Lower Healthcare Access and Its Association With Individual Factors and Health-Related Quality of Life in Adults With Rare Diseases in Switzerland.

Objectives: This study aims to determine clusters of access to healthcare among adults with rare diseases in Switzerland, identify associated individual characteristics of access, and impact on health-related quality of life (HRQoL). Methods: Swiss adults (N = 341) diagnosed with a rare disease completed an online survey including the Perception of Access to Healthcare Questionnaire (PAHQ) and Short Form Health Survey (SF-12). We employed partition around medoids algorithm to identify patient clusters based on the PAHQ. Various sociodemographic/disease-related factors and HRQoL were assessed. Results: We identified two patient clusters: higher (n = 227) and lower access (n = 114). Significantly associated with lower access were an unstable disease course (p < 0.05), increased number of misdiagnoses (p < 0.05), and diseases affecting the nervous system (p < 0.01). Membership in the lower access cluster was significantly associated with worse HRQoL (p < 0.05). Conclusion: Findings highlight the need for comprehensive assessment of healthcare access in adults with rare diseases and identifies potential targets for tailored interventions.

[Management of pediatric immune thrombocytopenia].

The new guidelines for pediatric immune thrombocytopenia (ITP) not only include changes to the name and staging of the disease, but also introduce the modified Buchanan's bleeding score for the assessment of bleeding symptoms. Treatments should aim to improve patients' health-related quality of life (HRQoL) based on a multidimensional assessment of not only platelet counts but also bleeding symptoms, as well as activity level, lifestyle, and access to healthcare. First-line therapy includes intravenous immunoglobulin therapy (IVIG) and short-term corticosteroids. Second-line therapy includes thrombopoietin receptor agonists, rituximab, and splenectomy. Many novel agents are also in development, with splenic-derived tyrosine kinase (Syk), Bruton's kinase (BTK), and fetal Fc receptor (FcRn) attracting attention as target molecules. Future developments in the treatment of pediatric ITP are eagerly awaited.

Risk and Protective Factors of Psychosocial Functioning in Survivors of Childhood Cancer: Results of the DCCSS-LATER Study.

OBJECTIVE: This study examines the association between psychosocial risk and protective factors and a wide range of psychosocial outcomes including emotional, social, cognitive, and physical domains in childhood cancer survivors (CCS). METHODS: CCS from the Dutch Childhood Cancer Survivor Study (DCCSS)-LATER cohort (diagnosed 1963-2001) part 2 (age ≥ 18 years, diagnosed < 18 years, ≥ 5 years since diagnosis) completed questionnaires on psychosocial risk and protective factors (Benefit and Burden Scale, Illness Cognition Questionnaire, Rosenberg Self-Esteem Scale, and Impact of Cancer Scale), and psychosocial outcomes (Hospital Anxiety and Depression Scale, Self-Rating Scale for Post-Traumatic Stress Disorder, TNO-AZL Questionnaire for Adult Health-Related Quality of Life, and Short Form-36). Associations were assessed with regression analysis, adjusting for attained age, sex, number of health conditions, and time since diagnosis, while correcting for multiple testing (p < 0.004). RESULTS: A total of 1382 CCS participated, all diagnosed ≥ 15 years ago. The mean age of participating CCS was 36 years, and 51% were female. Perceived benefit and burden, acceptance, and helplessness, self-esteem and social support were associated with the psychosocial outcomes. In the models including all psychosocial factors, most associations with psychosocial outcomes were seen for self-esteem (10×), and perceived burden (9×). Self-esteem (all ß ≤ 0.47) and perceived burden (all ß ≤ 0.38) demonstrated strongest associations of medium/large size. CONCLUSIONS: Perceptions of childhood cancer, illness cognitions, self-esteem, and social support play a role in explaining psychosocial functioning in CCS, outweighing the influence of socio-demographic and medical variables. Addressing negative perceptions and reducing feelings of helplessness, while promoting acceptance, self-esteem, and social support, could provide intervention targets for CCS who encounter psychosocial challenges.

Quality of life and functional outcome of rectal cancer patients: A prospective cohort study.

AIM: In the last decade, the Netherlands has implemented various diagnostic and treatment strategies to enhance rectal cancer outcomes. This study, using data from the Prospective Dutch ColoRectal Cancer (PLCRC) cohort, investigates whether these multidisciplinary advancements have translated into improved health-related quality of life (HRQoL) and functional outcomes for the general Dutch rectal cancer population. METHODS: Patients with Stage I-III rectal cancer enrolled in the PLCRC cohort were included. HRQoL and functional outcomes were assessed 1 and 2 years after diagnosis using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), EORTC QLQ Colorectal Cancer 29 and the Low Anterior Resection Syndrome score. HRQoL and functional outcomes were compared based on year of diagnosis (2014-2019). RESULTS: A total of 1294 patients were included. Two years after diagnosis, patients diagnosed in 2019 (n = 392) had a clinically relevant higher score on physical (8.2, 95% CI 4.1-12.3), role (13.5, 95% CI 7.3-19.7) and social functioning (5.8, 95% CI 0.3-11.2) compared to those diagnosed in 2014 (n = 65). Additionally, patients diagnosed in 2019 experienced less fatigue 2 years after diagnosis compared to those diagnosed in 2014 (-8.6, 95% CI -14.1 to -3.0). The Low Anterior Resection Syndrome score showed no differences. CONCLUSION: The findings of this study suggest that over the past decade rectal cancer patients in the Netherlands have witnessed improvements in HRQoL across various domains. Most probably, the improvement is due to a combination of implementation of population screening, a more restrictive neoadjuvant radiotherapy policy and advances in minimally invasive surgery and organ preserving treatment options.

The impact of negative COVID-19 experiences on cancer survivors' health-related quality of life and psychological distress: a moderated mediation model.

Introduction: Cancer survivors experienced poorer health-related quality of life (HRQoL) and greater psychological distress during the COVID-19 pandemic than those without cancer. However, the underlying mechanisms that may explain how negative experiences during the pandemic are associated with distress and HRQoL remain unknown. We examined whether psychosocial risk factors (i.e., healthcare disruption, disruption to daily activities and social interaction [DDASI], and financial hardship) mediated the relationship between negative COVID-19-related experiences and cancer survivors' HRQoL and psychological distress (i.e., depressive symptoms, and anxiety) and whether the mediating effects were moderated by psychosocial protective factors (i.e., stress management ability and social support). Methods: A total of 9,651 cancer survivors completed a questionnaire assessing negative COVID-19-related experiences, psychosocial and practical experiences, and HRQoL. Conditional process analysis was used to evaluate the proposed moderated mediation models. Results: Participants had a mean age of 63.8 years (SD = 12.3) and were mostly non-Hispanic White (82.3%). DDASI and financial hardship mediated the relationship between negative COVID-19-related experiences and cancer survivor's HRQoL and psychological distress. Stress management ability buffered the indirect effect of DDASI on cancer survivors' HRQoL and psychological distress. Social support buffered the indirect effect of financial hardship on HRQoL and depressive symptoms. Conclusion: Financial resources and social interactions may buffer negative effects of major disruptions such as the COVID-19 pandemic. Future studies should assess the longitudinal impact of these associations.

Reliability and validity of a Hindi version of the Oral Health Impact Profile (OHIP-EDENT-H) for edentulous subjects: A Cross-Sectional study.

Aim: The study aimed to translate the OHIP-EDENT into Hindi and assess its validity and reliability. Methods: The study included 150 participants whose demographic information was collected using the Modified Kuppuswamy Socio-economic Scale. The Oral Health Impact Profile in Edentulous (OHIP-EDENT) was translated into Hindi using the standard forward-backward method. Test-retest reliability was assessed using the Intra-class Correlation Coefficient (ICC) and internal consistency using Cronbach's alpha. The Kaiser-Meyer-Olkin (KMO) test and Bartlett's test of sphericity coefficient were used to conduct Exploratory Factor Analysis (EFA) and confirm the Construct validity. To establish Convergent validity, the relationship between the global question and the OHIP-EDENT-H subscale scores was observed. Results: The data was analyzed with a confidence level of 95 %, and statistical significance was interpreted as a p-value of less than 0.05. The Cronbach's alpha score for OHIP-EDENT-H was 1.00, indicating high internal consistency. The corrected item-total correlations ranged from 0.665 to 0.923, and the total ICC score was 0.763, demonstrating good reliability. The subscales' intra-class correlation coefficient values ranged from 0.968 to 0.997, indicating high reliability. However, items 4, 6, 13, 17, 18, and 19 had factor loadings below the acceptable threshold of 0.40 in the factor analysis. Additionally, the total and subscale scores of the OHIP-EDENT-H showed significant correlations with global question, with correlation coefficients ranging from 0.665 to 0.923. Conclusion: The Hindi version of OHIP-EDENT is a reliable and valid tool for evaluating the OHRQoL of Hindi-speaking edentulous individuals.

The 'AfterBurn' Longitudinal Exploratory (ABLE) study: Cohort profile.

With advanced medical treatments more burns patients survive their injuries, requiring a better understanding of the quality-of-life outcomes experienced after the burn. The aims of the ABLE study cohort profile are to describe the baseline characteristics. The study included participants aged 18 years or over, admitted to a single state-wide burns centre in Queensland, Australia with a 'major burn injury'. Baseline survey data were collected either in person or by telephone within 28 days of the injury and participants were followed up with in-person, or telephone interviews at 3-, 6- and 12-months post-burn. Injury and burns treatment information were collected from medical records or the hospital database and surveys collected demographic and psychosocial data. Health-Related Quality of Life (HRQoL) data were collected using the 12-item Short Form Survey - version 1 (SF-12v1) and the Burn Specific Health Scale - Brief version (BSHS-B). Of the 274 participants recruited, 71.5 % (N = 196) remained enrolled in the study at 12 months post-burn. The median age for the cohort was 41.5 years (range 18 to 87 years) and the majority were male (72 %) as well as non-Indigenous Australians (76 %). The median Total Body Surface Area (TBSA) burned was 4.75 % (IQR=1.5-10.13) with a range of 0.1 % to 79.75 %. The largest percentage of participants sustained a flame burn (N = 129, 47.1 %) followed by scald (N = 56, 20.4 %) then contact burn (N = 54, 19.7 %). Fewer participants were injured by chemical (N = 14, 5.1 %), friction (N = 11, 4 %), and electrical burns (N = 6, 2.2 %). There were 37.9 % participants who reported having been diagnosed with a psychological condition prior to their burn injury. These findings provide an overview of baseline characteristics with a greater understanding of personal, environmental, burn injury and burn treatment factors in a state-wide burn centre.

Health-related Quality of Life Assessment in Renal Cell Cancer: A Scoping Review.

BACKGROUND AND OBJECTIVE: In oncology, patient-reported outcome measures (PROMs) capturing health-related quality of life (HRQOL) play an increasing role in clinical trials, drug approval, and policy making. This scoping review aimed to identify and elaborate on HRQOL-focussed PROMs used in renal cell cancer (RCC) clinical trials. METHODS: MEDLINE, Web of Science, PsychINFO, Academic Search Elite, CINAHL, Embase, and the Cochrane Library were searched systematically for original peer-reviewed articles on clinical trials including RCC patients and using PROMs, published between 1950 and 2023. Prespecified trial characteristics and information on the PROMs used were extracted. Frequencies and proportions of categorical data, and ranges and medians of continuous variables were calculated. KEY FINDINGS AND LIMITATIONS: Of the 48 unique studies included, the majority followed a randomised controlled design (34, 71%) and evaluated systemic treatments (38, 79%). The trials used 27 different PROMs (max = 6, median = 2), of which only 4 (15%) were developed specifically for kidney cancer patients. Of the trials, 46% did not use any RCC-specific PROM. European Quality of Life-5 Dimensions (EQ-5D), European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30), Functional Assessment of Cancer Therapy Kidney Symptom Index (FKSI) -15/19-item version, FKSI-Disease Related Symptoms, and Functional Assessment of Cancer Therapy-General (FACT-G) were the most frequently used questionnaires, with pain, ability to work, fatigue, worry, and sleep quality being the most commonly assessed issues. CONCLUSIONS AND CLINICAL IMPLICATIONS: A variety of PROMs are used in RCC patients, hindering interpretability across trials. The PROMs used differ in terms of both the domains assessed and how the issues are translated into questionnaire items. Though RCC-specific PROMs exist, these have flaws in terms of relevance to patients. To answer predefined relevant HRQOL research questions, revised RCC-specific PROMs and standardisation of their integration into clinical trials are warranted. PATIENT SUMMARY: Researchers are more and more interested in the health-related quality of life of kidney cancer patients and use questionnaires to measure it. This review shows that there are many different health-related quality of life questionnaires that are used in different combinations in clinical trials for kidney cancer patients. This makes it very difficult to compare these study results and draw reliable conclusions for the actual clinical treatment. It was even found that some of the questionnaires used do not capture things that patients actually consider important (eg, emotional issues such as dealing with thoughts about cancer and depression). Therefore, more work needs to be done to develop questionnaires that ask what is really important to kidney cancer patients' health-related quality of life. If these questionnaires are used in a consistent way in clinical trials, the results can be better compared. This will help treat kidney cancer patients in the best possible way.

High-Frequency 10-kHz Spinal Cord Stimulation Provides Long-term (24-Month) Improvements in Diabetes-Related Pain and Quality of Life for Patients with Painful Diabetic Neuropathy.

BACKGROUND: The SENZA-PDN study evaluated high-frequency 10-kHz spinal cord stimulation (SCS) for the treatment of painful diabetic neuropathy (PDN). Over 24 months, 10-kHz SCS provided sustained pain relief and improved health-related quality of life. This report presents additional outcomes from the SENZA-PDN study, focusing on diabetes-related pain and quality of life outcomes. METHODS: The SENZA-PDN study randomized 216 participants with refractory PDN to receive either conventional medical management (CMM) or 10-kHz SCS plus CMM (10-kHz SCS + CMM), allowing crossover after six months if pain relief was insufficient. Postimplantation assessments at 24 months were completed by 142 participants with a permanent 10-kHz SCS implant, comprising 84 initial and 58 crossover recipients. Measures included the Brief Pain Inventory for Diabetic Peripheral Neuropathy (BPI-DPN), Diabetes-Related Quality of Life (DQOL), Global Assessment of Functioning (GAF), and treatment satisfaction. RESULTS: Over 24 months, 10-kHz SCS treatment significantly reduced pain severity by 66.9% (P < .001; BPI-DPN) and pain interference with mood and daily activities by 65.8% (P < .001; BPI-DPN). Significant improvements were also observed in overall DQOL score (P < .001) and GAF score (P < .001), and 91.5% of participants reported satisfaction with treatment. CONCLUSIONS: High-frequency 10-kHz SCS significantly decreased pain severity and provided additional clinically meaningful improvements in DQOL and overall functioning for patients with PDN. The robust and sustained benefits over 24 months, coupled with high participant satisfaction, highlight that 10-kHz SCS is an efficacious and comprehensive therapy for patients with PDN.

Breast-cancer specific comprehensive archive of Patient-Reported Outcome Measures (PROMs) for clinical research and clinical practice in oncology: Results from the PRO4All project.

BACKGROUND: Inclusion of patient-reported outcomes (PROs) in oncology clinical trials is strongly recommended. However, selecting the most appropriate patient-reported outcome measures (PROMs) is not easy. This study aimed to develop a breast cancer (BC) specific comprehensive archive of PROMs. METHODS: As part of the PRO4All project, we identified available PROMs in oncology by searching facit.org, eortc.org, eprovide.mapi-trust.org, PubMed, ema.europa.eu (European Public Assessment Reports) and published reviews. For this analysis, only BC tools were extracted. We described information about PROM name, type of questionnaire, questionnaire variant(s), recall period, number of items, and presence of minimum clinically important difference (MCID) reference in literature. Then, we assigned each item to a specific domain according to a predefined taxonomy of 38 items for outcome classification. RESULTS: We identified and analyzed 383 PROMs. Of these, 29 were BC specific, but 2 were excluded because the questionnaires description was not available. 6 (22.2 %) were variants of another questionnaire. All questionnaires were self-reported. In 6 cases (22.2 %) the recall period to consider was the "last week". The mean number of items per questionnaire was 25.81 (range 6-71). 602 items were assigned to an outcome domain: emotional functioning/wellbeing in 26.6 % of cases, physical functioning in 14.1 %, delivery of care in 10.8 %, and general outcomes in 10.5 %. MCID reference was found only in 4 (14.8 %) cases. CONCLUSIONS: The newly developed archive represents a useful tool to optimize the use of PROMs in the evaluation of treatments in BC patients, promoting a patient-centered approach both in clinical research and practice.

A national cross-sectional survey on community spinal cord injury individuals profiles, health-related quality of life and support services in China: implications for healthcare and rehabilitation.

BACKGROUND: Spinal cord injury (SCI) results in severe, permanent functional changes and has become a global health priority due to its high incidence, cost, and disability rate. Current national epidemiological data on SCI in China are limited and outdated. This study aimed to provide a comprehensive, national cross-sectional investigation of SCI epidemiology in China. METHODS: This cross-sectional study included 3055 SCI participants aged 8 to 78 years, conducted from May to September 2023. Data collected encompassed demographic characteristics, employment status, etiology, years lived with disability (YLD), family structure, caregiving status, income, health insurance, paralysis type, and health-related quality of life (HRQoL). Descriptive statistics analyses were used to assess demographic and injury characteristics. Group differences were assessed using t-tests, one-way ANOVA and Chi-square tests. Significant factors were examined using multivariate regression analysis. RESULTS: The majority (88.9%) of respondents were aged 15 to 59 years, with a male-to-female ratio of 2.36:1. Car accidents caused 45.4% of tetraplegia cases, falls caused 35.9% of paraplegia cases, and myelitis was the leading cause of non-traumatic SCI. Among paraplegia participants, 65.5% had complete SCI, while 53.1% of tetraplegia participants had incomplete SCI. Functional improvement was reported by 9.58% of participants. Half (50.3%) of the respondents were unemployed, and 75% had incomes below the national average. HRQoL was significantly lower in the SCI population compared to controls, mainly influenced by injury site, income, age and etiology (p < 0.05). CONCLUSIONS: SCI participants in China exhibit low HRQoL and reemployment rates. Accessible community and vocational rehabilitation programs, alongside robust public medical services, are essential for enhancing reemployment and HRQoL among SCI participants, reducing the overall disease burden.

Health-related quality of life in adults with Marfan syndrome.

BACKGROUND AND AIM: Marfan syndrome is a rare genetic connective tissue disorder. Research on health-related quality of life in Swedish patients is lacking. We aimed to examine health-related quality of life in patients with Marfan syndrome with respect to reference values, sex, and age. METHODS: Using the registry for adult CHD, Sahlgrenska University Hospital/Östra Hospital, between 1 April 2009 and 31 January 2023, we identified 1916 patients. Of these, we included 33 patients aged ≥18 years who were diagnosed with Marfan syndrome and had completed the 36-item Short-Form Health Survey. RESULTS: The median age was 32 years (interquartile range 25.5-47.0) and 22 (66.7%) were men. Patients with Marfan syndrome had significantly lower values than reference values for all scales in the Short-Form Health Survey except bodily pain, role-emotional, and the physical component summary score. For both men and women with Marfan syndrome, vitality was the subscale with the greatest percentage difference in comparison with healthy reference values (82% in women and 73% in men). Furthermore, men reported significantly higher vitality levels than women (62.5 points, interquartile range 43.8-75.0 vs. 35 points, interquartile range 10.0-65.0, p = 0.026). CONCLUSION: Adults with Marfan syndrome in Sweden showed lower health-related quality of life levels in comparison with reference values for most Short-Form Health Survey scales, and there were differences between patients with Marfan syndrome in terms of sex and age.

Oral health-related quality of life among women with temporomandibular disorders and hypermobile Ehlers-Danlos syndrome or hypermobility spectrum disorder.

BACKGROUND: People with hypermobile Ehlers-Danlos syndrome (hEDS) or hypermobility spectrum disorders (HSDs) are at greater risk of developing temporomandibular disorders (TMDs), perhaps due to the general joint hypermobility. There is, however, no information on how oral health-related quality of life (OHRQoL) is affected in people with hEDS or HSD with TMD. The authors' aim was to assess OHRQoL via the 14-item, short version Oral Health Impact Profile (OHIP-14), as well as associated risk factors in women with TMD symptoms and confirmed hEDS or HSD. METHODS: A digital questionnaire was sent to members of The Swedish National EDS Association who reported having a confirmed or suspected EDS or HSD diagnosis in the health care system from January through March 2022. Then, a sample of 133 women with confirmed hEDS or HSD and TMD symptoms was constructed, and information on the following variables was collected: TMD symptoms, age, general health, oral health-related factors, comorbid symptoms, and psychological factors. Linear regression analysis was conducted to investigate the association between these variables and the OHIP-14 summary score as the outcome. RESULTS: Most participants reported TMD pain symptoms (93.9%), temporomandibular joint clicking (89.5%), and crepitation (55.6%). The mean (SD) total OHIP-14 summary score was 21.0 (13.2). Oral function had the lowest impact (2.0 [2.4]) and orofacial pain had the highest impact on OHRQoL (3.9 [2.5]). Self-reported bruxism, poor general health, and comorbid symptoms were significantly associated with impaired OHRQoL. CONCLUSIONS: Women with confirmed hEDS or HSD and TMD symptoms have a considerably impaired OHRQoL. PRACTICAL IMPLICATIONS: The multidimensional phenomenon of OHRQoL in this group needs to be considered in management strategies.

Socio-economic inequalities in health-related quality of life and the contribution of cognitive impairment in Australia: A decomposition analysis.

BACKGROUND: The distributional effects of cognitive impairment on inequalities in health-related quality of life (HRQoL) are not well studied. This relationship has not been studied in any Australian health inequality literature. Therefore, this study aims to examine how cognitive impairment affects the distribution of HRQoL across various socio-economic classes amongst older Australians. METHODS: Data for this study was collected from the Household, Income and Labour Dynamics in Australia (HILDA) survey. The final analysis consisted of 5,247 and 5,614 unique individuals from wave 2012 and wave 2016, respectively. An ordinary least squares (OLS) regression model was used to investigate the relationship between cognitive impairment and HRQoL. Additionally, the Wagstaff-Doorslaer-Watanabe standard concentration index was used to examine socioeconomic inequality in HRQoL. RESULTS: The findings revealed pro-rich inequalities in HRQoL, as indicated by the concentration indices of 0.029 and 0.025 for wave 12 and wave 16, respectively. Additionally, the results showed that mild cognitive impairment accounted for 7.60% and 9.03%, respectively, of pro-rich socioeconomic inequality in HRQoL in 2012 and 2016. CONCLUSION: People from lower socioeconomic status (SES) groups tend to have lower HRQoL compared to those from higher SES. This leads to a greater disparity in HRQoL based on SES. Cognitive impairment positively contributed to this inequality in HRQoL. Therefore, it is critical to incorporate cognitive impairment into the design of interventions to reduce socioeconomic inequality in HRQoL.

Immediate Versus Delayed Exercise on Health-related Quality of Life in Patients Initiating Androgen Deprivation Therapy: Results from a Year-long Randomised Trial.

BACKGROUND AND OBJECTIVE: An array of treatment-related toxicities result from androgen deprivation therapy (ADT) in patients with prostate cancer (PCa), compromising function and health-related quality of life (HRQoL). Exercise has been demonstrated to counter a number of these adverse effects including decreased HRQoL; however, when exercise should be initiated is less clear. This study aims to examine whether commencing exercise when ADT is initiated rather than later during treatment is more effective in countering adverse effects on HRQoL. METHODS: Men with PCa (48-84 yr) initiating ADT were randomised to immediate exercise (IMEX; n = 54) or delayed exercise (DEL; n = 48) for 12 mo. IMEX consisted of 6 mo of supervised resistance/aerobic/impact exercise commenced at the initiation of ADT with 6 mo of follow-up. DEL consisted of 6 mo of usual care followed by 6 mo of the same exercise programme. HRQoL was assessed using the Short Form-36 at baseline and 6 and 12 mo. Intention to treat was utilised for the analyses that included group × time repeated-measures analysis of variance using log transformed data. KEY FINDINGS AND LIMITATIONS: There were a significant group × time interaction for the physical functioning domain (p = 0.045) and physical component summary score (p = 0.005), and a significant time effect for bodily pain (p < 0.001) and vitality domains (p < 0.001), with HRQoL maintained in IMEX and declining in DEL at 6 mo. Exercise in DEL reversed declines in vitality and in the physical component summary score, with no differences at 12 mo compared with baseline. Limitations include treatment alterations during the intervention. CONCLUSIONS AND CLINICAL IMPLICATIONS: Concurrently initiating exercise and ADT in patients with PCa preserves HRQoL, whereas exercise initiated while on established ADT regimens reverses declines in some HRQoL domains. PATIENT SUMMARY: To avoid initial treatment-related adverse effects on health-related quality of life, exercise medicine should be initiated at the start of treatment.

Huntington Disease Health Related Quality of Life, Function and Well Being: The Patient's Perspective.

BACKGROUND: Limited information is available on patients' experience living with Huntington's disease (HD). The primary objective of this study was to assess the health-related quality of life and well being of patients with HD. METHODS: A non-interventional, cross-sectional study was conducted in 17 hospitals-based movement disorders units in Spain. Patients aged ≥ 18 years, genetically HD diagnosed [with a diagnostic confidence level score of 4, and an Independence Scale (IS) score ≥ 70] were included. The primary variables were the Huntington's Disease Health-related Quality of Life (HDQLIFE) scores and results of the Satisfaction with Life Scale (SWLS). Secondary outcomes include the Unified HD Rating Scale (UHDRS), Beck Hopelessness Scale (BHS), Stigma Scale for Chronic Illness (SSCI-8), Beck Depression Inventory-Fast Screen (BDI-FS) and Problem Behaviours Assessment for HD short Version (PBA-S). RESULTS: A total of 102 patients were included. The mean age (SD) was 53.1 (12.1) years and 56% were male. Most of the patients (99.0%) showed motor symptoms (87.3%), behavioural and psychiatric disturbances (59.8%), or cognitive impairment (20.6%). HDQLIFE domain score means (SD) includes concern with death and dying 45.97 (9.60) end-of-life planning 37.91 (8.84), and meaning and purpose 44.74 (9.05). SWLS score mean was 24.25 (7.33). Depressive symptoms were found in 37.4% of patients and moderate-to-severe feelings of hopelessness in 32.9%. The prevalence of stigma was 55.9% (n = 57). CONCLUSION: HD impacted quality of life, with prevalent motor, psychiatric symptoms and cognitive impairment. Patient perspectives may provide complementary information to implement specific interventions.

Health utilities of patients with epilepsy in a Canadian population.

OBJECTIVE: Health state utilities are required to obtain quality adjusted life years, a common metric that informs clinical decision-making at individual, group, and health policy levels. Health state utilities are different from health-related quality of life, and their distribution across patients with epilepsy, as well as the factors that impact them, have not been studied in depth. We aimed to describe the distribution of health state utilities in people with epilepsy and the impact of different combinations of clinical and demographic factors on health state evaluation. METHODS: We performed a retrospective analysis of patients' data prospectively collected in the Calgary Comprehensive Epilepsy Program registry. Patient-reported health state utilities were measured using the 5-level EuroQol 5-Dimension scale (EQ-5D-5L) completed at their initial assessment. EQ-5D-5L index scores were derived via the time trade-off approach based on Canadian norms, and their distribution across different health states and patient characteristics was obtained. The Tobit regression model was used to evaluate the determinants of EQ-5D-5L index scores. RESULTS: Of 1446 patients included in this analysis, 724 (50.5%) were female. The median (interquartile range) Canada-normed EQ-5D-5L index score was .87 (.71-.91). Patients with significantly lower health utilities were more likely to be female (p = .008), to be older (p = .034), to be unmarried (p = .013), to have failed to achieve 1-year seizure freedom (p < .001), to have no postsecondary education (p = .028), to be depressed (p < .001), to have antiseizure medication side effects (p = .001), to be unemployed (p < .001), and to be unable to drive (p < .001). A look-up table of health utilities based on combinations of clinical-demographic characteristics was produced. SIGNIFICANCE: Health utility estimates for combinations of different health states in people with epilepsy attending specialty clinics are now available. These can help guide clinical decision-making in routine clinical practice, economic evaluations of treatment interventions, and health care policies.

The Development and Content Validation of the Sjögren's Related Quality of Life Instrument (SRQoL).

INTRODUCTION: Several clinical outcome assessment (COA) instruments assess Sjögren's disease (Sjögren's) symptoms, but do not provide comprehensive assessment of the health-related quality of life (HRQoL) impact of Sjögren's. This study aimed to develop a patient-reported outcome (PRO) instrument for the assessment of HRQoL, intended for use in clinical trials and clinical practice in the assessment of treatment benefit. METHODS: Review of study sponsor proprietary data and qualitative interviews informed the development of a conceptual model, the Sjögren's Related Quality of Life (SRQoL) and patient global impression of severity (PGI-S) and change (PGI-C) items. Combined concept elicitation and cognitive debriefing interviews with patients with Sjögren's explored their HRQoL impact experience and content validity of the SRQoL and PGI items. RESULTS: Twenty participants were interviewed about their Sjögren's experience. Following inductive analysis of interviews, concepts were categorized into eight domains: emotional well-being (e.g., worry and stress; n = 20/20; 100%), sleep (e.g., daytime sleepiness and waking up during the night; n = 20/20; 100%), activities of daily living (e.g., difficulty looking at screens and difficulty driving; n = 20/20; 100%), cognition (e.g., concentration difficulties and word finding difficulties; n = 19/20; 95.0%), physical functioning (e.g., difficulty walking and difficulty exercising; n = 19/20; 95.0%), social and family functioning (e.g., dependent on others and relationship difficulties; n = 17/20; 85.0%), work (n = 15/20; 75.0%), and sexual functioning (n = 12/20; 60.0%). SRQoL and PGI items, instructions, response options, and recall period were well understood and relevant to participants. CONCLUSIONS: The SRQoL is a new PRO instrument to assess Sjögren's impact on HRQoL, developed in accordance with regulatory guidance. This study provides considerable insight into the patient experience of Sjögren's and evidence to support the content validity of the SRQoL. Future research should evaluate the psychometric properties of the SRQoL to support its use in clinical trials and clinical practice and further validate its use as an assessment of treatment benefit.

Patient and physician completed questionnaires used in clinical trials and clinical practice have previously focused on symptoms. These questionnaires do not cover the full experience of living with Sjögren's, such as differences in symptoms between individual patients, and their use has also been known to result in inaccurate reports. Another way of thinking about patients' experiences is to use a questionnaire to ask about the impact Sjögren's has on patients' health-related quality of life, meaning how Sjögren's might affect their life in different ways including physically, emotionally, and socially. The goal of this study was to develop a new questionnaire (named the Sjögren's-Related Quality of Life [SRQoL] questionnaire) to measure the impact that Sjögren's has on patients' lives. Interviews were conducted with people with Sjögren's to explore their understanding of the new questionnaire and how relevant the content is to their experience of the condition. The results of this study suggest that the SRQoL includes relevant and easy-to-understand questions, suitable for use in future clinical trials and clinical practice, to measure if patients' HRQoL improves with treatment. Future research is needed to confirm that the questionnaire accurately measures the impact of Sjögren's on HRQoL for use with patients with Sjögren's.

The use of health utility in cost-utility analysis: A systematic review in substance use disorders.

BACKGROUND AND AIM: We aim to identify within-trial and modelled Cost-Utility Analysis (CUA) in substance use disorders (SUD) and review the applicability assessment associated with health utility used in modelled CUA. STUDY DESIGN AND METHODS: We searched Medline, Embase, EconLit and the Pharmaceutical Benefits Advisory Committee (PBAC) databases. A global systematic literature search was undertaken to determine the CUA of SUD interventions. Key characteristics of the studies and use of health utility were described. The applicability assessment associated with health utility used in modelled CUA was reviewed using The Health Utility Application Tool (HAT). RESULTS: The final review retrieved 49 CUA (14 within-trial and 35 modelled CUA). Three major health utility measurements were used - standard gamble, EQ-5D-5L and SF-6D. EQ-5D-5L was mainly used in within-trial CUA, whereas standard gamble, EQ-5D-5L and SF-6D were equally cited in modelled CUA and within-trial CUA. Twenty-nine articles using modelled CUA citing health utilities from published literature were assessed. Only half and one-third of CUA studies described the type of quality-of-life measure and value sets used in health utility studies, respectively. Only two-thirds showed the authors addressed questions about the similarities in clinical conditions, and health state description between health utility studies and economic evaluation studies. CONCLUSION: Justifications for chosen health utilities in modelled CUA studies were mostly absent in SUD. We suggested health economists use the HAT to make judgements when assessing health utility from published estimates. The use of this tool will increase the reliability of economic evaluation carried out to assist government and policymakers in making informed decisions around health topics.

Hindi Translation of Oral Health-Related Quality of Life (OHRQoL) Questionnaire and its Validation for Oral Submucous Fibrosis Patients: A Pilot Study.

Background: Oral Submucous fibrosis has a substantial burden on the Indian population; thus there is a substantial need for a validated questionnaire assessing QoL in Hindi. Thus, a prospective cross-sectional study was designed to translate and validate the English version of the OHR QoL-OSF questionnaire into Hindi. Methodology: We followed the guidelines suggested by the European Organisation for Research and Treatment of Cancer (EORTC) for the translation procedure and subsequently validated the translated questionnaire with reliability and validity. Results: A total of 50 patients were included in this pilot study. Cronbach's alpha for 17 items was 0.87 signifying high reliability. All questions had an I-CVI of 1.00, demonstrating complete agreement among the content experts regarding the relevance and clarity of items in the questionnaire. Convergent validity was adequately established for all factors except for factor 3 for which it was around 0.43. The intraclass coefficient was 0.803 showing good agreement between the test-retest results. Conclusion: The Hindi version of the OHR QoL questionnaire is a reliable instrument having good reliability, good content, good acceptability, and good responsiveness, and thus appears to be a valid instrument. Large studies with large sample sizes can be conducted in the future. Supplementary Information: The online version contains supplementary material available at 10.1007/s12663-023-01927-2.