Measuring What Matters for Children: A Systematic Review of Frequently Used Pediatric Generic PRO Instruments

Abstract

Objective To provide an assessment of the quality of the most frequently used self-reported, generic patient-reported outcome measures (PROMs) that measure health-related quality of life (HRQoL) in children against the good research practices recommended by ISPOR task force for the pediatric population. Method Literature search was conducted on OvidSP database to identify the generic pediatric PROMs used in published clinical studies. The quality of PROMs used in more than ten clinical studies were descriptively evaluated against the ISPOR task force’s good research practices. Results Six PROMs were evaluated, namely Pediatric Quality-of-Life inventory 4.0 (PedsQL), Child Health Questionnaire (CHQ), KIDSCREEN, KINDL, DISABKIDS and Child Health and Illness Profile (CHIP). All PROMs, except KIDSCREEN, had versions for different age ranges. Domains of physical, social, emotional health and school activities were common across all the instruments, while domains of family activities, parent relations, independence, and self-esteem were not present in all. Children’s input was sought during the development process of PROMs. Likert scales were used in all the instruments, supplemented with faces (smileys) in instruments for children under 8 years. KIDSCREEN and DISABKIDS were developed in a European collaboration project considering the cross-cultural impact during development. Conclusion The comparison of the instruments highlights differences in the versions for different pediatric age groups. None of the PROMs fulfill all the good research practices recommended by the ISPOR task force. Further research is needed to define which age-appropriate domains are important for older children and adolescents.

Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review

“…Also, only a minority of HRQoL‐research included children <4 years of age. This results in a knowledge gap on the child's gross motor development, vaccinations and initiation of prophylaxis in severe haemophilia, 11 as well as the effect of BDs on the bidirectional influence between infants and caregivers and early disease acceptance 78 . For this purpose, we advise to use a generic questionnaire that is, designed to measure HRQoL in young children between 0−4 years old, such as ITQOL 79 .…”

“…This results in a knowledge gap on the child's gross motor development, vaccinations and initiation of prophylaxis in severe haemophilia, 11 as well as the effect of BDs on the bidirectional influence between infants and caregivers and early disease acceptance. 78 For this purpose, we advise to use a generic questionnaire that is, designed to measure HRQoL in young children between 0−4 years old, such as ITQOL. 79 Finally, the distribution of countries shows that children from Africa and South America, and to a lesser extent Asia, are underrepresented.…”

“…We advise to use validated existing questionnaires after translation, taking different cross-cultural perspectives into account. 78,[80][81][82] 2. Strive for homogenous study groups or present results separately.…”

Towards a broader concept of wellbeing in evaluating paediatric quality of life

“…An immature minor can assent; and respect for children's developing autonomy confers an ethical obligation on HCPs to provide them with age and cognition appropriate material to inform their decisions. Certain paediatric PROMs allow children to self‐report at age 4 14 . However, developmental factors may limit a child's ability to understand concepts, for example those of data collection and storage.…”

“…Certain paediatric PROMs allow children to self-report at age 4. 14 However, developmental factors may limit a child's ability to understand concepts, for example those of data collection and storage. Most children do not appreciate the emergence of precision medicine and data insecurity as a risk for future de-identification.…”