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Home or away from home: comparing clinician and patient/family-centered outcomes relevant to the care of pediatric acute myeloid leukemia during periods of neutropenia
Investigator (PI): Aplenc, Richard
Performing Organization (PO): (Current): Children's Hospital of Philadelphia / (215) 590-3800
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2015
Final Year: 2020
Record Source/Award ID: PCORI/CER-1409-22827
Funding: Total Award Amount: $1,890,698
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Leukemia is the most common form of cancer in children. Acute myeloid leukemia (AML) is the most life-threatening form of leukemia, requiring the most intensive chemotherapy treatment. While advances in chemotherapy have improved AML survival, chemotherapy exposure results in significant side effects. Notably, each course of chemotherapy destroys a child's white blood cells, called neutrophils, leaving the child in a state of neutropenia for three or more weeks. During neutropenia, the child is at significant risk for life-threatening bloodstream infections, which can result in delays in starting the next course of chemotherapy as well as in significant morbidity and mortality. Given the concern for infection during neutropenia after AML chemotherapy, some doctors decide to keep their patients in the hospital for close monitoring. Other doctors worry that the prolonged hospital stay for neutropenia observation is too challenging for patients and thus allow their patients to go home, advising them to return to the hospital if there is a concern for infection. It is not known if keeping the child in the hospital reduces the risk of infection or improves the chance that the patient will get to their next course of chemotherapy on time. Furthermore, there are limited data comparing these two management options on the quality of life or other patient-centered outcomes for the children and their caregivers. The proposed study includes three aims focused on comparing physician-directed and patient-centered outcomes between AML patients with neutropenia who remain in the hospital for infection observation and those who are discharged home. The physician-directed outcomes include bacterial bloodstream infections and delays in starting the next round of chemotherapy. These outcomes will be measured in approximately 385 AML patients with neutropenia from 9 pediatric hospitals (aim 1). The patient-centered outcomes will be informed by semistructured in-person interviews of over 50 AML patients and their caregivers. A medical sociologist will perform these interviews over a six-month period (aim 2). Patient-centered outcomes identified from these interviews, coupled with validated quality-of-life surveys, will then be measured in 125 AML patients with neutropenia and their caregivers, and compared between inpatient and outpatient management groups (aim 3). In all our comparisons we will use established statistical approaches so that the two groups under comparison are similar, resulting in fair comparisons. Finally, this proposal is inclusive of key stakeholders: a family consultant, a director of programs and services from a large cancer advocacy foundation, and the Patient Advocacy Committee from the Children's Oncology Group. These study members have been integrated in milestones throughout the study to ensure efficient and appropriate dissemination of study results to physicians, patients, and caregivers in the pediatric oncology community.
MeSH Terms:
  • Antineoplastic Agents /*therapeutic use
  • Bacterial Infections /complications
  • /therapy
  • Child
  • Health Care Costs
  • Hospitals, Pediatric
  • Humans
  • Length of Stay
  • Leukemia, Myeloid, Acute /complications
  • /*drug therapy
  • Multicenter Studies as Topic
  • Neutropenia /*therapy
  • Outcome Assessment, Health Care
  • Patient-Centered Care
  • Pediatrics /*methods
  • Risk
Country: United States
State: Pennsylvania
Zip Code: 19104
UI: 20153568
CTgovId: NCT02774850
Project Status: Ongoing
Record History: ('2018: Project extended to 2020',) ('2017: Project extended to 2019',)