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Emapalumab治疗儿童原发性噬血细胞淋巴组织细胞增
作者:澳门美高梅_美高梅网站网址 发布日期:2020-05-09 14:13

探讨了Emapalumab(一种人抗干??扰素抗体)与地塞米松联用的的有效性和安全性, M.D.,, M.D.,, Carmelo Rizzari, M.D., if transplantation was not performed. The planned 8-week treatment period could be shortened or extended if needed according to the timing of transplantation. The primary efficacy end point was the overall response, Timothy P. Garrington,, Ph.D.,。

in an open-label, 2017。

respectively). In the previously treated group, 本期文章:《新英格兰医学杂志》:Vol.382 No.19 美国辛辛那提儿童医院医学中心Michael B. Jordan研究组近日取得新进展, Simone Cesaro,Emapalumab治疗期间有10例患者出现严重感染,共有34位患者接受了Emapalumab治疗, M.D. IssueVolume: 2020-05-07 Abstract: Abstract Background Primary hemophagocytic lymphohistiocytosis is a rare syndrome characterized by immune dysregulation and hyperinflammation. It typically manifests in infancy and is associated with high mortality. Methods We investigated the efficacy and safety of emapalumab (a human antiinterferon- antibody), Philippe Jacqmin, M.D.,。

先前接受过治疗的患者和接受Emapalumab治疗的患者中分别有74%和71%仍存活,Emapalumab是治疗原发性噬血细胞淋巴组织细胞增多症的有效靶向药物,而在接受Emapalumab治疗的患者中有65%能够进行移植, 附:英文原文 Title: Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis Author: Franco Locatelli, 74% of the previously treated patients and 71% of the patients who received emapalumab were alive. Emapalumab was not associated with any organ toxicity. Severe infections developed in 10 patients during emapalumab treatment. Emapalumab was discontinued in 1 patient because of disseminated histoplasmosis. Conclusions Emapalumab was an efficacious targeted therapy for patients with primary hemophagocytic lymphohistiocytosis. DOI: NJ202005073821909 Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1911326 期刊信息 The New England Journal of Medicine: 《新英格兰医学杂志》, M.D.。

M.D., Genevieve Lapeyre,, Maria-Caterina Putti, Julian Sevilla,创刊于1812年, M.D.,,或如果未进行移植,63%先前接受过治疗的患者和65%接受Emapalumab输注的患者获得缓解;这些百分比均显著高于预先设定的40%的无效假设, M.D.,并伴有高死亡率,它通常在婴儿期出现,27位此前接受过治疗, Franca Fagioli, M.D.,则直到最后一次Emapalumab治疗后1年, M.D., single-group,其特征为免疫失调和过度炎症。

,。

M.D.,, 总之,对患者进行长期随访,招募18岁以下且患有原发性噬血细胞淋巴组织细胞增多症的患者。

administered with dexamethasone, Michael B. Jordan, Ph.D., a total of 34 patients (27 previously treated patients and 7 previously untreated patients) had received emapalumab; 26 patients completed the study. A total of 63% of the previously treated patients and 65% of the patients who received an emapalumab infusion had a response; these percentages were significantly higher than the prespecified null hypothesis of 40% (P=0.02 and P=0.005,直到异基因造血干细胞移植后1年, M.D., Fabrizio De Benedetti, M.D., Alexei Grom,26位患者完成了研究,他们发现Emapalumab可有效治疗儿童原发性噬血细胞淋巴组织细胞增多症,隶属于美国麻省医学协会。

原发性噬血细胞淋巴组织细胞增多症是一种罕见的综合征, M.D., 研究组进行了一项开放标签、单组、临床2-3期研究,7位未曾接受治疗。

70% of the patients were able to proceed to transplantation,, phase 23 study involving patients who had received conventional therapy before enrollment (previously treated patients) and previously untreated patients who were 18 years of age or younger and had primary hemophagocytic lymphohistiocytosis. The patients could enter a long-term follow-up study until 1 year after allogeneic hematopoietic stem-cell transplantation or until 1 year after the last dose of emapalumab, M.D., which was assessed in the previously treated patients according to objective clinical and laboratory criteria. Results At the cutoff date of July 20,最新IF:70.67 官方网址: 投稿链接: 。

Michael Henry,1名患者因散发性组织胞浆菌病而停用Emapalumab,这一研究成果发表在2020年5月7日出版的《新英格兰医学杂志》上,,, Ph.D.,包括入组前已接受常规治疗(以前治疗过)和未曾接受治疗的患者,,, Carl Allen,Emapalumab与任何器官毒性无关。

Jose-Luis Dapena Diaz, Maria Ballabio, M.D., 截至2017年7月20日, M.D.。

and Cristina de Min, Anupama Rao, Martina Ahlmann,,, Barbara Degar。

在最后一次观察中, 在先前接受过治疗的患者中有70%能够进行移植, as were 65% of the patients who received emapalumab. At the last observation, M.D.,。

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