Japanese value set for the EORTC QLU-C10D: A multi-attribute utility instrument based on the EORTC QLQ-C30 cancer-specific quality-of-life questionnaire.

Purpose: This study aimed to develop a Japanese value set for the EORTC QLU-C10D, a multi-attribute utility measure derived from the cancer-specific health-related quality-of-life (HRQL) questionnaire, the EORTC QLQ-C30. The QLU-C10D contains ten HRQL dimensions: physical, role, social and emotional functioning, pain, fatigue, sleep, appetite, nausea, and bowel problems.

Methods: Quota sampling of a Japanese online panel was used to achieve representativeness of the Japanese general population by sex and age (≥ 18 years).

How to select and understand guidelines for patient-reported outcomes: a scoping review of existing guidance.

Background: Over the past few decades, patient-reported outcomes (PROs) have been used to understand patient health conditions better. Therefore, numerous PRO measures (questionnaires) and guidelines or guidance have been developed. However, it is challenging to select target guidance from among the many available guidance and to understand the chosen guidance.

Perceptions regarding the concept and definition of patient-reported outcomes among healthcare stakeholders in Japan with relation to quality of life: a cross-sectional study.

Patient-reported outcomes (PROs) are frequently used in a variety of settings, including clinical trials and clinical practice. The definition of PRO and quality of life (QOL) and their relationship have been concluded through discussions among experts that has been the premise of PRO guidelines are not clearly stated in the guidelines. Therefore, the definition of PRO, especially in relation to QOL, is sometimes explained simply, as "PRO includes QOL," but this complicated matters.

Cost-Effectiveness Analysis of Nivolumab Plus Ipilimumab Combination Therapy as First-line Treatment for Advanced Renal Cell Carcinoma in Japan.

Objectives: The purpose of this study is to examine the cost-effectiveness of nivolumab (NIVO) plus ipilimumab (IPI) combination therapy (NIVO + IPI) compared with the sunitinib (SUN) therapy for Japanese patients with advanced renal cell carcinoma from the perspective of a Japanese health insurance payer.

Methods: A lifetime horizon was applied, and 2% per annum was set as the discount rate. The threshold was set as $ 75 000 per quality-adjusted life-year (QALY) gained.

Cost-effectiveness analysis of nivolumab plus chemotherapy vs chemotherapy for patients with unresectable advanced or metastatic HER2-negative gastric or gastroesophageal junction or esophageal adenocarcinoma in Japan.

Background: This study aimed to evaluate the cost-effectiveness of nivolumab plus chemotherapy (NIVO + Chemo) compared with chemotherapy monotherapy (Chemo) for patients with advanced or metastatic HER2-negative gastric or gastroesophageal junction or esophageal adenocarcinoma (GC/GEJC/EAC) in Japan from the perspective of healthcare payer.

Methods: A partitioned survival analysis model was developed to predict costs and quality-adjusted life years (QALYs) for NIVO + Chemo and Chemo. The time horizon of the model was set to 38 years.

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.

Cost-Effectiveness Analysis of Universal Screening for Biliary Atresia in Japan.

Objective: To evaluate the cost-effectiveness of universal newborn screening using stool color card or direct bilirubin (DB) testing when comparing with no screening for biliary atresia in Japanese setting.

Study Design: A decision analytic Markov microsimulation model was developed to evaluate the universal screening for biliary atresia. Our screening strategies included stool color card, DB, or no screening.

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.

Cost-Effectiveness of First-Line Nivolumab Plus Ipilimumab Combination Therapy in Advanced Non-Small-Cell Lung Cancer in Japan.

Background And Objective: In Japan, indications for nivolumab have been expanded to include the combination therapy with ipilimumab in various cancers. This study aimed to evaluate the cost-effectiveness of combination therapy of nivolumab plus ipilimumab (NIV + IPI) for patients with advanced non-small-cell lung cancer (NSCLC), comparing it with platinum-doublet chemotherapy in Japanese settings.

Methods: A partitioned survival model was developed to predict costs and quality-adjusted life-years (QALYs) in a NIV + IPI arm and a chemotherapy arm.

Cost-Effectiveness of Trastuzumab With or Without Chemotherapy as Adjuvant Therapy in HER2-Positive Elderly Breast Cancer Patients: A Randomized, Open-Label Clinical Trial, the RESPECT Trial.

Background And Objective: Trastuzumab is a standard care as adjuvant chemotherapy (AdjCT) for patients with human epidermal growth factor receptor 2 (HER2)-positive primary breast cancer (BC) in Japan. However, no reports have evaluated its economics for patients with HER2-positive BC over 70 years of age. The objective of this study was to evaluate the cost-effectiveness of HER2-targeted trastuzumab + chemotherapy in Japan, comparing it with trastuzumab monotherapy.

Direct health care cost of treatment and medication of biliary atresia patients using the National Database of Health Insurance Claims and Specific Health Checkups.

Background: Treatment of biliary atresia (BA), which typically requires an initial surgical intervention called the Kasai procedure (KP) and possible liver transplant (LT) afterwards, is quite resource-intensive and would affect patients and families for a lifetime; yet a comprehensive view of the economic burden has not been reported. We estimated direct health care costs from the public payer perspective using the National Database of Health Insurance Claims and Specific Health Checkups of Japan.

Methods: Children newly diagnosed at ages 0 days to 4 years between April 2010 and September 2019 were identified.

Cost-Effectiveness of Nab-Paclitaxel and Gemcitabine Versus Gemcitabine Monotherapy for Patients with Unresectable Metastatic Pancreatic Cancer in Japan.

Objectives: The purpose of this study was to evaluate the cost-effectiveness of nab-paclitaxel and gemcitabine (GnP) compared with gemcitabine monotherapy (G) for patients with unresectable metastatic pancreatic cancer in Japan from the perspective of healthcare payer.

Methods: A partitioned survival analysis model was developed to predict costs and quality-adjusted life years (QALYs) for GnP and G. The time horizon of the model was set at 20 years.

Impact of chemotherapy on cognitive functioning in older patients with HER2-positive breast cancer: a sub-study in the RESPECT trial.

Purpose: To investigate whether postoperative adjuvant trastuzumab plus chemotherapy negatively affected cognitive functioning during the post-chemotherapy period compared with trastuzumab monotherapy in older patients with HER2-positive breast cancer.

Methods: In the randomized RESPECT trial, women aged between 70 and 80 years with HER2-positive, stage I to IIIA invasive breast cancer who underwent curative operation were randomly assigned to receive either 1-year trastuzumab monotherapy or 1-year trastuzumab plus chemotherapy. Cognitive functioning was assessed using the Mini-Mental State Examination (MMSE) test at enrollment and 1 and 3 years after initiation of the protocol treatment.

Economic Evaluation of First-Line Pertuzumab Therapy in Patients with HER2-Positive Metastatic Breast Cancer in Japan.

Objective: The purpose of this analysis was to evaluate the cost effectiveness of the combination of pertuzumab, trastuzumab, and docetaxel (PTD) for the treatment of patients with human epidermal growth factor receptor-2 (HER2)-positive breast cancer in Japan.

Methods: A partitioned survival analysis model was developed to predict costs and quality-adjusted life-years (QALYs) in a PTD arm and a trastuzumab plus docetaxel (TD) arm. Direct medical costs were considered from the perspective of the Japanese healthcare system.

Response Shift-Adjusted Treatment Effect on Health-Related Quality of Life in a Randomized Controlled Trial of Taxane Versus S-1 for Metastatic Breast Cancer: Structural Equation Modeling.

Objective: We investigated the quantification of the response shift-adjusted treatment effect on quality-of-life (QOL) data in a randomized controlled trial of taxane versus S-1 for patients with metastatic breast cancer (SELECT-BC).

Methods: This study was a secondary data analysis of a previously published trial. The response shift-adjusted treatment effect on health-related QOL (HRQOL) data measured by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) was estimated using structural equation modeling techniques in addition to quantifying the "true" treatment effect.

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.

[Breast Cancer].

Patients' quality of life(QOL)and/or patient-reported outcomes(PRO)has recently been paid much attention as one of the major health outcomes of breast cancer treatments in Japan. In this report, through a systematic literature review for the evidence of QOL/PRO assessments in clinical study undergoing drug treatments of breast cancer in these several years, I introduced the results of the limited qualified papers from 2017. Moreover, I introduced the results of the large multi-center Japanese survey for alopecia induced by chemotherapy in the recent published paper.

Psychometric properties of the Japanese version of the EQ-5D-Y by self-report and proxy-report: reliability and construct validity.

Purpose: This study aimed to assess psychometric properties of the Japanese version of the EQ-5D-Y (3 levels) with a focus on feasibility, reliability, and construct validity.

Methods: Respondents were recruited from the general populations of three cities in Japan. First, children and adolescents responded to the EQ-5D-Y and PedsQL by self-report.

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.

A multicenter survey of temporal changes in chemotherapy-induced hair loss in breast cancer patients.

Purpose: Many breast cancer patients suffer from chemotherapy-induced hair loss. Accurate information about temporal changes in chemotherapy-induced hair loss is important for supporting patients scheduled to receive chemotherapy, because it helps them to prepare. However, accurate information, on issues such as the frequency of hair loss after chemotherapy, when regrowth starts, the condition of regrown hair, and the frequency of incomplete hair regrowth, is lacking.

Effect of ruxolitinib therapy on the quality-of-life of Japanese patients with myelofibrosis.

Objectives: Myelofibrosis (MF) is associated with a significant symptom burden that severely impacts patient quality-of-life (QoL). Ruxolitinib, a potent Janus kinase 1 (JAK1)/JAK2 inhibitor, led to substantial improvements in splenomegaly, MF-associated symptoms, and QoL in the phase 3 COMFORT studies, proving superior to placebo and best available therapy. This study evaluated the effect of ruxolitinib on symptoms and QoL in Japanese patients with MF.

Cost-effectiveness analysis of the introduction of S-1 therapy for first-line metastatic breast cancer treatment in Japan: results from the randomized phase III SELECT BC trial.

Background: This study evaluated the cost-effectiveness of replacing standard intravenous therapy (taxane) with oral S-1 therapy for first-line metastatic breast cancer treatment.

Methods: This cost-effectiveness analysis was based on data from a randomized phase III trial (SELECT BC). As cost-effectiveness was a secondary endpoint of the SELECT BC trial, some of the randomized patients participated in an EQ-5D survey (N = 391) and health economic survey (N = 146).