Pediatric Acute Myeloid LeukemiaAim 3NCT02777021
Home Away From Home - Quality of Life Surveys
An observational study of Pediatric Acute Myeloid Leukemia, run by Children's Hospital of Philadelphia at 15 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Pediatric Acute Myeloid Leukemia
- How many people
- 154 participants
- Where
- 15 sitesUnited States
- Study dates
- Sep 2016to 18 Jul 2019
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
- 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.
- Parental/caregiver informed consent and, if appropriate, child assent.
Who cannot join
- Patients being treated for relapsed AML
- Patients with Acute Promyelocytic Leukemia (APML)
- Patients undergoing stem cell transplant (SCT)
- Patients receiving reduced intensity frontline chemotherapy
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital of Colorado
Aurora, Colorado, 80045
Dana-Farber Cancer Institute/Boston Children's Hospital
Boston, Massachusetts, 02215
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Children's Medical Center of Dallas
Dallas, Texas, 75235
Children's Hospital of Michigan
Detroit, Michigan, 48201
Texas Children's Hospital
Houston, Texas, 77030
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Primary Children's Hospital
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
Nemours/Alfred I DuPont Hospital for Children
Wilmington, Delaware, 19803
About this study
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
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.
Read the 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.
- Conditions
- Pediatric Acute Myeloid Leukemia
- Keywords
- Pediatric, Acute Myeloid Leukemia, Quality of Life, Neutropenia Management, Patient Centered Outcomes, Chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. | |
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 (1)
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.
Measured: 1 Course of Chemotherapy (Approximately 30-40 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2016 (estimated)
- Study startSep 2016
- Primary completion18 Jul 2019
- Study completion18 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pediatric Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 154 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- 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
- Responsible party
- Sponsor
- Investigators
- Richard Aplenc, MD, PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02777021
- Sponsor's protocol ID
- 15-012103
- Acronym
- Aim 3
- Status last verified
- Dec 2019
- First submitted
- 16 May 2016
- Last updated
- 23 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No: only aggregate and de-identified data will be shared upon appropriate request.
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02777021
- Acronym / sponsor ID
- Aim 3 / 15-012103
- Conditions
- Pediatric Acute Myeloid Leukemia
- Keywords
- Pediatric, Acute Myeloid Leukemia, Quality of Life, Neutropenia Management, Patient Centered Outcomes, Chemotherapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 154 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- 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
- Study start
- Sep 2016
- Primary completion
- 18 Jul 2019 (actual)
- Study completion
- 18 Jul 2019 (actual)
- First posted
- 19 May 2016 (estimated)
- Results
- Posted 23 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (8 listed)
- Participants will be enrolled as patient-caregiver dyads. The patient must be:
- Participants will be enrolled as patient-caregiver dyads. The caregiver must be:
- Parental/caregiver informed consent and, if appropriate, child assent.
Key exclusion criteria (4 listed)
- Patients being treated for relapsed AML
- Patients with Acute Promyelocytic Leukemia (APML)
- Patients undergoing stem cell transplant (SCT)
- Patients receiving reduced intensity frontline chemotherapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management
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.
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.
1 secondary outcome
Responses From Structured Patient-centered Outcome Surveys
Time frame: 1 Course of Chemotherapy (Approximately 30-40 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. only aggregate and de-identified data will be shared upon appropriate request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Aplenc, MD, PhD principal investigator
Children's Hospital of Philadelphia
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Acute Myeloid Leukemia, run by Children's Hospital of Philadelphia at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare Differences in HRQOL Scores Between Outpatient Versus Inpatient Management (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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2019.