RESUMO
OBJECTIVES: Determine the effectiveness and cost of defibrotide in patients with severe hepatic sinusoidal obstruction syndrome following haematopoietic stem cell transplantation in a tertiary hospital. METHODS: A retrospective observational study. Adults or children treated with defibrotide at a mean dose of 6.8 mg/kg every 6 hours, until the total bilirubin levels are normalised, were included. Main endpoint was complete response, defined as normalised total serum bilirubin levels and resolution of multiple organ failure. Secondary endpoints were survival by 100 days post-transplant, influence of risks factors in effectiveness and cost of treatment. RESULTS: 51 patients (36 adults and 15 children) received defibrotide; median dose of defibrotide administered was 25.19 mg/kg/day (10.0-100.3). Complete response was achieved in 19 (37.3%) patients. By 100 days post-transplant complete response was achieved in 18 (35.3%) patients and the survival rate was 29 (56.9%) patients. There were no significant differences in effectiveness between adults versus paediatrics and between patients who presented risk factors. The mean cost of treatment per patient was 32 916, mean costs in adults was 104 292 and 17 394 in children. CONCLUSION: Regarding the results of our study, it is convenient to identify the patients who will be able to benefit from this treatment according to clinical conditions, medical history and prognosis. Given the great economic impact of defibrotide, we consider that more cost-effectiveness studies are required. TRIAL REGISTRATION NUMBER: EPA-OD, number LBM-DEF-2016-01.
RESUMO
BACKGROUND AND OBJECTIVE: The objective of the study was to describe the trends of utilization, supply and prevalence of hormone replacement therapy (HRT) in Spain during the period 2000-2014. METHODS: Annual prevalence of HRT use including the 95% CI was calculated for women aged≥40 using individual data from the national population-based database BIFAP. Annual and total-period consumptions were expressed in defined daily doses (DDD) per 1,000 inhabitants per day and were obtained from the databases of medications dispensed in community pharmacies and charged through official prescriptions to the Spanish National Health System. RESULTS: In the year 2000, overall HRT consumption was 33.12 DDDs/1000 inhabitans/day: 19.81 for oestrogen only, 6.88 for tibolone and 6.44 for combined oestrogen and progestagen. In 2014 overall HRT consumption was 5.32 DDDs/1000 inhabitans/day: 1.08 for oestrogen only, 1.61 for tibolone and 2.62 for combinations of oestrogen and progestagen. The marketed presentations of HRT decreased by 46.9%. Prevalence of HRT use in women aged≥40 in BIFAP was 7.19% (95% CI 6.97-7.40) in 2001 and 0.21% (95% CI 0.20-0.22) in 2014. Women aged 40-45 registered the highest prevalence of use in 2014: 0.71% (95% CI 0.66-0.76). CONCLUSIONS: A sharp decline in the consumption and prevalence of HRT has been observed in Spain since the publication of the Women's Health Initiative and Million Women Study and the regulatory measures taken restricting conditions of use, showing a similar trend to that of other western countries.