Haemophilia Experiences, Results and Opportunities (HERO) study: treatment-related characteristics of the population.

The HERO (Haemophilia Experiences, Results and Opportunities) quantitative surveys collected information on characteristics and perceptions of adult persons with haemophilia (PWH) and parents of children with haemophilia. The aim of this article is to describe the perceptions of PWH and parents on psychosocial aspects related to treatment. Two online surveys (one for PWH, one for parents) were conducted in 10 countries. Among 675 PWH respondents, 77% reported having responsibility for their own care; 72% of 561 parent respondents had the main responsibility for their son. PWH were most commonly treated on demand (45% of 648 adults using factor concentrate), with 32% on regular prophylaxis and 23% treated on demand with short-term prophylaxis (e.g. for sports/physiotherapy). Children were most often treated with prophylaxis (65% of 549 children using factor concentrate), with 26% treated on demand and 8% treated on demand with short-term prophylaxis. Factor was generally used as instructed at home. Some respondents (41% PWH; 30% parents) had difficulties/concerns with factor availability/affordability. PWH reported more bleeds in the last 12 months than parents reporting their son's bleeds (mean 17.8 vs. 8.7). Both PWH and parents generally perceived that overall, their (their son's) haemophilia was well controlled. Results differed by country. The HERO study captured new, patient-based data regarding many facets of life relevant to PWH, including treatment. The information conveyed in this article largely represents new insights regarding perceptions of treatment and provides initial benchmark statistics for further research.

Haemophilia Experiences, Results and Opportunities (HERO) Study: influence of haemophilia on interpersonal relationships as reported by adults with haemophilia and parents of children with haemophilia.

Evidence delineating the effects of haemophilia on interpersonal relationships is sparse and largely outdated, failing to reflect the impact of current treatment strategies. HERO (Haemophilia Experiences, Results and Opportunities) was commenced to garner a more comprehensive understanding of psychosocial issues facing persons with haemophilia (PWH). This article describes the findings of the quantitative HERO survey relating to the influence of haemophilia on interpersonal relationships of adult PWH, and parents/caregivers of children with haemophilia. Separate questionnaires were completed by adult PWH and parents of minor children from 10 countries, including satisfaction with support from partners, family, friends and other social contacts; disclosure of haemophilia and carrier status and family dynamics. A total of 675 PWH and 561 parents completed the survey. Over half of PWH (57%) and parents (84%) were married. Most PWH were satisfied with support from partners (94%), family (90%) and friends (85%), with lower percentages reported among those with inhibitors. Most parents were likewise satisfied with support from partners (88%) and family (83%). Whereas PWH were reticent to disclose their diagnosis beyond family and friends, parents were more likely to share their son's diagnosis, and most were satisfied with the support from their son's peers (74%), teachers (83%) and other adults in supervisory roles (85%). PWH and parents surveyed were satisfied overall with the support they received from partners, family, friends and social contacts. Relationships are affected by haemophilia in various ways, and particularly affected in terms of disease burden, age and social life.

Haemophilia Experiences, Results and Opportunities (HERO) Study: survey methodology and population demographics.

Psychosocial factors have a significant impact on the quality of life of persons with haemophilia (PWH). The Haemophilia Experiences, Results and Opportunities (HERO) initiative was developed to provide a greater understanding of the psychological components which influence the lives of PWH. This article describes the HERO methodology and the characteristics of respondents. Two online surveys (one for adult PWH ≥18 years and one for parents of children <18 years with haemophilia) were developed by an international advisory board and conducted in 10 countries. The surveys included demographic and treatment characteristics, relationships, sexual intimacy, quality of life, barriers to treatment and sources of information. A total of 675 PWH [age, median (range) 36 (18-86 years)] and 561 parents [39 (23-68 years)] completed the survey. PWH/parents reported haemophilia A (74%/76%), B (13%/16%) or with inhibitors (13%/8%). Spontaneous joint bleeding was reported in 76%/52% of PWH/children with haemophilia A, 67%/47% with haemophilia B and 93%/76% with inhibitors. Median number of bleeds (interquartile range) was 7 (2-20) for PWH and 4 (2-10) for children in the past year. Most PWH and children were treated with factor concentrate. PWH reported arthritis (49%) and HIV/HCV infections (18%/43%) related to haemophilia. Most PWH and parent respondents had received formal education (85%/89%) and were employed full- or part-time (60%/72%). HERO is one of the largest multinational studies focused on psychosocial issues in haemophilia, including historical and treatment information that will allow for multivariate analyses of determinants of health in haemophilia.

The cost effectiveness of rapid-acting insulin aspart compared with human insulin in type 2 diabetes patients: an analysis from the Japanese third-party payer perspective.

OBJECTIVES: The Nippon Ultra-Rapid Insulin and Diabetic Complication Evaluation Study (NICE Study) (NCT00575172) was a 5-year, open-label, randomised controlled trial which compared cardiovascular outcomes in Japanese type 2 diabetes patients intensively treated with regular human insulin or insulin aspart (NovoRapid; Novo Nordisk A/S, Bagsvaerd, Denmark), a rapid-acting insulin analogue. The aim of the present analysis was to evaluate the cost effectiveness of insulin aspart versus regular human insulin from the perspective of a Japanese third-party healthcare payer. RESEARCH DESIGN AND METHODS: A discrete event-simulation model was developed in Microsoft Excel to assess the within-trial cost effectiveness and make longer-term clinical projections in patients treated with regular human insulin or insulin aspart. In addition to severe hypoglycaemia, the model captured myocardial and cerebral infarction events and percutaneous coronary intervention and coronary artery bypass graft procedures. Within-trial mortality, incidence of severe hypoglycaemia and cardiovascular event probabilities were derived from the annual rates observed during the trial period, while post-trial outcomes were calculated using the event rates from the trial, adjusted for increasing patient age. Event costs were accounted from the healthcare payer perspective and expressed in 2008 Japanese yen (JPY), while health-related quality of life (HRQoL) was captured using event and state utilities. Future costs and clinical benefits were discounted at 3% annually. Life expectancy, quality-adjusted life expectancy, cardiovascular event rates and costs were evaluated over 5- and 10-year time horizons and sensitivity analyses were performed to assess variability in model outcomes. RESULTS: Over 5 years of treatment, insulin aspart dominated human insulin both in incremental life expectancy and in incremental quality-adjusted life-years (QALYS). Insulin aspart was associated with a small improvement in discounted life expectancy of 0.005 years (4.688 vs. 4.684 years) and an increase of 0.023 quality-adjusted life-years (QALYs) (3.800 vs. 3.776 QALYs) when compared with regular human insulin. Insulin aspart also incurred lower costs (JPY 481,586 vs. 594,717, difference -113,131) which resulted from the decreased incidence of cardiovascular events with insulin aspart (0.013 events per patient year vs. 0.030 on regular human insulin). Breakdown of costs indicated that pharmacy costs were higher with insulin aspart (JPY 346,608 vs. 278,468), but these costs were more than offset by the reduced costs associated with cardiovascular complications and hypoglycaemia over 5 years of treatment (JPY 134,978 vs. 316,249). Sensitivity analysis showed that insulin aspart was still cost-effective in the case where only 18% of the within-trial cardiovascular and mortality benefit over regular human insulin was captured in the model (assuming a willingness-to-pay threshold of JPY 5,000,000). LIMITATIONS: The NICE study cohort was relatively small (n = 325), meaning that caution should be exercised when calculating and interpreting the incremental cost-effectiveness ratio. Also, despite the differences in cardiovascular risk profile between the Japanese and UK populations, UKPDS-derived risk equations were used to project MI outcomes and PCI and CABG procedures and UKPDS HRQoL scores were applied to all health states. While these risk formulas and HRQoL utilities may not be directly applicable to the Japanese population, no equivalent Japanese-specific data are currently available. CONCLUSIONS: In a Japanese type 2 diabetes population, prescribing rapid-acting insulin aspart significantly reduced cardiovascular complications over 5- and 10-year time horizons, resulting in increased quality of life and decreased costs when compared with human insulin.