Multi-centre, prospective, randomized, open-label, blinded-endpoint, non-inferiority trial of low- versus moderate-Dose Aspirin Plus Immunoglobulin (DAPI) for prevention of coronary artery abnormalities in Kawasaki disease - DAPI

开始日期2023-06-23

申办/合作机构-

100 项与人血丙种球蛋白相关的临床结果

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100 项与人血丙种球蛋白相关的转化医学

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100 项与人血丙种球蛋白相关的专利（医药）

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249

项与人血丙种球蛋白相关的文献（医药）

2024-07-01·American Heart Journal

Different dose aspirin plus immunoglobulin (DAPI) for prevention of coronary artery abnormalities in Kawasaki disease: Study protocol for a multi-center, prospective, randomized, open-label, blinded end-point, non-inferiority trial

Article

作者: Zhang, Xu ; Li, Ming ; Li, Wei ; Xie, Xiaofei ; Yuan, Jia ; Zhang, Li ; Li, Jianbin ; Wang, Yanfei ; Li, Fengxiang ; Wu, Yujian ; Hu, Lin ; Huang, Ping ; Xia, Shuliang ; Wang, Zhouping

BACKGROUNDKawasaki disease is a pediatric acute systemic vasculitis that specifically involves the coronary arteries. Timely initiation of immunoglobulin plus aspirin is necessary for diminishing the incidence of coronary artery abnormalities (CAAs). The optimal dose of aspirin, however, remains controversial. The trial aims to evaluate if low-dose aspirin is noninferior to moderate-dose in reducing the risk of CAAs during the initial treatment of Kawasaki disease.METHODSThis is a multi-center, prospective, randomized, open-label, blinded endpoint, noninferiority trial to be conducted in China. The planned study duration is from 2023 to 2026. Data will be analyzed according to intention-to-treat principles. Participants are children and adolescents under the age of 18 with Kawasaki disease, recruited from the inpatient units. A sample size of 1,346 participants will provide 80% power with a one-sided significance level of 0.025. Qualifying children will be randomized (1:1) to receive either intravenous immunoglobulin (2 g/kg) plus oral moderate-dose aspirin (30-50 mg·kg^-1·d^-1) until the patient is afebrile for at least 48 hours, or immunoglobulin plus low-dose aspirin (3-5 mg·kg^-1·d^-1) as initial treatment. The primary outcome will be the occurrence of CAAs at 8 weeks after immunoglobulin infusion. Independent blinded pediatric cardiologists will assess the primary endpoint using echocardiography.CONCLUSIONSThere is a shortage of consensus on the dose of aspirin therapy for Kawasaki disease due to the lack of evidence. The results of our randomized trial will provide more concrete evidence for the efficacy and adverse events of low- or moderate-dose aspirin in the acute phase of Kawasaki disease.TRIAL REGISTRATIONwww.chictr.org.cn: ChiCTR2300072686.

2024-04-09·Blood Advances

Immunoglobulin replacement vs prophylactic antibiotics for hypogammaglobulinemia secondary to hematological malignancy

Article

作者: Dendle, Claire ; Degelia, Amber ; Pepperell, Dominic ; Morrissey, C. Orla ; Wood, Erica M. ; Weinkove, Robert ; Keegan, Anastazia ; Gilbertson, Michael ; Weston, Helen ; Crispin, Philip ; Waters, Neil ; McQuilten, Zoe K. ; Wellard, Cameron ; Thao, Le Thi Phuong ; Pullon, Humphrey ; Johnston, Anna ; van Tonder, Tina ; Trotman, Judith ; Reynolds, John

AbstractImmunoglobulin replacement and prophylactic antibiotics are commonly used to prevent infections in patients with secondary hypogammaglobulinemia due to hematological malignancies but have never been directly compared. In this randomized controlled feasibility trial conducted in 7 hospitals in Australia and New Zealand, we enrolled patients with secondary hypogammaglobulinemia with either a history of recurrent/severe infection or an immunoglobulin G level <4 g/L. Participants were randomized in a 1:2 ratio to immunoglobulin (0.4 g/kg per 4 weeks IV) or daily antibiotics (trimethoprim-sulfamethoxazole 160 mg/800 mg or, if contraindicated, 100 mg doxycycline) for 12 months. Participants allocated to antibiotics were allowed to crossover after grade ≥3 infections. The primary outcome was proportion of patients alive on the assigned treatment 12 months after randomization. Between August 2017 and April 2019, 63 patients were randomized: 42 to antibiotics and 21 to immunoglobulin. Proportion of participants alive on allocated treatment at 12 months was 76% in the immunoglobulin and 71% in the antibiotic arm (Fisher exact test P=.77; odds ratio, 0.78; 95% CI, 0.22-2.52). The lower quartile for time to first major infection (median, not reached) was 11.1 months for the immunoglobulin and 9.7 months for the antibiotic arm (log-rank test, P=.65). Three participants in the immunoglobulin and 2 in the antibiotic arm had grade ≥3 treatment-related adverse events. A similar proportion of participants remained on antibiotic prophylaxis at 12 months to those on immunoglobulin, with similar rates of major infections. Our findings support the feasibility of progressing to a phase 3 trial. Trial registration #ACTRN12616001723471.

2024-03-05·Journal of Chest Surgery

Successful Heart Transplantation Despite Rhesus Blood Type Mismatch: A Case Report

作者: Kim, Ji Hong ; Shin, Yu-Rim

Matching for the rhesus (Rh) blood group is currently not taken into account in the organ allocation system. However, in Rh-mismatched transplantation, the primary concern is the potential for RhD-negative recipients to develop sensitization and produce anti-D anti-bodies if they receive a transfusion of RhD-positive blood. It is estimated that over 80% of RhD-negative recipients may experience Rh allosensitization when exposed to RhD-positive blood, although this occurrence is less common in recipients of solid organs. In theory, RhD-negative recipients who receive organs from RhD-positive donors are at risk of alloimmunization and the production of anti-D antibodies, which could complicate future blood product transfusions. However, our understanding of the impact of donor-recipient Rh mismatch on transplant outcomes, particularly in heart transplantation, is limited. We report a case of successful Rh-mismatched heart transplantation, which was effectively managed through the use of preoperative RhD immunoglobulin and plasmapheresis.

项与人血丙种球蛋白相关的新闻（医药）

2023-11-07

·BioSpace

KORU Medical Systems, Inc. Receives FDA 510(k) Clearance for FREEDOM60® Infusion System With Hizentra® 50 ML Prefilled Syringes

MAHWAH, N.J.--(BUSINESS WIRE)-- KORU Medical Systems, Inc. (NASDAQ: KRMD) ("KORU Medical" or the "Company"), a leading medical technology company focused on the development, manufacturing, and commercialization of innovative and easy-to-use specialty subcutaneous infusion solutions that improve quality of life for patients, today announced that it received U.S. Food and Drug Administration (FDA) clearance for use of the FREEDOM60® Infusion System with Hizentra® 50 mL prefilled syringes (PFS). “Being the first and only FDA-cleared infusion system for use with prefilled syringes demonstrates KORU Medical’s continued commitment to enhance the patient experience for all patients self-administering subcutaneous immunoglobulin (SCIg) therapy in the home,” said Linda Tharby, KORU Medical’s President and CEO. “Prefilled syringes are proven to provide a significant improvement in ease of use for all patients, and in administering higher doses of immunoglobulin, including for PIDD and CIDP patients. We are now able to deliver PFS benefits to Hizentra® patients through our FREEDOM™ Infusion System portfolio - the FREEDOM60® Infusion System with 50 mL PFS and the FreedomEdge® with 20 mL PFS. We believe the unlocking of these PFS benefits through use with the FREEDOM™ Infusion System could lead to even greater opportunity to expand SCIg use in the home.” The FREEDOM™ Infusion System is the first and only infusion system cleared for use with prefilled syringes and, given the majority of patients are prescribed doses of 50 mL or greater, this clearance for FREEDOM60® significantly expands the benefits of PFS use, especially for those diagnosed with conditions requiring significantly higher doses of immunoglobulin. When using prefilled syringes with the FREEDOM™ Infusion System, KORU Medical has demonstrated that patients can experience up to an 80% reduction in drug preparation tasks when compared to using vials. The FDA approved the Hizentra® 50 mL prefilled syringe in April 2023, and CSL Behring has announced an early 2024 availability. Hizentra® is the most prescribed subcutaneous immunoglobulin and the first to be available in prefilled syringes. About KORU Medical Systems KORU Medical Systems develops, manufactures, and commercializes innovative and easy-to-use subcutaneous drug delivery systems that improve quality of life for patients around the world. The FREEDOM™ Infusion System currently includes the FREEDOM60® and FreedomEdge® Syringe Infusion Drivers, Precision Flow Rate Tubing™ and HIgH-Flo Subcutaneous Safety Needle Sets™. The FREEDOM™ Infusion System is used for infusions administered in the home and alternate care settings. For more information, please visit . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. All statements that are not historical fact are forward-looking statements and can be identified by words such as "believe," “could” and "opportunity". Actual results may differ materially from these statements due to potential risks and uncertainties such as, among others, timing of Hizentra® 50 mL prefilled syringe availability, patient acceptance of PFS with the FREEDOM™ Infusion System, and those risks and uncertainties included under the captions "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2022 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, which are on the SEC and available on our website at and on the SEC website at . All information provided in this release and in the attachments is as of November 7, 2023. Undue reliance should not be placed on the forward-looking statements in this press release, which are based on information available to us on the date hereof. We undertake no duty to update this information unless required by law. Hizentra® is a registered trademark of CSL Behring AG. Hizentra® is manufactured by CSL Behring AG and distributed by CSL Behring LLC.

上市批准

2023-03-30

·药智网

50亿“单品之王”落选，90%原研撤退！这20个原研药，终端竞争格局如何？

万众瞩目的第八批国采，在美丽的海南岛开标，有人欢喜，有人愁。第八批国采，共涉及40个药品品种，除了左炔诺孕酮片落标外，剩余39个品种中选，品种中选率高达97.5%。本次参加申报的企业共有251家，共计366个产品，其中174家企业的252个产品获得拟中选资格，投标企业中选比例约70%。而与火热的国内中标药企形成鲜明对比的是，原研/进口企业的落选。据统计，本次国采涉及原研/进口20个品种，其中18个落选，2个中标。50亿“单品之王”落选，90%原研撤退！这20个原研药，终端竞争格局如何？50亿“单品之王”落选，90%原研撤退据网上公开信息整理，本次40个药品品种中，共有20个品种涉及原研/进口企业。在本轮国采中，仅有2家原研企业拟中标，分别为Organon的米氮平片和Alfasigma的左卡尼汀注射液。以涉及原研/进口企业品种中标情况测算，仅10%的原研中标，低得可怜。大部分的原研/进口企业虽然参与投标却落选，与国内企业热切的参与度形成鲜明的对比。表1 原研/进口产品在第八批国采的中标情况分析（根据网传信息手工整理，如有错漏以官方为准）是因为原研产品在中国公立医院市场已经被蚕食了吗？还是产品的市场份额可有可无？我们从中国城市公立医院终端竞争格局逐个分析。对阿加曲班注射液而言，竞争格局不算激烈。田边三菱制药仅占2.3%的市场份额，市场基本是被天津药物研究院药业占领，落标可以理解。表2 中国公立医院阿加曲班品牌年度销售格局（单位：万元）对醋酸阿托西班注射液而言，辉凌医药的市场霸主地位虽然已易主，但是仍然占据26.7%的市场份额，妥妥的3亿元大品种，落选实属可惜。表3 中国公立医院阿托西班品牌年度销售格局（单位：万元）对丙氨酰谷氨酰胺注射液而言，费森尤斯卡比华瑞制药依旧手握市场份额的No.1,超过4亿元的大品种，难道有其他路径可以保持销售屹立不倒？表4 中国公立医院丙氨酰谷氨酰胺品牌年度销售格局（单位：万元）对非洛地平缓释片而言，阿斯利康拥有着绝对的优势碾压着一众国产仿制企业，接近13亿的超级大品种，超过70%的市场份额，却静默错过本轮次的集采，实属难以理解。表5 中国公立医院非洛地平品牌年度销售格局（单位：万元）对骨化三醇软胶囊而言，罗氏屈居第二，市场份额被正大制药超过，但是市场上基本呈现也就是双雄争霸的局面，本次罗氏的“冷眼相看”，难道是要低下高贵的头颅服软？表6 中国公立医院骨化三醇品牌年度销售格局（单位：万元）对甲钴胺注射液而言，卫材手握7亿的销售额，“一览众山小”，但是对于这个魅力无限的大品种，已有接近20家过评，难保后续20家的追兵的追堵围击。表7 中国公立医院甲钴胺品牌年度销售格局（单位：万元）对磷酸奥司他韦干混悬剂而言，流感神药的故事始终占据着热点，东阳光又笑傲群雄，对于供不应求的大单品而言，罗氏进可攻退可守，策略不足为奇。表8 中国公立医院奥司他韦品牌年度销售格局（单位：万元）对氯沙坦钾氢氯噻嗪片而言，默沙东和北京福元医药不相伯仲，任何一点风吹草动，都可能成为霸主之争的胜负手。本次默沙东的失标，有可能让北京福元医药拉开差距。表9 中国公立医院氯沙坦钾氢氯噻嗪片品牌年度销售格局（单位：万元）对米氮平而言，虽然不算是一个超大品种，但是Organon为了保住榜首的地位，选择了中标，也感受到原研企业为保住过亿元品种的决心。表10 中国公立医院米氮平品牌年度销售格局（单位：万元）对那屈肝素钙注射液而言，作为首次纳入国采的肝素品种，Aspen占据将近一半公立医院销售的市场份额，依旧没有表现出很热切维护市场地位的决心。表11 中国公立医院那屈肝素钙注射液品牌年度销售格局（单位：万元）对于注射用生长抑素而言，又是一个黄金大单品，原研默克手握8亿元的“蛋糕”，本次并没有选择通过国采放量拉动量价齐升。表12 中国公立医院注射用生长抑素品牌年度销售格局（单位：万元）对于熊去氧胆酸而言，Dr.Falk Pharma GmbH一骑红尘，虽然入局者多，但原研始终把握着市场的主导权。作为第八批国采中，原研药品在公立医院为数不多超10亿元的黄金大单品，Dr.Falk的选择让人有点摸不着头脑。“光脚企业”一定会趁机而上。表13 中国公立医院熊去氧胆酸品牌年度销售格局（单位：万元）对于依诺肝素钠注射液而言，赛诺菲拥有着绝对的优势，占据了半壁江山，接近14亿元的大品种，也默默退出国采的竞争行列。要依靠品牌和推广维持被抢夺的市场，赛诺菲要做出更大的努力。表14 中国公立医院依诺肝素钠注射液品牌年度销售格局（单位：万元）对于左卡尼汀注射液而言，Alfasigma随保持着领先的位置，但优势并不明显，后面的东北制药在奋起直追，所以本轮次国采中，Alfasigma作为唯二的原研企业，选择中标来深化竞争优势。表15 中国公立医院左卡尼汀注射液品牌年度销售格局（单位：万元）对于注射用氨曲南而言，BMS在中国公立医院中早已经不见了踪迹了，原研反而成了“光脚企业”，但是BMS却没有作为“光脚企业”的勇气。表16 中国公立医院注射用氨曲南品牌年度销售格局（单位：万元）对于甲硝唑氯化钠注射液而言，Baxter（百特）早已被挤到三甲开外，仅维持了2000万元的销售规模，有点“食之无味，落之可惜”的意味，Baxter的选择也就没那么惊奇。表17 中国公立医院甲硝唑氯化钠注射液品牌年度销售格局（单位：万元）对于注射用达托霉素而言，默沙东早已不见踪迹，已经失去的市场，也就不再回首，也不想重新出发。表18 中国公立医院注射用达托霉素品牌年度销售格局（单位：万元）对于注射用伏立康唑而言，又是属于为数不多原研企业超10亿元的黄金超级大单品，而且拥有着相对的市场优势。可能是辉瑞对自身实力的信心，可以在不参加国采的前提下，继续维持该霸主地位。表19 中国公立医院注射用伏立康唑品牌年度销售格局（单位：万元）对于注射用哌拉西林钠他唑巴坦钠而言，这是一块“香饽饽”抗感染复方大品种，是多家企业的拳头大产品。本轮次中惠氏制药早已失去争夺市场的雄心。表20 中国公立医院注射用哌拉西林钠他唑巴坦钠品牌年度销售格局（单位：万元）对于注射用头孢哌酮钠舒巴坦钠而言，辉瑞公司“了不得”，单品超过50亿元，是第八批国采当之无愧的单品之王。别说仿制药了，创新药要实现年销售超50亿元都不容易。就是这么一个拥有着绝对市场优势的产品，辉瑞不屑于入局国采打价格战进行竞争，也就为其他“光脚企业”创造蚕食市场的良机。表21 中国公立医院注射用头孢哌酮钠舒巴坦钠品牌年度销售格局（单位：万元）为何手握大市场的原研在国采落选？本轮集采中，大部分原研/进口企业均是手握市场大筹码，仅有个别是被仿制药企反超逐渐退出竞争舞台。但是，他们纷纷“撤退”，意欲何为？难道让大市场拱手让人？原因中，除了行业流传已久的，外资药企需要权衡药物国内外定价差异，总部很难授权其开极低的价格竞争集采，降价幅度无法与国内企业保持一致这个原因外，好像也没有特别合理的解释。如果说由于其强势的市场地位确让其在失去集采市场后能支撑一段时间，而非像很多国内企业一样不进集采就是死局，但是每一轮次的集采，周期基本是3年，3年之久，足以发生很多故事，支撑不住的时候，难道就放弃这块市场？也有人说，对于这些外企巨头而言，集采品种都是专利过期原研药，基本属于其存量市场，降价入选即使保住了市场份额，但是利润仍然大幅降低。他们更看重的是利润指标，因为利润指标关乎着他们股市价格的晴雨表。还有最后一个可能性，这些外企巨头整体的成本，确实不如国内企业。国内企业这些年的发展，已然建立完善的产业链，具备较强硬的价格话语权。呈现出的效果，就有点像印度的仿制药企在美国犹如“秋风扫落叶”。展望无论参与与否，国采的洪流奔流不息，医保局的意志不容置疑。原研企业的撤退，可能是国内企业实现进口替代的一个契机。至于要不要抓住，能不能抓住，就看每一家企业的魄力了。PS：药智网公众号【ID:yaozh008】后台回复【第八批国采】，即可获取本文图表excel格式文档注：数据来源网络，手动统计难免有误，欢迎大家留言指正。声明：本内容为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 茴香转载开白 | 马老师 18996384680（同微信）商务合作 | 张武龙 13368443108（同微信）阅读原文，是昨天最受欢迎的文章哦

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100 项与人血丙种球蛋白相关的药物交易

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


EHA2022	N/A	多发性骨髓瘤	2,445	IMMUNOGLOBULIN (Patients who received Ig)	鑰積網積獵網衊憲衊膚(顧遞醖淵願淵製齋顧鏇) = 窪選襯願艱襯願構糧構範襯願夢鏇襯膚壓艱鹹 (繭鹽範襯糧餘憲遞憲蓋 )	-	2022-05-12
				IMMUNOGLOBULIN (Patients who did not receive Ig)	鑰積網積獵網衊憲衊膚(顧遞醖淵願淵製齋顧鏇) = 膚淵構鑰鏇糧顧壓製遞範襯願夢鏇襯膚壓艱鹹 (繭鹽範襯糧餘憲遞憲蓋 )
P5.044 (AAN2016)	N/A	慢性炎症性脱髓鞘性多发性神经病 \| 多发性骨髓瘤	-	IVIG	(鑰網選選選範淵淵觸鑰) = 繭艱憲鑰鑰網夢繭壓鬱壓簾製繭簾範製觸積壓 (獵夢積製憲觸鹽淵獵齋 ) 更多	-	2016-04-05
				Immunoglobulin	(鑰網選選選範淵淵觸鑰) = 選構壓衊築窪觸醖襯鑰壓簾製繭簾範製觸積壓 (獵夢積製憲觸鹽淵獵齋 ) 更多
SP153 (ERA2015)	N/A	膜性肾小球肾炎 IgG \| IgM \| IgA	61	IMMUNOGLOBULIN	(鑰繭範願範製觸築繭繭) = 遞網積鹹壓構獵構鏇艱淵製鹹窪積鹹衊醖築觸 (膚繭觸鏇鑰網夢淵艱鬱 )	不佳	2015-05-21
				IMMUNOGLOBULIN	(築憲糧顧壓繭獵膚衊願) = 淵遞襯艱艱鬱簾願衊鏇衊鏇艱遞選鹹醖鏇壓網 (積窪蓋鑰積淵網鏇構淵 ) 更多