Results Summary
What was the research about?
Acute myeloid leukemia, or AML, is a type of blood cancer. Although 20 percent of children with leukemia have AML, AML causes more than half of all deaths from leukemia in children.
Doctors use multiple courses of chemotherapy to treat AML. After each course, children with AML are at high risk for life-threatening infections. These infections can also delay the next course of chemotherapy.
After chemotherapy, some children with AML recover at the hospital and others recover at home, depending on the hospital. No one knows how recovery in the hospital or at home affects the risk of infections. Some children and their parents may prefer recovery at home if it is safe.
In this study, the research team compared health records of children with AML who went home with those who stayed in the hospital to recover from chemotherapy. The team wanted to learn whether recovery at home could be a suitable option.
What were the results?
Recovering at home didn’t negatively affect children with AML. Children who went home to recover from chemotherapy didn’t have more bloodstream infections, delays in starting their next course of chemotherapy, or worse quality of life than those who stayed in the hospital.
What did the research team do?
The research team looked at health records from 554 children with AML who were 19 years old and younger and who received chemotherapy at 1 of 17 health centers. The team classified children as recovering at home or at the hospital. Children discharged from the hospital within three days after ending a course of chemotherapy were recovering at home. Children who stayed for more than three days were recovering in the hospital.
To assess delays in treatment, the research team looked at health record data for up to 50 days after the start of each chemotherapy course. The team looked at the results of blood tests to identify the presence of infections.
A subset of 97 parents whose children were in the study completed surveys about their child’s health-related quality of life. Parents took the surveys at the start of a chemotherapy course and again after their child recovered.
Patients and family members with experience with AML helped design the study and gave input during the study.
What were the limits of the study?
The research team didn’t have data on how severe the children’s bloodstream infections were, which is important to know when comparing recovery options.
Future research could continue to look at when recovery at home would be suitable. Also, studies could compare the severity of bloodstream infections among children with AML who recover in the hospital versus at home.
How can people use the results?
Parents of a child with AML and their doctors can use the results when considering recovery from chemotherapy at home or in the hospital.
Professional Abstract
Objective
To compare the effectiveness of outpatient versus inpatient post-chemotherapy neutropenia management in reducing bacteremia incidence and delays in starting subsequent chemotherapy courses and improving health-related quality of life among children with acute myeloid leukemia (AML)
Study Design
| Design Elements | Description |
|---|---|
| Design | Observational: cohort study |
| Population | 554 children with AML who received standard intensive frontline chemotherapy |
| Interventions/ Comparators |
|
| Outcomes | Bacteremia incidence, time to subsequent chemotherapy courses, health-related quality of life |
| Timeframe | Up to 50-day follow-up for study outcomes following each chemotherapy course |
This study included retrospective and prospective analyses that compared the effectiveness of outpatient versus inpatient neutropenia management in reducing bacteremia incidence and delays in chemotherapy courses and improving health-related quality of life among children with AML.
The retrospective analysis examined electronic medical records from 554 children with AML who were 19 years old and younger and who received standard intensive frontline chemotherapy at 1 of 17 hospitals across the United States. Researchers categorized children discharged within three days of completing a course of chemotherapy as receiving outpatient neutropenia management and children who remained in the hospital for more than three days as receiving inpatient neutropenia management. Researchers compared the course-specific incidence of bacteremia and time to the start of the next chemotherapy course between the two groups of children. Positive blood cultures identified bacteremia occurrence.
The prospective analysis examined a subset of 97 caregivers whose children were in the study. Caregivers completed questionnaires about their child’s health-related quality of life at the start of a chemotherapy course and again after their child recovered from neutropenia.
Patients and caregivers with experience with AML helped design the study and provided feedback throughout the study.
Results
Children who received outpatient neutropenia management had similar overall bacteremia incidence across chemotherapy courses compared with those who received inpatient management (23.8% for outpatient versus 29.0% for inpatient; p=0.082), with no delays in starting subsequent chemotherapy courses (30.7 days versus 32.8; p=0.031) and comparable health-related quality of life scores (2.8 point difference in scores; p=0.558).
Limitations
The research team could not collect the data needed to compare the severity of infections between the two groups of children, which is an important factor to consider when comparing inpatient versus outpatient neutropenia management.
Conclusions and Relevance
In this study, outpatient neutropenia management did not impose excess clinical risk on children with AML or negatively affect their health-related quality of life compared with inpatient neutropenia management. Clinicians and caregivers can use these results when considering where children with AML should recover after receiving chemotherapy.
Future Research Needs
Future research could continue to explore when outpatient neutropenia management may be a suitable option. Studies could also compare the severity of infections among children with AML who receive outpatient versus inpatient neutropenia management.
Final Research Report
View this project's final research report.
Journal Citations
Related Journal Citations
Peer-Review Summary
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers asked how the researchers know that the population of patients treated at 17 centers in the United States were nationally representative. The researchers said they believe the population is representative because the contributing centers were geographically diverse, included patients who were part of clinical trials and patients who were not part of clinical trials, and the study population had clinical and demographic characteristics similar to those in large cooperative trials.
- The reviewers requested more information about who makes the choice whether chemotherapy recovery for pediatric acute myeloid leukemia occurs in the hospital or at home. Although the report indicated that this was the hospital’s choice, the reviewers asked if the choice is influenced by patients, parents, and clinicians, or solely determined by hospitals. The researchers said the standard practice at each institution determines discharge strategy. Patient characteristics and patient and family preferences typically do not influence whether patients recover within hospitals or at home after chemotherapy, or which management strategy is used.
- Given that individual hospitals determine patient discharge strategy, the reviewers expressed concern that there may be specific practices at hospitals or characteristics of patients at individual institutions that could confound the results. The researchers agreed this is an important issue and said that in order to try to mitigate such confounding factors, they captured detailed information on hospital-specific variables and standard practices. The researchers used statistical methods to address some potential confounders but acknowledged that, as for any observational study, there could have been confounding factors that they were unable to measure or measured imperfectly.
- The reviewers asked why the report presents parents’ economic concerns as part of their qualitative results. The researchers acknowledged that economic concerns were not the primary finding from their qualitative study, but when parents discussed their stress over caring for their children recovering from chemotherapy, financial pressures were an important component of parents’ anxiety. The researchers also acknowledged that patients who were discharged to recover at home had lower parental education and income than patients who recovered in hospitals. The researchers pointed out that if, as assumed, poorer, less educated, and larger families have a higher baseline risk for infection, then the risk of infection for recovering at home compared with at a hospital may be lower than measured.