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首个血友病A凝血因子Ⅷ替代治疗的周制剂efanesoctocog alfa
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作者 黄倩倩 林丽蓉 《中国新药与临床杂志》 北大核心 2025年第6期430-434,共5页
efanesoctocog alfa是首个突破血管性血友病因子(vWF)限制的长效凝血因子Ⅷ药物,能提供更持久的出血保护,防止出血发作,可减少频繁输注凝血因子Ⅷ,由美国食品和药品管理局批准上市,用于成人和儿童血友病A(先天性凝血因子Ⅷ不足)的常规... efanesoctocog alfa是首个突破血管性血友病因子(vWF)限制的长效凝血因子Ⅷ药物,能提供更持久的出血保护,防止出血发作,可减少频繁输注凝血因子Ⅷ,由美国食品和药品管理局批准上市,用于成人和儿童血友病A(先天性凝血因子Ⅷ不足)的常规预防和按需治疗。临床试验显示,efanesoctocog alfa半衰期长,耐受性良好,显著降低血友病A患者出血频率,支持每周1次给药,长期安全性高,最常见的不良反应为头痛和关节痛。 展开更多
关键词 efanesoctocog alfa 血友病A 凝血因子Ⅷ 血管性血友病因子
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用于成年患者法布雷病治疗的长效制剂pegunigalsidase alfa-iwxj
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作者 刘彬 杜小莉 《中国新药杂志》 CAS CSCD 北大核心 2024年第18期1875-1878,共4页
法布雷病(Fabry disease,FD)是一种与X染色体关联的伴性遗传的溶酶体贮积病,收录在我国第一批罕见病目录(2018)中。pegunigalsidase alfa-iwxj(商品名:Elfabrio^(TM))是美国FDA批准的第1款用于成年患者法布雷病治疗的长效制剂,其在体内... 法布雷病(Fabry disease,FD)是一种与X染色体关联的伴性遗传的溶酶体贮积病,收录在我国第一批罕见病目录(2018)中。pegunigalsidase alfa-iwxj(商品名:Elfabrio^(TM))是美国FDA批准的第1款用于成年患者法布雷病治疗的长效制剂,其在体内平均半衰期长达80 h,仅需每2周静脉输注1次。临床研究证实,pegunigalsidase alfa-iwxj对成年法布雷病患者的心肾功能改善效果不劣于第1代酶替代治疗(enzyme replacement therapy,ERT)药物。在使用期间需注意监测药品不良反应的发生以及抗药性抗体的滴度。本文就pegunigalsidase alfa-iwxj的作用机制、药动学、临床疗效、安全性等方面进行述评。 展开更多
关键词 法布雷病 溶酶体贮积症 酶替代疗法 长效制剂 pegunigalsidase alfa-iwxj
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凝血因子Ⅹa直接抑制剂的特异性逆转剂:andexanet alfa 被引量:1
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作者 赵淑娟 洪雪姣 +3 位作者 马培志 赵飞 陈博雅 曹晶晶 《中国新药与临床杂志》 CAS CSCD 北大核心 2019年第1期14-18,共5页
andexanet alfa是凝血因子Ⅹa直接抑制剂的特异性逆转剂,于2018年5月3日由美国食品和药物管理局批准上市,用于逆转利伐沙班、阿哌沙班的抗凝作用。andexanet alfa需弹丸式静脉注射,随后给予2 h持续静脉输注的给药方案,具有良好的安全性。
关键词 andexanet alfa 抗凝药 出血 解毒药
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评价蔡氏公式用于接受Turoctocog alfa治疗的中国重型血友病A经治患者的准确性 被引量:3
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作者 蔡力生 帕拉赛提·阿地力 +3 位作者 杜新 刘贝妮 吴优 杨仁池 《血栓与止血学》 CAS 2022年第6期1214-1220,共7页
目的在接受重组人凝血因子Ⅷ(rhFⅧ)Turoctocog alfa治疗的中国重型血友病A经治患者中评价蔡氏公式的准确性。方法纳入临床试验Guardian 7(NCT02938585)中国重型血友病A经治患者,均接受Turoctocog alfa 50±5 IU/kg单剂量输注,按不... 目的在接受重组人凝血因子Ⅷ(rhFⅧ)Turoctocog alfa治疗的中国重型血友病A经治患者中评价蔡氏公式的准确性。方法纳入临床试验Guardian 7(NCT02938585)中国重型血友病A经治患者,均接受Turoctocog alfa 50±5 IU/kg单剂量输注,按不同年龄组(<12岁和≥12岁)方案抽取血样并采用显色法测定rhFⅧ半衰期T_(1/2)-G。根据基线和第3小时后2个时间点的FⅧ浓度,应用蔡氏公式计算T_(1/2)-C。基于由T_(1/2)-C计算的第Ⅲ相PK曲线,估算峰浓度(C_(max)⁃C)、第Ⅲ相任意时间点血药浓度(C_(t)⁃C)和增量回收率(IVR⁃C)。对由蔡氏公式计算得到的PK参数与实际PK参数进行配对Wilcoxon符号秩和检验、Pearson相关系数分析。结果共纳入17例经治患者(年龄3~44岁),其中15例患者的FⅧ浓度可用于蔡氏公式计算。T_(1/2)-C、C_(max)⁃C的标准差(s)相对于各自的平均值(X)均较小,T_(1/2)-C、C_(max)⁃C与实际检测的T_(1/2)⁃G、C_(max)⁃G柱状图均基本重合,T_(1/2)-C与T_(1/2)⁃G、C_(max)⁃C与C_(max)⁃G数据之间差异均无统计学意义(分别为P=0.2293和P=0.5591)且均有高相关性(分别为r=0.958和r=0.987;P<0.01)。蔡氏公式计算得到的第Ⅲ相PK曲线与实际检测的PK模拟曲线高度重合。结论在接受Turoctocog alfa治疗的中国重型血友病A经治患者中,蔡氏公式可用于计算第Ⅲ相PK曲线,对重型血友病A个体化预防治疗有一定指导作用。 展开更多
关键词 血友病A 蔡氏公式 重组人凝血因子Ⅷ Turoctocog alfa 个体化预防治疗
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关于脑内灌注Cerliponase alfa治疗CLN2疾病的综述
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作者 王丹 孙婷婷 +1 位作者 孟雪莲 陈长兰 《辽宁大学学报(自然科学版)》 CAS 2019年第3期237-243,共7页
迟发性婴儿型2型神经元蜡样脂褐质沉积症(late infantile neuronal ceroid lipofuscinosis type 2,CLN2)又称为三肽基肽酶-1(TPP1)缺乏症,是Batten病的一种.Cerliponase alfa(Brineura^TM)是一种用于治疗CLN2的药物.文章综述了CLN2的病... 迟发性婴儿型2型神经元蜡样脂褐质沉积症(late infantile neuronal ceroid lipofuscinosis type 2,CLN2)又称为三肽基肽酶-1(TPP1)缺乏症,是Batten病的一种.Cerliponase alfa(Brineura^TM)是一种用于治疗CLN2的药物.文章综述了CLN2的病理特征、TPP1酶和cerliponase alfa(Brineura^TM)的使用方法和注意事项,为CLN2疾病的进一步研究提供参考. 展开更多
关键词 cerliponase alfa(Brineura TM) CLN2 TPP1酶 酶替代疗法 脑室灌注
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ALFA LAVAL S型分油机故障分析及处理 被引量:1
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作者 贾小平 于风卫 《航海技术》 2015年第6期52-54,共3页
某大型集装箱船主机型号为MAN B&W 10K98MC,缸径为980 mm,冲程为2 400 mm,额定功率为45 100 k W,额定转速为97 r/min。该船配备6台ALFA LAVAL S型分油机,具体配置情况见表1。分油机控制单元为EPC-50。
关键词 alfa 分油机 故障分析 S型 大型集装箱船 额定功率 额定转速 控制单元
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Alfa Laval MMPX 304与Westfalia OSD 6-0136全自动排渣式分油机的介绍
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作者 张统光 《中国水运(下半月)》 2010年第12期150-154,共5页
详细分析Alfa Laval MMPX 304与Westfalia OSD 6-0136全自动排渣式分油机的结构特点、工作原理以及各自控制单元如何程序化地实现分油机的分油、自动排渣与监控报警。简述有比重环与无比重环的分油机在实际应用中的区别,并寄希望分油机... 详细分析Alfa Laval MMPX 304与Westfalia OSD 6-0136全自动排渣式分油机的结构特点、工作原理以及各自控制单元如何程序化地实现分油机的分油、自动排渣与监控报警。简述有比重环与无比重环的分油机在实际应用中的区别,并寄希望分油机在设计上有更大的改进。 展开更多
关键词 alfa Laval MMPX Westfalia OSD 分油机 工作原理
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FXa抑制剂特异性拮抗剂Andexanet alfa的研究进展 被引量:1
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作者 邱小妙 张蕊 +3 位作者 郝春华 王维亭 赵专友 孙双勇 《山东医药》 CAS 2018年第12期105-108,共4页
口服抗凝药物(NOACs)抗凝效果好,且对正常的凝血级联反应几乎没有影响,仅有很小的内源性促凝血和抗凝血生物活性。但是,由于缺乏有效的拮抗剂,NOACs在使用过程中受到很大的限制。Andexanet alfa是美国Portola医药公司开发的一种重组凝... 口服抗凝药物(NOACs)抗凝效果好,且对正常的凝血级联反应几乎没有影响,仅有很小的内源性促凝血和抗凝血生物活性。但是,由于缺乏有效的拮抗剂,NOACs在使用过程中受到很大的限制。Andexanet alfa是美国Portola医药公司开发的一种重组凝血因子Xa(FXa)诱导分子,与天然的FXa类似,可竞争性抑制直接和间接FXa抑制剂,有希望成为广谱的针对FXa抑制剂类药物的解毒剂。目前,临床前和临床试验已经初步证实Andexanet alfa可抑制直接和间接FXa抑制剂活性,且具有良好的使用安全性。 展开更多
关键词 FXa抑制剂特异性拮抗剂 Andexanet alfa 重组凝血因子Xa抑制剂
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Sebelipase alfa——用于治疗溶酶体酸性脂肪酶缺乏症的药物 被引量:1
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作者 宋岐 马威 +1 位作者 李沁园 刘敏 《临床药物治疗杂志》 2017年第1期71-74,共4页
Sebelipase alfa是治疗罕见病溶酶体酸性脂肪酶缺乏症(LAL-d)的药物,该病是由基因突变导致的慢性渐进性代谢疾病,能导致患者生长障碍、肝脂异常堆积,肝脏纤维化和肝硬化等。Sebelipase alfa作为首个对症治疗溶酶体酸性脂肪酶缺乏的生物... Sebelipase alfa是治疗罕见病溶酶体酸性脂肪酶缺乏症(LAL-d)的药物,该病是由基因突变导致的慢性渐进性代谢疾病,能导致患者生长障碍、肝脂异常堆积,肝脏纤维化和肝硬化等。Sebelipase alfa作为首个对症治疗溶酶体酸性脂肪酶缺乏的生物制剂,被FDA授予孤儿药资格。 展开更多
关键词 Sebelipase alfa 溶酶体酸性脂肪酶缺乏 临床研究 孤儿药
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asfotase alfa用于罕见病低碱性磷酸酶血症的文献综述
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作者 刘清扬 后子靖 +4 位作者 葛育 王少红 刘鑫 张波 唐彦 《中国新药杂志》 CAS CSCD 北大核心 2023年第24期2526-2530,共5页
低碱性磷酸酶血症(hypophosphatasia, HPP)是一种罕见的以骨骼和(或)牙齿矿化障碍,伴有血清碱性磷酸酶活性降低为特征的遗传性内分泌系统疾病。asfotase alfa(AA)作为重组骨靶向人非特异性碱性磷酸酶获得美国FDA孤儿药认证,并于2015年... 低碱性磷酸酶血症(hypophosphatasia, HPP)是一种罕见的以骨骼和(或)牙齿矿化障碍,伴有血清碱性磷酸酶活性降低为特征的遗传性内分泌系统疾病。asfotase alfa(AA)作为重组骨靶向人非特异性碱性磷酸酶获得美国FDA孤儿药认证,并于2015年上市用于HPP的治疗。本文就AA的作用机制、药动学特点、有效性、安全性及用法用量进行综述,为该药物用于HPP患者提供参考和证据支持。 展开更多
关键词 低碱性磷酸酶血症 罕见病 孤儿药 asfotase alfa
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Psychiatric side effects of pegylated interferon alfa-2b as compared to conventional interferon alfa-2b in patients with chronic hepatitis C 被引量:5
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作者 Michael R Kraus Arne Sch(a|¨)fer +1 位作者 Herbert Csef Michael Scheurlen 《World Journal of Gastroenterology》 SCIE CAS CSCD 2005年第12期1769-1774,共6页
AIM: To assess systematically the spectrum and extent of depressive symptoms comparing patient groups receiving peginterferon or conventional interferon.METHODS: Ninety-eight patients with chronic hepatitis C and inte... AIM: To assess systematically the spectrum and extent of depressive symptoms comparing patient groups receiving peginterferon or conventional interferon.METHODS: Ninety-eight patients with chronic hepatitis C and interferon-based therapy (+ribavirin) were consecutively enrolled in a longitudinal study. Patients were treated with conventional interferon alfa-2b (48/98patients; 5 MIU interferon alfa-2b thrice weekly) or peginterferon alfa-2b (50/98 patients; 80-150 μgpeginterferon alfa-2b) in combination with weight-adapted ribavirin (800-1 200 mg/d). Repeated psychometric testing was performed before, three times during and once after antiviral therapy: Depression was evaluated by the Hospital Anxiety and Depression Scale (HADS), anger/hostility by the Symptom Checklist-90 Items Revised (SCL-90-R).RESULTS: Therapy with pegylated interferon alfa-2bproduces comparable scores for depression (ANOVA:P = 0.875) as compared to conventional interferon.Maximums of depression scores were even higher and cases of clinically relevant depression were frequent during therapy with peginterferon. Scores for anger/hostility were comparable for both therapy subgroups.CONCLUSION: Our findings suggest that the extent and frequency of depressive symptoms in total are not reduced by peginterferon. Monitoring and management of neuropsychiatric toxicity especially depression have to be considered as much as in antiviral therapy with unmodified interferon. 展开更多
关键词 Pegylated interferon Hepatitis C Side effects DEPRESSION Interferon alfa
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Peginterferon alfa-2a for the treatment of chronic hepatitis C in the era of direct-acting antivirals 被引量:11
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作者 Yan Huang Ming-Hui Li +1 位作者 Min Hou Yao Xie 《Hepatobiliary & Pancreatic Diseases International》 SCIE CAS CSCD 2017年第5期470-479,共10页
BACKGROUND: The availability of novel direct-acting antivirals (DAAs) represents a new era of curative hepatitis C virus (HCV) treatment, with over 95% of patients infected with HCV genotype 1 achieving sustained viro... BACKGROUND: The availability of novel direct-acting antivirals (DAAs) represents a new era of curative hepatitis C virus (HCV) treatment, with over 95% of patients infected with HCV genotype 1 achieving sustained virological response (SVR). Nevertheless, the majority of patients globally are unable to access these treatments because of cost and infrastructure constraints and, thus, remain untreated and uncured. DATA SOURCE: Relevant articles of peginterferon (PegIFN)-based treatments in HCV and sofosbuvir-based treatments, simeprevir, daclatasvir/asunaprevir, ritonavir-boosted paritaprevir/ombitasvir/dasabuvir, and grazoprevir/elbasvir, were searched in PubMed database, including general population and special population. RESULTS: PegIFN in combination with ribavirin remains an important and relevant option for some patients, achieving SVR rates of up to 79% in genotype 1 and 89% in genotype 2 or 3 infections, which increases for patients with favorable IL28B genotypes. Triple therapy of DAA plus PegIFN/ribavirin is effective in treating difficult-to-cure patients infected with HCV genotype 3 or with resistance-associated variants. Owing to its long history in HCV management, the efficacy, tolerability and long-term outcomes associated with PegIFN alfa-2a are well established and have been validated in large-scale studies and in clinical practice for many populations. Furthermore, emerging data show that IFN-induced SVR is associated with lower incidences of hepatocellular carcinoma compared with DAAs. On the contrary, novel DAAs have yet to be studied in special populations, and long-term outcomes, particularly tumor development and recurrence in patients with cirrhosis and/or hepatocellular carcinoma, and reactivation of HBV in dually infected patients, are still unclear. CONCLUSION: In this interferon-free era, PegIFN-based regimens remain a safe and effective option for selected HCV patients. 展开更多
关键词 chronic hepatitis C direct-acting antivirals hepatitis C virus peginterferon alfa-2a RIBAVIRIN
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ALFA LAVAL S系列分油机操作及管理要点 被引量:2
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作者 胡刚 《航海技术》 2015年第5期54-58,共5页
0引言 为降低航运成本,很多船舶使用380cSt甚至更差的燃油。380cSt燃油不仅黏度高、密度大,而且渣滓多,使用前必须经过净化处理以除去其中的水分和杂质。燃油的净化质量对柴油机工作的可靠性和使用寿命有很大影响。分油机是船舶净化燃... 0引言 为降低航运成本,很多船舶使用380cSt甚至更差的燃油。380cSt燃油不仅黏度高、密度大,而且渣滓多,使用前必须经过净化处理以除去其中的水分和杂质。燃油的净化质量对柴油机工作的可靠性和使用寿命有很大影响。分油机是船舶净化燃油和滑油的关键设备。分油机中的分离筒经过增速装置后, 展开更多
关键词 分油机 alfa 管理要点 操作 净化处理 航运成本 使用寿命 燃油
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Serum proteins in chronic hepatitis B patients treated with peginterferon alfa-2b 被引量:4
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作者 Sunida Kuakarn Poorichaya SomParn +3 位作者 Pisit Tangkijvanich Varocha Mahachai Visith Thongboonkerd Nattiya Hirankarn 《World Journal of Gastroenterology》 SCIE CAS 2013年第31期5067-5075,共9页
AIM: To study the differential protein profile in serum of hepatitis B patients.METHODS: Serum samples were obtained from patients with chronic hepatitis B who were receiving peginterferon alfa-2b.The serum samples we... AIM: To study the differential protein profile in serum of hepatitis B patients.METHODS: Serum samples were obtained from patients with chronic hepatitis B who were receiving peginterferon alfa-2b.The serum samples were subjected to albumin depletion and analyzed by two-dimensional gel electrophoresis(2-DE).Differentially expressed protein spots were identified by electrospray ionizationquadrupole time-of-flight mass spectrometry.Alpha2-HS-glycoprotein,complement component C3c and CD5 antigen were further analyzed by an enzymelinked immunosorbent assay and immunonephelometry.RESULTS: Nineteen patients with HBeAg-positive chronic hepatitis B(CHB) were studied.These patients were followed for at least 1 year after treatment and were classified according to their treatment response: responders(n = 9) and non-responders(n = 10).2-DE and MS/MS analysis were performed to compare the serum proteins before initiating peginterferon alfa2b.From the quantitative analysis of the 2-D gel,7 proteins were detected between the two groups at different levels before treatment.Among these potential candidates,serum levels of alpha-2-HS-glycoprotein,complement component C3c and CD5 antigen-like precursor were further analyzed.In the validation phase,23 subjects,9 sustained responders and 14 nonresponders,were recruited.Interestingly,the levels of alpha-2-HS-glycoprotein and complement component C3c were elevated in the serum of the non-responders compared to the responders.CONCLUSION: Serum alpha-2-HS-glycoprotein and complement component C3c may be potential serum biomarkers in predicting the treatment response of peginterferon alfa-2b in patients with CHB prior to treatment. 展开更多
关键词 PROTEOMICS PEGINTERFERON alfa-2b CHRONIC HEPATITIS B Alpha-2-HS-glycoprotein SERUM
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1-Deoxynojirimycin Content and Alfa-Glucosidase Inhibitory Activity and Heat Stability of 1-Deoxynojirimycin in Silkworm Powder 被引量:8
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作者 Kazuhisa Yatsunami Kohji Murata Tsutomu Kamei 《Food and Nutrition Sciences》 2011年第2期87-89,共3页
Silkworm powder containing 1-deoxynojirimycin (DNJ) has α-glucosidase inhibitory activity and is promising as a complementary and alternative medicine (CAM) agent in Japan. Silkworm powder produced in Korea was extra... Silkworm powder containing 1-deoxynojirimycin (DNJ) has α-glucosidase inhibitory activity and is promising as a complementary and alternative medicine (CAM) agent in Japan. Silkworm powder produced in Korea was extracted with 75% ethanol. The extract was derivatized with 9-fluorenylmethyl chloroformate (FMOC-Cl), and DNJ-FMOC content was measured by HPLC. Then, alfa-glucosidase inhibition by silkworm and mulberry powder in pig liver crude enzyme was assayed using 4-nitrophenyl-alfa-D-glucopyranoside as a substrate. Silkworm powder DNJ content (0.39 to 0.58%) was higher than that in mulberry powder (0.08 to 0.12%). The alfa-glucosidase inhibitory activity of silkworm powder was more potent than that of mulberry leaves and green tea. Silkworm powder DNJ was stable upon heating to 121?C for up to 15 min. 展开更多
关键词 SILKWORM Bombyx Mori 1-DEOXYNOJIRIMYCIN alfa-Glucosidase INHIBITOR Heat Stability
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Effect of Recycling Cycles on the Mechanical and Damping Properties of Short Alfa Fibre Reinforced Polypropylene Composite 被引量:2
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作者 Fatima Ezzahra El Abbassi Mustapha Assarar +3 位作者 Rezak Ayad Hamid Sabhi Stephane Buet Nouzha Lamdouar 《Journal of Renewable Materials》 SCIE 2019年第3期253-267,共15页
This paper aims at studying the effect of recycling on the static and dynamic properties of short alfa fibre reinforced polypropylene.For this purpose,alfa fibres reinforced composites were elaborated by an injection ... This paper aims at studying the effect of recycling on the static and dynamic properties of short alfa fibre reinforced polypropylene.For this purpose,alfa fibres reinforced composites were elaborated by an injection moulding process and were subjected to different mechanical recycling cycles.Then,non-recycled and recycled materials were subjected to static tests and Dynamic Mechanical Analysis(DMA)to evaluate the effect of recycling on their behaviour.Besides,the effects of alkali and salt water treatments on the static and dynamic properties of the alfa composite were also investigated.The obtained results show that tensile and flexural properties of alfa fibres reinforced composites decrease during recycling cycles.Moreover,the recycling induces a drop in the storage modulus and enhances the loss factor of these composites.The composites with alfa fibre especially the alkali treated composite show the same resistance to recycling as composites with hemp fibres.Further,SEM observations indicate a decrease in the fibres dimension with the recycling cycles,especially for alfa fibres,which can explain the decrease in the properties of the alfa composite during recycling operations. 展开更多
关键词 alfa fibre hemp fibre eco-composites RECYCLING mechanical properties dynamic properties
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Alfa Laval进口板式热交换器的修复处理 被引量:1
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作者 傅敬强 闫高伦 +1 位作者 王礼梅 陈芍桦 《石油化工设备》 CAS 2012年第4期90-92,共3页
对运行了一个多月的进口板式热交换器泄漏原因进行了分析,并采用了行之有效的处理办法,对类似设备泄漏的修复具有一定的借鉴作用。
关键词 板式热交换器 alfa Laval 泄漏 修复
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乙烯装置ALFA开关球阀的技术改造
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作者 刘立忠 《自动化仪表》 CAS 北大核心 2003年第11期65-66,共2页
关键词 乙烯装置 alfa开关球阀 技术改造 裂解装置
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Alfa-laval S系列净油机工作原理及典型故障分析(上) 被引量:2
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作者 耿佳东 李成 《航海技术》 北大核心 2010年第5期51-54,共4页
具有120多年历史的ALFA LAVAL公司的三大核心技术之一——离心分离技术,其产品广泛应用于海运、电力等行业的燃油、润滑油的净化处理,在全球市场中一直走在行业的前列,处于领先地位。
关键词 典型故障分析 工作原理 净油机 离心分离技术 alfa 净化处理 全球市场 润滑油
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