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BACKGROUD: A huge amount of data about psychosocial issues of people with haemophilia (PwH) are available; however, these materials are fragmentary and largely outdated, failing to reflect the impact of current treatment strategies. AIM: Describing the influence of illness on psychosocial aspects of adult PwH (≥18 years) and caregivers of children with haemophilia (CPwH) without inhibitors, in Italy. METHODS: Surveys (for adult PwH, CPwH and haemophilia specialists) were developed by a multidisciplinary working group and conducted from November 2019 to June 2020. RESULTS: A total of 120 PwH without inhibitors and 79 CPwH completed the survey. Adult patients reported a significant impairment in many psychosocial aspects, including working activities, relations with family members and social relations. Caregivers generally reported better scores in all aspects of the survey. Mobility, Pain and Mental health domains of EQ-5D were the most frequently impaired in both patients and caregivers, reducing the perceived quality of life. Genetic counselling was an important issue, 53% of CPwH declaring unawareness of their carrier status, as well as the psychological support offered by the reference center, 67.0% of respondents reporting that no psychological support was provided at the time of diagnosis communication. CONCLUSION: This study provides information about PwH's and CPwH's point of view in the current scenario of continuous innovations in haemophilia treatment and management furthermore, updated insights on psychosocial problems faced by patients and caregivers are reported.
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Hemofilia A , Adulto , Niño , Humanos , Hemofilia A/terapia , Calidad de Vida , Cuidadores/psicología , Encuestas y Cuestionarios , ItaliaRESUMEN
INTRODUCTION: Considering the advances in haemophilia management and treatment observed in the last decades, a new set of value-based outcome indicators is needed to assess the quality of care and the impact of these medical innovations. AIM: The Value-Based Healthcare in Haemophilia project aimed to define a set of clinical outcome indicators (COIs) and patient-reported outcome indicators (PROIs) to assess quality of care in haemophilia in high-income countries with a value-based approach to inform and guide the decision-making process. METHODS: A Value-based healthcare approach based on the available literature, current guidelines and the involvement of a multidisciplinary group of experts was applied to generate a set of indicators to assess the quality of care of haemophilia. RESULTS: A final list of three COIs and five PROIs was created and validated. The identified COIs focus on two domains: musculoskeletal health and function, and safety. The identified PROIs cover five domains: bleeding frequency, pain, mobility and physical activities, Health-Related Quality of Life and satisfaction. Finally, two composite outcomes, one based on COIs, and one based on PROIs, were proposed as synthetic outcome indicators of quality of care. CONCLUSION: The presented standard set of health outcome indicators provides the basis for harmonised longitudinal and cross-sectional monitoring and comparison. The implementation of this value-based approach would enable a more robust assessment of quality of care in haemophilia, within a framework of continuous treatment improvements with potential added value for patients. Moreover, proposed COIs and PROIs should be reviewed and updated routinely.
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Hemofilia A , Humanos , Hemofilia A/tratamiento farmacológico , Calidad de Vida , Estudios Transversales , Atención Médica Basada en Valor , Evaluación de Resultado en la Atención de SaludRESUMEN
BACKGROUND AND PURPOSE: Essential tremor (ET) is one of the most common neurological disorders, but information on treatment pattern is still scant. The aim of this study was to describe the demographic and clinical characteristics, treatment patterns, and determinants of drug use in patients with newly diagnosed ET in France and the United Kingdom. METHODS: Incident cases of ET diagnosed between January 1, 2015 and December 31, 2018 with 2 years of follow-up were identified by using The Health Improvement Network (THIN®) general practice database. During the follow-up, we assessed the daily prevalence of use and potential switches from first-line to second-line treatment or other lines of treatment. Logistic regression models were conducted to assess the effect of demographic and clinical characteristics on the likelihood of receiving ET treatment. RESULTS: A total of 2957 and 3249 patients were selected in the United Kingdom and France, respectively. Among ET patients, drug use increased from 12 months to 1 month prior the date of index diagnosis (ID). After ID, nearly 40% of patients received at least one ET treatment, but during follow-up drug use decreased and at the end of the follow-up approximately 20% of patients were still on treatment. Among treated patients, ≤10% maintained the same treatment throughout the entire follow-up, nearly 20% switched, and 40%-75% interrupted any treatment. Results from the multivariate analysis revealed that, both in France and the United Kingdom, patients receiving multiple concomitant therapies and affected by psychiatric conditions were more likely to receive an ET medication. CONCLUSION: This study shows that ET is an undertreated disease with a lower-than-expected number of patients receiving and maintaining pharmacological treatment. Misclassification of ET diagnosis should be acknowledged; thus, results require cautious interpretation.
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Temblor Esencial , Humanos , Temblor Esencial/diagnóstico , Temblor Esencial/tratamiento farmacológico , Temblor Esencial/epidemiología , Atención Primaria de Salud , Reino Unido/epidemiología , Francia/epidemiología , Estudios RetrospectivosRESUMEN
The European Liver and Intestine Transplant Association, ELITA, promoted a Consensus Conference involving 20 experts across the world which generated updated guidelines on HBV prophylaxis in liver transplant candidates and recipients. This study explores the economic impact associated with the implementation of the new ELITA guidelines. To this aim, a condition-specific cohort simulation model has been developed to compare new and historical prophylaxis, including only pharmaceutical cost and using the European perspective. The target population simulated in the model included both prevalent and incident cases, and consisted of 6,133 patients after the first year, that increased to 7,442 and 8,743 patents after 5 and 10 years from its implementation. The ELITA protocols allowed a cost saving of around 235.65 million after 5 years and 540.73 million after 10 years; which was mainly due to early HIBG withdrawal either after the first 4 weeks or after the first year post Liver Transplantation (LT) depending on the virological risk at transplantation. Results were confirmed by sensitivity analyses. The money saved by the implementation of the ELITA guidelines would allow healthcare decision makers and budget holders to understand where costs could be reduced and resources re-allocated to different needs.
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Hepatitis B , Trasplante de Hígado , Humanos , Antivirales/uso terapéutico , Hepatitis B/prevención & control , Quimioterapia CombinadaRESUMEN
Background and Objectives: This study aimed to assess the effectiveness and costs associated with pharmacokinetics-driven (PK) prophylaxis based on the myPKFiT® device in patients affected by hemophilia A (HA) in Italy. Materials and Methods: An observational retrospective study was conducted in three Italian hemophilia centers. All patients with moderate or severe HA, aged ≥ 18 years, capable of having PK estimated using the myPKFiT device, and who had had a clinical visit between 1 November 2019 and 31 March 2022 were included. Differences in clinical, treatment, health resources, and cost data were assessed comparing post-PK prophylaxis with pre-PK. The incremental cost-effectiveness ratio (ICER) was estimated as cost (EUR) per bleed avoided. Results: The study enrolled 13 patients with HA. The mean annual bleeding rate decreased by -1.45 (-63.80%, p = 0.0055) after the use of myPKFiT®. Overall, the consumption of FVIII IU increased by 1.73% during follow-up compared to the period prior the use of the myPKFiT. Prophylaxis based on the myPKFiT resulted in an ICER of EUR 5099.89 per bleed avoided. Conclusions: The results of our study support the idea that the use of PK data in clinical practice can be associated with an improvement in the management of patients, as well as clinical outcomes, with a reasonable increase in costs.
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Hemofilia A , Humanos , Hemofilia A/complicaciones , Hemofilia A/tratamiento farmacológico , Análisis de Costo-Efectividad , Estudios Retrospectivos , Recursos en Salud , ItaliaRESUMEN
BACKGROUND: Sarcopenia gained importance in the evaluation of patients with chronic respiratory diseases, including idiopathic pulmonary fibrosis (IPF), since it may impact negatively on clinical outcomes. AIM: Aim of this study is to evaluate the prevalence and factors associated with sarcopenia, defined according to the European Working Group on Sarcopenia in Older People 2 (EWGSOP2) 2019 definition, and to evaluate the prevalence of the single criteria that define the EWGSOP2 definition (muscle strength, muscle quantity and physical performance), in a cohort of consecutive patients with IPF prospectively followed up in 9 hospitals in Northern Italy between December 2018 and May 2021. METHODS: Enrolled patients underwent an extensive pulmonary and nutritional assessment, including bioelectrical impedance analysis, dynamometry and 4-m gait speed test, both at IPF diagnosis and at 6-month follow-up. RESULTS: Out of the 83 patients (81% males, mean age 72.5 years) with IPF at disease diagnosis enrolled in the study, 19 (22.9%) showed sarcopenia, including 2 (2.4%) with severe sarcopenia, 5 (6.0%) with confirmed sarcopenia and 12 (14.5%) with probable sarcopenia. Sarcopenia was associated with a significantly higher severity of the disease and sedentary lifestyle, while no differences were observed in regards to body mass index, history of weight loss and comorbidities between patients with and without sarcopenia. Out of the 64 patients without sarcopenia at baseline, 16 cases showed alteration of muscle quantity and/or physical performance. In the 51 patients with complete data at 6-month follow-up, there were no cases of severe sarcopenia, 1 case (2.0%) showed confirmed sarcopenia, while the prevalence of probable sarcopenia was 19.6% (10 cases). No differences in regards to antifibrotic treatment received and onset of gastrointestinal side effects were observed between patients with and without sarcopenia at follow-up. CONCLUSIONS: The prevalence of sarcopenia in patients with IPF both at diagnosis and at 6-month follow-up was low but not negligible and was associated with higher severity of the disease and sedentary lifestyle. In IPF patients, a comprehensive diagnostic work-up including all the criteria defining the EWGSOP2 definition might be more useful than a series testing for prompt recognition of nutritional and physical performance abnormalities.
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Fibrosis Pulmonar Idiopática , Sarcopenia , Anciano , Femenino , Fuerza de la Mano/fisiología , Humanos , Fibrosis Pulmonar Idiopática/diagnóstico , Fibrosis Pulmonar Idiopática/epidemiología , Masculino , Prevalencia , Estudios Prospectivos , Sarcopenia/diagnóstico , Sarcopenia/epidemiologíaRESUMEN
The need to reduce the burden of injections, and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half-life (EHL-rFVIII) in comparison with standard half-life products (SHL-rFVIII). Lack of head-to-head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between-product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL-rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL-rFVIII as a comparator. In the frame of these clinical studies, all the EHL-rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between-product differences in terms of reduction in injection frequencies. Further, between-product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL-rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real-life data.
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Factor VIII/uso terapéutico , Hemofilia A , Semivida , Hemofilia A/tratamiento farmacológico , Humanos , Plasma , Proteínas Recombinantes/uso terapéutico , Resultado del TratamientoRESUMEN
BACKGROUND: A shift from a standard to a personalized prophylaxis has been increasingly adopted in patients with severe haemophilia A (SHA). This approach has raised the likelihood of a significant variability in the prophylactic approaches and the relative Factor VIII (FVIII) consumptions. The aim of our study was to assess the treatment variability of SHA patients without inhibitors and on prophylaxis regimen in Italy. MATERIAL AND METHODS: Data reported in the National Registry of Congenital Coagulopathies (NRCC) were analysed to assess treatment distribution within SHA patients without inhibitors, focussing on FVIII consumption in 2017, associated with prophylaxis regimen. The analysis was stratified based on age groups and Italian regions to describe the variability of FVIII consumption in Italy. RESULTS: In 2017, the Registry reported the therapeutic plans of 1068 SHA patients without inhibitors on prophylaxis. The mean (95% CI) individual consumption ranges from 123 127 IU (99 736-146 518) in the age group 0-6 years to 345 000 IU (336 000-354 000) in the age group >20 years. A significant FVIII consumption variability was identified within the adult population. Regions with less than 50 patients reported the higher variability in mean FVIII consumption per patient-year within the different age groups. Similar difference in FVIII consumption variability was reported also in the age groups comparing "low," "middle" and "high" patient volume regions. DISCUSSION: A reliable estimation of FVIII consumption for patients' treatment is necessary to manage and plan the appropriate budget and keep treatment's costs affordable. However, without the implementation of a methodology aiming to assess the overall value produced by these FVIII consumptions, the scenario will keep driven by FVIII consumptions, its costs and the budget available. An effort by haemophilic community, haemophilia treatment centres and institutions is required to develop and share this cultural shift in improving haemophilia management and assessment.
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Quimioprevención/métodos , Costos de los Medicamentos/estadística & datos numéricos , Factor VIII/uso terapéutico , Hemofilia A/tratamiento farmacológico , Medicina de Precisión/métodos , Sistema de Registros , Adolescente , Adulto , Factores de Edad , Quimioprevención/economía , Niño , Preescolar , Esquema de Medicación , Factor VIII/economía , Femenino , Hemofilia A/sangre , Hemofilia A/economía , Hemofilia A/patología , Humanos , Lactante , Recién Nacido , Italia , Masculino , Medicina de Precisión/economía , Índice de Severidad de la EnfermedadRESUMEN
UNLABELLED: In the current context of rising health care costs and decreasing sustainability, it is becoming increasingly common to resort to decision analytical modeling and health economics evaluations. Decision analytic models are analytical tools that help decision makers to select the best choice between alternative health care interventions, taking into consideration the complexity of the disease, the socioeconomic context, and the relevant differences in outcomes. We present a brief overview of the use of decision analytical models in health economic evaluations and their applications in the area of liver diseases. The aim is to provide the reader with the basic elements to evaluate health economic analysis reports and to discuss some limitations of the current approaches, as highlighted by the case of the therapy of chronic hepatitis C. To serve its purpose, health economics evaluations must be able to do justice to medical innovation and the market while protecting patients and society and promoting fair access to treatment and its economic sustainability. CONCLUSION: New approaches and methods able to include variables such as prevalence of the disease, budget impact, and sustainability into the cost-effectiveness analysis are needed to reach this goal. (Hepatology 2016;64:1331-1342).
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Tecnología Biomédica/economía , Análisis Costo-Beneficio , Hepatopatías , Técnicas de Apoyo para la Decisión , Costos de la Atención en Salud , Humanos , Hepatopatías/economía , Modelos EconómicosRESUMEN
BACKGROUND: The adjuvanted recombinant zoster vaccine (RZV), consisting of varicella-zoster virus glycoprotein E (gE) and the AS01B adjuvant system, effectively prevents herpes zoster (HZ). In the absence of a well-defined correlate of protection, it is important to monitor the RZV immune response, as a proxy of clinical effectiveness. METHODS: This systematic review examined post-vaccination parameters: humoral and cell-mediated immunity, avidity index, geometric mean concentration of antibody (GMC), and immunity persistence. The meta-analysis used a random-effects model, and subgroup and meta-regression analyses were conducted. RESULTS: Among 37 included articles, after one month from RZV-dose 2, the pooled response rate for anti-gE humoral immunity was 95.2% (95%CI 91.9-97.2), dropping to 77.6% (95%CI 64.7-86.8) during immunosuppression. The anti-gE cell-mediated immunity-specific response reached 84.6% (95%CI 75.2-90.9). Varying factors, such as age, sex, coadministration with other vaccines, prior HZ, or live-attenuated zoster vaccine, did not significantly affect response rates. RZV induced a substantial increase in gE avidity. Immunity persistence was confirmed, with more rapid waning in the very elderly. CONCLUSIONS: This systematic review indicates that RZV elicits robust immunogenicity and overcomes immunocompromising conditions. The findings underscore the need for further research, particularly on long-term immunity, and have the potential to support HZ vaccination policies and programs.
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AIM: In 2022, in Italy, general practitioners (GPs) have been allowed to prescribe SGLT2i in Type 2 Diabetes (T2D) under National Health Service (NHS) reimbursement. In the pivotal clinical trial named DECLARE-TIMI 58, dapagliflozin reduced the risk of hospitalization for heart failure, CV death and kidney disease progression compared to placebo in a population of T2D patients. This study evaluated the health and economic impact of dapagliflozin for T2D patients who had or were at risk for atherosclerotic cardiovascular disease in the Italian GPs setting. METHODS: A budget impact model was developed to assess the health and economic impact of introducing dapagliflozin in GPs setting. The analysis was conducted by adopting the Italian NHS perspective and a 3-year time horizon. The model estimated and compared the health outcomes and direct medical costs associated with a scenario with dapagliflozin and other antidiabetic therapies available for GPs prescription (scenario B) and a scenario where only other antidiabetic therapies are available (scenario A). Rates of occurrence of cardiovascular and renal complications as well as adverse events were captured from DECLARE-TIMI 58 trial and the literature, while cost data were retrieved from the Italian tariff and the literature. One-way sensitivity analyses were conducted to test the impact of model parameters on the budget impact. RESULTS: The model estimated around 442.000 patients eligible for the treatment with dapagliflozin in the GPs setting for each simulated year. The scenario B compared to scenario A was associated with a reduction in the occurrence of cardiovascular and renal complication (-1.83%) over the 3 years simulated. Furthermore, the scenario A allowed for an overall cost saving of 102,692,305: 14,521,464 in the first year, 33,007,064 in the second and 55,163,777 in the third. The cost of cost of drug acquisition, the probability of cardiovascular events and the percentage of patients potentially eligible to the treatment were the factor with largest impact on the results. CONCLUSIONS: The use of dapagliflozin in GPs setting reduce the number of CVD events, kidney disease progression and healthcare costs in Italy. These data should be considered to optimize the value produced for the T2D patients who had or were at risk for atherosclerotic cardiovascular disease.
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BACKGROUND: This study was aimed at estimating the appropriate price of tucatinib plus trastuzumab and capecitabine (TXC), as third-line treatment, in HER2+ breast cancer (BC) patients from the Italian National Health System (NHS) perspective. METHODS: A partitioned survival model with three mutually exclusive health states (i.e., progression-free survival (PFS), progressive disease (PD), and death) was used to estimate the price of tucatinib vs trastuzumab emtansine (TDM-1), considering a willingness to pay (WTP) of 60,000 EUR. Data from the HER2CLIMB trial, the Italian population, and the literature were used as input. The model also estimated the total costs and the life-years (LY) of TXC and TDM1. Deterministic and probabilistic (PSA) sensitivity analyses were conducted to evaluate the robustness of the model. RESULTS: In the base case scenario, the appropriate price of tucatinib was 4828.44 EUR per cycle. The TXC resulted in +0.28 LYs and +16,628 EUR compared with TDM-1. Results were mainly sensitive to therapy intensity variation. In PSA analysis, TXC resulted cost-effective in 53% of the simulations. Assuming a WTP ranging 20,000-80,000 EUR, the tucatinib price ranged from 4090.60 to 5197.41 EUR. CONCLUSIONS: This study estimated the appropriate price for tucatinib according to different WTP in order to help healthcare decision makers to better understand the treatment value.
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BACKGROUND: There are no data to assess the value associated with a treat-to-target (T2T) strategy based on tight control of mild-moderate ulcerative colitis (UC). AIM: To assess the cost-effectiveness of a T2T approach based on the normalisation of clinical signs and faecal calprotectin (FC) METHODS: A decision analytical Markov model was developed to compare T2T algorithm combining clinical symptoms and FC levels to define treatment response and the possible switch to the next treatment line (T2T-FC), and the reference strategy based only on symptoms. The model included five treatment lines and was conducted from the Italian national health service (NHS) perspective using a 3-year time horizon. The model calculated the incremental cost-effectiveness ratio as per relapse avoided. Deterministic and probabilistic sensitivity analyses were conducted. RESULTS: The cost-effectiveness analysis produced an increased time spent by a patient in clinical remission and FC ≤ 100 level (+0.177 years; about 2 months) and a decreasing number of relapses (-0.1937; -20.9%) per patient using a T2T-FC approach compared to only symptoms. Furthermore, the T2T-FC was associated with higher cost (+1795). The ICER estimated was 9263 per relapse avoided. These results were confirmed by sensitivity analyses. CONCLUSIONS: T2T-FC approach resulted in a higher benefit for mild-moderate UC patients in terms of time in remission and incidence of relapse but was associated with higher costs. Clinical trials and real-world clinical studies are needed to provide additional data on the cost-benefit of this approach.
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Colitis Ulcerosa , Humanos , Colitis Ulcerosa/diagnóstico , Colitis Ulcerosa/tratamiento farmacológico , Análisis Costo-Beneficio , Medicina Estatal , Análisis de Costo-Efectividad , RecurrenciaRESUMEN
BACKGROUND: Recent developments improved outcomes in patients with autoimmune diseases. Biologics were approved as first-line treatment in selected naïve patients with plaque psoriasis (PsO), psoriatic arthritis (PsA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA). Among them, secukinumab was most recently approved for treatment of active nr-axSpA in adults. In this work, we assessed the budget impact of new secukinumab treatment options in the Italian market. METHODS: A cross-indication budget impact model was designed to estimate the effects of adding secukinumab in the Italian market from the National Health System perspective over a 3-year period. The model included all adults with PsO, PsA, AS and nr-axSpA, treated with biologics or biosimilars. It compared costs between two scenarios, secukinumab availability or absence, for the four diseases combined and taken individually. A sensitivity analyses was conducted. RESULTS: There were 68,121 adult patients treated with biologics in 2021 and 68,341 in 2023. The budget impact analysis (BIA) on all indications showed a cost reduction of 33.7 million (- 1.5%) over 3 years with the introduction of secukinumab. PsA patients had the highest saving (- 34.9 million), followed by PsO patients (- 7.8 million). Cost saving in PsO patients was balanced by increased budget reported in AS patients (+ 8.0 million). In nr-axSpA patients, secukinumab reported no significant budget increase (+ 1.0%). CONCLUSION: This BIA accounted for the new indication of secukinumab in nr-axSpA patients, reporting no significant changes in the required budget and adding an effective treatment option. Considering all indications, secukinumab is a sustainable treatment option.
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The successful deployment of safe and effective vaccines against coronavirus disease 2019 (COVID-19) has been crucial in reducing the global disease burden. Owing to the need for vaccination series over time, continuous observational studies are needed to estimate the COVID-19 vaccine response in real-world conditions. In particular, the detection, assessment, and understanding of adverse effects following immunization (AEFI) with a COVID-19 vaccine are crucial to better address vaccination strategies. Therefore, this study aimed to investigate the risk of repeated AEFI post-administration of a booster dose of mRNA COVID-19 vaccine in a sample of healthcare workers (HCWs) in an Italian teaching hospital. The data on any local and systemic AEFI were studied in multivariate Poisson regression analyses to model the association between the incidence of each postvaccination symptom and its prior reporting after the administration of the previous doses. Overall, compared with the primary vaccination series, the majority of post-third dose AEFI were less reported. The results from multivariable models showed that the likelihood of reporting an AEFI after the third dose was higher in those who experienced the same postvaccination symptom after the second dose (all AEFI except for itch at injection site) and, although not significant for all AEFI, after the first dose. Any associations with age, gender, smoking habits, previous SARS-CoV-2 infection and other characteristics, as well as the health impact of AEFI were also assessed. Taken together, the results from this research support reframe AEFI symptoms as signals of a robust postvaccination reaction as well as of common vaccine response, and they add important data to inform booster vaccination strategies in HCWs and, extensively, in the adult population.
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Surveillance costs and appropriateness of surgery of "low-risk" BD-IPMNs are relevant issues. In this study we evaluated the rate of correct indication for pancreatectomy defined as high grade dysplasia (HGD) at histology in 961 patients who underwent surveillance for a median of 5.1 years. Undertreatment and overtreatment were defined as invasive cancer and low grade dysplasia (LGD) at histology, respectively. Of the 66 patients (6.9%) who were operated, only 16 (23.8%) had a HGD while 40 (59.7%) had a LGD and 10 (14.9%) an invasive cancer, without differences regarding timing of surgery. The mean surveillance cost was 194.9 ± 107.6 per patient-year, with a median cost of 277.1 ± 148.2 in the correct surgery group compared with 222.7 ± 111.6 and 197 ± 102.7 in the overtreatment and undertreatment groups. The surveillance mean cost from diagnosis to surgery was 854.8. Rate of appropriate surgery in BD-IPMNs under surveillance is low.
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Adenocarcinoma Mucinoso , Carcinoma Ductal Pancreático , Neoplasias Pancreáticas , Humanos , Carcinoma Ductal Pancreático/patología , Adenocarcinoma Mucinoso/patología , Estudios Retrospectivos , Neoplasias Pancreáticas/patología , Riesgo , PancreatectomíaRESUMEN
INTRODUCTION: Clinical trials confirmed the beneficial effects of adding pertuzumab (P) to the combination of trastuzumab-chemotherapy (TC) in the (neo)adjuvant setting of high-risk HER2-positive early breast cancer (HER2+BC). We evaluated the clinical, economic and societal impact of adding pertuzumab to neoadjuvant TC combination (TPC) in Italy. METHODS: A cost-consequence analysis comparing TPC vs. TC was performed developing a cohort-based multi-state Markov model to estimate the clinical, societal and economic impact of the neoadjuvant therapy of TPC versus TC in HER2+BC at high-risk of recurrence. The model works on a cycle length of 1 month and 5-years-time horizon. Literature review-based data were used to populate the model. The following clinical and economic outcomes were estimated: cumulative incidence of loco-regional/distant recurrences, life of years and QALY and both direct and indirect costs (). Finally, sensitivity analyses were performed. RESULTS: TPC was associated with a 75,630 saved of direct costs. Specifically, it was associated with an initial increase of treatment costs (+4.8%) followed by reduction of recurrence management cost (-20.4%). TPC was also associated with an indirect cost reduction of 1.40%, as well as decreased incidence of distant recurrence (-20.14%), days of work lost (-1.53%) and days lived with disability (-0.50%). Furthermore, TPC reported 10,47 QALY gained (+2.77%) compared to TC. The probability to achieve the pathological complete response (pCR) was the parameter that mostly affected the results in the sensitivity analysis. CONCLUSION: Our findings suggested that TPC combination could be a cost-saving option in patients with HER2+BC at high-risk of recurrence.
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Neoplasias de la Mama , Humanos , Femenino , Neoplasias de la Mama/patología , Terapia Neoadyuvante/métodos , Receptor ErbB-2/análisis , Trastuzumab/uso terapéutico , Italia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéuticoRESUMEN
Therapies for psoriatic arthritis were inadequate until a short time ago. Nonsteroidal antiinflammatory drugs are helpful in relieving symptoms but do not prevent joint damage. Traditional disease-modifying antirheumatic drugs are used to control symptoms, but there is no evidence that they prevent or significantly slow the progression of structural damage in peripheral joints. The introduction of tumor necrosis factor-α (TNF-α) blocking agents has opened new horizons. These drugs lessen signs and symptoms of inflammation, enhance functional capacity and quality of life, and inhibit structural joint damage. On the other hand, TNF-α blockers are very costly and not easily available to all patients, whether they rely on a national health system or on private insurance. Pharmacoeconomic studies on these drugs so far have shown that they are cost-effective on both the musculoskeletal and skin manifestations of psoriatic disease, offering good value for money.
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Antiinflamatorios/economía , Antiinflamatorios/uso terapéutico , Artritis Psoriásica/tratamiento farmacológico , Artritis Psoriásica/economía , Costos de los Medicamentos , Artritis Psoriásica/diagnóstico , Artritis Psoriásica/inmunología , Análisis Costo-Beneficio , Accesibilidad a los Servicios de Salud/economía , Humanos , Articulaciones/efectos de los fármacos , Articulaciones/inmunología , Articulaciones/patología , Años de Vida Ajustados por Calidad de Vida , Factores de Tiempo , Resultado del Tratamiento , Factor de Necrosis Tumoral alfa/antagonistas & inhibidores , Factor de Necrosis Tumoral alfa/metabolismoRESUMEN
Background: Epidemiological investigations define the prevalence and distribution of the various types of malocclusions, and can help to identify etiological factors and set the most correct orthodontic therapy. Aim: The goal of this study was to verify the prevalence and distribution of various types of malocclusions in the pediatric population. Methods: The study was performed on a sample of 350 patients (ages 5-9) being treated at the ASST Grande Ospedale Metropolitano Niguarda in Milan. A comparison was presented with one similar epidemiological investigation conducted 22 years earlier by the same researchers. The values of the malocclusion indices were reported from the cephalometric analyzes of the patients and were differentiated on the basis of gender and ethnicity. Results: The predominant traits of malocclusion in the general population of the analyzed sample were: skeletal class II (47.43%), hyper-divergence (40.86%), maxillary retrusion (46%), mandibular retrusion (66%), maxillary hypoplasia (50%), mandibular hypoplasia (49.14%), Wits index > 2 mm (22.57%); overjet > 4 mm (31.1%) and overbite > 4 mm (24.86%). Substantial differences were found between Italian patients and patients belonging to different ethnic groups in almost all parameters, and between the male and female genders in some of them. Patients in the 2000 study had a higher prevalence of Class II and hyper-divergence. Conclusions: This epidemiological investigation can suggest different approaches in setting the orthodontic treatment plan based on the ethnic group of the patient taken in charge and encourage more specific and large-scale analytical studies on the subject.
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Maloclusión Clase II de Angle , Maloclusión , Sobremordida , Humanos , Femenino , Masculino , Niño , Preescolar , Maloclusión/epidemiología , Sobremordida/terapia , Maloclusión Clase II de Angle/terapia , Cefalometría , PrevalenciaRESUMEN
BACKGROUND: Siponimod is an effective treatment for patients with secondary progressive multiple sclerosis (SPMS), with active disease evidenced by relapses or imaging features characteristic of multiple sclerosis inflammatory activity, however there is a need to evaluate its economic value and sustainability compared to other disease modifying-therapies (DMTs). OBJECTIVE: To estimate the siponimod cost-effectiveness profile and its relative budget impact compared with other DMTs, by using the Italian National Healthcare System perspective. METHODS: We performed: 1) a cost-effectiveness analysis (CEA) vs interferon beta-1b using an analytical Markov model and a life time-horizon, and 2) a budget impact analysis by using 3-years time-horizon. The results were reported as incremental cost-effectiveness ratio (ICER) and net-monetary benefit (NMB) for CEA, using a willingness to pay threshold of 40,000 per QALY gained, and as difference in the overall budget (Euro) between the scenario with and without siponimod for budget impact. RESULTS: In the base case scenario siponimod resulted cost-effective compared with interferon beta-1b 28,891 per QALY. Overall, the market access of siponimod was associated to an increased budget of about 3 millions (+0.9%) in the next 3 years simulated. CONCLUSION: Compared to interferon beta-1b, siponimod seems to be cost-effective in SPMS patients and sustainable, with less than 1% overall budget increased in the next 3 years. Future studies need to confirm our results in the real word setting and in other countries.