Journal of Pediatric Hematology/Oncology最新文献

Ganglioneuroblastoma intermixed (GNB-I) is a rare pediatric tumor with favorable outcomes when treated primarily with surgical resection. In patients for whom surgical intervention is not feasible, chemotherapy is often considered, although its efficacy remains controversial. This retrospective study examined 12 patients with nonmetastatic GNB-I at Phoenix Children's Hospital. Four patients received chemotherapy but showed no significant tumor reduction postchemotherapy, and all required surgery afterward. Chemotherapy led to notable toxicities, including febrile neutropenia and anaphylactic reaction. These findings suggest chemotherapy offers limited benefit while posing significant risks of cytotoxic adverse events for unresectable GNB-I, and surgery should be prioritized. Further longitudinal studies are needed to confirm these results.

Synovial sarcoma is the most common nonrhabdomyosarcoma soft tissue sarcoma in children and adolescents, typically associated with more favorable outcomes compared with adults. However, the role of 18 F-fluorodeoxyglucose ( 18 F-FDG) PET imaging in pediatric synovial sarcoma is underexplored. This study investigates the prognostic value of pretreatment maximum standardized uptake value (SUVmax) on 18 F-FDG PET imaging in 15 pediatric patients with synovial sarcoma who underwent PET/CT or PET/MRI and MRI. SUVmax of the primary tumors were analyzed for their association with overall survival (OS) and progression-free survival (PFS). The median SUVmax was 4.64 (range: 1.66 to 12.46). Although higher SUVmax tended to be associated with poorer OS and PFS, no statistically significant associations were found. MRI findings also showed no significant association with outcomes. Our result highlights that tumors with low SUVmax can still exhibit aggressive behavior. High-grade tumors and those larger than 5 cm were associated with higher SUVmax, and were more frequently located in axial regions. These findings suggest integrating SUVmax with tumor grade, tumor size and location may enhance risk stratification and support more individualized treatment strategies in pediatric synovial sarcoma.

Children with cancer frequently experience gastrointestinal symptoms such as mucositis, nausea, vomiting, and feed intolerance, impairing nutritional intake and often necessitating parenteral nutrition (PN). However, standardized criteria guiding PN initiation in pediatric oncology remain poorly defined. This study examined PN use in pediatric oncology patients, identifying key indications, patterns, and clinical considerations. A retrospective cohort study was conducted on pediatric oncology patients who received PN at a tertiary cancer center in New Zealand between June 2018 and December 2021. Data on demographics, diagnosis, nutritional status, PN indications, duration, concurrent enteral nutrition (EN), and clinical outcomes were analyzed using descriptive statistics. PN was prescribed in 108 episodes for 62 children (mean age: 7.3 y). The primary indication was mucositis (59%), followed by neutropenic enteritis (19%). PN duration was longer in hematologic malignancies than in solid tumors ( P =0.014). Malnutrition was present in 44% of patients at PN initiation. Using hospital guidelines, 77% trialed EN before PN, and EN duration before PN was deemed adequate in 62%. Concurrent EN was more common in nonhematologic (98%) than hematologic diagnoses (93%; P =0.003). PN remains essential in pediatric oncology, particularly for patients with severe mucositis. Clearer PN initiation criteria are needed to minimize overuse and associated risks.

Purpose: Mixed phenotype acute leukemia (MPAL) represents an uncommon but heterogenous disease, often posing both a diagnostic and therapeutic challenge. The purpose of this retrospective study was to analyze the overall survival, event-free survival, and severity of associated complications after allo-HSCT in children with MPAL, and provide feasible recommendations for the treatment of MPAL patients.

Patients and methods: We retrospectively analyzed a total of 14 pediatric patients with MPAL who received allo-HSCT at our center between January 2010 and June 2024.

Results: In terms of immunophenotype, coexpression of myeloid and B-lymphoid antigens was observed in 10 patients (71.4%), and myeloid and T-lymphoid antigens in 4 (28.6%). Chromosomal abnormalities were found in 8 patients (57.1%) and BCR/ABL(+) was the most common fusion gene (3/14; 21.4%). All 14 patients underwent allo-HSCT after achieving the CR1 (78.6% with MRD-negative status pretransplantation). Among the 14 transplanted children, the OS rate was 92.9% and the EFS rate was 85.7%. No significant difference in OS, EFS, and CIR rates between children with Haplo-HSCT and those with MSD-HSCT (P>0.05). The rate of acute GVHD was 57.1% (8/14), and the rate of chronic GVHD was 71.4%, of which 90% were assessed as mild cGVHD, with the skin being the most common organ involved in cGVHD. Only one patient developed TA-TMA and died from transplant-related complications.

Conclusion: The children with MPAL who received allo-HSCT after MRD-negative CR often had a favorable disease control. Compared with patients receiving conventional chemotherapy, pediatric patients who received allo-HSCT showed a significant improvement in OS, EFS, and CIR rates. Although the incidence of cGVHD was relatively high, most of them were assessed as mild with no significant impact on daily activities.

Background: Keratin-positive giant cell-rich tumor (KPGCT) is a rare bone and soft tissue neoplasm, with pediatric and metastatic cases being exceedingly uncommon.

Observation: We report a 1.5-month-old male infant presenting with multifocal metastatic disease involving the skull, adrenal glands, vertebrae, mandible, soft tissue, and long bones. Histopathologic evaluation confirmed KPGCT, molecular testing was negative for HMGA2 rearrangement. Treatment with imatinib resulted in marked regression of lesions and clinical improvement without toxicity.

Conclusions: This case expands the clinical spectrum of pediatric KPGCT and suggests that imatinib may be an effective treatment option in infants with advanced disease, even canonical HMGA2::NCOR2 fusion absent.

The implementation of pediatric protocols for the treatment of adolescents and young adults (AYA) has led to an improvement in the survival rate of patients with acute leukemia (AL). However, the incidence of complications is higher in this population than in children. The objective of this study is to compare the occurrence of infectious complications during periods of severe neutropenia in children, adolescents, and young adults. This retrospective single-center observational study compares the incidence of infections during severe neutropenia episodes and the management of these between 2 groups (children and AYA) over a 4-year period (2017 to 2021). A total of 54 patients and 243 severe neutropenia episodes were examined, and 63 infections were identified. The incidence of infection was significantly higher in the AYA population (40% vs. 23%, P=0.02), but this difference was linked to a higher frequency of acute myeloid leukemia. However, AYA population presents more catheter-related infections (12% vs. 2%, aOR: 6.4, 95% CI: 1.7-25.1, P<0.01) and infections of the skin and soft tissues (14% vs. 4%, aOR: 3.9, 95% CI: 1.2-12.8, P=0.03) irrespective of the type of leukemia. In conclusion, this study reveals that AYA had more infectious complications than children, particularly more catheter, skin, and soft tissue infections.

Measles encephalitis results in devastating epilepsia partialis and/or mortality in the majority of children treated with intense chemotherapy for hematolymphoid malignancies. Although many agents have been studied in the treatment of Measles encephalitis with variable results, there are no effective strategies to mitigate the significant morbidity associated with measles encephalitis till date and treatment has remained largely supportive. In this report, a novel combination of antivirals have been shown to abrogate the morbid sequelae of Measles encephalitis with eventual complete recovery in a child with B-acute lymphoblastic leukemia.

Vitamin K deficiency bleeding (VKDB) is a well-known entity in the newborn period, classically presenting during the first week of life. VKDB causing massive bleeding beyond 3 months of age, in an otherwise healthy child, is extremely rare. We present a rare case of massive intracranial bleed in an infant who had received routine vitamin K prophylaxis at birth. His blood investigations revealed severe anemia with a grossly deranged coagulation profile and high proteins induced by vitamin K absence (PIVKAII). His coagulation parameters normalised within 12 hours of Vitamin K and a single dose of FFP. He was a well-nourished child with no evidence of malabsorption. He had no risk factors for VKDB except for extended exclusive breastfeeding. His coagulation profile remained normal at the 6-month review, also. VKDB should be considered outside the typical age group in a child with delayed introduction of complementary feeds.

Osteosarcoma is the most common primary malignancy of bone in children. Stereotactic body radiotherapy (SBRT) is an ablative technique that can overcome radioresistance. The use of SBRT in treating osteosarcoma bone metastases is understudied. Osteosarcoma patients with bony metastases from a single institution were retrospectively reviewed. Treatment response was evaluated per RECIST 1.1 criteria. Adverse effects were evaluated via the Common Terminology Criteria for Adverse Events (CTCAE) grading scale. Thirteen lesions from 9 patients were treated with SBRT. The median time to follow-up was 9.5 months (range 3 to 20.2 mo). Mean pretreatment volume was 48.7 cm 3 . Median delivered dose was 40 Gy in 5 fractions (range 30 Gy in 5 fractions to 48 Gy in 8 fractions). Twelve lesions (92%) showed stable disease (SD). One (8%) lesion showed progressive disease (PD) after 40 Gy in 5 fractions. Local control was 100% at 6 months and 87.5% at 12 months. Pretreatment pain was reported in 78% of patients. Seventy-one percent reported improvement in pain. There were no acute grade ≥3 toxicities observed. SBRT offers promising local control rate in the treatment of osteosarcoma bone metastases with a limited acute side-effect profile. Further studies with a longer follow-up time and larger cohorts are warranted.