Current medical research and opinion
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Objective: Clinical guidelines recommend the use of endocrine therapy (ET) in advanced hormone receptor positive (HR+) human epidermal growth factor receptor type 2 negative (HER2-) breast cancer (BC) patients in the absence of visceral disease or ET resistance. Furthermore, studies indicate similar response and survival rates using ET or cytotoxic chemotherapy (CT). Methods: Herein, we assessed clinical characteristics, type of systemic therapy and survival rates of advanced HR + HER2-BC patients in our database. ⋯ Several patients in our institution receive CT without indication. The increase in ET usage over time can be attributed to better and faster immunohistochemical detection methods for Estrogen Receptor (ER), changes in educational and government policies, and a wider variety of ET options. Finally, clinical trials have failed to demonstrate a substantial benefit of CT over ET in this setting.
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Objective: This study aimed to describe the current status of lung cancer in Spain, including patient characteristics and in-hospital mortality, and to revise disease management and the direct medical costs of secondary care. Methods: A retrospective observational study was set to analyse anonymized primary and secondary care records of patients admitted with lung cancer in Spain between 2011 and 2016. Data were obtained from the Primary Care Dataset and the Centralised Hospital Discharge Database. ⋯ Cost per patient was higher in those diagnosed with a squamous cell carcinoma. Conclusions: Preventive and early detection measures are recommended, continuing to focus on females. In parallel, a multidisciplinary approach could optimize patient journey considering the presence of disease comorbidities, although its role in lung cancer mortality should be further explored.
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Meta Analysis Comparative Study
The importance of considering differences in study and patient characteristics before undertaking indirect treatment comparisons: a case study of siponimod for secondary progressive multiple sclerosis.
Background: Indirect treatment comparisons (ITCs) provide valuable evidence on comparative efficacy where head-to-head clinical trials do not exist; however, differences in patient populations may introduce bias. Therefore, it is essential to assess between-trial heterogeneity to determine the suitability of synthesizing ITC results. We provide an illustrative case study in multiple sclerosis (MS) where we assess the feasibility of conducting ITCs between siponimod and interferon beta-1b (IFN β-1b) and between siponimod and ocrelizumab. ⋯ ITCs were not feasible between siponimod and ocrelizumab because study designs and patient populations were too dissimilar to conduct a reliable ITC. Conclusions: This study highlights the importance of conducting a detailed feasibility assessment before undertaking ITCs to illuminate when excessive between-trial heterogeneity would cause biased results. MAIC was performed for siponimod and IFN β-1b in the absence of a head-to-head trial and was considered a more valid approach than a traditional ITC to examine comparative effectiveness.
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Meta Analysis Comparative Study
Matching-adjusted indirect treatment comparison of siponimod and other disease modifying treatments in secondary progressive multiple sclerosis.
Background: Siponimod, interferon beta-1a (IFNβ-1a), IFNβ-1b and natalizumab have been evaluated as treatments for secondary progressive multiple sclerosis (SPMS) in separate randomized controlled trials (RCTs), but not head-to-head. These trials included heterogeneous patient populations, which limits the use of standard network meta-analysis (NMA) for indirect treatment comparison (ITC) of relative efficacy. Matching-adjusted indirect comparison (MAIC) aims to correct these cross-trial differences. ⋯ For annualized relapse rate (ARR), with the exception of natalizumab, siponimod was numerically but not statistically superior to all comparators. Conclusions: EXPAND provides evidence of the efficacy of siponimod compared with placebo, and these MAICs complement this by demonstrating improved efficacy of siponimod relative to DMTs. Siponimod offers a significant therapeutic advance that may slow disease progression compared to other DMTs in an EXPAND-like population with secondary progressive disease.
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Review
Health utility estimation in children and adolescents: a review of health technology assessments.
Objective: Health utility estimates for children and adolescents are critical for cost-utility analyses informing health technology assessment (HTA) authorities' decisions governing access to pediatric treatments. However, in a recent review, only 29% of published pediatric cost-utility models used a utility measure validated for children. We examined utility estimates used in pediatric HTAs. ⋯ When estimating pediatric utility weights, alternative measures should be reviewed for suitability given the model population and health condition. Pediatric and adult utility estimates should be applied appropriately as patients age over time, and caregiver and/or family member utility should be included, where relevant. Gaps exist in utility measures for children aged <4 years and caregivers.