136 research outputs found
Supplemental Material - Various phenotypes of <i>LRBA</i> gene with compound heterozygous variation: A case series report of pediatric cytopenia patients
No full textSupplemental Material for Various phenotypes of LRBA gene with compound heterozygous variation: A case series report of pediatric cytopenia patients by Jiafeng Yao, Hao Gu, Wenjun Mou, Zhenping Chen, Jie Ma, Honghao Ma, Nan Li, Rui Zhang, Tianyou Wang, Jin Jiang and Runhui Wu in International Journal of Immunopathology and Pharmacology</p
Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial
No full textBackground: fitusiran, a subcutaneous investigational small interfering RNA therapeutic, targets antithrombin to rebalance haemostasis in people with haemophilia A or haemophilia B, irrespective of inhibitor status. We evaluated the efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors. Methods: this multicentre, randomised, open-label phase 3 study was done at 26 sites (primarily secondary or tertiary centres) in 12 countries. Men, boys, and young adults aged 12 years or older with severe haemophilia A or haemophilia B with inhibitors previously treated with on-demand bypassing agents were randomly assigned (2:1) to receive once-a-month 80 mg subcutaneous fitusiran prophylaxis (fitusiran prophylaxis group) or to continue with bypassing agents on-demand (bypassing agents on-demand group) for 9 months. The primary endpoint was mean annualised bleeding rate during the efficacy period in the intention-to-treat population estimated by negative binomial model. Safety was assessed as a secondary endpoint in the safety population. This trial is complete and is registered with ClinicalTrials.gov, NCT03417102. Findings: between Feb 14, 2018, and June 23, 2021, 85 participants were screened for inclusion, of whom 57 (67%; 57 [100%] men; median age 27·0 years [IQR 19·5–33·5]) were randomly assigned: 19 (33%) participants to the bypassing agent on-demand group and 38 (67%) participants to the fitusiran prophylaxis. Negative binomial model-based mean annualised bleeding rate was significantly lower in the fitusiran prophylaxis group (1·7 [95% CI 1·0–2·7]) than in the bypassing agents on-demand group (18·1 [10·6–30·8]), corresponding to a 90·8% (95% CI 80·8–95·6) reduction in annualised bleeding rate in favour of fitusiran prophylaxis (p<0·0001). 25 (66%) participants had zero treated bleeds in the fitusiran prophylaxis group versus one (5%) in the bypassing agents on-demand group. The most frequent treatment-emergent adverse event in the fitusiran prophylaxis group was increased alanine aminotransferase in 13 (32%) of 41 participants in the safety population; there were no increased alanine aminotransferase treatment-emergent adverse events in the bypassing agents on-demand group. Suspected or confirmed thromboembolic events were reported in two (5%) participants in the fitusiran prophylaxis group. No deaths were reported. Interpretation: subcutaneous fitusiran prophylaxis resulted in statistically significant reductions in annualised bleeding rate in participants with haemophilia A or haemophilia B with inhibitors, with two-thirds of participants having zero bleeds. Fitusiran prophylaxis might show haemostatic efficacy in participants with haemophilia A or haemophilia B with inhibitors; therefore, the therapeutic might have the potential to improve the management of people with haemophilia. </p
P1619: LOME PILOT STUDY: A MULTICENTER CONTROLLED RANDOMIZED CLINICAL TRIAL OF LOW- VERSUS INTERMEDIATE-DOSE IMMUNE TOLERANCE INDUCTION WITH RECOMBINANT FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) IN CHINA
No full textThe Progress in Research in Hereditary Thrombotic Thrombocytopenic Purpura in Children
No full textHereditary thrombotic thrombocytopenic purpura (hTTP) in children is a rare but severe and fatal thrombotic microangiopathy. The etiology of the disease is the persistent severe deficiency of the enzyme ADAMTS13 gene mutation, resulting in microangiopathic hemolytic anemia, thrombocytopenia, neuropsychiatric symptoms, fever, and renal involvement. Different from adults, children with hTTP present earlier onset of the disease and are more likely to develop long-term complications in brain and kidney, so that the need for preventive replacement therapy is more urgent. This article reviews the research progress of hTTP in children
Severe congenital neutropenia associated with the ELANE gene in Chinese children: case report
No full textDevelopment and Application of Comprehensive Evaluation System in the Treatment of Children with Hemophilia in China
No full textBeing a hereditary bleeding disorder, hemophilia is characterized by spontaneous bleeding, especially joint bleeding. The treatment outcome is a comprehensive evaluation system of the following three aspects: bleeding, musculoskeletal structure (imaging), and function-activity participation. Multidisciplinary testing and corresponding scales are needed in the assessment. Among them, the quality of life assessment of hemophilia patients is particularly important, through general questionnaires and hemophilia-specific questionnaires. Canadian hemophilia outcomes-Kids' life assessment tool(CHO-KLAT), a special quality of life assessment tool for children with hemophilia, is the most widely used. This paper briefly describes the development and application of comprehensive evaluation system of the children with the disease
A Visualized Analysis of the Research Current Hotspots and Trends on Innovation Chain Based on the Knowledge Map
No full textThe continued increase in global trade protectionism, refinement of labor division, high innovation cost, and development of information technology have led to many enterprises actively being engaged in innovation to improve their national economic competitiveness. Although significant research has been carried out on this by numerous academic institutions, little is known about innovation trends in Chinese enterprises. In the existing methods of literature research, the scientific knowledge map, which, based on bibliometrics, is an effective tool for management knowledge, can visually describe the knowledge resources and their carriers under the state of time series and provides a new way for literature analysis. In this paper, the CiteSpace tool was used to map knowledge domains. A total of 459 and 5645 studies published between 2010 and 2020 were downloaded from the CNKI and Web of Science databases. By analyzing the keywords “co-occurrence matrix”, “author cooperation networks”, and “high-frequency cited literatures”, we found the differences of the research current, hotspots, and trends both in China and the world, but we were not limited to these. The research results are as follows: In China: (1) There were 759 nodes in the map of key authors, which shows that innovation chain research in China is still in the early stages. In addition, the layout of author nodes was relatively scattered while density was low; therefore, it was hard to form clusters. There is a need to strengthen academic cooperation to improve research on innovation chains. (2) From the keyword network analysis map of the innovation chain, we found that the Chinese research hotspots were: innovation chain, industrial chain, collaborative innovation, scientific and technological innovation, innovation-driven, technological innovation, strategic emerging industries, innovation ecosystem, and integration of industry and education, among other fields. In the world: (1) Most academic studies on the innovation chain have been published in different fields; these journals are about production, operation, management science, and economy, among others. These findings show that the innovation chain has received attention from multiple disciplines, and, therefore, it belongs to an interdisciplinary research field. Studies from different fields have analyzed the innovation chain from their own research perspectives. Therefore, current research outcomes on the innovation chain are difficult to unify. (2) The most important authors and key studies were analyzed. According to the co-citation map, studies on the “innovation chain” with high co-citation frequencies were not studies on the innovation chain but had the innovation chain as a theme or a concept without in-depth research on the innovation chain. (3) Through co-citation and cluster analysis of keywords, we found that international studies on the “innovation chain” are more focused on the global value chain, blockchain technology, strategic analysis, sustainable development, and absorptive capacity among other fields. Research frontier themes were mainly communication technology, continuous operation management, technological change, ecological innovation, supply chain integration, Industry 4.0, logistics innovation, nanotechnology, circular economy, and supply chain innovation, among other fields. Therefore, international scholars focus more on: technological issues related to innovation, using advanced communication technology, blockchain technology, and nanotechnology to improve innovation abilities. Moreover, they insist on sustainable development in the process of innovation, advocating for green innovation and ecological innovation. Finally, results of the visualization show that current research is mainly focused on innovation, not the innovation chain. Therefore, experts in this field should pay more attention to the study of structural stability and knowledge mobility of the innovation chain
Synthesis of Good Electrical Conductivity of CoCe-BTC/PEDOT for Ultrahigh Selectivity of NO2 Detection
No full textMetal–organic frameworks (MOFs) have broad application prospects in the development of efficient, sensitive and single select gas sensors. However, in order to construct a chemical resistance gas sensor based on MOFs, the problem of poor conductivity of MOFs must be solved. In this work, we synthesized CoCe-BTC, which based on the organic ligands of trimesic acid (H3BTC) by the water bath method and prepared CoCe-BTC/PEDOT composite film on an interdigital electrode by the spin coating process. Compared with pure MOF material, the conductivity of CoCe-BTC/PEDOT is significantly improved. Under a dry room temperature environment and N2 as the carrier gas, the response of the sensor to NO2 is about 1.2 times that of pure PEDOT and has a shorter response time. It has great repeatability and selectivity and shows a dynamic response with the change of NO2 gas concentration (5–50 ppm)
CUHK electronic theses & dissertations collection
No full textLi, Runhui.Thesis Ph.D. Chinese University of Hong Kong 2015.Includes bibliographical references (leaves ).Abstracts also in Chinese.Title from PDF title page (viewed on ...)
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