Salvage Therapy Efficacy is Modified by Risk Group at Diagnosis in Patients With Relapsed Rhabdomyosarcoma.

Background: Patients with relapsed rhabdomyosarcoma (RMS) are treated with varying approaches and have a poor overall survival (OS). We performed an observational comparison of salvage regimens exploring whether high-dose alkylator combinations were associated with longer OS.

Procedure: We categorized 110 patients with relapsed RMS from five institutions into two groups, those treated with regimens including a high-dose alkylator (Group A) and those treated without a high-dose alkylator (Group B).

Comparing the Diagnostic Yield of Germline Exome Versus Panel Sequencing in the Diverse Population of the Texas KidsCanSeq Pediatric Cancer Study.

Purpose: To evaluate the relative diagnostic yield of clinical germline genomic tests in a diverse pediatric cancer population.

Patients And Methods: The KidsCanSeq study enrolled pediatric cancer patients across six sites in Texas. Germline analysis included both exome sequencing and a therapy-focused pediatric cancer gene panel.

Teaching and Safety-Net Hospital Penalization in the Hospital-Acquired Condition Reduction Program.

Importance: The Hospital-Acquired Condition Reduction Program (HACRP) evaluates acute care hospitals on the occurrence of patient safety events and health care-associated infections. Since its implementation, several studies have raised concerns about the overpenalization of teaching and safety-net hospitals, and although several changes in the program's methodology have been applied in the last few years, whether these changes reversed the overpenalization of teaching and safety-net hospitals is unknown.

Objective: To determine hospital characteristics associated with HACRP penalization and penalization reversal.

Child Health and the Pediatric Hematology-Oncology Workforce: 2020-2040.

Pediatric hematology-oncology (PHO) is 1 of the oldest recognized pediatric subspecialities. PHO physicians care for infants, children, adolescents, and young adults with all types of cancer and nonmalignant blood conditions, in many cases temporarily assuming the role of a primary care physician because of the complexity and intensity of treatment. However, the number of clinically active PHO subspecialists needed to care for children in the United States remains unknown.

Price Increases Versus Upcoding As Drivers Of Emergency Department Spending Increases, 2012-19.

Recent studies document a substantial increase in emergency department (ED) spending in the past decade, even though the number of ED visits per capita has remained relatively stable. Price increases and upcoding are sometimes cited as possible explanations, but their relative impacts are not known. We analyzed Blue Cross Blue Shield claims for patients of all ages who received care in EDs in five states in 2012 and 2019.

Parent-Reported Clinical Utility of Pediatric Genomic Sequencing.

Background And Objectives: Genomic sequencing (GS) is increasingly used for diagnostic evaluation, yet follow-up care is not well understood. We assessed clinicians' recommendations after GS, parent-reported follow-up, and actions parents initiated in response to learning their child's GS results.

Methods: We surveyed parents of children who received GS through the Clinical Sequencing Evidence Generating Research consortium ∼5 to 7 months after return of results.

Lessons learned while starting multi-institutional genetics research in diverse populations: A report from the Clinical Sequencing Evidence-Generating Research (CSER) consortium.

Background: Increasing diversity in clinical trial participation is necessary to improve health outcomes and requires addressing existing social, structural, and geographic barriers. The Clinical Sequencing Evidence-Generating Research Consortium (CSER) included six research projects to enroll historically underrepresented/underserved (UR/US) populations in clinical genomics research. Delays and project re-designs emerged shortly after work began.

Conducting clinical genomics research during the COVID-19 pandemic: Lessons learned from the CSER consortium experience.

Clinical research studies have navigated many changes throughout the COVID-19 pandemic. We sought to describe the pandemic's impact on research operations in the context of a clinical genomics research consortium that aimed to enroll a majority of participants from underrepresented populations. We interviewed (July to November 2020) and surveyed (May to August 2021) representatives of six projects in the Clinical Sequencing Evidence-Generating Research (CSER) consortium, which studies the implementation of genome sequencing in the clinical care of patients from populations that are underrepresented in genomics research or are medically underserved.

Financial Implications of Short Stay Pediatric Hospitalizations.

Background: Observation status (OBS) stays incur similar costs to low-acuity, short-stay inpatient (IP) hospitalizations. Despite this, payment for OBS is likely less and may represent a financial liability for children's hospitals. Thus, we described the financial outcomes associated with OBS stays compared to similar IP stays by hospital and payer.

Is Detection of Relapse by Surveillance Imaging Associated With Longer Survival in Patients With Rhabdomyosarcoma?

Background: We investigated whether surveillance imaging had an impact on post-relapse survival in patients with rhabdomyosarcoma (RMS). We hypothesized that relapse detected by imaging (group IM) would be associated with longer survival compared with relapse detected with a clinical sign or symptom (group SS).

Materials And Methods: We performed an observational multi-institutional study in 127 patients with relapsed RMS comparing overall survival (OS) after relapse using Kaplan-Meier and Cox proportional hazards analyses.

US private payers' perspectives on insurance coverage for genome sequencing versus exome sequencing: A study by the Clinical Sequencing Evidence-Generating Research Consortium (CSER).

Purpose: There is limited payer coverage for genome sequencing (GS) relative to exome sequencing (ES) in the U.S. Our objective was to assess payers' considerations for coverage of GS versus coverage of ES and requirements payers have for coverage of GS.

Rising drug cost impacts on cost-effectiveness of 2 chemotherapy regimens for intermediate-risk rhabdomyosarcoma: A report from the Children's Oncology Group.

Background: The Children's Oncology Group clinical trial for intermediate risk rhabdomyosarcoma randomized participants to a combination of vincristine, dactinomycin, and cyclophosphamide (VAC) alone or VAC alternating with vincristine plus irinotecan (VAC/VI). Clinical outcomes were similar, but toxicity profiles differed. This study estimates the cost differences between arms from the health care system's perspective.

A safety and feasibility trial of I-MIBG in newly diagnosed high-risk neuroblastoma: A Children's Oncology Group study.

Introduction: I-meta-iodobenzylguanidine ( I-MIBG) is effective in relapsed neuroblastoma. The Children's Oncology Group (COG) conducted a pilot study (NCT01175356) to assess tolerability and feasibility of induction chemotherapy followed by I MIBG therapy and myeloablative busulfan/melphalan (Bu/Mel) in patients with newly diagnosed high-risk neuroblastoma.

Methods: Patients with MIBG-avid high-risk neuroblastoma were eligible.

Longitudinal Trends in Costs for Hospitalizations at Children's Hospitals.

Objectives: Children's hospitals are increasingly focused on value-based improvement efforts to improve outcomes and lower costs. Such efforts are generally focused on improving outcomes in specific conditions. Examination of cost drivers across all admissions may facilitate strategic prioritization of efforts.

Exome sequencing compared with standard genetic tests for critically ill infants with suspected genetic conditions.

Purpose: As exome sequencing (ES) is increasingly used as a diagnostic tool, we aimed to compare ES with status quo genetic diagnostic workup for infants with suspected genetic disorders in terms of identifying diagnoses, survival, and cost of care.

Methods: We studied newborns and infants admitted to intensive care with a suspected genetic etiology within the first year of life at a US quaternary-referral children's hospital over 5 years. In this propensity-matched cohort study using electronic medical record data, we compared patients who received ES as part of a diagnostic workup (ES cohort, n = 368) with clinically similar patients who did not receive ES (No-ES cohort, n = 368).

Care of Pediatric High-Cost Hospitalizations Across Hospital Types.

Background: High-cost hospitalizations (HCHs) account for a substantial proportion of pediatric health care expenditures. We aimed to (1) describe the distribution of pediatric HCHs across hospital types caring for children and (2) compare characteristics of pediatric HCHs by hospital type.

Methods: Cross-sectional analysis of all pediatric hospitalizations in the 2012 Kids' Inpatient Database.

Blood product administration during high risk neuroblastoma therapy.

The increasing intensity of high-risk neuroblastoma (HR NB) treatment over the last decades has resulted in improved survival at the expense of prolonging therapy and exposure to additional potentially toxic agents. Anemia and thrombocytopenia requiring transfusion are common during therapy for HR NB. Risks of cumulative red blood cell and platelet transfusions are incompletely defined in pediatric oncology patients, however, risks of transfusional iron overload are well described in other populations.

Factors Influencing Family Burden in Pediatric Hematology/Oncology Encounters.

Purpose: Caring for a child with cancer or hematologic disease places unique stress on a family unit. Families' subjective experience of this care-related burden mediates the relationship between cost and health-related outcomes. While financial costs are well described for families of pediatric hematology/oncology patients, it is unclear how cost and other factors each contribute to families' overall experience of care-related burden.

Using the Delphi method to identify clinicians' perceived importance of pediatric exome sequencing results.

Purpose: Information obtained from clinical exome sequencing (ES) may impact clinical care or other aspects of a patient's life. Little is known about clinicians' perceptions regarding either the value of ES results or which among various outcomes are most relevant to determine value. This study aims to assess clinicians' opinions of the importance of ES results for medical decision making and identify a set of outcomes to be measured in future ES evaluations.

Payer and race/ethnicity influence length and cost of childhood cancer hospitalizations.

Background: Health disparities related to race, ethnicity, socioeconomic status, and insurance status impact quality, access, and health outcomes for children. Medicaid is a proxy for poverty and restricted access to health care. The goal of this study was to determine if there are discrepancies in the length and cost of hospitalizations between admissions covered by Medicaid or commercial insurance for pediatric patients with cancer.

Neuroblastoma of the Bone and Bone Marrow Without an Apparent Primary Site: Report of 4 Cases With Long-term Follow-up.

Children with neuroblastoma rarely present with metastatic disease without identifiable primary tumors. We describe the clinical and histopathologic characteristics of 4 patients aged 1, 7, 7, and 11 years with neuroblastoma involving bone or bone marrow without an apparent primary site. One patient presented with a periorbital bone lesion, 1 presented with a distal femoral lesion, and 2 presented with diffuse bone marrow involvement.