Quality of survival assessment in European childhood brain tumour trials, for children aged 5 years and over

Abstract

A 'core plus' approach is suggested in which core assessments (both direct and indirect tests) are recommended for all clinical trials. The core component is a relatively brief screening assessment that, in most countries, is a sub-component of routine clinical provision. The 'plus' components enable the addition of assessments which can be selected by individual countries and/or tumour-, age-, and location-specific groups. The implementation of a QoS protocol common to all European clinical studies of childhood brain tumours is also discussed.

Quality of survival and cognitive performance in children treated for medulloblastoma in the PNET 4 randomized controlled trial

“…This lack of association contrasts with the strong correlations observed between the different domains conceptualized under the term QoS, which typically includes questionnaire-based information relative to health status, behavior, executive function, and HRQoL 11,16 . The robust association between these different constructs suggests the existence of a common factor sustaining the significant co-variance observed among these measures.…”

“…HRQoL is questionnaire-based and includes physical, social, cognitive, and emotional functioning dimensions 11,16 .…”

“…academic achievement, independence, professional and social integration, activity limitations or participation restrictions), and self-and/or proxy-reported HRQoL [10][11][12][13][14][15] . Awareness of the importance of multidimensional assessments based on different informants has led to efforts among European countries to reach consensus regarding the domains of functioning and measures to be included in clinical trials 16 aimed at evaluating the effects of brain tumors and their treatment. This international agreement was intended to increase robustness of data collection in clinical trials to support better-informed treatment and rehabilitation decisions.…”

“…This consensus established that the assessment of QoS in clinical trials should include demographic, endocrine, and other medical information, direct measures of cognitive functions and questionnairebased assessments of health status, behavior, HRQoL, and executive functioning in everyday life 16 .…”

Neuropsychological Outcome of Children Treated for Standard Risk Medulloblastoma in the PNET4 European Randomized Controlled Trial of Hyperfractionated Versus Standard Radiation Therapy and Maintenance Chemotherapy

“…This limitation justifies caution in the interpretation of the results and generalization of these findings. Importantly, these results highlight the urgent need for an international consensus in the measures used to assess cognitive ability 34 . Moreover, participants were slightly younger at diagnosis than non-participants.…”