Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease.
Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease.
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艾曲波帕用于治疗与结缔组织病相关的难治性血小板减少症
DOI：
10.1038/s41598-021-84493-2
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发表时间：
2021-03-09
影响因子：
4.6
通讯作者：
Chen S
中科院分区：
文献类型：
--
作者：
Wang J;Dai M;Fu Q;Chen S
为了评估艾曲泊帕治疗结缔组织病（CTD）-免疫性血小板减少症（ITP）的疗效和安全性，我们进行了这项单中心回顾性观察性研究，纳入了2018年1月至2019年8月期间接受艾曲泊帕治疗的难治性CTD-ITP患者。分析基线时患者的特征以及药物的有效性和安全性。使用单变量分析（如卡方检验或非参数检验）和多重对应分析（MCA）方法分析缓解的预测因素。共有15名难治性CTD-ITP患者入选该研究。入选时的中位年龄为40.6岁。开始艾曲泊帕治疗时的中位血小板计数为11.53 × 109/L。中位缓解时间为3.42周。完全缓解（CR）率和总有效率随时间延长而下降。在单变量分析中，与CTD-ITP患者中艾曲泊帕应答相关的因素为原发病、WBC计数、血红蛋白水平和骨髓结果特征。此外，MCA表明，难治性CTD-ITP患者对艾曲波帕的不良反应与原发病SS、中度至重度贫血、白细胞减少症和骨髓穿刺显示再生障碍性贫血、缺乏巨核细胞或巨噬细胞活化综合征（MAS）密切相关。总之，艾曲泊帕在CTD相关血小板减少症患者中有效且耐受性良好。使用艾曲泊帕时应考虑一些因素，包括原发病、血液检查和骨髓组织学。
To assess the efficacy and safety of eltrombopag in connective tissue disease (CTD)-immune thrombocytopenia (ITP), we conducted this single-center retrospective observational study, including patients with refractory CTD-ITP who were treated with eltrombopag between January 2018 and August 2019. The characteristics of patients at baseline, and the efficacy and safety of the drug were analyzed. The predictors for a response were analyzed using a univariate analysis such as Chi-square or nonparametric test and a multiple correspondence analysis (MCA) method. A total of 15 patients with refractory CTD-ITP were included in the study. Their median age at the time of inclusion was 40.6 years. The median platelet count at initiation of eltrombopag was 11.53 × 109/L. The median remission time was 3.42 weeks. The complete remission (CR) and overall response rate decreased with time. The factors that associated with response to eltrombopag in patients with CTD-ITP were protopathy, WBC counts, levels of hemoglobin, and characteristics of bone marrow findings in univariate analysis. In addition, MCA indicated that a poor response to eltrombopag in patients with refractory CTD-ITP was closely associated with a protopathy with SS, medium to severe degree of anemia, leukopenia, and bone marrow aspiration showing aplastic anemia, an absence of megakaryocytes or macrophage activation syndrome (MAS). In conclusion, eltrombopag was effective and well-tolerated in patients with CTD-associated thrombocytopenia. Some factors should be considered in the use of eltrombopag, including the protopathy, blood test, and bone marrow histology.
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影响因子：
--
作者：
Petri, Michelle;Orbai, Ana-Maria;Alarcon, Graciela S.;Gordon, Caroline;Merrill, Joan T.;Fortin, Paul R.;Bruce, Ian N.;Isenberg, David;Wallace, Daniel J.;Nived, Ola;Sturfelt, Gunnar;Ramsey-Goldman, Rosalind;Bae, Sang-Cheol;Hanly, John G.;Sanchez-Guerrero, Jorge;Clarke, Ann;Aranow, Cynthia;Manzi, Susan;Urowitz, Murray;Gladman, Dafna;Kalunian, Kenneth;Costner, Melissa;Werth, Victoria P.;Zoma, Asad;Bernatsky, Sasha;Ruiz-Irastorza, Guillermo;Khamashta, Munther A.;Jacobsen, Soren;Buyon, Jill P.;Maddison, Peter;Dooley, Mary Anne;van vollenhoven, Ronald F.;Ginzler, Ellen;Stoll, Thomas;Peschken, Christine;Jorizzo, Joseph L.;Callen, Jeffrey P.;Lim, S. Sam;Fessler, Barri J.;Inanc, Murat;Kamen, Diane L.;Rahman, Anisur;Steinsson, Kristjan;Franks, Andrew G., Jr.;Sigler, Lisa;Hameed, Suhail;Fang, Hong;Ngoc Pham;Brey, Robin;Weisman, Michael H.;McGwin, Gerald, Jr.;Magder, Laurence S.
通讯作者：
Magder, Laurence S.
影响因子：
5
作者：
LaMoreaux, Brian;Barbar-Smiley, Fatima;Madhoun, Hareth
通讯作者：
Madhoun, Hareth
影响因子：
3.3
作者：
Liu, Xiaofan;Hou, Ming;Yang, Renchi
通讯作者：
Yang, Renchi
DOI：
10.3760/cma.j.issn.0578-1426.2019.04.009
发表时间：
2019-04-01
期刊：
Zhonghua nei ke za zhi
影响因子：
--
作者：
Xue, Y;Xu, D;Zeng, X F
通讯作者：
Zeng, X F
影响因子：
7.2
作者：
Sourial, Nadia;Wolfson, Christina;Zhu, Bin;Quail, Jacqueline;Fletcher, John;Karunananthan, Sathya;Bandeen-Roche, Karen;Beland, Francois;Bergman, Howard
通讯作者：
Bergman, Howard