Validation of the caregiver Pediatric Patient‐Reported Outcomes Version of the Common Terminology Criteria for Adverse Events measure

Abstract

Background Despite improvements in survival rates, cancer treatments have significant side effects that affect the quality of life of children and their families. When an ill child cannot self‐report symptoms (eg, he or she is too ill), caregiver (parent) reporting becomes critical. This study evaluates the validity and reliability of the caregiver‐reported Pediatric Patient‐Reported Outcomes version of the Common Terminology Criteria for Adverse Events (Ped‐PRO‐CTCAE [Caregiver]) measure. Methods A diverse sample of caregivers with children receiving treatment at 9 oncology centers completed the Ped‐PRO‐CTCAE [Caregiver] measure, the Patient‐Reported Outcomes Measurement Information System® (PROMIS®) Parent Proxy measures, the Lansky Play‐Performance Scale (PPS), medication use questions, and Global Impressions of Change (GIC). Construct validity (including convergent, discriminant, and known groups validity and responsiveness over time) and reliability (stability) were examined. Results A majority of the 473 caregivers were female (85%), non‐Hispanic White (61%), and married (75%). Symptoms assessed with the Ped‐PRO‐CTCAE [Caregiver] and PROMIS Parent Proxy measures were strongly correlated (e.g., r for pain = 0.78; r for fatigue = 0.78; and r for depression = 0.83). Most of the Ped‐PRO‐CTCAE [Caregiver] item mean scores distinguished among PPS function levels and between children who did take medications for symptom control and children who did not. Changes in Ped‐PRO‐CTCAE [Caregiver] item mean scores were responsive to GIC over time. Test‐retest evaluation found moderate to high agreement (57.8%‐93.3%) over time. Conclusions This study found strong evidence for the convergent and discriminant validity, known groups validity, responsiveness, and stability of the Ped‐PRO‐CTCAE [Caregiver] measure in a large and diverse sample of caregivers. The caregiver perspective provides a valuable and unique insight into the experiences of children and adolescents undergoing cancer treatment. Lay Summary Despite advances in cancer treatments, children and adolescents continue to suffer from treatment side effects, including pain, nausea, fatigue, and emotional distress, that can adversely affect quality of life for children and their families. Although it is best for children to report how they are feeling, there are times when a child may be too young or too ill to self‐report. This study provides critical evidence for a new type of questionnaire that allows the caregiver or parent to report accurately what the child is experiencing. This measure can be used to improve adverse event reporting and child cancer care.

Symptom screening via screen: Real‐time electronic tracking of pediatric patient‐reported outcomes

“…Moreover, easier accessibility was shown to enhance the utility of PROs, as 21% of symptom reports led to a change in clinical care.The validity, responsiveness, and stability of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) instrument offer a new and proven way for children to report how they are feeling and for proxy caregivers to parallel-report on children's symptoms. 3,4 It is worth recognizing that children with cancer participated as codevelopers in the PRO-CTCAE instrument through extensive cognitive interviews. 5 Now, reading of the instrument's application in real-time feedback for medical teams harkens to the progressing state of PRO science as true translational research from development to implementation.Because of the amount of time that oncology teams already spend in front of computer screens instead of face to face with patients, there may be initial skepticism at the concept of electronic data reporting resulting in improved human communication.…”

“…The validity, responsiveness, and stability of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) instrument offer a new and proven way for children to report how they are feeling and for proxy caregivers to parallel-report on children's symptoms. 3,4 It is worth recognizing that children with cancer participated as codevelopers in the PRO-CTCAE instrument through extensive cognitive interviews. 5 Now, reading of the instrument's application in real-time feedback for medical teams harkens to the progressing state of PRO science as true translational research from development to implementation.…”

Opening Pandora’s Box

“… 46 The use of proxies and alternative approaches to data collection can reduce selection bias and missing data but can also introduce measurement error. Researchers can learn from pediatrics where the use of proxies for PCOM is well-established, 47 to consider the advantages and disadvantages of the different approaches to data collection and sources of data. When >1 approach to data collection is used, the team should conduct sensitivity analyses to understand the impact on study findings.…”

One Voice Is Good, But More Is Better: Symptom and Toxicity Reporting in Pediatric Oncology

“…However, there has been recognition of the value of the patient and/or family caregiver perspective 1 . Significant advances include new self-report and/or caregiver-report measures, such as measures of a single symptom, multiple symptoms, or larger item libraries allowing investigators or clinicians the selection of appropriate subset of symptoms or adverse events specific to certain cancer therapies 2,3 . Over time, studies have documented discrepancies between what the child and/or adolescent reports and what the clinicians or caregivers report in terms of presence and intensity of symptoms and toxicities.…”