PMID- 33552043 OWN - NLM STAT- MEDLINE DCOM- 20210503 LR - 20210503 IS - 1664-3224 (Electronic) IS - 1664-3224 (Linking) VI - 11 DP - 2020 TI - Efficacy and Safety of Eculizumab in the Treatment of Transplant-Associated Thrombotic Microangiopathy: A Systematic Review and Meta-Analysis. PG - 564647 LID - 10.3389/fimmu.2020.564647 [doi] LID - 564647 AB - BACKGROUND: Transplant-associated thrombotic microangiopathy (TA-TMA) is a dangerous and life-threatening complication in patients undergoing hematopoietic stem cell transplantation (HSCT). Eculizumab has been used in the treatment of TA-TMA, and several studies have confirmed the benefit of Eculizumab in patients with TA-TMA. However, the results remain controversial. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of Eculizumab for TA-TMA. MATERIALS AND METHODS: We searched PubMed and Embase for studies on the efficacy and safety of Eculizumab in TA-TMA patients. Efficacy outcomes consisted of overall response rate (ORR), complete response rate (CRR), and survival rate at the last follow-up (SR). Safety outcomes were adverse events (AEs), including infection, sepsis, impaired liver function, infusion reactions, and death. RESULTS: A total of 116 patients from six studies were subjected to meta-analysis. The pooled estimates of ORR, CRR, and SR for TA-TMA patients were 71% (95% CI: 58-82%), 32% (95% CI: 11-56%), and 52% (95% CI: 40-65%), respectively. Only one patient presented with a severe rash, and infection was the most common AEs. The main causes of death were infection and GvHD. CONCLUSION: Current evidence suggests that Eculizumab improves SR and ORR in patients with TA-TMA and that Eculizumab is well tolerated. However, the number of studies is limited, and the findings are based mainly on data from observational studies. Higher quality randomized controlled trials and more extensive prospective cohort studies are needed. CI - Copyright (c) 2021 Zhang, Zhou, Qi, Miao, Zhang, Wu and Han. FAU - Zhang, Rui AU - Zhang R AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. FAU - Zhou, Meng AU - Zhou M AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. AD - National Clinical Research Center for Hematologic Diseases, Suzhou, China. FAU - Qi, Jiaqian AU - Qi J AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. AD - National Clinical Research Center for Hematologic Diseases, Suzhou, China. FAU - Miao, Wenjing AU - Miao W AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. FAU - Zhang, Ziyan AU - Zhang Z AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - National Clinical Research Center for Hematologic Diseases, Suzhou, China. FAU - Wu, Depei AU - Wu D AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. AD - National Clinical Research Center for Hematologic Diseases, Suzhou, China. AD - State Key Laboratory of Radiation Medicine and Protection, Soochow University, Suzhou, China. FAU - Han, Yue AU - Han Y AD - Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China. AD - Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China. AD - Institute of Blood and Marrow Transplantation, Suzhou, China. AD - Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, China. AD - National Clinical Research Center for Hematologic Diseases, Suzhou, China. AD - State Key Laboratory of Radiation Medicine and Protection, Soochow University, Suzhou, China. LA - eng PT - Journal Article PT - Meta-Analysis PT - Research Support, Non-U.S. Gov't PT - Systematic Review DEP - 20210120 PL - Switzerland TA - Front Immunol JT - Frontiers in immunology JID - 101560960 RN - 0 (Antibodies, Monoclonal, Humanized) RN - 0 (Complement Inactivating Agents) RN - A3ULP0F556 (eculizumab) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Antibodies, Monoclonal, Humanized/*adverse effects MH - Child MH - Child, Preschool MH - Complement Inactivating Agents/*adverse effects MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Infant MH - Male MH - Middle Aged MH - Observational Studies as Topic MH - Survival Rate MH - Thrombotic Microangiopathies/*drug therapy/*etiology/mortality MH - Treatment Outcome MH - Young Adult PMC - PMC7856300 OTO - NOTNLM OT - Eculizumab OT - efficacy OT - hematopoietic stem cell transplantation OT - meta-analysis OT - safety OT - terminal complement inhibitor OT - transplant-associated thrombotic microangiopathy COIS- The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. EDAT- 2021/02/09 06:00 MHDA- 2021/05/04 06:00 PMCR- 2020/01/01 CRDT- 2021/02/08 05:36 PHST- 2020/05/22 00:00 [received] PHST- 2020/11/30 00:00 [accepted] PHST- 2021/02/08 05:36 [entrez] PHST- 2021/02/09 06:00 [pubmed] PHST- 2021/05/04 06:00 [medline] PHST- 2020/01/01 00:00 [pmc-release] AID - 10.3389/fimmu.2020.564647 [doi] PST - epublish SO - Front Immunol. 2021 Jan 20;11:564647. doi: 10.3389/fimmu.2020.564647. eCollection 2020.