Quality-of-Life Assessment in Pediatric Advanced Cancer: Development of the Patient-Reported Outcome Measure Advance QoL.

A recent measure was developed to assess the Quality of Life (QoL) of young people with advanced cancer and is available for parents and professionals (Advance QoL). The present study aimed to elaborate self-reported versions for children and adolescents with advanced cancer. We adopted a four-phase research plan: (1) to elaborate the Advance QoL questionnaire for youth (8-12 and 13-18 years old) with a team of young research partners; (2) to evaluate the understandability of these versions in a sample of 12 young patients from the target population using cognitive interviews; (3) to assess social validity in the same group using a questionnaire and the content validity index (CVI); and (4) to refine the questionnaires according to these results. Four major themes were identified: (1) issues affecting the understanding of the tool; (2) issues that did not affect the understanding of the tool; (3) modifications to improve the tool; and (4) positive features of the tool. Advance QoL was well received, and feedback was positive. Adjustments were made according to young people's comments and two self-reported versions are now available. It is essential to measure the key domains of QoL in advanced cancer. Advance QoL self-report versions will help target the specific needs of young people with this condition and their families.

A systematic review of the assessment of the clinical utility of genomic sequencing: Implications of the lack of standard definitions and measures of clinical utility.

PURPOSE: Exome sequencing (ES) and genome sequencing (GS) are diagnostic tests for rare genetic diseases. Studies report clinical utility of ES/GS. The goal of this systematic review is to establish how clinical utility is defined and measured in studies evaluating the impacts of ES/GS results for pediatric patients. METHODS: Relevant articles were identified in PubMed, Medline, Embase, and Web of Science. Eligible studies assessed clinical utility of ES/GS for pediatric patients published before 2021. Other relevant articles were added based on articles' references. Articles were coded to assess definitions and measures of clinical utility. RESULTS: Of 1346 articles, 83 articles met eligibility criteria. Clinical utility was not clearly defined in 19% of studies and 92% did not use an explicit measure of clinical utility. When present, definitions of clinical utility diverged from recommended definitions and varied greatly, from narrow (diagnostic yield of ES/GS) to broad (including decisions about withdrawal of care/palliative care and/or impacts on other family members). CONCLUSION: Clinical utility is used to guide policy and practice decisions about test use. The lack of a standard definition of clinical utility of ES/GS may lead to under- or overestimations of clinical utility, complicating policymaking and raising ethical issues.

Quality of life in childhood advanced cancer: from conceptualization to assessment with the Advance QoL tool.

BACKGROUND: Advanced childhood cancer, a condition with no available cancer-focused treatment options, greatly impacts Quality of Life (QoL). We need appropriate assessment strategies to select adapted treatment targets, improve care and optimize communication. Our first goal was to identify the domains of patients' QoL by combining for the first time the perspectives of patients and parents with previously collected reports in professionals. Our second goal was to develop a simple QoL assessment tool and optimize its format and content for use in the childhood advanced cancer population. METHODS: To identify QoL domains, we conducted qualitative interviews with 7 young patients (4 girls, 3 boys, aged 13 ± 4 yrs) and 9 parents (7 mothers, 2 fathers) from our treatment centre. We used inductive thematic content analysis to code and categorize respondents' viewpoints. The first version of the tool (Advance QoL) was then drafted, and structured feedback was collected through interviews and a survey with 15 experts. We computed content validity indices. RESULTS: Apart from the physical, psychological, and social domains, participants insisted on four original themes: autonomy, pleasure, the pursuit of achievement, and the sense of feeling heard. This was in line with the categories found in a preliminary study involving professionals (PMID: 28137343). Experts evaluated the tool as clear, relevant, acceptable, and usable. They formulated recommendations on instructions, timeframe, and item formulations, which we implemented in the refined version. CONCLUSIONS: Advance QoL is an innovative tool targeting key life domains in childhood advanced cancer. It is focused on preserved abilities and targets of care. The refined version is appropriate for adult respondents within families and professionals. Future studies will develop versions for young ages to collect the experience of patients themselves. This will open on future reliability, validity, sensitivity, and implementation studies.