Pediatric Hematology and Oncology最新文献

This study assessed the timeline from the initial presentation of symptoms of major cancers to the arrival at and diagnosis by the reference center for pediatric oncology in Barretos, São Paulo, Brazil. The goal was to examine the flow of care within the healthcare system and identify disparities indicative of inequitable access to health services. We conducted a cross-sectional analysis of patient data over eight years. Quantitative variables were examined using median values, T-tests, Kruskal-Wallis tests, ANOVA, Bonferroni adjustments, and pairwise comparisons. Categorical variables were analyzed using the Chi-square test, with a significance threshold set at 5%. A total of 508 patients with malignant cancers were studied. Patients with hematologic cancers experienced the shortest median times from initial symptom perception (2 days), to the first healthcare visit (1 day), onward referral to a specialized center (30 days), and final diagnosis (35 days). This is in contrast to patients with central nervous system cancers (2, 3, 90, and 112.5 days respectively) and bone cancers (20, 7, 60, and 90 days respectively). Families from municipalities ranked lower on the GeoSES socioeconomic scale reported longer durations before recognizing initial symptoms (p-value = 0.037). Low initial diagnostic suspicion and systemic delays are primary obstacles that need addressing to enhance the diagnosis and treatment of pediatric cancer in Brazil's most populous state.

Acute promyelocytic leukemia (APL) is an uncommon subtype of acute myeloid leukemia that is associated with hemorrhagic complications and early death. Our primary objective was to describe the frequency of hemorrhagic complications and outcomes associated with APL during the initial admission using the Pediatric Health Information System (PHIS) database. We performed a retrospective cross-sectional analysis of children with APL not in remission. Demographic characteristics, resource utilization, and outcomes were compared between those with and without hemorrhagic complications. Out of 173 patients with APL, hemorrhagic complications (intracranial, gastrointestinal, or pulmonary) occurred in 32%. Children with hemorrhagic complications were more likely to have acute respiratory failure, coagulopathy, stroke, and cerebral edema. Children with hemorrhagic complications experienced more ICU admissions and mechanical ventilation, and they also received transfusions, low molecular weight heparin, hydroxyurea, and rasburicase more frequently. The median length of stay and mortality rate was not different between the groups. The median length of stay of children who died was significantly lower (p = 0.003) as the majority (78%) who died death occurred within 7 days. Hemorrhagic complications occurred in a 3^rd of patients with the initial admission of APL, and most deaths occurred during the first week.

Kaposi sarcoma (KS) is a common childhood cancer in Malawi, but few studies have explored clinical characteristics of relapsed disease. We aimed to characterize clinical patterns of relapse to improve treatment and, ultimately, long-term survival in patients with pediatric KS. A retrospective cohort study was conducted among patients ages <19 years of age at time of KS diagnosis in Lilongwe, Malawi between August 1, 2010 and March 15, 2020. Specifically, emphasis was placed on patients who had relapsed disease and excluded patients with refractory disease or those who died whilst receiving front-line treatment. Salvage therapy typically involved an intensified chemotherapy regimen compared to front-line therapy - namely nonliposomal doxorubicin plus bleomycin/vincristine or paclitaxel monotherapy. One-hundred and ninety patients with pediatric KS were included in this analysis, 50 of whom experienced relapse (26%). Older median age was associated with occurrence of relapse (10 vs. 6.7 years, p-value = 0.004). Median time from diagnosis to first relapse was 10.6 months (range 2.3-49 months). Three-year post-relapse overall survival (OS) for the entire cohort was 60% with a median follow-up time of 4.7 years after relapse. Survival was significantly higher for patients who relapsed with the woody edema clinical phenotype of pediatric KS versus those with visceral/disseminated disease - 3-year OS 79% (95% CI 62-100) vs. 29% (14-61). These data demonstrate potential for continued survival after KS relapse in the pediatric population and identify subsets of high-risk patients. The higher mortality observed in patients with visceral/disseminated KS highlights the need for improved therapeutic strategies.

Autoimmune hemolytic anemia (AIHA) is rare inchildren. Little is known about the efficiency of red blood cells (RBCs) transfusions and the risk of reactions among children with severe AIHA. This article describes the clinical features and outcomes in AIHA children after RBCs transfusions. A retrospective study was conducted among hospitalized AIHA children from July 2019 to October 2024 in a tertiarycare medical center in China. Twenty-six patients received 47 RBCs transfusions, with a median age at diagnosis of 59.5 months. Sixteen patients had secondary AIHA, with 8 systemic lupus erythematosus patients. Only 7 RBCs transfusions were compatible with both major and minor cross-matching. After transfusions, the median hemoglobin levels increased from 43 to 67 g/L. All patients were treated with steroids and 24 patients were treated with intravenous immunoglobulin before RBCs transfusions. No adverse reaction was observed. The median follow-up period was 39 months and the median steroid duration was 14 months. Nine patients experienced relapses after reducing steroid dosage. In conclusion, systemic lupus erythematosus is the main causes of secondary severe AIHA in children. Blood transfusions after steroids and intravenous immunoglobulin, even with incompatible RBCs, are safe and efficient for for children with severe AIHA.

The management of non-neutropenic fever in pediatric oncology varies widely, with many providers choosing to administer empiric antibiotics. We conducted a quality improvement intervention seeking to decrease empiric antibiotic administration in pediatric oncology patients with a central venous catheter presenting in the outpatient setting with non-neutropenic fever assessed to be low-risk for bacteremia. Over a 1-year period, empiric antibiotics were administered at the index visit in 9.6% of episodes, compared with 97% prior to the intervention. In patients not receiving empiric antibiotics, the bacteremia rate was 2.3% and composite event rate 5.1%, similar to pre-intervention baseline. These data suggest that empiric antibiotics can be withheld in low-risk patients with non-neutropenic fever.

Pediatric cancer survivorship presents significant challenges globally, with varying survival rates and survivorship care models across different income settings. This present study focuses on the psycho-social outcomes of pediatric cancer survivors attending an After Completion Therapy (ACT) Clinic in a low-middle-income country, where survivorship care resources are limited compared to high-income countries. The study included 394 pediatric cancer survivors, aged 18 years and above, who had completed two years of disease-free survival post-treatment. Study data was collected from the ACT Clinic's survivorship proforma standardized by the experts in the field of Psycho-oncology and pediatric cancer survivorship, NCCN Distress Thermometer, and Visual Analog Pain Scale. Statistical analysis included descriptive statistics and chi-square tests to assess associations between demographic, clinical, and psycho-social variables. The majority of survivors were male (69.3%) hailing from rural areas (68.3%), with a mean age of 21 years. Haematological malignancies (61.3%) were predominant, treated mainly with chemotherapy (79%). Moderate distress was reported by 53% of female survivors. Cancer diagnosis (χ²(9) = 19.642, p < 0.020) and treatment modality (χ²(9) = 17.888^a, p < 0.036) are significantly influenced by distress levels. Academic and occupational status did not show a significant impact on distress but observed a notable percentage of challenges in post-treatment normalcy. Pediatric cancer survivors attending the ACT Clinic in resource-limited setting face substantial psycho-social challenges, influenced by gender, occupation, cancer type, and treatment history. The study emphasis the critical need for gender-sensitive and holistic survivorship care programs which should be tailored to address these challenges comprehensively. There is a need for more healthcare collaborations, mental health support, educating care givers on the importance of survivorship and improving advocacy through survivors support group activities. These programs can enhance the framework of survivorship care in low middle-income countries.

Alpelisib was recently approved by the FDA for the management of pediatric patients with PIK3CA-related overgrowth spectrum. However, this medication was approved in the absence of pediatric pharmacokinetic data, as a fixed 50 mg dose, with no consideration of weight, the primary pharmacokinetically relevant covariate. This raises concerns regarding potential under and over-exposure. Given this gap in information, we aimed to assess the effect of alpelisib in relation to drug exposure (clinical response and drug safety). Alpelisib plasma concentrations were obtained from eight patients under treatment for vascular malformations. Drug exposure determined with area under the curve (AUC) was correlated to drug effect determined by a decrease in the size of lesions and grade of adverse events. Analysis was performed retrospectively. Eight patients received oral alpelisib through the compassionate use program of Novartis. AUC revealed substantial variability (3036 to 16620 ug*h/L) and inversely correlated to weight. Alpelisib resulted in marked clinical improvement, reducing pain, resolving coagulopathy, and improving mobility. Volumetric MRI indicated a 17.4% decrease in targeted vascular anomaly volume after 6 months of alpelisib therapy (p < 0.05), although volume decrease did not correlate with AUC. Adverse events including insulin resistance (n = 8/8) and growth restriction (n = 1/8) were documented, with severity directly correlating to drug exposure. We observed significant weight-related variability in alpelisib plasma concentrations, suggesting that the FDA-approved fixed-dose regimen of alpelisib is not optimal for pediatric patients. Weight-based dosing and therapeutic drug monitoring should be considered to enhance alpelisib safety.

Reduced bone mineral density (BMD) has been reported during and after treatment of children with hematologic malignancies. However, little is known about the skeletal status of these patients at diagnosis. The aim of this study was to evaluate the skeletal profile of newly diagnosed pediatric patients with Acute lymphoblastic leukemia (ALL), Hodgkin Lymphoma (HL), and Non-Hodgkin Lymphoma (NHL). A case-control study included 50 children with ALL, 11 with HL, and 10 with NHL and compared them to 108 sex- and age-matched controls. Patients underwent bone metabolism evaluation and dual-energy X-ray absorptiometry (DXA) scan at the time of diagnosis. Seventy-one children were evaluated (43 boys) with a median age of 8.25 years (2.16-17.33 years). Twenty-one with ALL had bone pain (16 with a limp pain) at diagnosis. More than half (59.1%) of the patients were vitamin D sufficient (25-ΟΗ-D > 20 ng/ml). Patients had lower values of serum procollagen type I C-terminal propeptide (PICP), osteocalcin (OC), and tartrate-resistant acid phosphatase (bTRAP5b) (p < 0.001) than controls. A DXA scan was performed in 45 patients. Patients with ALL and Lymphoma had lower values of Lumbar Spine (L1-L4, LS) BMD Z-score (p < 0.001, p < 0.01, respectively) while those with ALL had lower values of Total Body Less Head (ΤBLH) BMD Z-score (p = 0.003) than controls. Skeletal health is adversely affected in pediatric patients with ALL and Lymphoma at diagnosis. These observations support bone health surveillance in cancer patients and timely intervention starting at the time of diagnosis.