Gradient Boosted Tree Approaches for Mapping European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Onto 5-Level Version of EQ-5D Index for Patients With Cancer.

OBJECTIVES: This study aimed to develop direct and response mapping algorithms from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 onto the 5-level version of EQ-5D index based on the gradient boosted tree (GBT), a promising modern machine learning method. METHODS: We used the Quality of Life Mapping Algorithm for Cancer study data (903 observations from 903 patients) for training GBTs and testing their predictive performance. In the Quality of Life Mapping Algorithm for Cancer study, patients with advanced solid tumor were enrolled, and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 and 5-level version of EQ-5D were simultaneously evaluated. The Japanese value set was used for direct mapping, whereas the Japanese and US value sets were used for response mapping. We trained the GBTs in the training data set (80%) with cross-validation and tested the predictive performance measured by the root mean squared error (RMSE), mean absolute error (MAE), and mean error in the test data set (20%). RESULTS: The RMSE and MAE in the test data set were larger in the GBT approaches than in the previously developed regression-based approaches. The mean error in the test data set tended to be smaller in the GBT approaches than in the previously developed regression-based approaches. CONCLUSIONS: The predictive performances in the RMSE and MAE did not improve by the GBT approaches compared with regression approaches. The flexibility of the GBT approaches had the potential to reduce overprediction and underprediction in poor and good health, respectively. Further research is needed to establish the role of machine learning methods in mapping a nonpreference-based measure onto health utility.

Impacts of the preceding cancer-specific health-related quality of life instruments on the responses to the subsequent EQ-5D-5L.

BACKGROUND: In clinical studies, the EQ-5D-5L is often employed with disease-specific health-related quality of life instruments. The questions in the former are more general than the latter; however, it is known that responses to general questions can be influenced by preceding specific questions. Thus, the responses to the EQ-5D-5L have the possibility of being influenced by the preceding disease-specific health-related quality of life instruments. This may lead to bias in the cost-effectiveness analysis results. Therefore, this study aimed to evaluate the impact of the preceding cancer-specific health-related quality of life instruments on the EQ-5D-5L responses. METHODS: We prepared questionnaire booklets containing the EQ-5D-5L, the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30, and the Functional Assessment of Cancer Therapy General with different orders. Using a quasi-randomized design, they were distributed to the patients undergoing drug therapy for advanced cancer, who were classified into three groups: Groups 1, 2, and 3 (the EQ-5D-5L placed first, second, and last, respectively). We compared the EQ-5D-5L index and the missingness of EQ-5D-5L among the groups. RESULTS: The mean EQ-5D-5L index was 0.796, 0.760, and 0.789 for groups 1 (n = 300), 2 (n = 306), and 3 (n = 331), respectively. The difference between Groups 2 and 1 was - 0.036 (95% CI - 0.065 to - 0.007; p = 0.015). The proportion of patients with an incomplete EQ-5D-5L was 0.11, 0.11, and 0.05 for Groups 1, 2, and 3, respectively. The difference of the proportions between group 3 and 1 and between 3 and 2 was - 0.06 (95% CI - 0.10 to - 0.02; p = 0.003) and - 0.06 (95% CI - 0.10 to - 0.02; p = 0.003), respectively. CONCLUSIONS: Although the EQ-5D-5L index differed according to the instrument orders, the difference size would not be considerably larger than the minimally important difference. The patients tended to complete the EQ-5D-5L when they were placed at the end of the questionnaire.

Characteristics of Elderly Patients with Heart Failure and Impact on Activities of Daily Living: A Registry Report from Super-Aged Society.

BACKGROUND: To assess the health care burden of elderly patients with heart failure (HF) in an aging Japanese community-based hospital, we investigated the outcomes of cardiac rehabilitation. METHODS AND RESULTS: We enrolled all patients with HF aged ≥65 years admitted to 3 hospitals in the Niigata Prefecture. We prospectively collected data on their hospital stays and for 2 years postdischarge. The cohort comprised 617 patients (46.5% men; mean age 84.7 years), 76.2% of whom were aged ≥80 years. Among these patients, 15.6% were nursing home residents, 57.7% required long-term care insurance, only 37.6% could walk unaided at the time of admission, and 70.5% required cardiac rehabilitation; age had no significant rehabilitative effect on the degree of improvement in activities of daily living (ADLs). Two years postdischarge, all-cause mortality, and HF rehospitalization were 41.1% and 38.6%, respectively. The ADL score at discharge was an independent prognostic factor for mortality. The incidence of mortality and rehospitalization was lower in elderly patients with preserved ADLs at discharge. CONCLUSIONS: Elderly patients with HF in our super-aged society were mainly octogenarians who required disease management and personalized care support. Although their ADL scores increased with comprehensive cardiac rehabilitation, improved scores at discharge were closely associated with prognosis.

Japanese Population Norms of EQ-5D-5L and Health Utilities Index Mark 3: Disutility Catalog by Disease and Symptom in Community Settings.

OBJECTIVES: This study aimed to establish the Japanese population norms of the EQ-5D-5L and Health Utilities Index Mark 3 (HUI3) and estimate the disutility associated with diseases and symptoms. METHODS: We performed a door-to-door survey of the general population by random sampling. The planned sample size was 10 000 residents (age ≥16 years) of 334 districts in Japan. In addition to the EQ-5D-5L and HUI3 questionnaires, questions regarding demographic factors and self-reported main diseases and symptoms were asked. The EQ-5D-5L and HUI3 responses were converted to index values on the basis of Japanese value sets. Summary values by age and sex were calculated to obtain Japanese normative values. A multiple linear model was used to examine relationships between these values and diseases and symptoms. RESULTS: We collected 10 183 responses from 334 districts. The mean EQ-5D-5L index values were 0.821 (male) and 0.774 (female) in the age group of 80 to 89 years, which were lower compared with 0.978 (male) and 0.967 (female) in the age group of 16 to 19 years. Similar trends were observed for the HUI3 values. Age, sex, household income, and education level had a significant influence on the values of both instruments. When measured with the EQ-5D-5L, Parkinson disease, dementia, and stroke were associated with the largest disutility (>0.2), and the disutility for depression was approximately 0.18. In contrast, the HUI3 disutility values for Parkinson disease and dementia were approximately 0.4. CONCLUSIONS: This study established the Japanese population norms of the EQ-5D-5L and HUI3, which can be used in healthcare decision making and contribute to a more reliable analysis of economic evaluations.

Psychological impact of the state of emergency over COVID-19 for non-permanent workers: a Nationwide follow-up study in Japan.

BACKGROUND: The outbreak of COVID-19 has caused mental health problems and increased unemployment due to the economic recession. This survey aimed to assess the psychological impact of the state of emergency. We estimated changes in mental health, quality of life, and unemployment experience for general workers during the first COVID-19 outbreak in Japan. METHODS: We conducted a nationwide follow-up study. During the periods of March 26 to April 6, 2020 and June 26 to July 2, 2020, we used the internet to survey general workers aged 15 to 59 years in Japan. The questionnaire items covered employment status and socioeconomic factors, and we used the Center for Epidemiologic Studies Depression Scale (CES-D) and EQ-5D-5L to assess depression and health-related quality of life (HR-QOL), respectively. The differences in outcomes of permanent and non-permanent workers were analyzed using propensity score analysis. A multiple linear regression analysis was performed to examine the relationship between unemployment and CES-D scores. RESULTS: We included 2351 subjects in the analysis. Changes in both CES-D scores and utility were not significantly different between the two groups. However, a significant difference was found regarding the rate of unemployment, which was associated with higher CES-D scores. CONCLUSIONS: The present study demonstrated that the mental health of non-permanent workers was not negatively affected following the state of emergency due to COVID-19 in Japan. Unemployment is an important factor that influences the mental health of general workers.

The relationship between preference-based health-related quality of life and lifestyle behavior: a cross-sectional study on a community sample of adults who had undergone a health check-up.

BACKGROUND: Preference-based Health-Related Quality of Life (HRQL) is one of the most important indicators for calculating QALY (Quality-Adjusted Life Years) in a cost-effectiveness analysis. This study aimed to collect data on healthy individuals' HRQL based on the preferences of Japanese people who had undergone a comprehensive health check-up, and to examine the influence of relevant factors, such as blood biochemical data and lifestyle behavior. METHODS: We conducted a cross-sectional study targeting people who had undergone a comprehensive health check-up in 2015. Participants were asked to respond to a medical interview sheet. We then examined the utility value, as well as lifestyle habits such as alcohol intake, smoking, and exercise. HRQL was examined using EQ-5D-5L. Using a multiple regression analysis, we examined the influence of related factors, such as lifestyle and biochemical test data. RESULTS: We collected 2037 responses (mean age = 54.98 years; 55.0% female). The average preference-based health-related HRQL was 0.936 ± 0.087. A total of 1167 people (57.2%) responded that they were completely healthy. The biochemical test data that were recognized to correlate with HRQL were hemoglobin, total cholesterol, creatinine, all of which were weak (r = - 0.045-0.113). The results of multiple regression analysis showed that significant facts were: being female, age (≧70 year-old), drinking alcohol (sometimes), activity (very often), and lack of sleep. CONCLUSIONS: The HRQL of participants who had undergone a comprehensive health check-up was generally high, and only declined for those over 70 years of age. It is suggested that preference-based HRQL is related to physical activity, and that decrease of activity and lack of sleep leads to a decrease in HRQL.

Mapping EORTC QLQ-C30 and FACT-G onto EQ-5D-5L index for patients with cancer.

BACKGROUND: To develop direct and indirect (response) mapping algorithms from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and the Functional Assessment of Cancer Therapy General (FACT-G) onto the EQ-5D-5L index. METHODS: We conducted the QOL-MAC study where EQ-5D-5L, EORTC QLQ-C30, and FACT-G were cross-sectionally evaluated in patients receiving drug treatment for solid tumors in Japan. We developed direct and indirect mapping algorithms using 7 regression methods. Direct mapping was based on the Japanese value set. We evaluated the predictive performances based on root mean squared error (RMSE), mean absolute error, and correlation between the observed and predicted EQ-5D-5L indexes. RESULTS: Based on data from 903 and 908 patients for EORTC QLQ-C30 and FACT-G, respectively, we recommend two-part beta regression for direct mapping and ordinal logistic regression for indirect mapping for both EORTC QLQ-C30 and FACT-G. Cross-validated RMSE were 0.101 in the two methods for EORTC QLQ-C30, whereas they were 0.121 in two-part beta regression and 0.120 in ordinal logistic regression for FACT-G. The mean EQ-5D-5L index and cumulative distribution function simulated from the recommended mapping algorithms generally matched with the observed ones except for very good health (both source measures) and poor health (only FACT-G). CONCLUSIONS: The developed mapping algorithms can be used to generate the EQ-5D-5L index from EORTC QLQ-C30 or FACT-G in cost-effectiveness analyses, whose predictive performance would be similar to or better than those of previous algorithms.

Factors contributing to the ceiling effect of the EQ-5D-5L: an analysis of patients with prostate cancer judged "no-problems".

PURPOSE: The goal of the present study was to determine factors related to a ceiling effect (CE) on the EQ-5D-5L among Japanese patients with prostate cancer (PC). METHODS: An existent cross-sectional observational study dataset was used. Patients were ≥ 20 years of age and diagnosed with PC. For CE determinants on the EQ-5D-5L, we excluded possible "full-health" patients flagged by the EQ-VAS (score = 100) and/or FACT-P (score = 156) instruments. We then divided them into binary variables: A CE group (EQ-5D-5L score = 1) and others (< 1). The associations between CE, sociodemographic and medical characteristics, and FACT-P subscale scores were examined using a multivariate LASSO selection followed by a binomial logistic regression analysis performed to calculate odds ratios (ORs) and 95% confidence intervals (CIs). RESULTS: A total of 362 patients were analyzed. The LASSO selection variables, including all obtained variables, were as follows: age, palliative treatment, FACT-P physical well-being, and PC subscale score. Statistically significant variables predicting CE were palliative treatment (OR 0.23; 95% CI 0.09-0.60), physical well-being (OR 1.54; 95% CI 1.34-1.76), and PC subscale (OR 1.08; 95% CI 1.03-1.14). CONCLUSIONS: This study revealed that palliative treatment and two FACT-P physical well-being and PC subscale scores were positively related to CE on the EQ-5D-5L. To our knowledge, this is the first study to examine predictors of CE on the EQ-5D-5L. The present results may be helpful for facilitating the consideration of "bolt-on" studies from the standpoint of PC patients.

Availability of EuroQol-5-Dimensions-5-Level (EQ-5D-5L) as health-related QOL assessment for Japanese systemic sclerosis patients.

Objective: To assess the functional disability in Japanese patients with systemic sclerosis (SSc) using the EuroQol-5-Domain-5-Level health questionnaire (EQ-5D-5L), which was developed in Europe to demonstrate the cost utility of treatments for non-specific diseases.Methods: The EQ-5D-5L and Disability Index of the Health Assessment Questionnaire (HAQ-DI), which is a questionnaire for the quality of life for rheumatic diseases, were completed by 109 Japanese patients with SSc, and the clinical findings and laboratory data were collected at the same time.Results: There was a correlation between the EQ-5D-5L score and HAQ-DI score. The EQ-5D-5L index score was affected by the % of predicted vital capacity (%VC), pulmonary arterial hypertension, and renal crisis. The %VC and renal crisis were also indicated as factors reducing the quality of life in the HAQ-DI. There was no difference in the EQ-5D-5L score between the SSc subtypes or among autoantibodies.Conclusion: Our single-center study demonstrated the EQ-5D-5L to be a valuable assessment tool for functional disability in Japanese SSc patients, similarly to the disease specific HAQ-DI.

Health utility and health-related quality of life of Japanese prostate cancer patients according to progression status measured using EQ-5D-5L and FACT-P.

PURPOSE: To obtain health utility data to allow for cost-effectiveness analysis in groups stratified by disease progression along with health-related quality of life (HRQoL) information in Japanese prostate cancer (PC) patients. METHODS: In this cross-sectional observational study, EuroQol-5 Dimension- 5 Level (EQ-5D-5L), EuroQol Visual Analog Scale (EQ-VAS), and Functional Assessment of Cancer Therapy-Prostate (FACT-P) measures were used to examine utility, VAS scores, and disease-specific HRQoL, respectively. Scores obtained were statistically examined for the correlation among measures and domains. Parameter estimates of statistically significant factors were assessed using generalized linear models (GLM). RESULTS: A total of 380 patients stratified by their disease progression status were analyzed. The numbers (%) of patients in groups stratified as having localized (L), localized progression (LP), distant metastatic (DM), and DM-castration-resistant PC (CRPC) were 275 (72.4), 40 (10.5), 27 (7.1), and 38 (10.0), respectively. EQ-5D-5L mean (standard deviation, SD) scores of L, LP, DM, and DM-CRPC in study participants were 0.87 (0.15), 0.86 (0.15), 0.85 (0.18), and 0.84 (0.17), respectively. The mean (SD) scores assessed by EQ-5D-5L, EQ-VAS, and FACT-P instruments were 0.86 (0.16), 74.6 (16.8), and 110.8 (19.6), respectively. Utility scores correlated well with FACT-P scores. Eastern Cooperative Oncology Group performance status had significant influences on all instruments' scores. CONCLUSIONS: We obtained health utility and HRQoL scores of Japanese PC patients stratified by disease progression in detail. Our results will be useful for establishing cost-effectiveness analyses in Japanese PC settings.

Comparison of Value Set Based on DCE and/or TTO Data: Scoring for EQ-5D-5L Health States in Japan.

BACKGROUND: The valuation study of the five-level version of the EuroQol five-dimensional questionnaire (EQ-5D-5L) involved composite time trade-off (cTTO) and a discrete choice experiment (DCE). The DCE scores must be anchored to the quality-of-life scale from 0 (death) to 1 (full health). Nevertheless, the characteristics of the statistical methods used for converting the EQ-5D-5L DCE results by using TTO information are not yet clearly known. OBJECTIVES: To present the Japanese DCE value set of the EQ-5D-5L and compare three methods for converting latent DCE values. METHODS: The survey sampled the general population at five locations in Japan. 1098 respondents were stratified by age and sex. To obtain and compare the value sets of the EQ-5D-5L, the cTTO and DCE data were analyzed by a linear mixed model and conditional logit, respectively. The DCE scores were converted to the quality-of-life scale by anchoring to the worst state using cTTO, mapping DCE onto cTTO, and a hybrid model. RESULTS: The data from 1026 respondents were analyzed. All the coefficients in the cTTO and DCE value sets were consistent throughout all the analyses. Compared with the cTTO algorithm, the mapping and hybrid methods yielded very similar scoring coefficients. The hybrid model results, however, produced a lower root mean square error and fewer health states with errors exceeding 0.05 than did the other models. The DCE anchored to the worst state overestimated the cTTO scores of almost all the health states. CONCLUSIONS: Japanese value sets based on DCE were demonstrated. On comparing the observed cTTO scores, we found that the hybrid model was slightly superior to the simpler methods, including the TTO model.

Japanese population norms for preference-based measures: EQ-5D-3L, EQ-5D-5L, and SF-6D.

PURPOSE: The purpose of this study was to measure the population norms for the Japanese versions of preference-based measures (EQ-5D-3L, EQ-5D-5L, and SF-6D). We also considered the relations between QOL score in the general population and socio-demographic factors. METHODS: A total of 1143 adult respondents (aged ≥ 20 years) were randomly sampled from across Japan using data from the Basic Resident Register. The health status of each respondent was measured using the EQ-5D-3L, EQ-5D-5L, and SF-6D, and responses regarding socio-demographic data as well as subjective diseases and symptoms were obtained. The responses were converted to a QOL score using Japanese value sets. RESULTS: The percentages of respondents with full health scores were 68 % (EQ-5D-3L), 55 % (EQ-5D-5L), and 4 % (SF-6D). The QOL score measured using the SF-6D was significantly lower than those measured using either EQ-5D score. The QOL score was significantly lower among respondents over the age of 60 years, those who had a lower income, and those who had a shorter period of education. Intraclass correlation coefficient showed a poor agreement between the EQ-5D and SF-6D scores. The differences in QOL scores between respondents with and those without any disease were 0.064 for the EQ-5D-3L, 0.061 for the EQ-5D-5L, and 0.073 for the SF-6D; these differences are regarded as between-group minimal important differences in the general population. CONCLUSION: The Japanese population norms of three preference-based QOL measures were examined for the first time. Such information is useful for economic evaluations and research examining QOL score.

Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan.

BACKGROUND: Patients with Gaucher disease (GD), a rare lysosomal storage disorder, have reduced health-related quality of life (HRQOL). A patient-reported outcome measure (PROM) for HRQOL developed for type 1 GD (GD1) is not appropriate for patients with neuronopathic GD (nGD) types 2 (GD2) and 3 (GD3). In this study, we developed a new PROM for use in all GD types. We previously reported the qualitative analysis of interviews with Japanese patients with nGD, which was used to create nGD-specific PROM items. Here we evaluated the full PROM combining the type 1 questionnaire with the new nGD-specific items. METHODS: Patients with confirmed GD were recruited (Association of Gaucher Disease Patients in Japan or leading doctors) for pre-testing (May 2021) or the main survey (October-December 2021). The PROM had three parts: Parts 1 and 2 were translated into Japanese from the pre-existing GD1 PROM, whereas Part 3 was newly developed. Patients (or their caregivers, where necessary) completed the PROM questionnaire on paper and returned it by mail. Mean scores were determined overall and by GD type. Inter-item correlations, content consistency (Cronbach's alpha), and test-retest reliability (Cohen's kappa; main survey only, taken 2 weeks apart) were calculated. RESULTS: Sixteen patients (three with GD1; six with GD2; seven with GD3) and 33 patients (nine with GD1; 13 with GD2; 11 with GD3) participated in the pre-test and main survey, respectively. All GD2 patients and one-third (6/18) of GD3 patients required caregivers to complete the questionnaire. Mean scores indicated that the burden was highest in GD2 and lowest in GD1. In the main survey, internal consistency was high (Cronbach's alpha = 0.898 overall, 0.916 for Part 3), and test-retest reliability was high for Part 3 (kappa > 0.60 for 13/16 items) but low for Part 1 (kappa < 0.60 for 12/15 items). CONCLUSIONS: We have developed a flexible and reliable PROM that can be tailored for use in all types of GD and propose using Parts 1 and 2 for GD1, Parts 2 and 3 for GD2, and Parts 1, 2, and 3 for GD3.

Perspectives on Aging and Quality of Life.

The aging of the world's population and the health problems accompanying it are becoming increasingly severe. Healthcare policies in developed countries focus on how to prevent and treat diseases associated with aging and how to maintain quality of life. Typical age-related diseases include deafness, cataracts, osteoarthritis, chronic obstructive pulmonary disease, diabetes mellitus, and dementia. Although the mechanisms by which these diseases develop differ, they are all caused by the accumulation of molecular and cellular damage over time. In addition, age-related diseases can cause a decline in physical and mental functions and the ability to perform activities of daily living, as well as the loss of roles in society and a sense of fulfillment in life. Therefore, there is a need for treatment and measures to accurately grasp and maintain quality of life. This review aims to introduce areas and representative papers expected to be contributed to the special issue of "Aging and Quality of Life".

Preferences for Rehabilitation in Persons with a History of Stroke: A Discrete Choice Experiment.

Objective: To investigate the preferences of persons with a history of stroke for various attributes of rehabilitation using a discrete choice experiment (DCE). Design: Cross-sectional study. Setting: A web-based survey. Participants: A total of 600 adults with a history of stroke who were not asked whether or not they had participated in previous rehabilitation. Intervention: None. Main Outcome Measures: Preference weights by attribute ie, treatment time (30 minutes, one hour, one and a half hours), treatment content (walking exercises, activities of daily living; ADL exercises), priority treatment of paralyzed limbs (upper extremity, lower upper extremity), treatment location (hospital visit, home visit), therapist gender, and out-of-pocket costs for stroke rehabilitation using discrete choice experiment. Results: The most common self-reported diagnosis was cerebral infarction (408 patients, 68%). The mean age was 62.0 ± 9.8 years, and 515 (85.8%) were male. Of the five attributes, excluding out-of-pocket costs, the highest relative importance score was treatment location (0.331), followed by treatment time (0.304). Among the rehabilitation programs, the statistically significant coefficients calculated were one hour of therapy (0.173, 95% CI = 0.088-0.258), hospital visits (0.241, 95% CI = 0.180-0.303), and female therapists (0.186, 95% CI = 0.125-0. 247). No significant differences were obtained regarding the treatment contents or the paralyzed limb to be treated. Conclusion: A discrete choice experiment revealed that persons with a history of stroke prefer a one-hour hospital rehabilitation program with a female therapist, with cost being a major consideration for rehabilitation. The results of this study may provide useful information for rehabilitation professionals.

Patient-reported outcomes in patients with primary immunodeficiency diseases in Japan: baseline results from a prospective observational study.

Introduction: Primary immunodeficiency diseases (PIDs) are rare inherited diseases resulting in impaired immunity. People with PID experience lower health-related quality of life (HR-QOL) and disease-related burdens in daily activities. This ongoing, prospective observational study aims to evaluate disease activity, treatment status, treatment-related burden, daily activities, and HR-QOL in patients with PID in Japan over a 1-year period. In this interim report (database lock: July 29, 2022), we present baseline results. Methods: Participants were enrolled between November 2021 and May 2022; data were collected four times/year per participant until May 2023 using an online electronic patient-reported outcomes system. Patients with PID and healthy volunteers aged ≥12 years, residing in Japan, and with access to a smartphone were eligible. HR-QOL (primary endpoint) was assessed by the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) and the Medical Outcomes Study 36-Item Short Form Health Survey (SF-36). Work productivity was assessed by the Work Productivity and Activity Impairment (WPAI) Questionnaire. Other aspects of PID and burden were assessed with a new questionnaire developed in-house. The study is registered at the University hospital Medical Information Network clinical trials registry (UMIN000045622). Results: The full interim analysis set comprised 71 patients with PID and 47 healthy volunteers. The most common International Union of Immunological Societies PID category was primary antibody deficiency (56.3% of patients). Complications were common, especially recurrent respiratory tract infections (63.4%). Most patients with PID were treated with immunoglobulin replacement therapy (73.2%); 22.4% of these patients had serum immunoglobulin levels <700 mg/dL. Among patients who did not undergo hematopoietic cell transplantation, EQ-5D-5L (n=67) and SF-36 (n=59) Physical and Mental Component Summary scores were significantly lower than in healthy volunteers (p < 0.001). WPAI absenteeism, work productivity loss, and activity impairment scores were significantly lower in 42 working patients with PID than in 37 working healthy volunteers (p < 0.05). Other results indicated that patients with PID experience substantial burdens related to medical visits, expenses, work, and daily activities. Discussion: This interim analysis confirms that patients with PID in Japan have lower HR-QOL and work productivity compared with healthy individuals and experience substantial limitations and burdens in their daily lives.

Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study.

Background: Gaucher disease (GD), a rare lysosomal storage disorder, is associated with considerable patient and caregiver burden. We examined the applicability of existing caregiver questionnaires and assessed the level of burden in caregivers of patients with GD. Methods: This cross-sectional, non-interventional study was conducted in Japan. Caregivers of patients with confirmed GD (any type) were recruited (patient association group and referral) for pre-testing (May 2021) or the main survey (October-December 2021). Caregivers completed the Caregiver Impact Questionnaire (CIQ; 30 items) and Zarit Caregiver Burden Interview (ZBI; 22 items) on paper. Total CIQ and ZBI scores and subscores were determined overall and by GD type. Inter-item correlations and test-retest reliability (2 rounds, 2 weeks apart) were calculated. The relationship between caregiving duration and caregiver burden was also analyzed. Results: Nine caregivers (type 2 [GD2]: n = 6; type 3 [GD3]: n = 3) and 25 caregivers (type 1 [GD1]: n = 2; GD2: n = 17; GD3: n = 6) completed the pre-test and main survey, respectively. In the main survey, mean total CIQ score, all CIQ subscores (except emotional function), and total ZBI score were highest in caregivers of patients with GD2 compared with caregivers of patients with GD1/GD3. High test-retest reliability (Kappa >0.6) was observed for 15 CIQ items and 16 ZBI items. CIQ and ZBI scores appeared to be positively correlated with each other and negatively correlated with caregiving duration. Conclusions: The CIQ and ZBI are applicable, reliable measures to assess burden in caregivers of patients with GD in Japan. Caregiver burden was highest in caregivers of patients with GD2 and decreased with caregiving duration.

Changes in Physical and Mental Functions in Patients with Cardiovascular Disease During the First Two Waves of COVID-19 in Japan.

BACKGROUND AND OBJECTIVES: The goal of this study was to determine whether changes in life-space mobility and quality of life (QoL) recovered in patients with cardiovascular disease (CVD) during the first two waves of the pandemic of Coronavirus disease 2019 (COVID-19) in Japan. METHODS: We performed a follow-up survey in 20 CVD patients using the Life-Space Assessment (LSA) scale and the five-level EuroQoL five-dimensional questionnaire (EQ-5D-5L), Japanese version, at three time points: January-March 2020 (before the first wave of the COVID-19 pandemic), July 2020 (following the first wave of the pandemic), and November 2020 (following the second wave of the pandemic). RESULTS: The LSA score in November 2020 (median [interquartile range], 90 [83.5-100] points) did not recover from the July 2020 score (83 [76.5-93] points). However, the EQ-5D-5L QoL score in November 2020 (0.89 [0.82-1]) had improved from that in July 2020 (0.80 [0.71-0.87]). CONCLUSION: The QoL of CVD patients might have been more affected by psychological factors rather than physical factors during the first two waves of the COVID-19 pandemic in Japan.

Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan.

BACKGROUND: Gaucher disease (GD) is a rare, autosomal recessive lysosomal storage disorder that adversely affects life expectancy and health-related quality of life (HRQOL). Although HRQOL questionnaires are available for type 1 GD, they are not suitable for patients with the neuronopathic types 2 and 3 GD who have neurological symptoms that develop during early childhood or adolescence. Here we report the development of a language-validated HRQOL questionnaire specifically for patients with neuronopathic types 2 and 3 GD in Japan, which is the first step toward HRQOL questionnaire provision for all types of GD in the future. METHODS: In February and March 2021, semi-structured interviews were conducted by the authors (supported by qualified interviewers) with patients and/or their caregivers (for patients < 16 years old) who were recruited from a Japanese patient association, the Association of Gaucher Disease Patients in Japan. Qualitative analysis of interview transcripts was used to identify major themes and key topics within those themes. Hierarchical cluster analysis and co-occurrence network analysis were performed to map relationships between commonly occurring words. The study is registered at the UMIN Clinical Trials Registry ( https://www.umin.ac.jp/ctr/index.htm [UMIN000042872]). RESULTS: Three main themes emerged from qualitative analysis: treatment status, patient burden, and social support systems. Key topics within each theme included hearing impairment, visual impairment, difficulty swallowing, difficulty speaking, involuntary movement of extremities, epileptic seizures, and body aches (treatment status); anxiety about symptoms, difficulty with exercise and work, anxiety about continuing treatment, anxiety about going out, and tiredness from hospital visit or treatment (patient burden); and dissatisfaction about government service, lack of social support, and information exchange in the patient association (social support systems). Commonly used words and the relationships between words identified through the hierarchical cluster and co-occurrence network analyses supported these themes and topics. CONCLUSIONS: The themes and topics identified in this analysis were specific to patients with types 2 and 3 GD and will be used to inform the development of a HRQOL questionnaire specifically for patients with all GD types.

Health-related quality of life in patients with alopecia areata: Results of a Japanese survey with norm-based comparisons.

Alopecia areata (AA) is a non-scarring hair loss disorder affecting approximately 2% of the global population. AA is reported to have a significant negative impact on the emotional and psychological well-being of the patients. This study aimed to evaluate the health-related quality of life (HRQoL) of Japanese patients with AA in comparison to the Japanese population norms (national standard values for Japanese) using Short Form Health Survey 36 Item Version 2.0 (SF-36v2). The study also aimed to access the negative effect of AA on patients' daily lives and the proportion of patients having anxiety and/or depression. This cross-sectional, non-interventional web-based survey study included 400 participants aged 17-84 years currently suffering from medically diagnosed AA. The assessment tools integrated in the online questionnaire included SF-36v2, the Dermatology Life Quality Index (DLQI), and the Hospital Anxiety and Depression Scale (HADS). All outcome measures from the tools were evaluated across the study population. SF-36v2 subscale scores for patients with AA revealed lower scores specifically for mental health (45.7 ± 10.1 points), social functioning (45.8 ± 10.9 points), vitality (46.2 ± 9.8 points), and role emotional (46.9 ± 11.6 points) as compared to the Japanese population norms of 50 ± 10 points each. The DLQI questionnaire-based analysis indicated that 32.1% of respondents showed a moderate to extremely large effect on their lives; and HADS-A (anxiety) and HADS-D (depression) scores categorized 46.0% and 41.8% respondents as doubtful-to-definite cases, respectively. Multivariate linear regression revealed that hair loss range, age, comorbidities, and depression significantly worsened DLQI scores. In conclusion, the results of this survey demonstrated that a significant decrease in the HRQoL scores was observed in Japanese patients with AA in comparison with the national norms. Hence, emphasis on mental health is crucial for AA management.