Pediatric Hematology and Oncology最新文献

Intrathecal (IT) chemotherapy is a highly effective treatment and prophylaxis for central nervous system (CNS) involvement in leukemia and lymphoma. Despite its therapeutic efficacy, IT chemotherapy has potential neurotoxicity risks, including acute and delayed symptoms that can severely affect patient outcomes. Effective management of acute, IT-related neurotoxicity requires a prompt, case-specific approach that considers symptom severity, the type of chemotherapeutic agent, and individual patient factors. This review examines a case-based approach to managing common scenarios of IT neurotoxicity and provides a structured guide for clinicians in assessing and addressing these complications.

Cutaneous melanoma (CM) is a rare occurrence in the pediatric population and suffers from a dearth of epidemiological data. This study aims to estimate the distribution and temporal trends of pediatric CM. Data specific to the pediatric (<20 years old) CM were extracted from the Global Burden of Disease (GBD) Study 2019, stratified by Socio-demographic Index (SDI) and WHO region. The data encompassed incidence, mortality, and disability-adjusted life-years (DALYs) representing the years of healthy life lost due to a pediatric CM diagnosis. Join point regression analysis and Quality of care index (QCI) were computed. In 2019, the global age-standardized incidence, mortality, and DALYs rates of pediatric CM were estimated at 0.13, 0.02, and 1.46 per 100,000 population, respectively. From 1990 to 2010, an increase in incidence was noted (0.95, 95% UI: 0.89 to 1.02), while mortality (-0.62, 95% UI: -0.71 to -0.53) and DALYs (-0.58, 95% UI: -0.67 to -0.50) exhibited a decline. The global QCI for pediatric melanoma in 2019 was 87, while Somalia was noted to have the lowest QCI (16). The incidence rate was predominantly observed in European regions and high SDI regions, whereas the disease burden was more pronounced in low SDI region and Africa regions. An age-related discrepancy was noted with pediatric CM being higher and more broadly distributed among western countries in children above the age of ten. This study highlights that pediatric CM remains rare but has a disproportionate global distribution, warranting targeted strategies to tackle this issue.

The 4th Symposium on Childhood Cancer Health Disparities was held at Texas Children's Hospital in Houston, Texas, on September 26, 2023. The symposium registered 94 attendees from different backgrounds (e.g. clinicians, epidemiologists, exposure assessment scientists, geospatial experts) with an interest in environmental health disparities of pediatric cancer susceptibility and treatment outcomes. The focus of the symposium was to provide an overview of the role of environmental risk factors in studies of pediatric cancer, introduce novel exposure assessment tools that can be applied to the field, and highlight opportunities to study the impact of environmental health disparities in pediatric cancer susceptibility and outcomes. This report summarizes the scientific content of the symposium and highlights priorities to advance the field.

Treatment of young children with brain tumors may require the use of high dose chemotherapy with alkylating agents to avoid craniospinal irradiation. The objective of this study is to describe the probability of primary gonadal failure (PGF) in children with a malignant intracranial tumor treated with high-dose alkylating agents in children diagnosed less than age 8 years who were treated with this radiation-sparing approach at our institution. Patient demographics, oncological and endocrine diagnoses, treatment modalities, and laboratory values were collected. Descriptive statistics, Kaplan Meier survival curves, regression analysis, and T-tests were used in data analysis. Eight of 18 (44%) developed PGF. The probability of developing PGF is 11% at 5 years, and 31% at 10 years. Cyclophosphamide equivalent dose (CED) was higher and duration of follow-up was longer in children with PGF. PGF is common in children who received CED without irradiation, but further studies are needed to correlate CED dose and time to onset of PGF.

This pilot study explored caregiver experiences caring for children with hematologic and oncologic diseases. Survey data were collected from caregivers (n = 85) of children with hematologic and oncologic diseases while participating in therapeutic camp programming. Caregivers of children with Sickle Cell Disease perceived their child's ability to self-regulate higher than caregivers of children with cancer, which impacted aspects of caregiver hope and wellness. Implications for multi-sector collaborations are provided. In assessing caregiver hope, perception of child self-regulation was associated with higher caregiver hope and wellness, which varied by illness. Caregivers of children with SCD had higher hope scores on both the subscales and total scores compared to caregivers of children with cancer.

Pediatric patients with sickle cell disease and vitamin D deficiency have worse clinical and laboratory outcomes. This study aims to quantify the prevalence of vitamin D deficiency in this population and identify possible risk factors for hypovitaminosis D by performing a cross-sectional study with children aged 3-18 years old with sickle cell disease. Sixty patients were evaluated, with a mean age of 10.80 + 4.21 years. The prevalence of vitamin D deficiency was 46.7% (21.02 ± 8.47 ng/mL). Patients were clustered into two groups regarding vitamin D deficiency (25-OH-D < 20 ng/mL). When comparing groups with and without vitamin D deficiency, age (p = 0.002) and season of 25-OH-D collection (p = 0.005) were statistically significant. Age presented OR 1.23 (95% CI: 1.07; 1.41/p = 0.004), as well as the season of the 25-OH-D collection with OR 5.21 (95% CI: 1.58; 17.14/p = 0.007) for autumn/winter assessment. After linear regression, an association was noted for age (β = -0.80/95% CI: -1.29; -0.320/p = 0.002), days of sun exposure (β = 0.83/95% CI: 0.07; 1.58/p = 0.032), and autumn/winter vitamin D assessment (β = -7.94/95% CI: -12.02; -3.85/p = 0.032). In conclusion, hypovitaminosis D is highly prevalent in this population; meanwhile, age, season of 25-OH-D collection, and days of sunlight exposure appeared as risk factors for deficiency.

Convenient multiday dosing of antiemetic regimens for the prevention of chemotherapy-induced nausea and vomiting (CINV) are needed in pediatric patients, who are more likely than adults to be treated with emetogenic chemotherapy over multiple consecutive days. Intravenous (IV) fosaprepitant is approved for the prevention of CINV in children aged 6 months and older. This open-label, single-arm study assessed the safety and tolerability of a 3-day fosaprepitant regimen (consecutive daily IV administration on days 1-3) plus a serotonin receptor antagonist with or without dexamethasone in pediatric patients (6 months to 17 years) receiving emetogenic chemotherapy. Study treatment was initiated at the start of a chemotherapy cycle (cycle 1); patients completing cycle 1 could participate in optional cycles 2 and 3. Primary endpoints included adverse events (AEs) and AE-related discontinuation during cycle 1.98/100. Patients completed cycle 1; 69 participated in optional cycles 2 and 3. The AE profile during cycle 1 was typical of cancer patients receiving emetogenic chemotherapy; 80/100 (80.0%) patients experienced ≥1 AE. AE rates were generally similar between patients aged 6 months to <2 years (11/15 patients [73.3%]), 2 to <6 years (22/30 [73.3%]), 6 to <12 years (24/25 [96.0%]), and 12-17 years (23/30 [76.7%]). Rates of drug-related AEs (4/100 [4.0%]) and AE-related discontinuations (2/100 [2.0%]) were low. Similar trends in safety outcomes were observed during cycles 2 and 3. No deaths were reported. The 3-day IV fosaprepitant regimen for the prevention of CINV was generally well tolerated in pediatric patients receiving emetogenic chemotherapy.

Pulmonary toxicity is one of the most common morbidities experienced by childhood cancer survivors (CCS). The aim of this study was to identify prevalence, pattern of dysfunction, and risk factors among CCS and compare with age and sex matched controls. Details of demographic and pulmonary-toxic treatment of CCS at least 2 years off-treatment were collected and a cross-sectional analysis of pulmonary function test (PFT) and risk factors was performed. Spirometry findings were categorized as normal, restrictive, or obstructive and diffusing capacity of carbon monoxide (DLCO) as normal or abnormal. PFT data of 192 CCS and 50 controls was analyzed. One or more abnormalities inspirometry or DLCO were observed among 112 (58.3%) CCS and 8 (16%) controls (p value <0.01). Abnormal PFT was more likely to be associated with older age at evaluation, longer follow-up, and use of chest-directed radiotherapy (p value 0.002, 0.02, 0.03). DLCO was the most common abnormality observed in 85 (44%) patients. Obstructive and restrictive patterns were observed in 66 (34.3%) and 42 (21.8%) survivors respectively. There was no correlation between any risk factor and specific pattern of pulmonary dysfunction. On univariate analysis age at evaluation >20 years, follow-up >10 years, cumulative bleomycin more than 120 mg/m², chest-directed radiotherapy, surgery, and female gender were found to be predictive for abnormal PFT. On multivariable analysis first four factors retained significance. High subclinical prevalence among CCS especially in older patients with longer follow-up mandates longitudinal follow-up to assess long-term pulmonary outcome and plan intervention strategies for this subset.

Measurable residual disease (MRD) is critical in guiding therapeutic strategies for B-cell acute lymphoblastic leukemia (B-ALL). This study evaluated the performance of a novel next-generation sequencing-based Celemics IGH assay (CM-IGH; Celemics, Seoul, Korea) compared with the LymphoTrack^® IGH FR1 assay (LT-IGH; Invivoscribe Technologies, USA) and multiparameter flow cytometry (MFC). A total of 31 diagnostic and 60 follow-up bone marrow aspirate samples, all from the same 31 pediatric patients with B-ALL, were analyzed using the CM-IGH and LT-IGH assays on the MiSeq platform, as well as MFC according to EuroFlow guidelines. Initial IGH clonality was detected in 83.9% of CM-IGH samples and 90.3% of LT-IGH samples (p = 0.060). MRD positivity rates in follow-up samples were 74.5% for CM-IGH, 61.1% for LT-IGH, and 56.7% for MFC. CM-IGH showed concordance rates of 78.3% with LT-IGH and 68.1% with MFC, while LT-IGH demonstrated an 81.5% concordance rate with MFC. The correlation coefficients (r) of MRD levels were 0.831 between CM-IGH and LT-IGH, 0.702 between CM-IGH and MFC, and 0.776 between LT-IGH and MFC. The CM-IGH assay demonstrates substantial concordance with LT-IGH and MFC in detecting MRD in pediatric patients with B-ALL, highlighting the complementary value of IGH clonality assays and MFC.