The PediQUEST Study: Evaluation of Pediatric Quality of Life and Evaluation of Symptoms Technology
|ClinicalTrials.gov Identifier: NCT01838564|
Recruitment Status : Completed
First Posted : April 24, 2013
Last Update Posted : April 29, 2013
|First Submitted Date ICMJE||April 19, 2013|
|First Posted Date ICMJE||April 24, 2013|
|Last Update Posted Date||April 29, 2013|
|Study Start Date ICMJE||November 2004|
|Actual Primary Completion Date||December 2009 (Final data collection date for primary outcome measure)|
|Current Primary Outcome Measures ICMJE
|Original Primary Outcome Measures ICMJE
||Child Distress [ Time Frame: 20 weeks ]
Child distress will be assessed through:
|Change History||Complete list of historical versions of study NCT01838564 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
|Original Secondary Outcome Measures ICMJE
||Child's quality of life [ Time Frame: 20 weeks ]
Trends of the PedsQL scores (total and subscales) over 20 weeks: Continuous variable. Range of PedsQL: 0-100. 0 worst quality of life.
|Current Other Outcome Measures ICMJE
||Satisfaction with PediQUEST [ Time Frame: At 4th PediQUEST administration ]
Satisfaction with the technology and with the feedback system will be assessed twice at the fourth and eight PediQUEST administration. Specifically we will assess consumers'impressions about the technology, easiness of use, relevance.
Among those receiving the feedback intervention we also assessed whether reports helped parents understand their children's feelings better, facilitated communication with doctors and overall satisfaction with the technology and feedback.
Finally, we also assessed providers satisfaction with feedback reports including their impression about the reports and whether reports affected their behaviors regarding symptom management.
|Original Other Outcome Measures ICMJE
|Brief Title ICMJE||The PediQUEST Study: Evaluation of Pediatric Quality of Life and Evaluation of Symptoms Technology|
|Official Title ICMJE||The PediQUEST Study: Evaluation of Pediatric Quality of Life and Evaluation of Symptoms Technology|
Over 50% of children dying from cancer still suffer from symptoms that could be effectively alleviated. The purpose of the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) Study was to evaluate whether providing feedback to families and providers about how the child is feeling improved child distress and quality of life (QoL) in children with advanced cancer.
PediQUEST is a computerized survey that asks the child and/or parents how the child has been feeling, i.e. whether the child had any physical or emotional symptoms, as well as how other aspects of life, such as school and friends, are going. After the survey is complete a report that summarizes patient/parent answers is printed. When a child reports moderate to high distress from any symptom an email is automatically sent to the primary providers (oncologist, nurse, and psycho-social clinician as well as the pain and palliative care services) alerting them about the child's distress.
In this study we evaluated whether using PediQUEST and providing printed reports to parents and providers reduced distress and improved quality of life in children with advanced cancer. In addition, we wanted to understand whether it was feasible to carry out a randomized controlled trial in children with advanced cancer. Finally, the data collected, will be used to describe the natural history of symptoms and quality of life as reported by the children.
Children enrolled in the study (or their parents) were asked to complete a PediQUEST survey at most once a week. A random half of the children received the feedback intervention, i.e. patients, parents, and providers received printed reports (and emails if the child was in distress). The other half only completed the PediQUEST surveys and did not receive reports. We analyzed data collected over 20 weeks of follow-up to see whether receiving PediQUEST reports had any effect on child distress and quality of life.
Introduction: Cancer is the leading cause of nonaccidental death in childhood.(Arias et al. 2003) In spite of significant scientific progress, one quarter of children with cancer still do not survive(Greenlee et al. 2000), and over 50% experience substantial suffering that could be alleviated.(Wolfe et al. 2000a) Pediatric palliative care is now an expected standard of care and research in this field has been established as a national priority by the Institute of Medicine (IOM).(Field et al. 2003) Earlier work by our group suggested that when parents and physicians recognized at an early stage that a child had no realistic chance of cure, they were more likely to integrate elements of effective palliation into the child's care. However, this early stage concordance about prognosis was rare.(Wolfe et al. 2000b) Communication and delivery of optimal care in the advanced stages of illness is especially challenging in children.(Shelton 1999) The anticipation of losing a child and physicians' own difficulties(Saunders 1979-80; Hilden et al. 2001) may hinder discussions about poor prognosis and impact on decision-making and integration of palliative care. Thus attention to symptom management and quality of life (QoL) in children with advanced cancer may be limited.
The collection of standardized symptom and QoL data has shown to enhance physician's recognition, knowledge and accuracy in the assessment and palliation of symptoms, and to increase patient-provider discussions about QoL issues and directly improve patient's QoL.(Detmar et al. 2002; Taenzer et al. 2000; Velikova et al. 2004) Whether these findings are applicable to children remains to be established.
One of the obstacles to routine symptom and QoL assessment in children is the need of developmentally adapted(Eiser et al. 2001b; Eiser et al. 2001c; Hain 1997) and parent-proxy tools(Eiser et al. 2001a), making the paper and pencil format extremely impractical. Computerized surveys offered a potential solution. Patient acceptance of such devices has been found to be high.(Buxton et al. 1998; Velikova et al. 2002; Velikova et al. 1999; Velikova et al. 2001) Our current project -the PediQUEST Study- consists of a pilot for a randomized controlled trial (RCT) aimed at assessing the effect of routinely reporting symptom and QoL data. We will use an innovative data collection strategy: a hand-held computer devise, the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) that readily administers a user-appropriate survey instrument and generates reports summarizing how the patient feels.
As far as we are aware of, this will be the first RCT conducted in children with advanced cancer to evaluate a palliative care intervention. Given the challenges with doing research in this population, we propose to simultaneously focus on several goals.
Aims: Aim 1: (Feasibility study) Assess the feasibility of conducting a randomized controlled supportive care trial (RCT) in children with advanced cancer. Aim 2: (PediQUEST Evaluation) Preliminarily assess the effect of routinely feeding back QoL and symptom data to families and primary oncologists, and alerting supportive care teams about highly distressing symptoms. Aim 3: (Descriptive study) Prospectively explore determinants of child's suffering and parental-physician discordance regarding prognosis and treatment goals in children with advanced cancer.
Design: Aim 1: Cohort survey study; Aim 2: Pilot prospective RCT; Aim 3: Cohort study embedded in an RCT.
Setting: Three large US pediatric cancer centers. Subjects: 104 children 2-years old and above with advanced cancer and their parents were enrolled between December 2004 and June 2009.
Follow-up: Patients were initially followed up for 9 months or until death whatever occurred first. However, an interim analysis of the first 29 patients showed that 75% were alive at nine months, suggesting the enrollment of a healthier cohort. We therefore changed the follow-up period to 3 months with the option of multiple re-enrollments until the end of data collection (December 2009) or death.
Feasibility study: All subjects approached for enrollment were invited to complete a one-time paper and pencil "Consent survey" regardless of their decision about whether to participate. All enrolled subjects were asked to complete a paper and pencil "Participation survey" after completing the study or dropping-out in order to explore determinants of attrition.
PediQUEST Evaluation and Descriptive studies: Symptom and QOL scores were tracked up to once a week. Surveys were embedded in the PediQUEST system and administered through tablet computers.
Participants' recruitment: Eligible children were identified through daily review of clinic roster with clinic staff including nurses, oncologists and/or nurse practitioners and verified with the patient's primary oncologist using a checklist sent over email. If after being introduced to the study and having had the opportunity to ask questions, child and parent(s) were willing to participate, parent(s) were asked to review and sign the informed permission/assent document covering the parent(s) and child's participation. A provider's completion of the eligibility checklist will indicate informed consent of providers.
Intervention: Patients were randomly allocated to receive routine feedback about symptoms and QOL. The feedback intervention had two components: (i) PQ-reports: printed reports were made available to providers (inserted in charts before the visit) and families (handed immediately after survey completion at clinic or ward). PQ-reports consisted of bar plots of PedsQL and MSAS symptom scores from current and four prior administrations, a summary highlighting changes since last report, and a list of available resources for symptom control (for families), or generic pain management recommendations (for providers). Training on how to interpret PQ-reports was offered to families at enrollment and on an annual basis to providers; (ii) PQ emails: were automatically generated if PQ-MSAS individual symptom scores were ≥70, or if on two consecutive administrations the patient or parent reported a score of ≥70 for any frequency, severity or distress item, or a PedsQL total score ≤ 40. E-mails were sent to primary providers (oncologist, nurse, and psychosocial clinician), the local palliative care service, and, when pain was reported, the pain service (or equivalent). No instructions were imparted on how to respond to emails. Families and providers of patients in the control arm of the study did not receive feedback reports. The allocation process was embedded in PediQUEST and blinded to investigators until the patient completed the first survey. Because of the nature of the intervention this was an open label study. As a token of our appreciation, small non-monetary incentives were provided to children, parents, and providers.
Outcomes: This study explores the feasibility of conducting an RCT assessing a supportive care intervention in children with advanced cancer (aim 1). It also provides estimates of consent and attrition rates, its determinants, and the burden associated with repeated measurements. In addition, this pilot study will preliminarily assess the impact of feedback on child's distress and quality of life. Main outcomes of this efficacy study (aim 2) are presented below. This pilot study will render sufficient data for accurate sample size calculation for future studies and serve to explore determinants of suffering in children with advanced cancer (aim 3).
PediQUEST Evaluation (Aim 2): To assess the effect of PediQUEST, the proportion of patients with unrelieved symptoms in each arm will be compared using odds ratios and associated chi-square tests. To assess the progression of reported levels of child's distress and QOL trends, we will run a mixed effects model that assumes a linear trend in the outcome over 20 weeks of follow up for each subject. The model will examine the difference in the average trend over time (slope) between the intervention and control group by including a fixed effect term that represents the interaction of treatment status and time since study entry. Lack of difference between slope coefficients will support the null hypothesis of no effect by the intervention. Single point secondary outcomes will be analyzed by comparing both arms with a t test or Wilcoxon rank-sum test for continuous outcomes and odds ratios and associated chi-square tests for proportions.
Sample Size and Power Calculations A formal sample size estimation was not possible given the lack of data on distribution of scores in an advanced cancer population or their variation over time. For this reason we originally proposed to recruit 120 patients and their parents, a sample size driven by practical rather than statistical considerations that would serve as a basis for future sample size calculations. We estimated that 60 subjects per arm would allow for an adequate balance between arms and likely provide sufficient power to detect large clinical effects for single point measurements.
|Study Type ICMJE||Interventional|
|Study Phase||Not Applicable|
|Study Design ICMJE||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Completed|
|Actual Enrollment ICMJE
|Original Actual Enrollment ICMJE||Same as current|
|Actual Study Completion Date||June 2010|
|Actual Primary Completion Date||December 2009 (Final data collection date for primary outcome measure)|
|Eligibility Criteria ICMJE||
|Ages||2 Years to 25 Years (Child, Adult)|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Listed Location Countries ICMJE||United States|
|Removed Location Countries|
|NCT Number ICMJE||NCT01838564|
|Other Study ID Numbers ICMJE||04-321
1K07CA096746-01 ( U.S. NIH Grant/Contract )
|Has Data Monitoring Committee||No|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement||Not Provided|
|Responsible Party||Joanne Wolfe, MD, Dana-Farber Cancer Institute|
|Study Sponsor ICMJE||Dana-Farber Cancer Institute|
|Collaborators ICMJE||National Cancer Institute (NCI)|
|PRS Account||Dana-Farber Cancer Institute|
|Verification Date||April 2013|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP