Pediatric Acute Myeloid Leukemia
Conditions
Keywords
Pediatric, Acute Myeloid Leukemia, Quality of Life, Neutropenia Management, Patient Centered Outcomes, Chemotherapy
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.
Interventions
None listed
Sponsors
Children's Healthcare of Atlanta
C.S. Mott Children's Hospital
Ann & Robert H Lurie Children's Hospital of Chicago
Arkansas Children's Hospital Research Institute
Children's Medical Center Dallas
Baylor College of Medicine
Primary Children's Hospital
Children's Hospital of Michigan
Lucile Packard Children's Hospital
St. Jude Children's Research Hospital
Seattle Children's Hospital
Patient-Centered Outcomes Research Institute
Alfred I. duPont Hospital for Children
Children's Hospital Colorado
Dana-Farber Cancer Institute
Children's Hospital of Philadelphia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
Yes
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management | 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. | 1 Course of Chemotherapy (Approximately 30-40 days). The primary outcome of interest was patient health-related quality of life (HRQOL) measured using the acute PedsQL™ 4.0 Generic Core Scales.31 These scales use a 7-day time frame. The multidimensional assessment includes items in four domains: physical functioning, emotional functioning, social functioning, and school functioning. Respondents document responses to each question using a 5-point Likert scale anchored by never a problem (0) to almost always a problem (4). PedsQL™ items were reverse scored and linearly transformed to a scale of 0 to 100 such that higher scores reflect better HRQOL. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Responses From Structured Patient-centered Outcome Surveys | 1 Course of Chemotherapy (Approximately 30-40 days) | Information from semi-structured patient outcome surveys completed by patients and caregivers will be collected to evaluate the relationship between neutropenia management strategy (inpatient versus outpatient) and outcomes reported by patients and caregivers. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Early Discharge Patients Patients receiving or having received chemotherapy for AML who are discharged to outpatient management within 3 days after chemotherapy completion. Subjects will complete a health related quality of life (HRQOL) survey at baseline and again at the start of the next treatment course. Survey questions will collect information such as patient race and educational level.
HRQOL questions will be scored on a 5-point Likert response scale (0=never a problem to 4=almost always a problem). The total score includes a 23 item multidimensional core (physical, emotional, social and school functioning); an 18 item multidimensional fatigue scale (general, sleep/rest and cognitive fatigue) and a 27 item multidimensional cancer module (pain, nausea, anxiety, communication, and cognitive problems). Total score is calculated with a lower score representing a higher health related quality of life. | 22 |
| Inpatient Management Patients Patients receiving or having received chemotherapy for AML who remain in the hospital more than 3 days after chemotherapy completion. Subjects will complete a health related quality of life (HRQOL) survey at baseline and again at the start of the next treatment course. Survey questions will collect information such as patient race and educational level.
HRQOL questions will be scored on a 5-point Likert response scale (0=never a problem to 4=almost always a problem). The total score includes a 23 item multidimensional core (physical, emotional, social and school functioning); an 18 item multidimensional fatigue scale (general, sleep/rest and cognitive fatigue) and a 27 item multidimensional cancer module (pain, nausea, anxiety, communication, and cognitive problems). Total score is calculated with a lower score representing a higher health related quality of life. | 75 |
| Total | 97 |
Baseline characteristics
| Characteristic | Inpatient Management Patients | Total | Early Discharge Patients |
|---|---|---|---|
| Age, Categorical <=18 years | 75 Participants | 97 Participants | 22 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Hispanic Ethnicity Hispanic Ethnicity | 13 Participants | 17 Participants | 4 Participants |
| Race/Ethnicity, Customized Hispanic Ethnicity Not Hispanic Ethnicity | 62 Participants | 80 Participants | 18 Participants |
| Race/Ethnicity, Customized Race Asian | 5 Participants | 7 Participants | 2 Participants |
| Race/Ethnicity, Customized Race Black | 6 Participants | 11 Participants | 5 Participants |
| Race/Ethnicity, Customized Race Not recorded in EMR | 2 Participants | 2 Participants | 0 Participants |
| Race/Ethnicity, Customized Race Other | 6 Participants | 11 Participants | 5 Participants |
| Race/Ethnicity, Customized Race White | 56 Participants | 66 Participants | 10 Participants |
| Region of Enrollment United States | 75 Participants | 97 Participants | 22 Participants |
| Sex: Female, Male Female | 38 Participants | 46 Participants | 8 Participants |
| Sex: Female, Male Male | 37 Participants | 51 Participants | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 0 | 0 / 0 |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management
1 Course of Chemotherapy (Approximately 30-40 days). The primary outcome of interest was patient health-related quality of life (HRQOL) measured using the acute PedsQL™ 4.0 Generic Core Scales.31 These scales use a 7-day time frame. The multidimensional assessment includes items in four domains: physical functioning, emotional functioning, social functioning, and school functioning. Respondents document responses to each question using a 5-point Likert scale anchored by never a problem (0) to almost always a problem (4). PedsQL™ items were reverse scored and linearly transformed to a scale of 0 to 100 such that higher scores reflect better HRQOL.
Time frame: 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.
Population: Participants who completed evaluable PedsQL follow-up surveys.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Early Discharge Patients | Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management | 70.1 score on a scale | Standard Deviation 18.9 |
| Inpatient Management Patients | Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management | 68.7 score on a scale | Standard Deviation 19.4 |
Secondary
Responses From Structured Patient-centered Outcome Surveys
Information from semi-structured patient outcome surveys completed by patients and caregivers will be collected to evaluate the relationship between neutropenia management strategy (inpatient versus outpatient) and outcomes reported by patients and caregivers.
Time frame: 1 Course of Chemotherapy (Approximately 30-40 days)