Pediatric Oncology Care Research

Pediatric Oncology Care Research | CPCE

Oncology care research at CPCE focuses on improving the outcomes of pediatric cancer patients by investigating and identifying ways to improve treatment. Through research into variation of care across institutions, genetic predictors of treatment responses, and risk factors for treatment complications, this work can provide invaluable education to pediatric oncologists.

Hospital Factors and Impact of Care in Pediatric and Young Adult Oncology Patients

Treatment of Acute Myeloid Leukemia

Oncology Care Published Research


Hospital Factors and Impact of Care in Pediatric and Young Adult Oncology Patients

Care in pediatric oncology is often multi-faceted and complex. Volume and outcome relationships, which analyze the correlation between the amount of patients seen or procedures performed by a clinician/hospital and outcomes for their patients, have been well-studied in other fields but not as extensively in pediatric oncology. Understanding whether variations in morbidity and mortality are influenced by provider and hospital volume are important to aid in improving care for all children with cancer.

To improve understanding as to what hospital and provider factors influence the care received at the individual patient level, we are analyzing retrospective cohort studies in large administrative databases, including Pediatric Health Information System (PHIS) and Premier Perspective Database. To date, an inverse relationship between pediatric oncology hospital volume and mortality in the first phase of therapy for pediatric acute lymphoblastic leukemia does not appear to be present.

What’s Next: Further work will be necessary to determine why institutions may differ including identification of best practices and potential recommendations for standardization of pediatric cancer care.

Funding:  Agency for Healthcare Research and Quality, National Research Service Awards, Alex’s Lemonade Stand

Please contact Jennifer Wilkes, MD, Instructor in Pediatric Oncology at CHOP, for more information about this line of research.


Treatment of Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that affects immature blood cell growth. The second most common form of leukemia in children, AML is found throughout the bloodstream or in an organ, and must be treated aggressively as soon as possible after diagnosis.

Led by Richard Aplenc, MD, PhD, MSCE, CPCE’s research efforts in this area aim to improve the outcomes of children with cancer, particularly AML, by determining the genetic predictors of treatment response (specifically relapse and infection risk) and studying the clinical epidemiology of risk factors for treatment complications and outcome disparities using administrative data sets, particularly focusing on disparities in outcomes and antibiotic and intensive care resource utilization.

Several studies have addressed clinically significant research questions by merging data from the Children’s Oncology Group (COG) clinical trials with an external administrative/billing data set (Pediatric Health Information System—PHIS). COG is the largest international pediatric cooperative oncology group, and enrolls approximately 4,400 patients annually in therapeutic trials.

In this line of research, we have utilized COG/PHIS data to determine that patients with newly diagnosed pediatric leukemia who are admitted to the hospital on weekends have a prolonged length of stay, increased time to chemotherapy and higher risk for respiratory failure than those admitted during the week.

Additionally, CPCE research found that the use of chemotherapy regimen ADE, as opposed to an alternative called DCTER, is associated with lower induction mortality in pediatric AML.

These COG/PHIS merged data have also revealed that acute renal failure (ARF) is relatively common in children with AML and the risk for ARF is greater in older patients, in African-American patients, and in patients with increased exposure to the antibiotics vancomycin and carbapenem.

Further, research efforts in this area of pediatric oncology education have led to the understanding that adverse events are underreported in clinical trials.

See the Published Research

Funding:  National Institute of Health, Alex’s Lemonade Stand

Please contact Richard Aplenc, MD, PhD, MSCE, CHOP Attending Physician, for more information about this line of research.


Pediatric Oncology Care Published Research

Treatment of Acute Myeloid Leukemia

Aplenc R., Fisher BT, Huang Y S, Li Y, Alonzo TA, Gerbing RB, Hall M, Bertoch D, Keren R, Seif AE, Sung L, Adamson PC, Gamis A. Merging of the National Cancer Institute-funded cooperative oncology group data with an administrative data source to develop a more effective platform for clinical trial analysis and comparative effectiveness research: a report from the Children's Oncology Group. Pharmacoepidemiol Drug Saf. 2012 May;21(2):37-43.

Fisher BT, Kavcic M, Li Y, Seif AE, Bagatell R, Huang YS, Zaoutis T, Torp K, Leckerman KH, Aplenc R. Antifungal prophylaxis associated with decreased induction mortality rates and resources utilized in children with new-onset acute myeloid leukemia. Clin Infect Dis. 2014 Feb;58(4):502-8.

Fisher BT, Zaoutis TE, Leckerman KH, Localio R, Aplenc R. Risk factors for renal failure in pediatric patients with acute myeloid leukemia: a retrospective cohort study. Pediatr Blood Cancer. 2010 Oct;55(4):655-61.

Goodman EK, Reilly AF, Fisher BT, Fitzgerald J, Li Y, Seif AE, Huang YS, Bagatell R, Aplenc R. Association of weekend admission with hospital length of stay, time to chemotherapy, and risk for respiratory failure in pediatric patients with newly diagnosed leukemia at freestanding U.S. children's hospitals. JAMA Pediatr. 2014 Oct;168(10):925-31.

Kavcic M, Fisher BT, Li Y, Seif AE, Torp K, Walker DM, Huang YS, Lee GH, Tasian SK, Vujkovic M, Bagatell R, Aplenc R. Induction mortality and resource utilization in children treated for acute myeloid leukemia at free-standing pediatric hospitals in the United States. Cancer. 2013 May;119(10):1916-23. 

Kavcic, M, Fisher BT, Seif AE, Li Y, Huang YS, Walker D, Aplenc R. Leveraging administrative data to monitor rituximab use in 2875 patients at 42 freestanding children's hospitals across the United States. J Pediatr. 2013 Jun;162(6):1252-8.

Maude SL, Fitzgerald JC, Fisher BT, Li Y., Huang YS, Torp K, Seif AE, Kavcic M, Walker DM, Leckerman KH, Kilbaugh TJ, Rheingold SR, Sung L, Zaoutis TE, Berg RA, Nadkarni VM, Thomas NJ, Aplenc R. Outcome of pediatric acute myeloid leukemia patients receiving intensive care in the United States. Pediatr Crit Care Med. 2014 Feb;15(2):112-20.

Miller TP, Troxel AB, Li Y, Huang YS, Alonzo TA, Gerbing RB, Hall M, Torp K, Fisher BT, Bagatell R, Seif AE, Sung L, Gamis A, Rubin D, Luger S, Aplenc R. Comparison of administrative/billing data to expected protocol-mandated chemotherapy exposure in children with acute myeloid leukemia: A report from the Children’s Oncology Group. Pediatr Blood Cancer. 2015 Jul;62(7):1184-9.

Seif AE, Walker DM, Li Y, Huang YS, Kavcic M, Torp K, Bagatell R, Fisher BT, Aplenc R. Dexrazoxane exposure and risk of secondary acute myeloid leukemia in pediatric oncology patients. Pediatr Blood Cancer. 2015 Apr;62(4):704-9.

Walker DM, Fisher BT, Seif AE, Huang YS, Torp K, Li Y, Aplenc R. Dexrazoxane use in pediatric patients with acute lymphoblastic or myeloid leukemia from 1999 and 2009: analysis of a national cohort of patients in the Pediatric Health Information Systems database. Pediatr Blood Cancer. 2013 Apr;60(4):616-20.