Clinical trial NCT02777021: Home Away From Home - Quality of Life Surveys
Official title: Home or Away From Home: Comparing Patient and Caregiver Reported Quality of Life (QoL) and Other Patient-centered Outcomes for Inpatient Versus Outpatient Management of Neutropenia in Children With AML
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pediatric Acute Myeloid Leukemia
Keywords: Pediatric
Acute Myeloid Leukemia
Quality of Life
Neutropenia Management
Patient Centered Outcomes
Chemotherapy
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
Collaborators: name: Children's Healthcare of Atlanta
name: C.S. Mott Children's Hospital
name: Ann & Robert H Lurie Children's Hospital of Chicago
name: Arkansas Children's Hospital Research Institute
name: Children's Medical Center Dallas
name: Baylor College of Medicine
name: Primary Children's Hospital
name: Children's Hospital of Michigan
name: Lucile Packard Children's Hospital
name: St. Jude Children's Research Hospital
name: Seattle Children's Hospital
name: Patient-Centered Outcomes Research Institute
name: Alfred I. duPont Hospital for Children
name: Children's Hospital Colorado
name: Dana-Farber Cancer Institute
First posted: 2016-05-19
Enrollment: 154
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include all AML patients who are receiving a planned chemotherapy course at any of the fifteen pediatric institutions across the US between June 1, 2016 and December 31, 2019. Patients discharged within 3 days after chemotherapy completion will be categorized as 'early discharge' to outpatient management during neutropenia. Patients remaining in the hospital more than 3 days after chemotherapy completion will be categorized as inpatient management.

Caregivers of these patients will also be included.

Brief summary: Treatment for pediatric acute myeloid leukemia (AML) involves intensive chemotherapy regimens that result in periods of profound neutropenia leaving patients susceptible to severe infectious complications. Infectious complications are the leading cause of treatment related mortality among AML patients, but there are little clinical data to inform whether management of neutropenia post AML chemotherapy should occur in an outpatient or inpatient setting. Further, no studies have been conducted that assess the impact of neutropenia management strategy on the quality of life of pediatric patients with AML and their caregivers.

Detailed description: This is a prospective observational cohort study, where the primary objective of this study is to compare patient and caregiver quality of life and other patient-centered outcomes for inpatient versus outpatient management of neutropenia in children with AML or MDS receiving standard intensive AML frontline chemotherapy.

Local study investigators (pediatric oncologists and study coordinators) at each of the fifteen participating pediatric institutions will communicate on a weekly basis with their inpatient leukemia service to identify AML or MDS patients potentially eligible for study enrollment. Once identified, study personnel will review each patient for study eligibility criteria. Three visits with the patient and their caregivers will occur: a screening visit, initial survey visit, and a follow-up visit.

Screening Visit: The eligibility criteria for participation will be confirmed prior to approaching for consent. Eligible patients interested in the study will be approached for consent at any time from AML/MDS diagnosis through last day of chemotherapy in the treatment course under study. In some cases, the patient's caregivers may not be present in the hospital to provide consent. In these cases, study personnel will obtain verbal consent from the caregivers and child assent (if appropriate).

Visit 1 will occur prior to the last day of chemotherapy administration in the course. This visit will include:

* 2 Brief demographic surveys to capture covariates unavailable in the medical record
* Baseline health-related quality of life (HRQOL) surveys
* A baseline financial toxicity assessment

Surveys will be administered via paper or a smart device and will last a total of 15-30 minutes per respondent. In the case that the child is 5 years of age or older, the child self-report and parent proxy-report scales will be separately administered to the child and caregiver, respectively. If the child is under 5 years of age, only the parent-proxy version will be administered. Only the caregiver completes the baseline financial toxicity assessment. We will provide a $25 gift card to each child-parent dyad upon completion of the baseline surveys

Visit 2 will occur within the period after absolute neutrophil count recovery and ideally prior to the start of the subsequent course of chemotherapy, but no later than the last day of chemotherapy in that next treatment course. This visit will include:

* Follow-up HRQOL surveys
* Patient-centered outcome survey developed previously from qualitative interviews of AML patients and their caregivers
* A follow-up financial toxicity assessment

Surveys will be administered via paper or a smart device and will last a total of 15-30 minutes per respondent. In the case that the child is 5 years of age or older, the child self-report and parent proxy-report scales will be administered to the child and caregiver, respectively. If the child is under 5 years of age, only the parent-proxy version will be administered. The follow-up financial toxicity assessment and the patient-centered outcome survey are completed by the caregiver only. We will provide a $25 gift card to each child-parent dyad upon completion of the follow-up surveys.

Eligibility criteria: Inclusion Criteria:

1. Participants will be enrolled as patient-caregiver dyads. The patient must be:

   * Less than 19 years of age at diagnosis.
   * Patient is English or Spanish literate.
   * Receiving chemotherapy for AML between June 1, 2016 and December 31, 2019.
2. Participants will be enrolled as patient-caregiver dyads. The caregiver must be:

   * English or Spanish literate.
   * The legal guardian of a patient receiving chemotherapy for AML between June 1, 2016 and December 31, 2019.
3. Parental/caregiver informed consent and, if appropriate, child assent.

Exclusion Criteria:

1. Patients being treated for relapsed AML
2. Patients with Acute Promyelocytic Leukemia (APML)
3. Patients undergoing stem cell transplant (SCT)
4. Patients receiving reduced intensity frontline chemotherapy

Primary outcomes:
measure: Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management
timeFrame: PedsQL assessments were administered at two points - at the start of the study-contributed chemotherapy course prior to the patient becoming neutropenic (baseline) and again within the period between neutropenia resolution (follow-up); follow-up reported.
Secondary outcomes:
measure: Responses From Structured Patient-centered Outcome Surveys
timeFrame: 1 Course of Chemotherapy (Approximately 30-40 days)
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Wilmington
country: United States
facility: Nemours/Alfred I DuPont Hospital for Children
state: Delaware
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H Lurie Children's Hospital of Chicago
state: Illinois
city: Boston
country: United States
facility: Dana-Farber Cancer Institute/Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: C.S. Mott Children's Hospital
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Dallas
country: United States
facility: Children's Medical Center of Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Richard Aplenc, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: