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.

Final Research Report

View this project's final research report.

More About This Research

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.

Conflict of Interest Disclosures

The COI disclosure form will be posted here soon.

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.

Project Information

Richard Aplenc, MD, PhD
The Children's Hospital of Philadelphia
$1,890,698
10.25302/01.2021.CER.140922827

Key Dates

40 months
April 2015
August 2020
2015
2020

Study Registration Information

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Last updated: October 20, 2021