The inclusion of health-related quality of life (HRQoL) impacts on informal carers in health technology assessments (HTAs) is lacking due, primarily, to a deficiency in evidence and methodological issues on how informal carer HRQoL is captured and incorporated into economic models. These issues are magnified in areas of significant burden, such as caring for children and adolescents with rare, progressive, life-limiting conditions. In this commentary we outline key challenges in measuring, and incorporating in HTA submissions, informal carer HRQoL data in rare, progressive, paediatric, life-limiting conditions and identify future research priorities in this space. We argue that: (i) a generic model of carer HRQoL is likely inadequate to characterise the HRQoL impacts in this population; (ii) the underlying evidence-base is underdeveloped, including understanding commonalities across conditions, impact beyond the primary carer, and differences over time; and (iii) methodological challenges in modelling informal carer HRQoL in cost-effectiveness analysis are inhibiting progress. A research agenda is proposed that addresses these challenges by focusing first on in-depth qualitative research to develop an appropriate, content valid ‘disease-group-specific’ model for understanding informal carer HRQoL in rare, progressive, paediatric, life-limiting conditions. This model can be used to inform the appropriate measurement of carer HRQoL in this population, which, alongside methodological research on addressing modelling challenges, can help to facilitate the recommended inclusion of informal carer HRQoL data in HTA submissions for children and adolescents living with rare, progressive, life-limiting conditions.

PurposeAlthough HIV is likely to be a couple-based issue among serodiscordant male couples due to cross-partner transmission, little is known about how they cope with HIV as a team. This study aimed to examine the dyadic coping process among serodiscordant couples.MethodsA dyadic daily diary study was used to answer our research questions. One hundred and forty-one Chinese HIV serodiscordant male couples completed measures of we-disease appraisal, common dyadic coping, quality of life, and relationship satisfaction for 14 consecutive days and ART adherence, attitudes toward PrEP, quality of life, and relationship satisfaction at a 2-month post-diary assessment. Computations were based on the common fate mediation model, using multilevel structural equation modeling.ResultsRegarding direct effect, we-disease appraisal was associated with both partners’ quality of life at the between-person level, post-diary quality of life, attitudes toward to PrEP, relationship satisfaction at both levels as well as post-diary relationship satisfaction. Common dyadic coping mediated the association between we-disease appraisal and both partners’ quality of life as well as relationship satisfaction at the within-person level. However, no mediating effects were observed at the between-person level.ConclusionsOur findings highlighted the importance of the daily dyadic coping process among HIV serodiscordant couples. Future interventions should direct resources toward fostering a shared disease appraisal and training couples in common dyadic coping strategies for their daily lives.

PurposeTo examine global and health-related quality of life (QOL) among parents of individuals with Fontan physiology and determine associations with sociodemographic, parent and child-related health, psychological, and relational factors.MethodsParents participating in the Australian and New Zealand Fontan Registry (ANZFR) QOL Study (N = 151, Parent Mean age = 47.9 ± 10.2 years, age range: 31.6–79.6 years, 66% women; child Mean age = 16.3 ± 8.8, age range: 6.9–48.7 years, 40% female) completed a series of validated measures. Health-related QOL was assessed using the PedsQL 4.0 Core Generic Scales for adults and global QOL was assessed using a visual analogue scale (0–10).ResultsMost parents (81%) reported good global QOL (≥ 6), consistent with broader population trends. Nearly one-third of parents (28%) reported at-risk health-related QOL (based on total PedsQL scores) with physical functioning most affected (44%). Psychological factors, including psychological stress and sense of coherence, emerged as the strongest correlates of global and health-related QOL, explaining an additional 16 to 30% of the variance (using marginal R2). Final models explained 35 and 57% and of the variance in global and health-related QOL, respectively (marginal R2). Relational factors, including perceived social support and family functioning contributed minimally when analyzed alongside psychological variables.ConclusionWhile parents of individuals with Fontan physiology report good global QOL, challenges in health-related QOL exist. We identified key psychological, sociodemographic, and health-related factors associated with parental QOL outcomes. These data may aid early identification of physical and psychosocial difficulties and guide targeted health resource allocation for this population.

PurposeCost-utility analyses examining the value of new vaccines for pneumococcal disease will require health state utilities as inputs. Existing utilities for pneumococcal infections in young children are limited. The purpose of this study was to estimate health state utilities associated with pneumococcal infections in young children.MethodsSix health state vignettes depicting infections due to Streptococcus pneumoniae were drafted based on published literature and clinician interviews. To address methodological challenges in estimating utilities for temporary infections in children 0–5 years of age, several time trade-off approaches were explored in a pilot study (N = 28 participants). In the subsequent utility elicitation study conducted in the UK, health states were valued using the best performing method from the pilot (10-year time horizon, with infections repeated annually) with adult general population respondents imagining a child 2–5 years of age.ResultsA total of 208 participants completed interviews (51.9% female; mean [SD] age = 41.0 [14.9] years). Mean (SD) utilities were 0.902 (0.092) for pneumonia requiring hospitalization, 0.901 (0.087) for bacteremia, 0.894 (0.103) for recurrent acute otitis media (AOM), 0.882 (0.107) for recurrent AOM treated with pressure equalization tubes, 0.878 (0.109) for bacteremic pneumonia, and 0.809 (0.145) for meningitis.ConclusionLower health state utilities were associated with health states that had longer treatment periods, required more invasive treatment, and described more severe infections. Utilities from this study can be used in models examining cost-effectiveness of pneumococcal vaccines. These results have methodological implications for future research estimating utilities associated with temporary pediatric health conditions.

PurposeTo test the comprehensibility and feasibility of the modified brief pain inventory (mBPI) and Fear of Pain Questionnaire for Children short-form (FOPQ-C-SF) adapted for children and young people with cerebral palsy (CP), with diverse cognitive and communication abilities. Improving assessment of pain interference (mBPI) and pain-related fear (FOPQ-C-SF) in CP can enhance quality of life by increasing access to under-utilised interventions targeting pain-related physical disability and mental health.MethodsA convergent mixed methods approach was used. Twenty-two people (5–30 years) with CP completed the adapted mBPI and FOPQ-C-SF in a cognitive interview, administered by pen/paper or TalkingMats®, an evidence-based visual communication framework. Cognitive interviewing approaches were adapted to optimise participation and expression for diverse cognitive and communication abilities. Quantitative data were analysed to report tool administration times, overall completion rates and communication effectiveness. Qualitative data were analysed by content analysis to determine further changes required to the tools.ResultsMedian administration times were 6.2 min (IQR = 5.3–7.6) for mBPI and 4.1 min (IQR = 2.7–4.9) for FOPQ-C-SF. All completed the mBPI. Three did not complete the FOPQ-C-SF due to fatigue, challenging behaviour and parent recommendation. Ten minor changes were identified.ConclusionThe adapted mBPI and FOPQ-C-SF are likely comprehensible and feasible for children and young people with CP, including those with diverse cognitive and communication abilities. It is likely most children and young people with CP can effectively communicate responses to both tools. The adapted tools will now undergo further psychometric testing, prior to becoming freely available for clinical and research use.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-03981-4.

PurposeInterpretation of changes on the individual level is often based on minimally important differences (MIDs) developed on the group level. We investigated the impact of applying different group-level MIDs (anchor-based and 10-point MIDs) to determine health-related quality of life (HRQoL) changes in glioma patients. We further explored directions and magnitudes of these changes and their relationship to response formats and types of scale.MethodsWe included 92 glioma patients at least 18 years old from a previously conducted randomized prospective study. We calculated changes in HRQoL (EORTC QLQ-C30 and QLQ-BN20) at individual levels over a two-week period and used anchor-based and 10-point MIDs to estimate if change is clinically meaningful; thereafter, we explored the direction and magnitude of changes.ResultsBetween 8.8% and 66.3% of the patients had actual changes in estimated scales. While 16.3%-60.9% and 8.8%-59.8% of the patients changed to a clinically relevant extent using anchor-based and 10-point MIDs in any scale, respectively. Changes were mostly in the functional than symptom scales and mostly minor, i.e., changes between ‘not at all’ and ‘a little’ or ‘a little’ and ‘quite a bit.’Conclusion10-point compared to anchor-based MIDs underestimates clinically relevant changes. Therefore, the application of different MIDs to the same research question can lead to diverse result interpretations. As most changes were minor, it could be argued if these reflect actual relevant changes for an individual or that the current response scale lacks sufficient differentiating ability, warranting further research over the best method to evaluate individual-level changes.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-04029-3.

PurposeIncorporating PROMs into routine clinical care comes with numerous challenges, including pressure to move patients through office visits with brief, efficient assessments. The purpose of our study was to select and evaluate very short PROMIS screeners with full-bank simulations and predictive accuracy analysis.MethodsWe identified candidate pairs of items for PROMIS Anxiety, Depression, Pain Interference, Fatigue, and Physical Function, on the basis of frequently selected CAT items at both normal and clinical thresholds. We then simulated full-bank responses for 10,000 patients with a T-score mean of 50 in Fatigue, Depression, Pain Interference, and Anxiety, but 45 for Physical Function. We scored candidate 2-item short-form pairs, but also evaluated a 1-item screener and a 4-item fixed CAT for comparison purposes. Clinical thresholds were set at 1 SD in the direction of worse health; diagnostic values were computed based on agreement with full-bank T-scores. We aimed for sensitivity and positive predictive values (PPV) above 0.80, considering also diversity and redundancy of content.ResultsAverage sensitivity of our chosen 2-item pairs across the five domains was 0.84 (range 0.80-0.89), while the average PPV was 0.82 (0.80-0.85). While the 1-item screeners for Anxiety and Depression performed poorly, they met or approached our criteria for Pain Interference, Fatigue and Physical Function. ConclusionFive 2-item customized PROMIS forms are available for use in routine clinical care, such as distress screening in oncology. Our study provides a useful complement to psychometric criteria, helping to minimize misclassification of patients in need during screening. Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-04015-9.

IntroductionThe EuroQol health and well-being instrument (EQ-HWB™) measures quality of life (QoL) outcomes in health, public health, and social care settings. Its conceptual framework is rooted in QoL theory, a multidimensional concept encompassing social, psychological, and physical aspects influenced by cultural factors. The content validity of the EQ-HWB remains unexplored in China. This study addressed this gap through qualitative interviews with Chinese laypeople, uncovering their QoL conceptual framework and comparing it with the EQ-HWB’s to evaluate how well it captures its intended outcomes.MethodsQuota sampling recruited respondents from two regions in China, ensuring diversity in age, gender, education, health conditions, and caregiving experience. Semi-structured qualitative interviews were conducted, transcribed verbatim, and analyzed by two coders using a thematic framework approach. The coders refined codes through consensus, removed irrelevant ones based on set criteria, and organized the remaining codes into sub-themes and themes to develop a Chinese QoL conceptual framework. Lastly, this Chinese QoL framework was compared with the EQ-HWB conceptual framework.ResultsThirty respondents were recruited and interviewed, achieving data saturation in the last three interviews. From 221 initial codes, 187 were retained to develop a conceptual framework comprising eight themes: feeling and emotion, cognition, self-identity, coping, physical sensation, relationship, activity, and mindset. This framework largely aligned with the EQ-HWB conceptual framework, except for the absence of the ‘mindset’ theme.ConclusionThe conceptual framework of the EQ-HWB is well-represented within the QoL framework. The findings support the content validity of the EQ-HWB among laypeople in the Chinese context.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-04038-2.

PurposeLittle is known about the effectiveness of health behaviour intervention in improving health-related quality of life (HRQOL) despite HRQOL being an important outcome in Type 2 diabetes (T2DM) management. This study examined the effectiveness of a culturally appropriate health behavioural intervention in improving HRQOL of people with T2DM in Nepal.MethodsA cluster randomized controlled trial was conducted among 481 people with T2DM from 30 randomly selected healthcare facilities in Kavrepalanchok and Nuwakot districts. The intervention group received 12 culturally tailored and group-based intervention sessions for six months whereas, the control group received usual care. The primary outcomes of this study were changes in the EuroQOL (EQ5D-3L) index score and EuroQOL visual analogue scale (EQVAS) score between baseline and six-month post-intervention. The intervention effect was assessed using generalized estimating equation models.ResultsAt six-months post-intervention, there was a positive and statistically significant effect on EQVAS (β1 = 3.61, 95%CI: 0.05, 7.17) in the intervention group compared to control group. No statistically significant effect was observed in EQ5D-3L index score. A statistically significant increase in EQVAS score of 0.5 was observed per session attended by the study participants (β1 = 0.49, 95%CI: 0.01, 0.98).ConclusionThe health behaviour intervention led to improved HRQOL. An increase in the number of intervention sessions attended was significantly associated with higher EQVAS scores, emphasizing the need for longer-term and engaging interventions that are well adhered to. Longer term assessment of change in HRQOL outcomes are needed when actual changes in HRQOL are more likely to be observed.Trial registrationAustralia and New Zealand Clinical Trial Registry (ACTRN12621000531819).Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-03971-6.

PurposeRegularly collected patient-reported outcome measures (PROMs) can facilitate early symptom detection and improve health outcomes. This explorative analysis aimed to investigate PROM adherence and factors associated with daily PROM completion among pediatric cancer patients.MethodsWe analyzed data from a prospective study at the Medical University of Innsbruck in which pediatric patients with cancer treated with chemotherapy completed daily PROMs via a web-based portal (ePROtect). We analyzed the PROM adherence during their first year of treatment descriptively and using a linear mixed model to evaluate factors associated with PROM adherence.ResultsFifty patients (42% female) with a median age of 10.7 years (IQR 7.1–15.4) were included in this analysis (analysis period 05/2020 to 05/2023). The mean adherence was 48.7% (SD 27.2), with the highest adherence during the first 30 days (77.1%). Significant predictive factors for lower adherence included time in the program (logarithmic; β = -0.093, p < 0.001) and admission to the intensive care unit (β = -0.224, p < 0.001). In contrast, inpatient stays (β = 0.035, p = 0.014) and self-reported pain (β = 0.087, p = 0.002) were significant predictors for higher PROM adherence. Occurrences of adverse events were not significantly associated with adherence.DiscussionOur findings suggest that continuous daily symptom monitoring is feasible over extended periods. PROM adherence stabilized over time despite an initial drop, with higher participation observed during inpatient stays and in weeks with self-reported pain. Future research should explore and evaluate strategies to improve adherence.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11136-025-04002-0.