The Children’s Hospital of Philadelphia can be a second home for families who have a child diagnosed with acute myeloid leukemia (AML) which usually requires several intensive rounds of chemotherapy during three to four-week inpatient hospital stays. Parents set up air mattresses to sleep in the hospital room. School tutors ensure schoolwork doesn’t fall behind. Although it can be a supportive, comforting environment during this distressing time, families often dearly miss their daily routines.
Part of the reason patients with AML stay in the hospital for many weeks is because chemotherapy depletes their neutrophils, which are the blood cell that are the first responders to fight organisms that cause infection. During the periods following treatment when neutrophils are low, called neutropenia, pediatric cancer patients can have a difficult time fighting off bloodstream infections and related complications.
Because of this risk, some physicians prefer that children with AML stay in the hospital for close monitoring following a course of treatment. Other doctors allow patients to go home and return to the hospital if a fever occurs.
However, physician-researchers do not have enough evidence to know with certainty if patients fare better or worse if they are sent home during periods of neutropenia. A collaborative, observational clinical trial being led by a study team at CHOP aims to shed light on how physicians could approach these patient discharge scenarios safely and consistently while also considering family preferences.
“There is a lot of complexity and interplay between medical outcomes and patient experiences,” said Richard Aplenc, MD, PhD, a CHOP pediatric oncologist and AML researcher who is the principal investigator. “We need to know about both sides of the street, in order to have the best and most informed discussions with families about what to do in this situation.”
The study, entitled “Home or Away From Home,” will compare 490 patients’ medical records to determine the risks/benefits of outpatient vs. inpatient management of neutropenia in children with AML. Fifteen U.S. pediatric hospitals from across the U.S. will participate. In addition to collecting data so that the study team can compare each discharge strategy’s medical outcomes including bloodstream infection and delays in subsequent courses of chemotherapy, several sites also will assess patient outcomes by surveying patients and families about their quality of life.
Julia Szymczak, PhD, a medical sociologist, postdoctoral fellow in the division of Infectious Diseases at CHOP, and staff member of the Center for Pediatric Clinical Effectiveness, will add a third dimension to the project by using anthropologic research methods. Over a six-month period, she will conduct semi-structured in-person interviews with focus groups to gather their personal perspectives.
For instance, will some families be willing to take on the risk of complications arising at home that could require a return to the hospital and staying in the intensive care unit? Or if a child is discharged home, could it place additional financial stress on working families who must balance caring for a sick child? Three patient advisory councils from Alex’s Lemonade Stand, the Children’s Oncology Group, and CHOP will help the study team connect with families and eventually disseminate the study’s findings.
“Whether most of their child’s chemotherapy experience is at home or most of it is in the hospital is a big deal for families, so we need to hear from patients and families about what is most important to them,” Dr. Aplenc said.
The study’s innovative combination of three different kinds of research — clinical epidemiology, quality of life, and medical anthropology — helped it gain the attention of the Patient-Centered Outcomes Research Institute (PCORI), which awarded the three-year project $1.8 million. PCORI is an independent, nonprofit organization authorized by Congress in 2010 to fund research to provide patients, caregivers, and clinicians with the evidence-based information they need to make better-informed healthcare decisions.
Read more about Dr. Aplenc’s pediatric AML research and a recent $1 million Hyundai Quantum Grant from Hyundai Hope on Wheels he received to advance new cancer treatments. Dr. Aplenc also is an associate professor of Pediatrics at the Perelman School of Medicine, University of Pennsylvania.