Thrombopoietin receptor agonists for refractory thrombocytopenia in patients after autologous hematopoietic stem cell transplantation

作者全名："Wang, Dan; Lang, Tao; Zeng, Hanqing; Zou, Zhongmin; Yang, Shijie; Cheng, Ting; Liu, Huanfeng; Zhu, Lidan; Xiang, Xixi; Yao, Han; Tang, Shuhan; Kong, Peiyan; Wei, Jin; Xiong, Jingkang; Gao, Lei; Zhang, Xi; Feng, Yimei"

作者地址："[Wang, Dan; Wei, Jin] North Sichuan Med Coll, Affiliated Hosp 1, Dept Oncol, Nanchong, Peoples R China; [Wang, Dan; Yang, Shijie; Cheng, Ting; Liu, Huanfeng; Zhu, Lidan; Xiang, Xixi; Yao, Han; Tang, Shuhan; Kong, Peiyan; Xiong, Jingkang; Gao, Lei; Zhang, Xi; Feng, Yimei] Army Med Univ, Med Ctr Hematol, Affiliated Hosp 2, Chongqing, Peoples R China; [Lang, Tao] Peoples Hosp Xinjiang Uygur Autonomous Reg, Dept Hematol, Urumqi, Peoples R China; [Zeng, Hanqing] Chongqing Med Univ, Dept Hematol, Affiliated Hosp 2, Chongqing, Peoples R China; [Zou, Zhongmin] Army Med Univ, Sch Mil Prevent Med, Dept Chem Def Med, Chongqing, Peoples R China"

通信作者："Wang, D (通讯作者)，North Sichuan Med Coll, Affiliated Hosp 1, Dept Oncol, Nanchong, Peoples R China.; Wang, D; Zhang, X; Feng, YM (通讯作者)，Army Med Univ, Med Ctr Hematol, Affiliated Hosp 2, Chongqing, Peoples R China."

来源：TRANSPLANT IMMUNOLOGY

ESI学科分类：IMMUNOLOGY

WOS号：WOS:001161937100001

JCR分区：Q3

影响因子：1.5

年份：2023

卷号：81

期号：　

开始页：　

结束页：　

文献类型：Article

关键词：Thrombopoietin receptor agonists/TPORAs; Autologous hematopoietic stem cell; transplantation/ASCT; Thrombocytopenia; Platelet count; Megakaryocyte count

摘要："Objective: Autologous hematopoietic stem cell (ASC) transplantation (ASCT) is an effective treatment method for patients with hematological disorders and malignant diseases. The patient's ASCs are harvested prior to radiotherapy/chemotherapy, cryopreserved and then transfused back after the high -dose radiotherapy/chemotherapy conditioning treatment. Since some patients develop thrombocytopenia after receiving ASCT, it is difficult for them to bear simultaneously the management of their original disease and thrombocytopenia. The present study aimed to evaluate the efficacy and safety of thrombocytopenia therapy with thrombopoietin receptor agonists (TPORAs) after ASCT. Methods: We retrospectively analyzed the clinical safety and efficacy of TPORA treatment for the enrolled 20 patients who developed thrombocytopenia after ASCT. The measured parameters were prolonged isolated thrombocytopenia (PIT), secondary failure of platelet recovery (SFPR) and other calculated response index. Patients with platelet count (PC) <= 50x109/L were treated with TPORA, namely with either eltrombopag (Elt), hetrombopag (Het), or avatrobopag (Ava). Results: The group of 20 patients, who received TPORA administration for their thrombocytopenia after ASCT, had a median age of 50 years (ranging between 17 and 60 years). The median administration time of TPORA application was 48 days (ranging from 7 to 451 days); an overall response rate (ORR) was 85% with no response in 15% of patients, while with complete response (CR) in 70% of patients and partial response (PR) in 15% of patients. The median platelet count was 19 x 109/L before TPORA treatment and increased to 87x109)/L after the treatment. The TPORA treatment was safe as only 4 patients (20%) displayed a mild transaminase elevation. No other reported side effects occurred, such as thrombosis, joint pain, diarrhea, and myelofibrosis. It was demonstrated that the short response time to TPORA treatment correlated to the fast platelet recovery, when the number of megakaryocytes in the bone marrow smear exceeded 35/4.5 cm2 under a low magnification of 100 times (p = 0.015). Conclusion: TPORA therapy for thrombocytopenia occurring after the radiotherapy/ chemotherapy-conditioned ASCT was well tolerated and effective for platelets recovery."

基金机构："National Natural Science Foundation of China [82020108004, 82270231]; Youth Project of National Health Commission [WKZX2023CX080001]; Chongqing Natural Science Foundation Innovative Group Program [cstc2021jcyj-cxttX0001]; General Project of Chongqing Natural Science [cstc2020jcyj-msxmX0448, cstc2020jcyj-msxmX1086]; Chongqing Science and Health Joint Major Project [2022DBXM003]"

基金资助正文："This work was supported by National Natural Science Foundation of China (No. 82020108004; No.82270231) , Youth Project of National Health Commission (WKZX2023CX080001) , Chongqing Natural Science Foundation Innovative Group Program (cstc2021jcyj-cxttX0001) , General Project of Chongqing Natural Science (cstc2020jcyj-msxmX0448; cstc2020jcyj-msxmX1086) , and Chongqing Science and Health Joint Major Project (2022DBXM003) ."