Prof James W. Varni & the PedsQL™ - Celebrating 25 years of partnership with Mapi Research Trust
Few pediatric instruments have the adoption, the peer-reviewed evidence base, or the global reach that have made the Pediatric Quality of Life Inventory™ (PedsQL™) the standard of care for patient-reported outcomes in pediatric populations.
The PedsQL™ began as a response to a specific gap: the absence of developmentally appropriate, child-centered measurement tools allowing young patients to reliably report their own experiences.
To mark 25 years of partnership between Mapi Research Trust and creator Professor James W. Varni, we spoke with him about the origins of the PedsQL™, the principles that have guided its development, and what its global reach has meant for pediatric care.
What gap in pediatric care drove the development of the PedsQL™?
The origins of the Pediatric Quality of Life Inventory™ (PedsQL™) began in 1985 with the development of the Varni/Thompson Pediatric Pain Questionnaire (the shortened version distributed as the PedsQL™ Pediatric Pain Questionnaire). My initial interest in pediatric pain began in 1976 during my Pediatric Psychology Postdoctoral Fellowship at the Johns Hopkins University School of Medicine, Department of Pediatrics and the John F. Kenney Institute after graduating with a Ph.D. from the Department of Psychology at the University of California, Los Angeles (UCLA).
At the time, I was working clinically with a young patient who was an inpatient at Johns Hopkins Hospital who was hospitalized because of severe burns. My clinical responsibility was to help her manage her pain more effectively. It became evident to me that even at a very young age, she was able to communicate her pain and was responsive to intervention.
Subsequent to my postdoctoral fellowship at Johns Hopkins, my first clinical position was at Children’s Hospital of Los Angeles and Orthopaedic Hospital of Los Angeles, were I was primarily responsible for pediatric and young adult patients with hemophilia who were experiencing moderate to severe pain. This work resulted in the first pediatric pain study in the international journal Pain in 1981, which remains the premier pain journal in the world.
My clinical work then led me to help pediatric patients with rheumatoid arthritis who were experiencing significant pain. In the research literature in the late 1960’s and early 1970’s, published articles stated that children with rheumatoid arthritis were not able to accurately self-report their pain experiences. As a pediatric psychologist working clinically with pediatric patients with rheumatoid arthritis, I knew that was wrong. In fact, when reviewing these early publications, I discovered that they were using patient self-report measures that were developed with adult patients, and which were clearly not developmentally appropriate for pediatric patients.
Hence, I recognized the urgent need to develop and validate a pediatric pain measurement approach that would be age appropriate for pediatric patients and was designed for pediatric self-report so that the voices of these young children would be heard and valued.
What principles guided you in designing the PedsQL™?
The Varni/Thompson Pediatric Pain Questionnaire first publication was again in the international journal Pain in 1987 with pediatric patients with rheumatoid arthritis, and this initial publication systematically began my lifelong evidence-based commitment to demonstrate empirically that children as young as 5 years of age could reliably self-report their pain experiences if given the opportunity to do so with a developmentally appropriate measurement model.
Eventually, these early clinical and research endeavors using the Pediatric Pain Questionnaire lead to the recognition that pediatric patients with cancer, who were dealing with not only pain but also numerous other symptoms which negatively impacted their health-related quality of life (HRQOL), deserved to have their voices heard regarding their daily life. Once again, my clinical work with pediatric patients with cancer lead me to recognize this need to hear their voices not only clinically, but to develop a measurement model so that even young patients could reliably self-report their symptoms and HRQOL and which would be available internationally to help children worldwide.
At the time, the only measures of HRQOL in oncology were for adult patients, again because the field did not believe that pediatric patients were capable of reporting their own HRQOL. Thus, the challenge was to demonstrate empirically that pediatric patients were reliable reporters of their HRQOL, starting with pediatric patients with cancer. Beginning in the late 1980’s and most of the 1990’s, I initiated the iterative development of the PedsQL™ Generic Core Scales, with subsequent publication of the PedsQL™ Measurement Model comprising the integration of the Generic Core Scales with disease-specific modules in the journal Medical Care in 1999.
From that early beginning, more research evidence accrued that demonstrated that children as young as 5 years of age could reliably self-report their HRQOL using the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scales as demonstrated empirically in peer-reviewed journal publications initially in Medical Care in 2001, and subsequently in numerous publications in Quality of Life Research, Health and Quality of Life Outcomes, and a broad diversity of PedsQL™ disease-specific modules published in peer-reviewed pediatric research journals.
The PedsQL™ is used worldwide. What has been most surprising or meaningful about its global adoption?
From the very beginning, it has been an overarching goal to make the PedsQL™ internationally available in the service of children worldwide. What has been most gratifying is that this vision has been widely achieved as evidenced by not only the thousands of requests for the PedsQL™ from clinicians and researchers in over 100 countries, but also in the widespread research publications by investigators worldwide in highly diverse peer-reviewed journal publications in multiple languages.
How do you see cross-cultural validation contributing to better pediatric care globally?
Clearly, the large number of PedsQL™ international translations, the easy availability of the PedsQL™ for clinicians and researchers worldwide through the Mapi Research Trust website, the numerous peer-reviewed research journal publications, have all resulted in better pediatric care globally given the internationally validated patient-reported outcome measurement methodology provided through using the PedsQL™ instruments from infants to young adults combining pediatric patient self-report and parent proxy-report where age appropriate. This measurement approach has resulted in the worldwide adoption of the PedsQL™ as the standard of care for assessing patient outcomes from infants to young adults utilizing the same measurement constructs across the age groups of infants, young children, children, adolescents, and young adults.
What distinguishes the PedsQL™ from other patient-reported outcome measures in its approach to measuring quality of life across developmental stages?
There are now 36 generic and disease-specific versions and modules for the PedsQL™, with additional versions and modules in the planning and development process. As stated on the PedsQL™ website, a principal vision for the PedsQL™ was and is to this day to “Quantify the QualitativeSM” in the daily lives of infants, young children, children, adolescents and young adults.
The PedsQL™ Measurement Model facilitates this determination of HRQOL across the age groups of infants, young children, children, adolescents, and young adults by creating a multidimensional HRQOL construct consistently measured across the age groups, from infancy through young adulthood, ensuring that HRQOL can be measured, tracked, compared and understood as they grow and mature across the developmental stages of daily life. In this way, the PedsQL™ measures constructs that are meaningful for all age groups, which is uniquely a characteristic of the PedsQL™ not available with other patient-reported outcome measures (PROMs).
What has the 25-year collaboration with Mapi Research Trust meant for the reach and scientific integrity of the PedsQL™?
The 25-year collaboration with the Mapi Research Trust has facilitated the global research and clinical enterprise of the PedsQL™ by providing an internationally available website in which the PedsQL™ can be conveniently downloaded with the country-specific languages and country-specific dialects that are most relevant to the clinical and research community across the globe.
Thanks to my collaboration with the Mapi Research Trust, I have been given the opportunity to focus my efforts on PedsQL™ research and development (R&D), which in collaboration with colleagues has resulted in a large number of peer-reviewed research journal publications supporting the evidence-based approach of the PedsQL™ generic and disease-specific measurement instruments demonstrating their reliability and validity across diverse populations. This continuous commitment to R&D will be the primary driver in future advances in PedsQL™ measurement of HRQOL in rare diseases and other pediatric groups in order to hear the voices of all children, adolescents and young adults in matters of their health and wellbeing.