Search

Background As a system of European Reference Networks (ERNs) emerges, the differences in quality of care for patients with rare cancers may increase at national level. We aimed to elucidate the processes and healthcare planning principles through which the reference centres (RCs) for rare cancers are embedded in national health systems. Methods We used a multiple case-study design based on the experiences of Czechia, Finland, France, Italy, Lithuania and Spain. Using sarcoma as an example of rare cancer, 52 semi-structured interviews were conducted during on-site visits, including a multidisciplinary group of professionals, Ministry of Health professionals, patient representatives and European policymakers. Results The comparative analysis showed substantial heterogeneity in the processes for formalizing RCs' status and in their levels of integration in the different health systems, but two models (centre-based and the network-based) can be envisaged at national level. RCs for rare cancers were legally established only in France and Spain. Expert clinicians cooperate in a structured way, using network mechanisms, in France and Italy, and these countries, plus Finland and Lithuania, had a referral system to facilitate patients' access from non-expert centres to RCs. Seven key healthcare planning principles in instituting RCs at the national level were identified. Conclusions The conditions governing patient access to treatment centres—whether RCs or not—are decided at the national level. It is advisable to progressively align the European and national levels so that the RCs that participate in the ERNs also play a significant role at the national level. [ABSTRACT FROM AUTHOR]

Estimating incidence of rare cancers is challenging for exceptionally rare entities and in small populations. In a previous study, investigators in the Information Network on Rare Cancers (RARECARENet) provided Bayesian estimates of expected numbers of rare cancers and 95% credible intervals for 27 European countries, using data collected by population-based cancer registries. In that study, slightly different results were found by implementing a Poisson model in integrated nested Laplace approximation/WinBUGS platforms. In this study, we assessed the performance of a Poisson modeling approach for estimating rare cancer incidence rates, oscillating around an overall European average and using small-count data in different scenarios/computational platforms. First, we compared the performance of frequentist, empirical Bayes, and Bayesian approaches for providing 95% confidence/credible intervals for the expected rates in each country. Second, we carried out an empirical study using 190 rare cancers to assess different lower/upper bounds of a uniform prior distribution for the standard deviation of the random effects. For obtaining a reliable measure of variability for country-specific incidence rates, our results suggest the suitability of using 1 as the lower bound for that prior distribution and selecting the random-effects model through an averaged indicator derived from 2 Bayesian model selection criteria: the deviance information criterion and the Watanabe-Akaike information criterion. [ABSTRACT FROM AUTHOR]

Background: Although trastuzumab (T) represents the standard of care for the adjuvant treatment of HER2‐positive early‐stage breast cancer, contrasting results are available about the cardiac toxicity associated to its use. We conducted a multiregional population‐based cohort investigation aimed to assess both the short‐ and long‐term cardiovascular (CV) outcomes in women with early breast cancer treated with T‐based or standard adjuvant chemotherapy (CT). Materials and Methods: We used health care use databases of six Italian regions, overall accounting for 42% of the Italian population. The study cohort was made by all women surgically treated for breast cancer who started a first‐line adjuvant T‐based or CT treatment. Patients treated with T were 1:2 matched to those treated with CT based on date of treatment start, age, and presence of CV risk factors. Short‐ and long‐term CV outcomes (heart failure and cardiomyopathy) were measured, respectively, after 1 year and at the end of follow‐up. Results: Among 28,599 women who met the inclusion criteria, 6,208 T users were matched to 12,416 CT users. After a mean follow‐up of 5.88 years, short‐ and long‐term cumulative CV risk were 0.8% and 2.6% in patients treated with T and 0.2% and 2.8% in those treated with CT, respectively. Adjusted hazard ratios were 4.6 (95% confidence interval [CI], 2.6–8.0) for short‐term and 1.2 (95% CI, 0.9–1.6) for long‐term CV risk. Discussion: In our large real‐world investigation, T‐associated cardiotoxicity was limited to the treatment period. The addition of T to adjuvant CT did not result in long‐term worsening of CV events. Implications for Practice: Adjuvant trastuzumab‐based chemotherapy represents the backbone therapy in patients with HER2‐positive early breast cancer. Although well tolerated, cardiovascular events can manifest during or after therapy because of treatment‐related toxicities. In this wide multicenter and unselected cohort, long‐term symptomatic cardiotoxicity was low and limited to the treatment period. The findings suggest that developing tools that would be adequately able to predict cardiac toxicity at an early stage remains an important area in which additional research efforts are needed. With breast cancer patients experiencing longer survival, emphasizing their overall health through management of late and long‐term treatment effects is becoming increasingly important. This article reports a multiregional real‐world population‐based cohort investigation carried out to compare short‐term and long‐term cardiovascular risk of symptomatic cardiovascular events in women with localized breast cancer treated with trastuzumab‐based or standard adjuvant therapy in clinical practice. [ABSTRACT FROM AUTHOR]