Symptom Screening for Hospitalized Pediatric Patients With Cancer: A Randomized Clinical Trial.

Importance: Pediatric patients with cancer experience severely bothersome symptoms during treatment. It was hypothesized that symptom screening and provision of symptom reports to the health care team would reduce symptom burden in pediatric patients with cancer.

Objective: To determine if daily symptom screening and provision of symptom reports to the health care team was associated with lower total symptom burden as measured by the Symptom Screening in Pediatrics Tool (SSPedi) compared to usual care among pediatric patients with cancer admitted to a hospital or seen in a clinic daily for at least 5 days.

Race and Ethnic Group Enrollment and Outcomes for Wilms Tumor: Analysis of the Current Era Children's Oncology Group Study, AREN03B2.

Background: To review race and ethnic group enrollment and outcomes for Wilms tumor (WT) across all 4 risk-assigned therapeutic trials from the current era Children's Oncology Group Renal Tumor Biology and Risk Stratification Protocol, AREN03B2.

Study Design: For patients with WT enrolled in AREN03B2 (2006 to 2019), disease and biologic features, therapeutic study-specific enrollment, and event-free (EFS) and overall (OS) 4-year survival were compared between institutionally reported race and ethnic groups.

Results: Among 5,146 patients with WT, no statistically significant differences were detected between race and ethnic groups regarding subsequent risk-assigned therapeutic study enrollment, disease stage, histology, biologic factors, or overall EFS or OS, except the following variables: Black children were older and had larger tumors at enrollment, whereas Hispanic children had lower rates of diffuse anaplasia WT and loss of heterozygosity at 1p.

Treatment of children with favorable histology Wilms tumor with extrapulmonary metastases: A report from the COG studies AREN0533 and AREN03B2 and NWTSG study NWTS-5.

Background: Patients with stage IV favorable histology Wilms tumor (FHWT) with extrapulmonary metastases (EPM) constitute a small subset of patients with FHWT. Because of their rarity and heterogeneity, optimal FHWT treatment is not well understood. Children's Oncology Group protocol AREN0533 assigned patients with FHWT and EPM to intensified chemotherapy, regimen M, after initial DD-4A chemotherapy.

Prognostic impact of lymph node involvement and loss of heterozygosity of 1p or 16q in stage III favorable histology Wilms tumor: A report from Children's Oncology Group Studies AREN03B2 and AREN0532.

Introduction: The prognostic impact of positive lymph nodes (LN+) and/or singular loss of heterozygosity (LOH) of 1p or 16q were assessed in children with stage III favorable histology Wilms tumor (FHWT) enrolled on AREN0532 or AREN03B2 alone.

Patients And Methods: A total of 635 stage III FHWT vincristine/dactinomycin/doxorubicin (DD4A)-treated patients met inclusion criteria. Event-free survival (EFS) and overall survival are reported overall and by LN sampling, LN status, LOH 1p, LOH 16q, and a combination of LN status and singular LOH.

Establishing Reliability and Validity of the FACE-Q Craniofacial Module for Pediatric Head and Neck Cancer.

We aimed to establish content validity and assess the psychometric properties of the FACE-Q Craniofacial Module, a patient-reported outcome measure, for use in pediatric and adolescent patients with head and neck cancer (HNC). To establish content validity (Part 1), between June 2017 and August 2019, cognitive interviews were conducted with survivors of pediatric HNC ( = 15), and input was obtained from clinical experts ( = 21). To examine item and scale performance (Part 2), Rasch Measurement Theory (RMT) analysis was performed using data from two international studies ( = 121).

Validation of co-Symptom Screening in Pediatrics Tool: a novel dyadic approach to symptom screening in pediatric patients receiving cancer treatment.

Background: Co-Symptom Screening in Pediatrics Tool (co-SSPedi) is a dyadic (child-guardian) approach to symptom assessment. Objectives were to evaluate the reliability and validity of co-SSPedi for pediatric patients receiving cancer treatments.

Methods: This multicenter study included dyads of patients aged 4-18 years of age with cancer or undergoing hematopoietic cell transplant and their guardians.

The NIH pediatric/young adult chordoma clinic and natural history study: Making advances in a very rare tumor.

Background: Chordomas are rare tumors arising from the skull base and spine, with approximately 20 pediatric chordoma cases in the Unitedn States per year. The natural history and optimal treatment of pediatric chordomas, especially poorly differentiated and dedifferentiated subtypes, is incompletely understood. Herein, we present findings from our first National Cancer Institute (NCI) chordoma clinic and a retrospective analysis of published cases of pediatric poorly differentiated chordomas (PDC) and dedifferentiated chordomas (DC).

Reductions in Bone Mineral Density Are Apparent Early in Children With Prevalent Osteonecrosis Lesions Following Leukemia Therapy.

Osteonecrosis (ON) is a serious complication of childhood acute lymphoblastic leukemia. We determined the prevalence of osteonecrotic lesions in our patient population by a one-time multisite magnetic resonance imaging (MRI) more than 1 year following leukemia therapy. MRI findings were evaluated in relationship to clinical factors (including longitudinal changes in bone mineral density [BMD]).

Tumor biology, biomarkers, and liquid biopsy in pediatric renal tumors.

The expansion of knowledge regarding driver mutations for Wilms tumor (WT) and malignant rhabdoid tumor of the kidney (MRT) and various translocations for other pediatric renal tumors opens up new possibilities for diagnosis and treatment. In addition, there are growing data surrounding prognostic factors that can be used to stratify WT treatment to improve outcomes. Here, we review the molecular landscape of WT and other pediatric renal tumors as well as WT prognostic factors.

High urinary CXCL10/Cr with onset of Burkitt lymphoma in a pediatric kidney transplant recipient.

Background: Urinary CXCL10/Cr is a promising diagnostic tool for early detection of TCMR in pediatric transplant recipients, and most studies focus on its utility in the context of localized allograft inflammation thus far. Other sources of inflammation that may be detected by CXCL10 are less clear.

Methods: We present a case review of a patient with BL, who was enrolled in a prospective trial of urinary CXCL10 monitoring.

Circulating Tumor DNA as a Biomarker in Patients With Stage III and IV Wilms Tumor: Analysis From a Children's Oncology Group Trial, AREN0533.

Purpose: The utility of circulating tumor DNA (ctDNA) analyses has not been established in the risk stratification of Wilms tumor (WT). We evaluated the detection of ctDNA and selected risk markers in the serum and urine of patients with WT and compared findings with those of matched diagnostic tumor samples.

Patients And Methods: Fifty of 395 children with stage III or IV WT enrolled on Children's Oncology Group trial AREN0533 had banked pretreatment serum, urine, and tumor available.

Complete resolution of alopecia totalis following chemotherapy for B-cell acute lymphoblastic leukemia.

Alopecia totalis (AT) is a cosmetically debilitating chronic disease characterized by non-scarring hair loss of the entire scalp. The clinical course of AT is highly unpredictable, and effective durable treatment options are limited. The resolution of alopecia has been reported post-autologous and allogeneic hematopoietic stem cell transplantation; however, no cases of AT remission following chemotherapy alone have been described.

Clinical response to nivolumab in an INI1-deficient pediatric chordoma correlates with immunogenic recognition of brachyury.

Poorly differentiated chordoma (PDC) is a recently recognized subtype of chordoma characterized by expression of the embryonic transcription factor, brachyury, and loss of INI1. PDC primarily affects children and is associated with a poor prognosis and limited treatment options. Here we describe the molecular and immune tumour microenvironment profiles of two paediatric PDCs produced using whole-genome, transcriptome and whole-genome bisulfite sequencing (WGBS) and multiplex immunohistochemistry.

Impact of the First Generation of Children's Oncology Group Clinical Trials on Clinical Practice for Wilms Tumor.

Refinements in surgery, radiation therapy, and chemotherapy since the mid-20th century have resulted in a survival rate exceeding 90% for patients with Wilms tumor (WT). Although this figure is remarkable, a significant proportion of patients continue to have event-free survival (EFS) estimates of <75%, and nearly 25% of survivors experience severe chronic medical conditions. The first-generation Children's Oncology Group (COG) renal tumor trials (AREN '0'), which opened to enrollment in 2006, focused on augmenting treatment regimens for WT subgroups with predicted EFS <75% to 80%, including those with the adverse prognostic marker of combined loss of heterozygosity (LOH) at chromosomes 1p/16q, pulmonary metastasis with incomplete lung nodule response after 6 weeks of chemotherapy, bilateral disease, and anaplastic histology.

Association between in-line filtration and Type I hypersensitivity reactions in pediatric oncology patients receiving intravenous etoposide.

The objective of this study was to describe hypersensitivity reactions with and without the use of in-line filters during intravenous etoposide therapy in pediatric oncology patients. This was a retrospective review of all patients treated in the Division of Oncology/Hematology/Bone Marrow Transplant at British Columbia Children's Hospital with intravenous etoposide between December 1, 2013 and February 1, 2018. Hypersensitivity reactions and anaphylaxis associated with etoposide infusions were compared over time, including 12 months prior to, 27 months during the use of, and for 12 months after the discontinuation of in-line filtration.

Feeling scared or worried self-report in children receiving cancer treatments using the Symptom Screening in Pediatrics Tool (SSPedi).

Introduction: The objectives of this study were to describe reports of bother for feeling scared or worried among children with cancer and pediatric hematopoietic stem cell transplant (HSCT) recipients, and to identify factors associated with it.

Methods: We included children receiving cancer treatments who were 8-18 years of age. Three patient types were enrolled: inpatients receiving active cancer treatment, outpatients receiving maintenance acute lymphoblastic leukemia chemotherapy, and outpatients in survivorship.

Etoposide-based treatment of adult HLH is associated with high biochemical response but poor survival outcomes.

Etoposide-based treatment is the standard of care for adult HLH in many centers, yet there remains a paucity of data regarding treatment outcomes. We conducted a retrospective study of 23 adults treated with etoposide-based therapy compared to 10 pediatric HLH cases at a single center. At diagnosis, the median serum ferritin was 20,071 µg/L and 937 µg/L in adults and children, respectively; median sIL-2r was 14,524 U/mL and 4,478 U/mL.

Augmentation of Therapy for Combined Loss of Heterozygosity 1p and 16q in Favorable Histology Wilms Tumor: A Children's Oncology Group AREN0532 and AREN0533 Study Report.

Purpose: In National Wilms Tumor Study 5 (NWTS-5), tumor-specific combined loss of heterozygosity of chromosomes 1p and 16q (LOH1p/16q) was associated with adverse outcomes in patients with favorable histology Wilms tumor. The AREN0533/AREN0532 studies assessed whether augmenting therapy improved event-free survival (EFS) for these patients. Patients with stage I/II disease received regimen DD4A (vincristine, dactinomycin and doxorubicin) but no radiation therapy.

The social impact of early psychological maturity in adolescents with cancer.

Objective: There is a growing need to assess the long-term quality of life (QOL) of pediatric oncology patients since many children now survive their disease. This paper highlights the subjective perspectives of pediatric cancer patients and specifically explores how experiencing cancer at a young age impacts adolescents in the areas of social functioning, peer relationships, and QOL. The findings emerged from a qualitative research study that explored how pediatric oncology patients ascribe meaning to their illness.

Severely bothersome fatigue in children and adolescents with cancer and hematopoietic stem cell transplant recipients.

Background: Objectives were to describe bothersome fatigue in children with cancer and hematopoietic stem cell (HSCT) recipients and to identify factors associated with severely bothersome fatigue.

Methods: We included children ages 8-18 years treated for cancer or HSCT recipients from three groups: [1] receiving active cancer treatment and admitted to hospital for at least 3 days, [2] attending outpatient clinic for acute lymphoblastic leukemia maintenance therapy, and [3] attending outpatient clinic following treatment completion. Fatigue was measured using the Symptom Screening in Pediatrics Tool (SSPedi); severely bothersome fatigue was defined as a lot or extremely bothersome fatigue (score of 3-4 on 0-4 scale).

Taste changes in children with cancer and hematopoietic stem cell transplant recipients.

Background: Objectives were to describe bothersome self-reported changes in taste in pediatric oncology and hematopoietic stem cell (HSCT) patients and to identify patient and treatment-related factors associated with bothersome taste changes.

Methods: We prospectively enrolled children and adolescents with cancer or pediatric HSCT recipients 8-18 years of age from three groups: inpatients receiving cancer treatments; outpatients in maintenance therapy for acute lymphoblastic leukemia (ALL); and outpatients in survivorship. Bothersome changes in taste was self-reported using the Symptom Screening in Pediatrics Tool (SSPedi); nausea was self-reported using the Pediatric Nausea Assessment Tool (PeNAT).