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Introduction: Previous literature suggests that allogeneic hematopoietic stem cell transplant (HCT) utilization rates are lower amongst Hispanic and Black compared to non-Hispanic White (NHW) cancer patients. However, no previous studies have focused explicitly on the pediatric and adolescent young adult (AYA) population. We sought to examine utilization of HCT by race/ethnicity using the California Cancer Registry (CCR) and the Office of Statewide Health Planning and Development (OSHPD) hospitalization database. We hypothesized that Black and Hispanic patients with acute leukemia less frequently receive HCT than NHW patients. Methods: Using population-based data from California, a retrospective cohort of patients aged 0-39 years with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) diagnosed between 2000 and 2015 was assembled. The primary exposure was a composite of race/ethnicity with NHW patients as the reference group. The primary outcome was receipt of first HCT, defined by diagnosis codes in OSHPD or treatment in the CCR. Logistic regression analyses were used to estimate odds ratios (OR) and corresponding 95% confidence intervals (CI). Multivariable models were adjusted for race/ethnicity, age, sex, year of diagnosis, leukemia type, insurance type at diagnosis, rurality, and neighborhood socioeconomic status at diagnosis (SES). Results: Among 7,183 patients (4,790 with ALL and 2,393 with AML), 21% (16% of ALL patients and 31% of AML patients) underwent HCT. Distributions of insurance type and neighborhood SES differed by race/ethnicity with a higher proportion of Black and Hispanic patients having Medicaid insurance and living in lower SES neighborhoods. In univariate analyses, Black and Hispanic race/ethnicity were associated with decreased likelihood of receiving HCT (OR 0.70 95% CI 0.53, 0.94 and OR 0.77 95% CI 0.68, 0.88, respectively) and Asian race was associated with increased likelihood of HCT (OR 1.29 95% CI 1.07, 1.56) compared to NHW patients. In the multivariable model, there was no statistically significant difference in receipt of HCT among Hispanic patients, but disparities persisted among Black patients (Table 1). Uninsured patients and those in the lowest SES quintile were also less likely to receive HCT, while older age and AML were associated with HCT receipt. In analyses stratified by age and leukemia type, we found that the disparities in receipt of HCT among Black patients was largely driven by patients >20 years old (adjusted OR 0.50 95% CI 0.33, 0.78). These analyses also revealed that the increased likelihood of HCT among Asian/PI patients was driven by patients 2 readmissions in the first year post-HCT v. 25%, p=0.005) compared to NHW patients. Conclusions: HCT is a potentially curative treatment for high-risk acute leukemia; thus the observed racial and SES disparities in receipt of HCT may contribute to disparities in leukemia survival. More detailed disease, treatment, and relapse data would provide a better understanding of the etiology of our findings and allow for reduction of existing disparities through improved access to HCT. Disclosures: Muffly: Servier: Research Funding; Amgen: Consultancy; Adaptive: Research Funding. Wun:Glycomimetics, Inc.: Consultancy.

Equal access to clinical trial enrollment is important to ensure that findings are generalizable to the broader population. This study aimed to evaluate disparities in enrollment on pediatric oncology clinical trials. We assessed the relationship between patient characteristics and enrollment on COG trial AAML1031 in a cohort of pediatric patients with AML in the Pediatric Health Information System. The associations of enrollment with outcomes were evaluated. Non-Hispanic Black patients, infants, and patients from zip codes with a lower proportion of poverty were less likely to enroll (30% vs. 61%, p = .004; 34% vs. 58%, p = .003; 46% vs. 58%, p = .02). On-therapy mortality was similar among enrolled and nonenrolled patients (7.3% vs. 8.9%, p = .47). Differences in early mortality were more pronounced among nonenrolled patients compared to enrolled patients (3.0% vs. 0.5%, p = .03). Understanding the etiology of these disparities will inform strategies to ensure balanced access to clinical trials across patient populations.

BackgroundBlack patients with acute myeloid leukemia (AML) are more likely to present with high acuity and consequently experience higher rates of induction mortality than white patients. Given the consistently identified racial disparities in overall survival (OS) among patients with AML, we aimed to evaluate whether there were sustained on-therapy racial differences in inpatient mortality, intensive care unit (ICU) requirements, or supportive care beyond initial induction.ProcedureWithin a retrospective cohort of 1239 children diagnosed with AML between 2004 and 2014 in the Pediatric Health Information System (PHIS) database who survived their initial course of induction chemotherapy, we compared on-therapy inpatient mortality, ICU-level care requirements, treatment course duration, cumulative length of hospital stay (LOS), and resource utilization after induction I by race.ResultsOver the period from the start of induction II through completion of frontline chemotherapy, there were no significant differences in mortality (adjusted odds ratios [OR], 1.01; 95% confidence intervals [CI], 0.41-2.48), ICU-level care requirements (adjusted OR, 0.93; 95% CI, 0.69-1.26), LOS (adjusted mean difference, 3.2 days; 95% CI, -2.3-9.6), or supportive care resource utilization for black patients relative to white patients. Course-specific analyses also demonstrated no differences by race.ConclusionAlthough black patients have higher acuity at presentation and higher induction mortality, such disparities do not persist over subsequent frontline chemotherapy treatment. This finding allows interventions aimed at reducing disparities to be directed at presentation and induction.

PURPOSE: Although the potential transformative effect of electronic health record (EHR) data on clinical research in adult patient populations has been very extensively discussed, the effect on pediatric oncology research has been limited. Multiple factors contribute to this more limited effect, including the paucity of pediatric cancer cases in commercial EHR-derived cancer data sets and phenotypic case identification challenges in pediatric federated EHR data. METHODS: The ExtractEHR software package was initially developed as a tool to improve clinical trial adverse event reporting but has expanded its use cases to include the development of multisite EHR data sets and the support of cancer cohorts. ExtractEHR enables customized, automated data extraction from the EHR that, when implemented across multiple hospitals, can create pediatric cancer EHR data sets to address a very wide range of research questions in pediatric oncology. After ExtractEHR data acquisition, EHR data can be cleaned and graded using CleanEHR and GradeEHR, companion software packages. RESULTS: ExtractEHR has been installed at four leading pediatric institutions: Children's Healthcare of Atlanta, Children's Hospital of Philadelphia, Texas Children's Hospital, and Seattle Children's Hospital. CONCLUSION: ExtractEHR has supported multiple use cases, including five clinical epidemiology studies, multicenter clinical trials, and cancer cohort assembly. Work is ongoing to develop Fast Health care Interoperability Resources ExtractEHR and implement other sustainability and scalability enhancements. [ABSTRACT FROM AUTHOR]

We develop a Bayesian semiparametric model for the impact of dynamic treatment rules on survival among patients diagnosed with pediatric acute myeloid leukemia (AML). The data consist of a subset of patients enrolled in a phase III clinical trial in which patients move through a sequence of four treatment courses. At each course, they undergo treatment that may or may not include anthracyclines (ACT). While ACT is known to be effective at treating AML, it is also cardiotoxic and can lead to early death for some patients. Our task is to estimate the potential survival probability under hypothetical dynamic ACT treatment strategies, but there are several impediments. First, since ACT is not randomized, its effect on survival is confounded over time. Second, subjects initiate the next course depending on when they recover from the previous course, making timing potentially informative of subsequent treatment and survival. Third, patients may die or drop out before ever completing the full treatment sequence. We develop a generative Bayesian semiparametric model based on Gamma Process priors to address these complexities. At each treatment course, the model captures subjects' transition to subsequent treatment or death in continuous time. G-computation is used to compute a posterior over potential survival probability that is adjusted for time-varying confounding. Using our approach, we estimate the efficacy of hypothetical treatment rules that dynamically modify ACT based on evolving cardiac function. [ABSTRACT FROM AUTHOR]

Background Targeted cancer drugs (TCDs) have revolutionized oncology but vary in clinical benefit and patient out-of-pocket (OOP) costs. The American Society of Clinical Oncology (ASCO) Value Framework uses survival, toxicity, and symptom palliation data to quantify the net health benefit (NHB) of cancer drugs. We evaluated associations between NHB, uptake, and spending on oral TCDs. Methods We conducted a retrospective cohort study of patients aged 18-64 years with an incident oral TCD pharmacy claim in 2012-2020 in a nationwide deidentified commercial claims dataset. TCDs were categorized as having high (>60), medium (40-60), and low (<40) NHB scores. We plotted the uptake of TCDs by NHB category and used standard descriptive statistics to evaluate patient OOP and total spending. Generalized linear models evaluated the relationship between spending and TCD NHB, adjusted for cancer indication. Results We included 8524 patients with incident claims for 8 oral TCDs with 9 first-line indications in advanced melanoma, breast, lung, and pancreatic cancer. Medium- and high-NHB TCDs accounted for most TCD prescriptions. Median OOP spending was $18.78 for the first 28-day TCD supply (interquartile range [IQR] = $0.00-$87.57); 45% of patients paid $0 OOP. Median total spending was $10 118.79 (IQR = $6365.95-$10 600.37) for an incident 28-day TCD supply. Total spending increased $1083.56 for each 10-point increase in NHB score (95% confidence interval = $1050.27 to $1116.84, P  <  .01 for null hypothesis H0 = $0). Conclusion Low-NHB TCDs were prescribed less frequently than medium- and high-NHB TCDs. Total spending on oral TCDs was high and positively associated with NHB. Commercially insured patients were largely shielded from high OOP spending on oral TCDs. [ABSTRACT FROM AUTHOR]

BackgroundTetrachloroethene (PCE) is a common environmental and occupational contaminant and an acknowledged neurotoxicant. From 1968 through 1983, widespread contamination of public drinking water supplies with PCE occurred in the Cape Cod region of Massachusetts. The source of the contamination was a vinyl liner applied to the inner surface of water distribution pipes.ObjectivesA retrospective cohort study (the Cape Cod Health Study) was undertaken to examine possible health consequences of early-life exposure to PCE-contaminated drinking water. This review describes the study methods and findings regarding the effects of prenatal and childhood exposure on neurologic outcomes during early adulthood, including vision, neuropsychological functioning, brain structure, risky behaviors, and mental illness. The review also describes the strengths and challenges of conducting population-based epidemiologic research in this unique setting.MethodsParticipants were identified by cross-matching birth certificates and water system data. Information on health outcomes and confounding variables was collected from self-administered surveys (n = 1689), neuropsychological tests (n = 63), vision examinations (n = 63), and magnetic resonance imaging (n = 42). Early-life exposure to PCE was estimated using a leaching and transport model. The data analysis compared the occurrence of each health outcome among individuals with prenatal and early childhood PCE exposure to unexposed individuals while considering the effect of confounding variables.FindingsThe study found evidence that early-life exposure to PCE-contaminated drinking water has long-term neurotoxic effects. The strongest associations were seen with illicit drug use, bipolar disorder, and post-traumatic stress disorder. Key strengths of the study were availability of historical data on affected water systems, a relatively high exposure prevalence and wide range of exposure levels, and little confounding. Challenges arose mainly from the historical nature of the exposure assessments.ConclusionsThe Cape Cod Health Study demonstrates how scientists can take advantage of unique "natural experiments" to learn about the health effects of environmental pollution. This body of work has improved our understanding of the long-term health effects of early-life exposure to this common environmental contaminant and will help risk assessors and policymakers ensure that drinking water supplies in the United States are safe for vulnerable populations.

Background: Pediatric acute myeloid leukemia (AML) chemotherapy increases the risk of life‐threatening complications, including septic shock (SS). An area‐based measure of social determinants of health, the social disorganization index (SDI), was hypothesized to be associated with SS and SS‐associated death (SS‐death). Methods: Children treated for de novo AML on two Children's Oncology Group trials at institutions contributing to the Pediatric Health Information System (PHIS) database were included. The SDI was calculated via residential zip code data from the US Census Bureau. SS was identified via PHIS resource utilization codes. SS‐death was defined as death within 2 weeks of an antecedent SS event. Patients were followed from 7 days after the start of chemotherapy until the first of end of front‐line therapy, death, relapse, or removal from study. Multivariable‐adjusted Cox regressions estimated hazard ratios (HRs) comparing time to first SS by SDI group. Results: The assembled cohort included 700 patients, with 207 (29.6%) sustaining at least one SS event. There were 233 (33%) in the SDI‐5 group (highest disorganization). Adjusted time to incident SS did not statistically significantly differ by SDI (reference, SDI‐1; SDI‐2: HR, 0.84 [95% confidence interval (CI), 0.51–1.41]; SDI‐3: HR, 0.70 [95% CI, 0.42–1.16]; SDI‐4: HR, 0.97 [95% CI, 0.61–1.53]; SDI‐5: HR, 0.72 [95% CI, 0.45–1.14]). Nine patients (4.4%) with SS experienced SS‐death; seven of these patients (78%) were in SDI‐4 or SDI‐5. Conclusions: In a large, nationally representative cohort of trial‐enrolled pediatric patients with AML, there was no significant association between the SDI and time to SS. Time to first septic shock event was not associated with the social disorganization index (SDI), an area‐based measure of social determination of health, in children with acute myeloid leukemia treated on two Children's Oncology Group trials. Septic shock–associated mortality is rare in children with acute myeloid leukemia, and differences by SDI were not able to be detected. [ABSTRACT FROM AUTHOR]

Background: Despite the widespread implementation of telemedicine, there are limited data regarding its impact on key components of care for patients with incurable or high‐risk cancer. For these patients, high‐quality care requires detailed conversations regarding treatment priorities (advance care planning) and clinical care to minimize unnecessary acute care (unplanned hospitalizations). Whether telemedicine affects these outcomes relative to in‐person clinic visits was examined among patients with cancer at high risk for 6‐month mortality. Methods: This retrospective cohort study included adult patients with cancer with any tumor type treated at the University of Pennsylvania who were newly identified between April 1 and December 31, 2020, to be at high risk for 6‐month mortality via a validated machine learning algorithm. Separate modified Poisson regressions were used to assess the occurrence of advance care planning and unplanned hospitalizations for telemedicine as compared to in‐person visits. Additional analyses were done comparing telemedicine type (video or phone) as compared to in‐person clinic visits. Results: The occurrence of advance care planning was similar between telemedicine and in‐person visits (6.8% vs. 6.0%; adjusted risk ratio [aRR], 1.25; 95% CI, 0.92–1.69). In regard to telemedicine subtype, patients exposed to video encounters were modestly more likely to have documented advance care planning in comparison to those seen in person (7.5% vs. 6.0%; aRR, 1.48; 95% CI, 1.03–2.11). The 3‐month risk for unplanned hospitalization was comparable for telemedicine compared to in‐person clinic encounters (21% vs. 18%; aRR, 1.06; 95% CI, 0.81–1.38). Conclusions: In this study, care delivered by telemedicine, compared to in‐person clinic visits, produced comparable rates of advance care planning conversations without increasing hospitalizations, which suggests that vulnerable patients can be managed safely by telemedicine. In this study, care delivered by telemedicine, compared to in‐person clinic visits, produced comparable rates of advance care planning conversations without increasing hospitalizations, which suggests that vulnerable patients can be managed safely by telemedicine. [ABSTRACT FROM AUTHOR]