Statistical Fragility of Findings From Randomized Phase 3 Trials in Pediatric Oncology.

Purpose: The fragility index (FI) is an adjunctive metric to facilitate the interpretation of p-values in clinical trials. The FI has not been studied in phase 3 trials in pediatric oncology.

Methods: PubMed was used to identify phase 3 pediatric oncology trials published between 1980 and 2020.

Pediatric transplant-associated thrombotic microangiopathy health care utilization and implications of eculizumab therapy.

The health care use (HCU) burden of transplant-associated thrombotic microangiopathy (TA-TMA) and its treatments are unknown. The objective of this study was to investigate inpatient costs associated with meeting criteria for TA-TMA in the first year after hematopoietic cell transplant (HCT). This institutional review board-approved retrospective multicenter study included serial children who underwent HCT from 1 January 2015 to 1 July 2019.

Risk factors and outcomes of melanoma in children and adolescents: A retrospective multicenter study.

Background: Pediatric melanoma presents with distinct clinical features compared to adult disease.

Objective: Characterize risk factors and negative outcomes in pediatric melanoma.

Methods: Multicenter retrospective study of patients under 20 years diagnosed with melanoma between January 1, 1995 and June 30, 2015 from 11 academic medical centers.

Phase 2 trial of palbociclib and ganitumab in patients with relapsed Ewing sarcoma.

Background: Ewing sarcoma (EWS) is an aggressive sarcoma with few treatment options for patients with relapsed disease. Cyclin-dependent kinase 4 (CDK4) is a genomic vulnerability in EWS that is synergistic with IGF-1R inhibition in preclinical studies. We present the results of a phase 2 study combining palbociclib (CDK4/6 inhibitor) with ganitumab (IGF-1R monoclonal antibody) for patients with relapsed EWS.

Tolerability of ifosfamide-containing regimen in patients with high-risk renal and INI-1-deficient tumors.

Background: Outcomes for children with high-risk renal (HRR) and INI-1-deficient (INI-) tumors are unacceptably poor. Concerns about excessive toxicity-as many are infants and/or undergo nephrectomy-have resulted in decreased chemotherapy dosing and omission of the nephrotoxic drug ifosfamide in collaborative group studies. As cause of death for children with these cancers remains overwhelmingly more from progressive disease rather than treatment toxicity, we examined the tolerability of an intensive ifosfamide-containing regimen.

Survival of Patients With Neuroblastoma After Assignment to Reduced Therapy Because of the 12- to 18-Month Change in Age Cutoff in Children's Oncology Group Risk Stratification.

Purpose: In 2006, Children's Oncology Group (COG) reclassified subgroups of toddlers diagnosed with neuroblastoma from high-risk to intermediate-risk, when the age cutoff for high-risk assignment was raised from 365 days (12 months) to 547 days (18 months). The primary aim of this retrospective study was to determine if excellent outcome was maintained after assigned reduction of therapy.

Patients And Methods: Children <3 years old at diagnosis, enrolled on a COG biology study from 1990 to 2018, were eligible (n = 9,189).

Clinical and biological features prognostic of survival after relapse or progression of INRGSS stage MS pattern neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project.

Background: Outcomes for patients with INRGSS metastatic special (MS) metastatic pattern neuroblastoma at initial diagnosis are well described. Prognosis after an initial event (relapse, progression, secondary malignancy) is unclear.

Methods: We investigated characteristics of MS pattern neuroblastoma patients in the International Neuroblastoma Risk Group database who subsequently experienced an event.

Hypothyroidism Is Correlated with Ventilator Complications and Longer Hospital Days after Coronary Artery Bypass Grafting Surgery in a Relatively Young Population: A Nationwide, Population-Based Study.

Background: Some research indicated that hypothyroidism has huge adverse effects for the metabolic, cardiovascular, respiratory, and immune systems. However, there is no confirmed conclusion for the effect of cardiovascular surgery. This cohort study aims to investigate the prognosis of hypothyroidism patient at the age under 65-year-old after coronary artery bypass grafting (CABG) surgery.

Molecular profiling identifies targeted therapy opportunities in pediatric solid cancer.

To evaluate the clinical impact of molecular tumor profiling (MTP) with targeted sequencing panel tests, pediatric patients with extracranial solid tumors were enrolled in a prospective observational cohort study at 12 institutions. In the 345-patient analytical population, median age at diagnosis was 12 years (range 0-27.5); 298 patients (86%) had 1 or more alterations with potential for impact on care.

Health Literacy and Clinical Outcomes Following Hematopoietic Stem-Cell Transplantation.

Purpose: To test associations between health literacy and clinical outcomes in patients undergoing hematopoietic stem-cell transplantation (HSCT).

Methods: English- and Spanish-speaking patients age ≥ 18 years were recruited while admitted for first allogeneic HSCT. Associations between low health literacy (Newest Vital Sign ≤ 3 or Short Test of Functional Health Literacy in Adults ≤ 22) and HSCT outcomes were evaluated.

Prevalence and Predictors of Iron Deficiency in Adolescent and Young Adult Outpatients: Implications for Screening.

Current screening guidelines may not be adequate to identify iron deficiency (ID) and iron deficiency anemia (IDA) in adolescent and young adults. Adolescent and young adult outpatients from 4 hospital-based clinics (N = 493) reported on diet, health, and bleeding, and had phlebotomy for iron and hematologic tests. We examined sex-specific factors associated with ID and IDA and ability of universal and risk factor-based screening using hemoglobin and hemoglobin plus ferritin to detect ID and IDA.

Symptoms and Suffering at End of Life for Children With Complex Chronic Conditions.

Context: Children with cancer and cardiac disease suffer with high symptom burden at end of life (EOL). Little is known about the EOL experience for children with other complex chronic conditions (CCCs).

Objectives: To evaluate symptoms and suffering at EOL for children with noncancer, noncardiac CCCs as well as parental distress related to child suffering.

A POETIC Phase II study of continuous oral everolimus in recurrent, radiographically progressive pediatric low-grade glioma.

Background: To evaluate efficacy, pharmacokinetics (PK) and pharmacodynamics of single-agent everolimus in pediatric patients with radiographically progressive low-grade glioma (LGG).

Methods: Everolimus was administered at 5 mg/m once daily as a tablet or liquid for a planned 48-week duration or until unacceptable toxicity or disease progression. Patients with neurofibromatosis type 1 were excluded.

Race Disparities in Proton Radiotherapy Use for Cancer Treatment in Patients Enrolled in Children's Oncology Group Trials.

This quality improvement study examines disparities in proton radiotherapy use in patients enrolled in Children’s Oncology Group Trials

Risk factors for transplant-associated thrombotic microangiopathy and mortality in a pediatric cohort.

Transplant-associated thrombotic microangiopathy (TA-TMA) is a well-recognized complication of hematopoietic cell transplantation (HCT). Diagnosis is challenging and in the absence of a tissue biopsy, TA-TMA is provisionally diagnosed by meeting clinical criteria. In this study, we describe the prevalence, outcomes, and risk factors for meeting 2 different diagnostic criteria for TA-TMA and for increased transplant-related mortality (TRM).

RBMY, a novel inhibitor of glycogen synthase kinase 3β, increases tumor stemness and predicts poor prognosis of hepatocellular carcinoma.

Unlabelled: Male predominance of hepatocellular carcinoma (HCC) occurs particularly among young children aged 6-9 years, indicative of a possible role of the Y chromosome-encoded oncogene in addition to an androgenic effect. The discovery of oncogenic activation of RBMY (RNA-binding motif on Y chromosome), which is absent in normal hepatocytes but present in male HCC tissues, sheds light on this issue. Herein, we report on a critical hepatocarcinogenic role of RBMY and its ontogenic origin.

A high serum interleukin-12p40 level prior to Kasai surgery predict a favourable outcome in children with biliary atresia.

Background: Biliary atresia (BA) is a paediatric cholestatic disease characterized by a progressive fibro-inflammation of the biliary tree. Current treatment of choice is to establish good bile flow via the Kasai operation.

Aims: We aimed to identify outcome-predictive serum biomarkers in BA infant.

Tumour necrosis factor-α promoter region polymorphisms affect the course of spontaneous HBsAg clearance.

Background: This study aimed to investigate the roles of tumour necrosis factor-α (TNF-α) gene polymorphisms in the spontaneous clearance of HBsAg after a hepatitis B virus (HBV) infection.

Methods: Polymorphisms in the TNF-α (-1031 T to C, -863 C to A, -857 C to T, -308 G to A and -238 G to A transition) gene were evaluated in 274 chronic HBV-infected patients and 194 patients with resolved HBV infection. The peripheral blood mononuclear cells (PBMC) isolated from 77 (28%) of the 274 chronic HBV-infected patients with negative HBeAg and positive antibody to HBeAg were stimulated with HBcAg.

Involvement of Ca2+ signaling in tachykinin-mediated contractile responses in swine trachea.

Neuropeptide tachykinins, present within sensory nerves, have been implicated as neurotransmitters involved in nonadrenergic and noncholinergic airway muscle contraction. The signal transduction pathways of tachykinins on muscle contraction and Ca2+ mobilization were investigated in swine trachea. Tachykinins, substance P (SP) and neurokinin A (NKA), concentration (1 nM to 1 microM)-dependently induced contractile responses with removal of epithelium, whereas neurokinin B (NKB) did not alter the muscle tension.