age at initiation. CVD was lower post-guideline (2.2/100py) versus pre-guideline (4.4/100py). However, MRI screening also decreased post-guideline, leading to lower CVD detection. Results suggest decreased CVD, including silent cerebral infarcts, in the era of early hydroxyurea use; further analyses addressing age at hydroxyurea initiation and relevant confounders are needed to confirm whether earlier hydroxyurea initiation improves protection against CVD.

We retrospectively analyzed the clinical outcomes of seven patients receiving palliative treatment with oraltemozolomide (TMZ)/etoposide (ETP) for recurrent or progressive neuroblastoma (NB). Patients received a median of three cycles of TMZ/ETP. One patient achieved complete response (CR), three showed stable disease, and three showed progressive disease. The median time to progression in the six patients without achieving CR was 82 days excluding one achieving CR for >222 days. Grade 3 hematological toxicities were common but manageable, and no patients experienced severe non-hematological toxicity. TMZ/ETP is a feasible palliative chemotherapy option for certain patients with recurrent or progressive NB.

Pediatric patients with progressive and refractory solid tumors face a challenging prognosis. Despite advancements in treatments like immunotherapy and targeted therapy, survival rates remain low for certain tumor types. Decision-making in these complex cases often necessitates a multidisciplinary approach, integrating risk-based management, precision medicine, and access to clinical trials. Artificial intelligence (AI) technologies, particularly large language models (LLMs), hold promise for improving clinical reasoning and decision support in pediatric oncology. This study evaluated the decision-making capabilities of five AI tools-ChatGPT, Gemini, Claude, Perplexity, and OpenEvidence-in six hypothetical cases of refractory or progressive pediatric solid tumors. Each AI tool was presented with two sequential queries: a request to generate potential treatment options and then a request to identify and justify the most appropriate option from its initial list. The AI tools generated a total of 124 treatment recommendations, with an average of 24.8 per tool. Clinical trial enrollment was the most frequently selected "best option," accounting for 55.2% of cases. Other notable recommendations included targeted therapy (17.2%), surgery (10.3%), chemotherapy (10.3%), best supportive care (10.3%), and immunotherapy (3.4%). Notably, the AI tools exhibited distinct tendencies in their decision-making approaches, with some favoring aggressive interventions and others emphasizing supportive or palliative care. AI tools demonstrate potential for assisting with complex treatment decisions in pediatric oncology, particularly by identifying clinical trial options. However, the observed variability in recommendations underscores the need for careful human oversight to ensure that AI-generated suggestions align with clinical evidence, patient and family preferences, and the overall goals of care. Future research should explore how AI tools can be further refined to incorporate nuanced patient-specific information and address the emotional and psychological impact of AI-assisted decision-making.

Hypoalbuminemia and cancer cachexia predict poor survival in adult cancers, with limited studies in pediatrics. Also, the serial recovery kinetics of serum albumin and its effect on cancer outcomes are unknown. This study investigated trends and determinants of serum-albumin at various time points of therapy and their influence on 3-year survival of pediatric cancers. It included cancer patients below 18yrs. Baseline systemic symptoms, stage/risk-group, nutritional status and serum-albumin at baseline, 3-months, 6-months and 12-months were recorded from hospital database along with outcomes like relapse/death and toxicity. Among 402 patients, hemato-lymphoid cancers (57.71%)were more common. Prevalence of hypoalbuminemia at baseline, 3 m, and 6 m was 46.52%, 26.45% and 22.0%, respectively. Patients with hypoalbuminemia at baseline (HR = 2.1; p = 0.000), 3 m (HR = 2.28; p = 0.000), or 6 m (HR = 2.02; p = 0.001) had lower overall-survival (OS) in univariate analysis, while on multivariate analysis, only risk group (HR = 1.74; p = 0.009) and 6 m albumin (HR = 3.33; p = 0.001) independently predicted OS. For event-free-survival (EFS) risk-group (HR = 1.59; p = 0.018) and 6 m-albumin (HR = 2.91; p = 0.001) were the only independent predictors. Out of various predictors of serum albumin, systemic-symptoms (OR = 8.01, p = 0.008) and baseline-malnutrition (OR = 2.08; p = 0.004) independently predicted baseline-hypoalbuminemia. Organomegaly measured as liver/spleen size(r=-0.39) and systemic inflammation measured as serum-globulin (r=-0.37) had negative correlation with baseline-albumin. In contrast, 6-month albumin was dependent only on baseline systemic symptoms (OR = 5.28; p = 0.007) and independent of baseline malnutrition (OR = 1.72; p = 0.091). We conclude that while baseline albumin is influenced by multiple factors, such as malnutrition, systemic symptoms, inflammatory state (hypergammaglobulinemia), and disease bulk (organomegaly), 6-month albumin is independent of baseline malnutrition and serves as a better predictor of the cancer-inflammation-cachexia-cascade and ultimate survival.

Late effects are a major concern after pediatric hematopoietic stem cell transplantation (HSCT), but data on health outcomes beyond early adulthood remain scarce. This study assessed long-term morbidity and mortality in one of the earliest childhood cancer cohorts treated with HSCT. The study comprised 52 male childhood cancer survivors (survival ≥5 years), treated with HSCT in 1983-2001 at a median age of 6.1 years, and 257 matched population controls. Finnish national healthcare registries provided data on prescription drug purchases, reimbursements for chronic diseases, hospitalizations, and late mortality. Median follow-up was 24.9 years. Compared to population controls, survivors had increased risk for diabetes (HR 16.4, 95% CI 4.33-61.8), severe hypertension (HR 11.4, 95% CI 3.42-37.8), purchases of lipid-lowering drugs (OR 13.5, 95% CI 4.87-41.3), and purchases of antihypertensives (OR 2.58, 95% CI 1.28-5.12). HSCT survivors had also higher risk for hospitalizations due to cardiovascular diseases (HR 6.13, 95% CI 1.52-24.7) and neurological conditions (HR 4.79, 95% CI 1.73-13.2). New diagnoses of epilepsy and secondary neoplasms emerged beyond 20 years post-transplantation. Chronic graft-versus-host disease was associated with increased purchases of thyroxine, growth hormone, and lipid-lowering drugs. Given the high risk for diabetes, hypertension, and dyslipidemia, proactive cardiovascular risk assessment is essential in long-term care after pediatric HSCT. The occurrence of late-onset complications underscores the importance of structured lifelong follow-up.

Pediatric Hodgkin lymphoma (HL) treatment has increasingly shifted toward response-adapted protocols, aiming to minimize radiotherapy and employ intensive chemotherapy such as OEPA/COPDAC. We retrospectively reviewed treatment outcomes and toxicities in pediatric HL patients treated with OEPA/COPDAC between 2015 and 2019 at a tertiary care center in Pakistan, a resource limited country, following the Euronet PHL-C1 protocol with radiotherapy reserved for inadequate interim responses. Clinical features, treatment-related toxicities, and hospital admissions were documented. The cohort included 20 patients with a median age of 12 years; 13 (65%) achieved complete remission after OEPA induction and avoided radiotherapy. Toxicities were frequent-15 (75%) after OEPA-1 and 13 (68%) after OEPA-2-most commonly gastrointestinal symptoms and febrile neutropenia. Hospitalization was required in 9 (45%) after the first cycle and 11 (58%) after the second, with one treatment-related death from febrile neutropenia. At median follow-up, overall survival was 95% (95% CI: 69.47-99.28%) and event-free survival was 85% (95% CI: 60.38-94.90%). These findings highlight that OEPA/COPDAC achieves high survival rates even in advanced-stage pediatric HL within low-resource settings. However, the substantial toxicity burden and frequent hospitalizations underscore the need for enhanced supportive care and further evaluation in larger, long-term studies.