BACKGROUND: Reslizumab, a biologic targeting IL-5, has been shown to reduce asthma exacerbations and maintenance oral corticosteroid use in randomized controlled trials and pre-post studies in... Show moreBACKGROUND: Reslizumab, a biologic targeting IL-5, has been shown to reduce asthma exacerbations and maintenance oral corticosteroid use in randomized controlled trials and pre-post studies in patients with severe eosinophilic asthma. However, real-world effectiveness data of reslizumab are scarce, and it is unknown whether reslizumab has added value after switching from another type 2 biologic.OBJECTIVE: To evaluate (1) the real-world effectiveness of reslizumab on severe asthma exacerbations, maintenance oral corticosteroid use, and overall treatment response, both in biologic-naive patients who initiated reslizumab and in those who switched from another type 2 biologic; and (2) physicians' experience with reslizumab treatment.METHODS: This observational real-world study evaluated data from 134 adults with severe eosinophilic asthma included in the Dutch severe asthma registry (RAPSODI), who initiated reslizumab treatment (4-weekly infusions, 0.3 mg/kg) before April 2020 and had follow-up data for 6 months and greater. Clinical asthma experts completed surveys on their experience with reslizumab treatment.RESULTS: Overall, reslizumab reduced the exacerbation rate (odds ratio [95% CI] = 0.10 [0.05-0.21]; P<.001), oral corticosteroid use (OR [95% CI], 0.2 [0.0-0.5]; P<.001), and maintenance dose (median [CI], 5.0 [0.0-10.0] to 0.0 [0.0-5.0]; P<.001), with comparable results in biologic-naive reslizumab initiators and switchers. The overall response to reslizumab was graded good or excellent in 59.2% of patients. The additive effectiveness of reslizumab after switching from another biologic was reflected in physicians' surveys.CONCLUSIONS: Real-world data show that reslizumab reduces severe asthma exacerbations and oral corticosteroid use in patients with severe eosinophilic asthma, both in biologic-naive reslizumab initiators and in those who switched from another type 2 biologic. This additional value of reslizumab was recognized by clinical asthma experts. (C) 2022 The Authors. Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology. Show less
Javaid, M.K.; Mordenti, M.; Boarini, M.; Sangiorgi, L.; Group, E.B.W.; Westerheim, I.; ... ; Grasemann, C. 2021
Background Understanding the natural history of rare bone and mineral conditions is essential for improving clinical practice and the development of new diagnostics and therapeutics. Recruitment... Show moreBackground Understanding the natural history of rare bone and mineral conditions is essential for improving clinical practice and the development of new diagnostics and therapeutics. Recruitment and long-term participation in registries are key challenges for researchers. Methods To understand the user needs, the European Reference Network on Rare Bone Diseases (ERN BOND) and European Patient Advocacy Groups developed and implemented a multinational survey about the patient's preferred database content and functionality through an iterative consensus process. The survey was disseminated by national and international patient groups and healthcare professionals. The findings were analysed using descriptive statistics and multivariate regression. Results There were 493 eligible responses from 378 adults, 15 children and 100 parents, guardians or carers (PGC) across 22 rare bone and mineral conditions. Osteogenesis imperfecta constituted 53.4% of responses. Contents related to improving treatment and medical services scored the highest and contents about anxiety and socializing scored less highly. Additional content was recommended by 205 respondents. Respondents preferred data entry by their Healthcare Provider (HCP). However, less than 50% of adults received followup from their specialist HCP at least annually and 29% were followed up as needed. Conclusions This survey of individuals, their family, guardians and carers has prioritised the key components for an EU-based rare bone and mineral condition research database. The survey highlights issues around collecting psychosocial impacts as well as measures of HCP trust. The survey demonstrated that using only specialist centre visits for data collection, while preferred by patients, will miss a substantial number of individuals, limiting generalisability. Combined HCP and patient platforms will be required to collect representative and complete natural history data for this patient group. Show less
Willems, S.A.; Kranenburg, F.J.; Cessie, S. le; Marang-van de Mheen, P.J.; Kesecioglu, J.; Bom, J.G. van der; Arbous, M.S. 2020
Introduction: Standardization of voice outcomes indicators (VOIs) is an important issue when it comes to evaluating and comparing surgical treatments for Unilateral Vocal Fold Paralysis (UVFP). In... Show moreIntroduction: Standardization of voice outcomes indicators (VOIs) is an important issue when it comes to evaluating and comparing surgical treatments for Unilateral Vocal Fold Paralysis (UVFP). In a recent review, 11 VOIs were found to represent 80% of the VOIs cited in the literature. A survey was launched among the European laryngologists to acquire surgeons' opinions on the above mentioned preselected VOIs.Material and method: The electronic survey took place between November and December 2016. Three general questions were asked about surgeon's practice setting(s) and experience. The eleven next questions concerned (a) surgeon's VOIs preference and (b) their estimates of post-operative target values, they would consider being satisfactory.Results: The response rate was 16% (50 surveys). The majority of responders worked in tertiary hospitals (50%), had 15 years of experience with UVFP and performed on average 20 UVFP related procedures a year. The VOIs that were favored by the responding surgeons were, in decreasing order of importance, Voice handicap Index (VHI-30), Maximum Phonation Time (MPT), GRBAS-I, Mean Airflow Rate (MeAF), Jitter and Shimmer. There was an excellent consensus on post-operative VOI target values between survey's results and the literature data, except for three VOIs that showed somewhat divergent tendencies (absolute VHI-30, Jitter and Shimmer).Conclusions: Three VOIs are favored by surgeons: VHI-30, MPT and GRBAS-I. Jitter and Shimmer, although very frequently reported and statistically valid in the literature, come last concerning surgeon's choice as VOI for UVFP treatment assessment. (C) 2019 Elsevier Masson SAS. All rights reserved. Show less
Borm, C.D.J.M.; Werkmann, M.; Visser, F.; Peball, M.; Putz, D.; Seppi, K.; ... ; Vries, N.M. de 2019
The BOADICEA breast cancer (BC) risk assessment model and its associated Web Application v3 (BWA) tool are being extended to incorporate additional genetic and non-genetic BC risk factors. From an... Show moreThe BOADICEA breast cancer (BC) risk assessment model and its associated Web Application v3 (BWA) tool are being extended to incorporate additional genetic and non-genetic BC risk factors. From an online survey through the BOADICEA website and UK, Dutch, French and Swedish national genetic societies, we explored the relationships between the usage frequencies of the BWA and six other common BC risk assessment tools and respondents' perceived importance of BC risk factors. Respondents (N=443) varied in age, country and clinical seniority but comprised mainly genetics health professionals (82%) and BWA users (93%). Oncology professionals perceived reproductive, hormonal (exogenous) and lifestyle BC risk factors as more important in BC risk assessment compared to genetics professionals (p values <0.05 to 0.0001). BWA was used more frequently by respondents who gave high weight to breast tumour pathology and low weight to personal BC history as BC risk factors. BWA use was positively related to the weight given to hormonal BC risk factors. The importance attributed to lifestyle and BMI BC risk factors was not associated with the use of BWA or any of the other tools. Next version of the BWA encompassing additional BC risk factors will facilitate more comprehensive BC risk assessment in genetics and oncology practice. Show less
As the de facto industry standard for software modeling, the Unified Modeling Language (UML) is used widely across various IT domains. UML__s wide acceptance is partly because the language offers... Show moreAs the de facto industry standard for software modeling, the Unified Modeling Language (UML) is used widely across various IT domains. UML__s wide acceptance is partly because the language offers flexibility and freedom in modeling software systems: 1) UML provides an extensive set of modeling notations that can be used to model various concepts; 2) UML can be used both in a casual and formal manners. In the context of model-driven software development, the degree of freedom in which UML is used raises an important issue related to model quality. Different styles and rigors in using UML affect the quality of the resulting models. It is then logical to think that the level of quality of the UML model may affect the quality of the resulting software. This thesis reports on a series of empirical studies performed to address a pivotal question concerning the benefits of UML modeling in software development, particularly from a quality perspective. The results of these empirical studies show that the use of UML provides benefits in terms of increased quality and productivity in software development. The availability of UML models also allows early prediction of defects in software systems. Such prediction is potentially useful for identifying and fixing defects early during software development, and for prioritizing testing. Show less