Rhabdoid tumor predisposition syndrome: A historical review of treatments and outcomes for associated pediatric malignancies.

Rhabdoid tumor predisposition syndrome (RTPS) is a rare disorder associated with malignant rhabdoid tumor of the kidney (RTK), atypical teratoid rhabdoid tumor (ATRT), and/or other extracranial, extrarenal rhabdoid tumors (EERT), and these pediatric malignancies are difficult to treat. Presently, most of the information regarding clinical manifestations, treatment, and outcomes of rhabdoid tumors comes from large data registries and case series. Our current understanding of treatments for patients with rhabdoid tumors may inform how we approach patients with RTPS.

Variability in Surveillance Strategies Following Resection of Sacrococcygeal Teratoma.

Introduction: Surveillance following sacrococcygeal teratoma (SCT) resection varies. The purpose of this study was to describe the clinical characteristics and outcomes of patients undergoing SCT resection and examine current institutional practices to detect recurrence.

Methods: A single-institution retrospective review of children who underwent resection of an SCT from January 1, 2010 to December 31, 2020 was performed.

Apixaban versus no anticoagulation for the prevention of venous thromboembolism in children with newly diagnosed acute lymphoblastic leukaemia or lymphoma (PREVAPIX-ALL): a phase 3, open-label, randomised, controlled trial.

Background: Paediatric patients with acute lymphoblastic leukaemia or lymphoma are at increased risk of venous thromboembolism resulting in increased mortality and morbidity. We hypothesised that apixaban, a direct oral anticoagulant, would safely reduce venous thromboembolism in this patient population.

Methods: PREVAPIX-ALL was a phase 3, open-label, randomised, controlled trial conducted in 74 paediatric hospitals in 9 countries.

Utilizing a Quality Improvement Strategy to Increase Faculty Engagement With Resident Learning Goals.

Background: Establishing and achieving learning goals (LGs) are important lifelong learning skills for residents. Faculty are critical in facilitation and achievement of residents' LGs, yet many have difficulty with this role in the busy inpatient setting.

Objectives: Our primary aim was to improve faculty engagement in resident LGs, targeting ≥80% of faculty achieving a mean score ≥goal, over a period of 1 year by setting prerotation expectations and stressing team-based faculty accountability during the rotation in the current inpatient learning environment.

Molecular Heterogeneity in Pediatric Malignant Rhabdoid Tumors in Patients With Multi-Organ Involvement.

Rhabdoid tumors (RTs) of the brain (atypical teratoid/rhabdoid tumor; AT/RT) and extracranial sites (most often the kidney; RTK) are malignant tumors predominantly occurring in children, frequently those with germline alterations. Here we present data from seven RTs from three pediatric patients who all had multi-organ involvement. The tumors were analyzed using a multimodal molecular approach, which included exome sequencing of tumor and germline comparator and RNA sequencing and DNA array-based methylation profiling of tumors.

EGFR internal tandem duplications in fusion-negative congenital and neonatal spindle cell tumors.

Next-generation sequencing (NGS) assays can sensitively detect somatic variation, and increasingly can enable the identification of complex structural rearrangements. A subset of infantile spindle cell sarcomas, particularly congenital mesoblastic nephromas with classic or mixed histology, have structural rearrangement in the form of internal tandem duplications (ITD) involving EGFR. We performed prospective analysis to identify EGFR ITD through clinical or research studies, as well as retrospective analysis to quantify the frequency of EGFR ITD in pediatric sarcomas.

Wilms Tumor (Nephroblastoma), Version 2.2021, NCCN Clinical Practice Guidelines in Oncology.

The NCCN Guidelines for Wilms Tumor focus on the screening, diagnosis, staging, treatment, and management of Wilms tumor (WT, also known as nephroblastoma). WT is the most common primary renal tumor in children. Five-year survival is more than 90% for children with all stages of favorable histology WT who receive appropriate treatment.

Gastroblastoma with a novel EWSR1-CTBP1 fusion presenting in adolescence.

Gastroblastomas are rare tumors with a biphasic epithelioid/spindle cell morphology that typically present in early adulthood and have recurrent MALAT1-GLI1 fusions. We describe an adolescent patient with Wiskott-Aldrich syndrome who presented with a large submucosal gastric tumor with biphasic morphology. Despite histologic features consistent with gastroblastoma, a MALAT1-GLI1 fusion was not found in this patient's tumor; instead, comprehensive molecular profiling identified a novel EWSR1-CTBP1 fusion and no other significant genetic alterations.

Functional fecal and urinary outcomes after sacrococcygeal mass resection in pediatric patients.

Background: Sacrococcygeal masses (SCM) are uncommon in children. The purpose of this study is to review the functional fecal and urinary outcomes following resection of SCM and to determine the impact of a multidisciplinary clinic (MDC) on these outcomes.

Methods: A retrospective review was performed of patients who underwent SCM resection between 1979 and 2019.

Longitudinal understanding of prognosis among adolescents with cancer.

Objective: Despite calls to increase prognosis communication for adolescents with cancer, limited research has examined their perceptions of prognosis as compared with their parents. We assessed adolescents' understanding of their prognosis relative to parents and oncologists.

Methods: Families of adolescents (aged 10-17) were recruited at two pediatric institutions following a new diagnosis or relapse.

Cabozantinib for relapsed neuroblastoma: Single institution case series.

Relapsed high-risk neuroblastoma has few effective therapies currently available or in development. Cabozantinib is an Food and Drug Administration approved multitargeted tyrosine kinase inhibitor for select adult malignancies with preclinical data suggesting efficacy against neuroblastoma. A safe and tolerable dose has been identified for children, but its efficacy remains unknown.

Improving Quality of Chest Computed Tomography for Evaluation of Pediatric Malignancies.

Introduction: Atelectasis is a problem in sedated pediatric patients undergoing cross-sectional imaging, impairing the ability to accurately interpret chest computed tomography (CT) imaging for the presence of malignancy, often leading to additional maneuvers and/or repeat imaging with additional radiation exposure.

Methods: A quality improvement team established a best-practice protocol to improve the quality of thoracic CT imaging in young patients with suspected primary or metastatic pulmonary malignancy. The specific aim was to increase the percentage of chest CT scans obtained for the evaluation of pulmonary nodules with acceptable atelectasis scores (0-1) in patients aged 0-5 years with malignancy, from a baseline of 45% to a goal of 75%.

First-in-Human Intravenous Seprehvir in Young Cancer Patients: A Phase 1 Clinical Trial.

Seprehvir (HSV1716) is an oncolytic herpes simplex virus-1 (HSV-1) previously demonstrated to be well tolerated in pediatric patients when administered intratumorally. To determine the safety of administering Seprehvir systemically, we conducted the first-in-human phase I trial of intravenous injection in young patients with relapsed or refractory extra-cranial solid cancers. We delivered a single dose of 5 × 10 infectious units (iu)/kg (maximum dose of 2 × 10) or 2.

Multifocal Appendiceal Carcinoid Tumor in an Adolescent: A Case Report and Review of the Literature.

Appendiceal carcinoid tumors in children and adolescents are rare. This report describes a case of a multifocal appendiceal carcinoid tumor identified incidentally following appendectomy in an adolescent. In this report, we describe the staging process and surgical management for focal and locally invasive appendiceal carcinoid tumors and highlight the rarity of multifocality in this location.

Renal Tumors in Children and Young Adults Older Than 5 Years of Age.

Renal masses are most common in children between ages 1 to 3 years, with less known about renal tumors in older children and young adults. The aim of this study was to review the presentation, demographics, histology, and outcomes in patients over 5 years of age with renal tumors compared with younger children. 111 renal tumors were diagnosed in patients 5 years of age and older (median, 7 y; range, 5 to 31 y) between 1950 and 2017 at a single institution.

Veno-occlusive disease after high-dose busulfan-melphalan in neuroblastoma.

Survival for high-risk neuroblastoma patients is still suboptimal. Although stem cell transplantation (SCT) is used, there is no consensus as to which conditioning regimen has the greatest efficacy and fewest toxicities. We assessed the incidence of and risk for hepatic veno-occlusive disease (VOD) for neuroblastoma patients who underwent autologous SCT with busulfan and melphalan (BuMel) at eight centers following Children's Oncology Group (COG)-based induction chemotherapy.

Fostering Strategies to Expand the Consumption of Edible Insects: The Value of a Tripartite Coalition between Academia, Industry, and Government.

Although many insect-based foods are nutritious and often an inexpensive option for human and domesticated animal consumption, there remains a negligible market for such foods in many countries. Several environmental and economic considerations underscore the potential value of insect-based foods, and emerging science suggests that diets incorporating such foods might also convey some genuine health benefits. However, if expanded markets for insect-based foods in cultures naïve to entomophagy are to be pursued, it will be important to develop multifaceted and coordinated strategies to ) delineate authentic health benefits, ) explore means of optimizing insect husbandry and food processing, ) examine cultural barriers to acceptance, ) formulate workable approaches to marketing, and ) address relevant food regulations.

Clinical efficacy of cabozantinib in two pediatric patients with recurrent renal cell carcinoma.

Advanced-stage renal cell carcinoma (RCC) carries a dismal prognosis for pediatric patients with few studied therapeutic options. Cabozantinib is a small molecule tyrosine kinase inhibitor against the oncoprotein MET. It is currently approved by the U.

Renal Tumors in Children Younger Than 12 Months of Age: A 65-Year Single Institution Review.

Wilms tumor (WT) is the most prevalent pediatric renal tumor and most commonly occurs between ages 1 and 5 years. Data are lacking on children younger than 12 months with renal tumors. The cancer registry at the authors' institution was queried to identify patients 12 months and younger with renal masses.

Global microRNA profiling for diagnostic appraisal of melanocytic Spitz tumors.

Background: Melanoma skin cancer remains the leading cause of skin cancer-related deaths. Spitz lesions represent a subset of melanocytic skin lesions characterized by epithelioid or spindled melanocytes organized in nests. These lesions occupy a spectrum ranging from benign Spitz and atypical Spitz lesions all the way to malignant Spitz tumors.

Classification of Indeterminate Melanocytic Lesions by MicroRNA Profiling.

Purpose: Identification of indeterminate melanocytic skin lesions capable of neoplastic progression is suboptimal and may potentially result in unnecessary morbidity from surgery. MicroRNAs (miRs) may be useful in classifying indeterminate Spitz tumors as having high or low risk for malignant behavior.

Methods: RNA was extracted from paraffin-embedded tissues of benign nevi, benign Spitz tumors, indeterminate Spitz tumors, and Spitzoid melanomas in adults (n = 62) and children (n = 28).

Pancreaticoduodenectomy outcomes in the pediatric, adolescent, and young adult population.

Background: Pancreatic malignancy and chronic pancreatitis are rare in the pediatric, adolescent, and young adult (AYA) population, making pancreas resections an infrequent procedure in this demographic. Only case reports and small case series exist in the literature describing surgical outcomes and complications in this population. The aim of this study was to review the surgical outcomes of pediatric/AYA patients undergoing pancreaticoduodenectomy (PD) at our institution.

Carcinoid tumors in children and adolescents: risk for second malignancies.

Background: Carcinoid tumors (CTs) are rare in the pediatric population and are generally noted as an incidental finding on histopathologic examination. Cure is usually achieved with wide surgical excision. Second primary malignancies (SPM) of the gastrointestinal tract after CTs have been reported in 13% to 33% of affected adults.

Nothing but NET: a review of norepinephrine transporter expression and efficacy of 131I-mIBG therapy.

Neuroblastoma is unique amongst common pediatric cancers for its expression of the norepinephrine transporter (NET), enabling tumor-selective imaging and therapy with radioactive analogues of norepinephrine. The majority of neuroblastoma tumors are avid for (123)I-metaiodobenzaguanidine (mIBG) on imaging, yet the therapeutic response to (131) I-mIBG is only 30% in clinical trials, and off-target effects cause short- and long-term morbidity. We review the contemporary understanding of the tumor-selective uptake, retention, and efflux of meta-iodobenzylguanidine (mIBG) and strategies currently in development for improving its efficacy.