Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen.

Patients with severe thalassemia commonly have a survival that is significantly shorter than that of the general population. Allogeneic hematopoietic stem cell transplantation (allo-SCT) is the only established treatment that is potentially curative, but it is limited by the availability of donors and the medical condition of the patient. To expand the donor pool to include haploidentical related donors, we introduced a program consisting of a pharmacologic pretransplant immune suppression phase (PTIS) and 2 courses of dexamethasone and fludarabine, followed by pretransplant conditioning with fludarabine-i.v. busulfan and post-transplant graft-versus-host disease (GVHD) prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. We transplanted 83 consecutive transfusion-dependent patients with thalassemia (median age, 12 years; range, 1 to 28 years) with a minimum follow-up of 6 months (median, 15 months; range, 7 to 53 months); the 3-year projected overall and event-free survival is over 96%, and there have been no secondary graft failures. Of the first 31 patients, we had 2 graft failures, both of them occurring in patients with extremely high titers of anti-donor-specific HLA antibodies (anti-DSAs), but after adjusting the PTIS to include bortezomib and rituximab for patients with high titers of anti-DSAs and using pharmacologic dose guidance for busulfan, we had no graft failures in the last 52 patients. Six (7%) of 83 patients developed severe GVHD. We conclude that this is a safe and efficacious approach to allogeneic SCT in thalassemia, yielding results comparable to those available for patients with fully matched donors.

Co-insurance and health care utilization in Japanese patients with rheumatoid arthritis: a discontinuity regression approach.

BACKGROUND: Co-insurance rates in Japan decrease when patients turn 70 years of age. We aim to compare changes in medical demand for Japanese patients with rheumatoid arthritis (RA) at age 70 prior to 2014, when there was a reduction in co-insurance rates from 30 to 10%, with changes in medical demand at age 70 after 2014 when co-insurance rates decreased from 30% to only 20%. METHODS: We used administrative data from large Japanese hospitals. We employed a discontinuity regression (RD) approach to control for unobserved endogeneity in the data. RESULTS: We identified a total of 7343 patients with RA, 4905 (67%) turned age 70 before April, and found that a 20% decrease in co-insurance was associated with increased utilization of more expensive biologic RA drugs, more outpatient visits and higher total medical costs. However, a 10% decrease in co-insurance for patients who turned 70 after 2014 did not significantly change demand for medical services. CONCLUSIONS: For the younger cohort, we did not observe any changes in medical demand after a price decrease. We therefore conclude that the economic goal of cost sharing, namely a behavioural change towards lower health-care utilization, is not achieved in this particular cohort of chronic patients.

Biologic treatment of Japanese patients with inflammatory bowel disease.

BACKGROUND: There is little information regarding the use of biologics in Inflammatory Bowel Disease (IBD) patients in Japan. The aim of this study was to determine the factors associated with the use of biologics in the treatment of Japanese patients with IBD. METHODS: An online survey was conducted among Japanese patients with IBD (n = 1035). Socioeconomic as well as treatment related information was collected. Logistic regression was applied to analyze the determinants of biologic treatment. RESULTS: Younger age (≤ 40 years vs. > 65 years; OR:0.24), time since diagnosis (< 2 years vs. < 15 years; OR: 4.16), surgical history (OR:1.98) and visiting university hospitals (university hospitals vs. clinics; OR: 0.47) were associated with biologic treatment for Japanese IBD patients. CONCLUSIONS: Currently, biologics have been used in younger IBD patients which may give rise to the presence of an age bias in biologic treatment. Further studies are required to confirm these results and to define appropriate IBD patients who should be treated with biologic agent.

Cultural beliefs, utility values, and health technology assessment.

BACKGROUND: Health-care utilities differ considerably from country to country. Our objective was to examine the association of cultural values based on Hofstede's cultural dimensions' theory with utility values that were identified using the time trade off method. METHODS: We performed a literature search to determine preference-based value algorithms in the general population of a given country. We then fitted a second-order quadratic function to assess the utility function curve that links health status with health-care utilities. We ranked the countries according to the concavity and convexity properties of their utility functions and compared this ranking with that of the Hofstede index to check if there were any similarities. RESULTS: We identified 10 countries with an EQ-5D-5L-based value set and 7 countries with an EQ-5D-3L-based value set. Japan's degree of concavity was highest, while Germany's was lowest, based on the EQ-5D-3L and EQ-5D-5L value sets. Japan also ranked first in the Hofstede long-term orientation index, and rankings related to the degree of concavity, indicating a low time preference rate. CONCLUSIONS: This is the first evaluation to identify and report an association between different cultural beliefs and utility values. These findings underline the necessity to take local values into consideration when designing health technology assessment systems.

Persistence rates and medical costs of biological therapies for psoriasis treatment in Japan: a real-world data study using a claims database.

BACKGROUND: Biological therapies (BTs) including infliximab (IFX), adalimumab (ADL), secukinumab (SCK) and ustekinumab (UST) are approved in Japan for the treatment of psoriasis. Although the persistence rates and medical costs of BTs treatment have been investigated in multiple foreign studies in recent years, few such studies have been conducted in Japan and the differences between patients who adhered to treatment and those who did not have not been reported. This study is aimed at investigating the persistence rates and medical costs of BTs in the treatment of psoriasis in Japan, using the real-world data from a large-scale claims database. METHODS: Claims data from the JMDC database (August 2009 to December 2016) were used for this analysis. Patient data were extracted using the ICD10 code for psoriasis and claims records of BT injections. Twelve-month and 24-month persistence rates of BTs were estimated by Kaplan-Meier methodology, and 12-month-medical costs before and after BT initiation were compared between persistent and non-persistent patient groups at 12 months. RESULTS: A total of 205 psoriasis patients treated with BTs (BT-naïve patients: 177) were identified. The 12-month/24-month persistence rates for ADL, IFX, SCK, and UST in BT-naïve patients were 46.8% ± 16.6%/46.8 ± 16.6%, 53.0% ± 14.9%/41.0% ± 15.5%, 55.4%/55.4% (95% CI not available) and 79.4% ± 9.9%/71.9% ± 12.2%, respectively. Statistically significant differences in persistence were found among different BT treatments, and UST was found to have the highest persistence rate. The total medical costs during the 12 months after BT initiation in BT-naïve patients were (in 1000 Japanese Yen): 2218 for ADL, 3409 for IFX, 465 for SCK, 2824 for UST (average: 2828). Compared with the 12-month persistent patient group, the total medical costs in the persistent group was higher (Δ:+ 118), but for some medications such as IFX or UST cost increases were lower for persistent patients. CONCLUSIONS: UST was found to have the highest persistence rate among all BTs for psoriasis treatment in Japan. The 12-month medical costs after BT initiation in the persistent patient group may not have increased as much as in the non-persistent patient group for some medications.

Productivity loss of caregivers of schizophrenia patients: a cross-sectional survey in Japan.

BACKGROUND: When a family member is diagnosed with schizophrenia, it causes stress to the caregiver that can eventually result in missed work days and lower work performance. AIM: This study aims at revealing productivity costs for caregivers of schizophrenia patients in Japan. METHOD: A cross-sectional survey of caregivers was conducted and resulted in 171 respondents. The assessment of work productivity included calculating the costs of absenteeism, presenteeism and total productivity costs. This was accomplished using the "Work Productivity and Activity Impairment Questionnaire" (WPAI). RESULTS: A relative majority of caregivers in the sample provided care for their spouse (47%), 18% cared for their brother or sister and 16% provided care for their child. Per capita productivity costs totaled JPY 2.42 million, with JPY 2.36 million (97%) of that amount being due to presenteeism. CONCLUSIONS: The burden on caregivers is substantial enough to warrant structured support programs aimed at maintaining careers' physical and mental health, helping them provide more effective care to schizophrenia patients and eventually increase productivity at work.

Productivity loss of Japanese patients with rheumatoid arthritis - A cross-sectional survey.

OBJECTIVE: The objective of this study was (1) to determine productivity costs due to absenteeism and presenteeism among Japanese workers with rheumatoid arthritis (RA), and (2) to identify additional factors associated with productivity loss among workers with RA. METHODS: An online survey of 500 RA Japanese patients was used. The Japanese version of the Stanford Health Assessment Questionnaire (J-HAQ) was used to measure patients' functional disability. The patient health questionnaire-9 item (PHQ-9) was used to measure symptoms and severity of depression. To assess work productivity the 'work productivity and activity impairment questionnaire' for rheumatoid arthritis (WPAI-RA), a six-item validated instrument was used. RESULTS: Percentages of absenteeism and presenteeism were found to be 1% and 23%, respectively. The annual combined productivity costs of both absenteeism and presenteeism was 7877 USD per patient. Factors significantly associated with a higher productivity loss were functional disability, depressive symptoms, and time since RA diagnosis, while age, and biological disease-modifying antirheumatic drugs (bDMARDs) treatment were significantly associated with a lower productivity loss. CONCLUSION: Treatment of RA with bDMARDs would likely result in decreased productivity loss among Japanese patients.

Shared Decision Making and Treatment Satisfaction in Japanese Patients with Inflammatory Bowel Disease.

BACKGROUND: Inflammatory bowel disease (IBD) is a range of complex disorders that imposes a high burden on patients' quality of life and on available health resources. Only 50% of those with this disease appear to be satisfied with the treatment methods. Therefore, this has a negative effect on their willingness to comply with treatment. OBJECTIVE: This study examined whether patients are more satisfied when they and their doctors agree on how much of their management decisions should be shared. PARTICIPANTS: A nationwide online survey of Japanese patients in treatment for IBD yielded 1,068 respondents. METHODS: A scoring system to match patients' preference for shared decision-making and their actual involvement in their treatment decisions was devised. To assess factors that were associated with treatment satisfaction, univariate and multivariate logistic regressions were performed. RESULTS: We found a clear correlation between the extent to which patients and their doctors agreed on decision-sharing and patients' overall satisfaction with their treatment. An excellent fit increased the odds ratio of being satisfied with the treatment by 16.48 (8.31-32.69). CONCLUSIONS: Physicians should expect best patient compliance when they and their patients are in agreement with the extent to which treatment decisions are shared. This in turn maximizes the likelihood of successful treatment outcome.

Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification.

Improving outcomes among class 3 thalassemia patients receiving allogeneic hematopoietic stem cell transplantations (HSCT) remains a challenge. Before HSCT, patients who were ≥ 7 years old and had a liver size ≥ 5 cm constitute what the Center for International Blood and Marrow Transplant Research defined as a very high-risk subset of a conventional high-risk class 3 group (here referred to as class 3 HR). We performed HSCT in 98 patients with related and unrelated donor stem cells. Seventy-six of the patients with age < 10 years received the more conventional myeloablative conditioning (MAC) regimen (cyclophosphamide, busulfan, ± fludarabine); the remaining 22 patients with age ≥ 10 years and hepatomegaly (class 3 HR), and in several instances additional comorbidity problems, underwent HSCT with a novel reduced-toxicity conditioning (RTC) regimen (fludarabine and busulfan). We then compared the outcomes between these 2 groups (MAC versus RTC). Event-free survival (86% versus 90%) and overall survival (95% versus 90%) were not significantly different between the respective groups; however, there was a higher incidence of serious treatment-related complications in the MAC group, and although we experienced 6 graft failures in the MAC group (8%), there were none in the RTC group. Based on these results, we suggest that (1) class 3 HR thalassemia patients can safely receive HSCT with our novel RTC regimen and achieve the same excellent outcome as low/standard-risk thalassemia patients who received the standard MAC regimen, and further, (2) that this novel RTC approach should be tested in the low/standard-risk patient population.

Systematic review and network meta-analysis in health technology assessment.

Conducting systematic review and meta-analysis (SR/MA) is a standard process for establishing evidences for health technology assessment. Quality assessment of studies included in SR/MA and SR/MA studies should be considered. This article provides recommendations on tools used for assessing the quality of studies included in each SR/MA and the quality of SR/MA. For assessing the quality of randomized controlled trial, we recommend a tool called "Risk of Bias", which focuses on random generation, allocation concealment, blinding and outcome reporting. For assessing the quality of observational study, the Newcastle Ottawa Scale (NOS) is recommended. The NOS consists of three different dimensions-selection, comparability and outcomes or exposure. Another tool which is recommended is the Down and Black scale. It focuses on the quality of reporting, validity, bias and confounding, and power of study. For assessing the quality of SR/MA, we recommend to use a checklist developed by Klassen et al, covering well-defined question, inclusion criteria, comprehensiveness, quality of included studies, reproducibility, and external validity. This article also provides a fundamental of network meta-analysis that should be considered where no direct evidence exists or when there is a need to compare multiple interventions at the same time.

The macroeconomic impact of a dengue outbreak: Case studies from Thailand and Brazil.

BACKGROUND: Dengue is spreading in (sub)tropical areas, and half of the global population is at risk. The macroeconomic impact of dengue extends beyond healthcare costs. This study evaluated the impact of dengue on gross domestic product (GDP) based on approaches tailored to two dengue-endemic countries, Thailand and Brazil, from the tourism and workforce perspectives, respectively. FINDINGS: Because the tourism industry is a critical economic sector for Thailand, lost tourism revenues were estimated to analyze the impact of a dengue outbreak. An input-output model estimated that the direct effects (on international tourism) and indirect effects (on suppliers) of dengue on tourism reduced overall GDP by 1.43 billion US dollars (USD) (0.26%) in the outbreak year 2019. The induced effect (reduced employee income/spending) reduced Thailand's GDP by 375 million USD (0.07%). Overall, lost tourism revenues reduced Thailand's GDP by an estimated 1.81 billion USD (0.33%) in 2019 (3% of annual tourism revenue). An inoperability input-output model was used to analyze the effect of workforce absenteeism on GDP due to a dengue outbreak in Brazil. This model calculates the number of lost workdays associated with ambulatory and hospitalized dengue. Input was collected from state-level epidemiological and economic data for 2019. An estimated 22.4 million workdays were lost in the employed population; 39% associated with the informal sector. Lost workdays due to dengue reduced Brazil's GDP by 876 million USD (0.05%). CONCLUSIONS: The economic costs of dengue outbreaks far surpass the direct medical costs. Dengue reduces overall GDP and inflicts national economic losses. With a high proportion of the population lacking formal employment in both countries and low income being a barrier to seeking care, dengue also poses an equity challenge. A combination of public health measures, like vector control and vaccination, against dengue is recommended to mitigate the broader economic impact of dengue.

Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control.

Patients with class 3 thalassemia with high-risk features for adverse events after high-dose chemotherapy with hematopoietic stem cell transplantation (HSCT) are difficult to treat, tending to either suffer serious toxicity or fail to establish stable graft function. We performed HSCT in 18 such patients age ≥7 years and hepatomegaly using a novel approach with pretransplant immunosuppression followed by a myeloablative reduced-toxicity conditioning regimen (fludarabine and i.v. busulfan [Flu-IV Bu]) and then HSCT. The median patient age was 14 years (range, 10 to 18 years). Before the Flu-IV Bu + antithymocyte globulin conditioning regimen, all patients received 1 to 2 cycles of pretransplant immunosuppression with fludarabine and dexamethasone. Thirteen patients received a related donor graft, and 5 received an unrelated donor graft. An initial prompt engraftment of donor cells with full donor chimerism was observed in all 18 patients, but 2 patients developed secondary mixed chimerism that necessitated withdrawal of immunosuppression to achieve full donor chimerism. Two patients (11%) had acute grade III-IV graft-versus-host disease, and 5 patients had limited chronic graft-versus-host disease. The only treatment-related mortality was from infection, and with a median follow-up of 42 months (range, 4 to 75), the 5-year overall survival and thalassemia-free survival were 89%. We conclude that this novel sequential immunoablative pretransplantation conditioning program is safe and effective for patients with high-risk class 3 thalassemia exhibiting additional comorbidities.

The effects of metformin on ovarian cancer: a systematic review.

OBJECTIVE: The potential therapeutic effects of metformin on several cancers were reported. However, the evidence of the effects of metformin on ovarian cancer is still limited and inconclusive. This systematic review and meta-analysis study aim to summarize the existing evidence of the therapeutic effects of metformin on ovarian cancer. METHODS: We performed systematic searches using electronic databases including PubMed and EMBASE until December 2012. Key words included "metformin" AND ("ovarian cancer" OR "ovary tumor"). All human studies assessing the effects of metformin on ovarian cancer were eligible for inclusion. All articles were reviewed independently by 2 authors with a standardized approach for the purpose of study, study design, patient characteristics, exposure, and outcomes. The data were pooled using a random-effects model. RESULTS: Of 190 studies retrieved, only 3 observational studies and 1 report of 2 randomized controlled trials were included. Among those studies, 2 reported the effects of metformin on survival outcomes of ovarian cancer, whereas the other 2 reported the effects of metformin on ovarian cancer prevention. The findings of studies reporting the effects on survival outcomes indicated that metformin may prolong overall, disease-specific, and progression-free survival in ovarian cancer patients. The results of studies reporting the effects of metformin on ovarian cancer prevention were meta-analyzed. It indicated that metformin tended to decrease occurrence of ovarian cancer among diabetic patients with the pooled odds ratio of 0.57 (95% confidence interval, 0.16-1.99). CONCLUSIONS: Our findings showed the potential therapeutic effects of metformin on survival outcomes of ovarian cancer and ovarian cancer prevention. However, most of the evidence was observational studies. There is a call for further well-conducted controlled clinical trials to confirm the effects of metformin on ovarian cancer survival and ovarian cancer prevention.

Cost utility analysis of reduced intensity hematopoietic stem cell transplantation in adolescence and young adult with severe thalassemia compared to hypertransfusion and iron chelation program.

BACKGROUND: Hematopoieticic stem cell transplantation is the only therapeutic option that can cure thalassemia disease. Reduced intensity hematopoietic stem cell transplantation (RI-HSCT) has demonstrated a high cure rate with minimal complications compared to other options. Because RI-HSCT is very costly, economic justification for its value is needed. This study aimed to estimate the cost-utility of RI-HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for adolescent and young adult with severe thalassemia in Thailand. METHODS: A Markov model was used to estimate the relevant costs and health outcomes over the patients' lifetimes using a societal perspective. All future costs and outcomes were discounted at a rate of 3% per annum. The efficacy of RI-HSCT was based a clinical trial including a total of 18 thalassemia patients. Utility values were derived directly from all patients using EQ-5D and SF-6D. Primary outcomes of interest were lifetime costs, quality adjusted life-years (QALYs) gained, and the incremental cost-effectiveness ratio (ICER) in US ($) per QALY gained. One-way and probabilistic sensitivity analyses (PSA) were conducted to investigate the effect of parameter uncertainty. RESULTS: In base case analysis, the RI-HSCT group had a better clinical outcomes and higher lifetime costs. The incremental cost per QALY gained was US $3,236 per QALY. The acceptability curve showed that the probability of RI-HSCT being cost-effective was 71% at the willingness to pay of 1 time of Thai Gross domestic product per capita (GDP per capita), approximately US $4,210 per QALY gained. The most sensitive parameter was utility of severe thalassemia patients without cardiac complication patients. CONCLUSION: At a societal willingness to pay of 1 GDP per capita, RI-HSCT was a cost-effective treatment for adolescent and young adult with severe thalassemia in Thailand compared to BT-ICT.

The effects of medication supply on hospitalizations and health-care costs in patients with chronic heart failure.

OBJECTIVES: Use of angiotensin-converting enzyme inhibitors or angiotensin receptor blockers results in decreased morbidity among patients with chronic heart failure (CHF). Undersupply of medication could result in inadequate control of CHF, whereas oversupply of medication could increase health-care costs and risks of toxicities. This study aimed to determine the effects of medication supplies on health-care costs and hospitalizations in patients with CHF receiving angiotensin-converting enzyme inhibitors or angiotensin receptor blockers. METHODS: We retrospectively examined the electronic database in a hospital in Thailand. Patients who were diagnosed with CHF and who received angiotensin-converting enzyme inhibitors or angiotensin receptor blockers in the year 2003 were included. Medication supplies were assessed by using the medication possession ratio (MPR). The Cox proportional hazard model was used to determine the association of medication supply (appropriate supply: MPR 0.8-1.2, oversupply: MPR > 1.2, undersupply: MPR < 0.8) with CHF-related and all-cause hospitalizations. Health-care costs were compared by using multiple linear regressions. All analyses were adjusted for propensity score and other variables. RESULTS: A total of 393 patients were included. Their mean age was 66 years, with 56% being females. Fifty-seven percent of the patients received an inappropriate -supply of medication. Undersupply of medication likely increased the risks of CHF-related hospitalization with an adjusted hazard ratio of 1.66 (95% confidence interval [CI] 0.80-3.46). The adjusted hazard ratio of undersupply and oversupply of medication for all-cause hospitalization was 1.13 (95% CI 0.74-1.73) and 3.19 (95%CI 0.66-15.47), respectively. The total health-care costs in the undersupply and oversupply groups were significantly greater than that in the appropriate-supply group: $49 (95% CI 32-66) and $103 (95% CI 32-173), respectively. CONCLUSIONS: Inappropriate medication supplies could increase the risks of CHF-related and all-cause hospitalizations. Both undersupply and oversupply of medication had significantly higher health-care costs.

Epidemiology and costs of dengue in Thailand: A systematic literature review.

BACKGROUND: Dengue is the fastest-spreading vector-borne viral disease worldwide. In Thailand, dengue is endemic and is associated with a high socioeconomic burden. A systematic literature review was conducted to assess and describe the epidemiological and economic burden of dengue in Thailand. METHODS: Epidemiological and economic studies published in English and Thai between 2011-2019 and 2009-2019, respectively, were searched in MEDLINE, Embase, and Evidence-Based Medicines reviews databases. Reports published by the National Ministry of Public Health (MoPH) and other grey literature sources were also reviewed. Identified studies were screened according to predefined inclusion and exclusion criteria. Extracted data were descriptively summarised and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. RESULTS: A total of 155 publications were included in the review (39 journal articles and 116 grey literature). Overall, dengue incidence varied yearly, with the highest rates per 100,000 population in 2013 (dengue fever (DF) 136.6, dengue haemorrhagic fever (DHF) 100.9, dengue shock syndrome (DSS) 3.58) and 2015 (DF 133.1, DHF 87.4, DSS 2.14). Peak incidence coincided with the monsoon season, and annual mortality was highest for DSS, particularly in the age group 15-24-year-olds. The highest dengue incidence rates were reported in children (10-14-year-olds) and young adults (15-24-year-olds), irrespective of dengue case definition. Economic and societal burdens are extensive, with the average cost per case ranging from USD 41 to USD 261, total cost per year estimated at USD 440.3 million, and an average of 7.6 workdays lost for DHF and 6.6 days for DF. CONCLUSIONS: The epidemiological, economic, and societal burden of dengue in Thailand is high and underreported due to gaps in national surveillance data. The use of expansion factors (EFs) is recommended to understand the true incidence of dengue and cost-benefit of control measures. Furthermore, as dengue is often self-managed and underreported, lost school and workdays result in substantial underestimation of the true economic and societal burden of dengue. The implementation of integrated strategies, including vaccination, is critical to reduce the disease burden and may help alleviate health disparities and equity challenges posed by dengue.

Treatment patterns and medical cost of systemic lupus erythematosus patients in Japan: a retrospective claims database study.

Aim: To describe the characteristics and medication treatment patterns, healthcare resource utilization (HRU), and associated costs in Japanese patients with systemic lupus erythematosus (SLE).Methods: Claims data from the Japan Medical Data Center (JMDC) database were used to identify patients with SLE-related claims from 2010 to 2017. Algorithms were developed to retrospectively categorize patients by disease severity, treatment experience, and SLE-related manifestations such as lupus nephritis and central nervous system lupus. Descriptive and multivariate analyses were used to describe treatment pattern and estimate HRU and associated costs for the SLE cohort overall and by disease severity and complications.Results: Among 4,733 eligible patients, 2,072 (43.8%) were treatment naïve, 2,214 (46.8%) were previously treated for SLE, and 447 (9.4%) did not receive any treatment. Mean (SD) age of the total SLE cohort was 45.2 (13.1) years and mean (SD) follow-up duration was 1,137.3 (758.0) d. Based on disease severity, 1,383 (29.2%) patients had mild, 2,619 (55.3%) patients had moderate, and 731 (15.4%) patients had severe SLE. Patients on glucocorticoids (both oral and parenteral) received add-on medications the most (35.5%, p < .001). Mean annual cost per SLE patient in the post-index period, inclusive of hospitalizations, outpatient visits, and pharmacy was ¥436,836; ¥1,010,772; and ¥2,136,780 for patients with mild, moderate, and severe SLE, respectively.Limitations: The database only captured information on patients up to 75 years of age. Due to the nature of the database, biases regarding conditions that attribute to the spectrum of SLE severity, flare incidences, or individual physical status cannot be ruled out.Conclusions: This study describes the treatment patterns and economic burden experienced by Japanese patients with SLE. The results highlight an unmet need to derive better treatment strategies for patients with SLE to effectively address the disease burden in Japan.

Treatment patterns and healthcare resource utilization in palmoplantar pustulosis patients in Japan: A claims database study.

BACKGROUND: Palmoplantar pustulosis (PPP) is a chronic, relapsing, inflammatory autoimmune condition, characterized by sterile pustules on the palms and soles. The treatment patterns of PPP and total health care resource utilization in Japan are not well described. Investigating these areas is needed to understand current PPP management in Japan. OBJECTIVE: To describe the characteristics, medication treatment and health care resource utilization patterns, and associated costs of PPP patients in Japan. METHODS: A retrospective analysis of insurance claims data was conducted using the Japan Medical Data Center database. Adult Patients with at least two claims with a PPP diagnosis from January 1, 2011 to March 30, 2017 and six months of follow-up after the first diagnosis were included. Patient characteristics described include age, gender, and comorbid conditions. Treatment patterns assessed include the types of treatment, sequence of treatment, and rates of discontinuation, switching, persistence and use of concomitant medications. RESULTS: A total of 5,162 adult patients met all inclusion criteria. Mean (SD) patient age was 49.7 (11.6) years and 43.2% were male. A total of 2441 patients (47.8%) received systemic non-biologic drugs during the entire follow up period, 2,366 (46.4%) were prescribed topical therapy, 273 (5.4%) were prescribed phototherapy, while 18 (0.4%) of patients with other autoimmune comorbidities were eligible for prescribed biologics. For treatment-naïve patients with mild PPP, topical therapy was most commonly (77.1%) prescribed, whereas in moderate to severe cases of PPP, systemic non-biologic drugs (65%) were most often used. The frequency of switching was similar (64.3% to 75.3%) across various therapies and treatment lines. CONCLUSION: This study describes the treatment patterns and health care resource utilization for Japanese PPP patients using a large claims database, and highlights an unmet need to derive better treatment strategies for PPP and address disease burden in Japan.

What are the volume and budget needs to provide chemotherapy to all children with acute lymphoblastic leukaemia in Thailand? Development and application of an estimation tool.

OBJECTIVE: Insufficient access to anticancer medicines may contribute to the wide survival differences of children with cancers across the globe. We developed a tool to estimate the volume of medicines and budget requirements to provide chemotherapy to children with acute lymphoblastic leukaemia (ALL). DESIGN: Development and application of an estimation tool. SETTING: Paediatric oncology hospital departments in Thailand. PARTICIPANTS: 318 children aged 0-14 years diagnosed with ALL and 215 children with undiagnosed ALL. INTERVENTIONS: Estimates of volume and budget requirements for administering a full course of chemotherapy for ALL and a further course for children who relapse, according to National Treatment Guidelines. PRIMARY AND SECONDARY OUTCOME MEASURES: Primary outcome measures were the volume (mg) and cost (US$) of medicines needed to treat children with ALL. For medicines whose main indication is paediatric ALL (asparaginase and 6-mercaptopurine), we estimated the difference between volume needed and actual sales in 2017 (secondary outcome). RESULTS: Ten anticancer medicines and four chemoprotective agents are needed for the treatment of paediatric ALL according to the Thai treatment guidelines. Of these 14 medicines, 13 are included in the WHO essential medicines list for children. All are available as generics. We estimated that essential chemotherapy and chemoprotective agents to treat all children diagnosed with ALL in Thailand in 2017 would cost US$ 814 952 (US$ 1 365 422 for diagnosed and undiagnosed children), which corresponds to 0.005% (0.008%) of the country's total health expenditure. The volumes of asparaginase and 6-mercaptopurine available on the Thai market in 2017 were more than sufficient (2.3 and 1.5 times the amounts needed, respectively) to treat all children diagnosed with ALL. CONCLUSIONS: Procuring sufficient quantities of essential medicines to treat children with ALL requires relatively modest resources. Medicine cost should not be a major barrier to ALL treatment in similar settings.

Shared Decision-Making and Patient Satisfaction in Japanese Rheumatoid Arthritis Patients: A New "Preference Fit" Framework for Treatment Assessment.

INTRODUCTION: We have developed a new framework to assess shared decision-making (SDM) as a tool to improve patient satisfaction. This framework is based on a "preference fit" index that relates SDM to patient treatment preferences and patient satisfaction in a sample of rheumatoid arthritis (RA) patients in Japan. METHODS: We surveyed 500 RA patients in Japan and explored the interactions between the treatment preference fit index, SDM, and overall patient satisfaction. RESULTS: Our new preference fit index reveals significant impact on patient satisfaction: the better the fit between SDM and patient preferences, the higher the patient satisfaction with the current treatment. Patients treated with biologic agents were more satisfied. Patients suffering from depression or migraines scored significantly lower both on our preference fit measure and for overall patient satisfaction. CONCLUSION: The association between depression and a low treatment preference fit suggests that depression may pose challenges to SDM and that doctors in Japan are less attuned to the SDM preferences of depressed patients. FUNDING: Janssen Pharmaceutical KK.