Journal of Pediatric Hematology/Oncology最新文献

Background: Pediatric germ cell tumor (GCT) with extensive cavo-atrial tumor thrombus presenting as an oncologic emergency is extremely rare.

Observation: A 13-month-old female child with sacrococcygeal GCT having inferior vena cava (IVC) and right atrial thrombus presenting with anasarca and respiratory distress is reported. Because of worsening symptoms post initiation of chemotherapy, tumor thrombus debulking from the right atrium and distal end of IVC was performed on day 9 on an emergency basis. Her symptoms improved, and after planned chemotherapy, tumor excision with coccygectomy could be done. The IVC thrombus remained inoperable due to sclerosis. She remains recurrent-free now 12 months post therapy.

The recent trial Pediatric Neuro-Oncology Consortium 003 (PNOC003) utilized a molecular tumor board to recommend personalized treatment regimens based on tumor sequencing results in children with DIPG. We separately developed the Central Nervous System Targeted Agent Prediction (CNS-TAP) tool, which numerically scores targeted anticancer agents using preclinical, clinical, and patient-specific data. We hypothesized that highly scored agents from CNS-TAP would overlap with the PNOC003 tumor board's recommendations. For each of the 28 participants, actionable genetic alterations were derived from PNOC003 genomic reports and input to CNS-TAP to identify the highest scoring agents. These agents were then compared with PNOC003 recommendations, with a resultant concordance percentage calculated. Overall, 38% of the total agents recommended by the tumor board were also selected by CNS-TAP, with higher concordance (63%) in a subanalysis including only targeted anticancer agents. Furthermore, nearly all patients (93%) had at least 1 drug chosen by both methods. We demonstrate overlap between agents recommended by CNS-TAP and PNOC003 tumor board, though this does not appear to improve survival. We do observe some discordance, highlighting strengths and limitations of each method. We propose that a combination of expert opinion and data-driven tools may improve targeted treatment recommendations for children with DIPG.

Introduction: Thrombocytopenia is a common clinical problem in cancer patients undergoing high-dose chemotherapy and autologous hematopoietic stem cell transplantation (HSCT). It can occur as prolonged isolated thrombocytopenia (PIT) or secondary failure of platelet recovery (SFPR) and may cause potentially fatal bleeding. However, data on the treatment of post-transplant thrombocytopenia is still lacking.

Methods: We reported our practices involving 15 pediatric patients who received eltrombopag (ELT) treatment for PIT and SFPR after autologous HSCT.

Results: The overall response was 78.5% (11/14), with 1 patient excluded due to noncompliance. The 12 surviving patients' median follow up was 699 days (range: 167 to 2250 d).

Conclusions: Our study indicates the efficacy and safety of ELT for treating PIT and SFPR after autologous HSCT in pediatric patients. However, more studies are needed to confirm these findings in children.

Objective: Childhood cancer treatment disrupts vaccination schedules and weakens or eliminates vaccine-induced immunity. In addition, post-treatment vaccine responses vary. This study aimed to assess post-treatment serum antibody levels and vaccine responses in children.

Methods: Pediatric patients treated at Hacettepe University between years 2015 and 2020, achieved remission after chemotherapy for lymphoma and solid tumors were included. Post-treatment vaccination status, serum antibody levels for hepatitis A (HAV), hepatitis B (HBV), varicella-zoster (VZV), measles-mumps-rubella (MMR), and changes in vaccine responses were retrospectively analyzed.

Results: The study included 533 patients. Post-treatment seronegativity rates were: measles (83.5%), HAV (64%), rubella (60.1%), HBV (48.5%), VZV (43.3%), and mumps (28%). Post-treatment antibody loss was observed for measles (47.1%), HAV (31.9%), HBV (31.4%), mumps (28.6%), VZV (21.7%), and rubella (11.4%). Seropositivity after 1 vaccine dose was seen with HAV (83.6%), rubella (82.9%), HBV (81.4%), VZV (63.5%), mumps (45.4%), and measles (33.3%). Seropositivity after 2 vaccine doses was achieved with HAV (98.8%), VZV (84.6%), rubella (80%), HBV (80%), measles (32.2%), and mumps (36.2%).

Conclusion: Post-treatment serological vaccine responses in children were lower than anticipated despite multiple doses. Given the potential need for periodic serological assessments and booster vaccinations, long-term follow-ups are planned.

Pediatric thrombocytopenia is frequently observed in critical care and oncology settings with an increased risk of bleeding and platelet transfusions. However, little is known about low platelets in childhood during seasonal influence. This study aimed to evaluate the frequency and severity of pediatric thrombocytopenia in the postflood period. The patients 1 to 18 years of age with thrombocytopenia (platelet count <150×109/L) were studied from August to December 2022 after institutional ethical approval (ERC-Path-2022-8044-23395). Data was collected from electronic health records and laboratory information systems. Of 2318 admitted patients, 192 (8.3%) including 128 males and 64 females had thrombocytopenia. The median (IQR) age was 12 (8 to 15) years. Mild, moderate, and severe thrombocytopenia were seen in 109 (56.8%), 76 (39.5%), and 7 (3.6%) patients, respectively. Concomitant leucopenia was observed in 77 of 192 patients (40.1%). Infection was the predominant cause of low platelets (N=175 or 91.1%). Only 15 patients (7.8%) had grade 1/2 bleeding. Overall, 176 patients (92%) were discharged in stable conditions and no mortality was observed. The frequency of pediatric thrombocytopenia in the noncritical and nononcological care settings was <10% and mostly observed in association with underlying infections. The frequency of bleeding manifestation and platelet transfusions was minimal in this group.

Hodgkin lymphoma with vanishing bile duct syndrome is a rare paraneoplastic syndrome and has never been studied in the pediatric population. The objectives of this study were to determine the clinical characteristics of this rare condition in children through a literature review, and a descriptive analysis of all published cases with the index case report. All reported cases fulfilling the inclusion criteria were found through a literature search, and analyzed in descriptive statistics. A total of 10 cases were included in the study with a median age of 9.5 years and a male-to-female ratio of 9:1. The median duration of symptoms was 5.5 weeks with 3 cases having jaundice before the symptoms of lymphoma. The median bilirubin level was 8.4 mg/dL. Seven cases received modified chemotherapy, and 5 used ursodeoxycholic acid. The survival rate was 50%. Normalization of liver functions after the lymphoma treatment was observed in 4 cases and was the only statistically significant factor ( P =0.01) associated with the outcome. This is a rare entity in the pediatric population with a guarded prognosis comparable to the adult counterparts but a marked male predominance.

Nijmegen breakage syndrome (NBS) is a rare primary immunodeficiency disease due to a pathogenic variant in the NBN gene causing impaired DNA repair and increased predisposition for lymphoid malignancy. By contrast, solid tumors have been rarely reported. Neuroblastoma (NB) is a rare childhood solid tumor, associated with the worse outcome if MYCN oncogene is amplified. We describe 2 young pediatric patients with NBS who developed high-risk NB. The first patient died shortly after chemotherapy was introduced. The second patient successfully received modified chemotherapy resulting in clinical remission lasting 2 years after an initial diagnosis of NB.

Objectives: This study aimed to develop machine learning (ML) prediction models for identifying bloodstream infection (BSI) and septic shock (SS) in pediatric patients with cancer who presenting febrile neutropenia (FN) at emergency department (ED) visit.

Materials and methods: A retrospective study was conducted on patients, younger than 18 years of age, who visited a tertiary university-affiliated hospital ED due to FN between January 2004 and August 2022. ML models, based on XGBoost, were developed for BSI and SS prediction.

Results: After applying the exclusion criteria, we identified 4423 FN events during the study period. We identified 195 (4.4%) BSI and 107 (2.4%) SS events. The BSI and SS models demonstrated promising performance, with area under the receiver operating characteristic curve values of 0.87 and 0.88, respectively, which were superior to those of the logistic regression models. Clinical features, including body temperature, some laboratory results, vital signs, and diagnosis of acute myeloblastic leukemia were identified as significant predictors.

Conclusions: The ML-based prediction models, which use data obtainable at ED visits may be valuable tools for ED physicians to predict BSI or SS.