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不同诊断标准下移植相关性血栓性微血管病的生存分析及血浆置换的疗效。

Survival analysis of transplant-associated thrombotic microangiopathy under different diagnostic criteria and the efficacy of plasma exchange.

机构信息

Medical School of Chinese PLA, Beijing, China (mainland).

Department of Hematology, The Fifth Medical Center, Chinese PLA General Hospital, Beijing, China (mainland).

出版信息

Ann Transplant. 2023 Jun 20;28:e939890. doi: 10.12659/AOT.939890.

DOI:10.12659/AOT.939890
PMID:37337423
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10290434/
Abstract

BACKGROUND Transplant-associated thrombotic microangiopathy (TA-TMA) is a serious complication of hematopoietic stem cell transplantation (HSCT). The efficacy and survival of plasma exchange (PE) for TA-TM have not been fully clarified. In addition, there is a lack of consensus on diagnostic criteria for TA-TMA.  MATERIAL AND METHODS We retrospectively analyzed 32 patients diagnosed with TA-TMA by different diagnostic criteria from January 2018 to February 2022 at the First Medical Center of the PLA General Hospital. RESULTS (1) The patients with TA-TMA treated with PE in this study had a remission rate of 42.8%, a 100-day OS of 47.6%, and a 6-month OS of 38.1%. The only factor affecting the response to PE treatment was the number of PE sessions (P = 0.047). (2) III-IV aGVHD prior to TA-TMA diagnosis (P = 0.002), renal or neurological dysfunction (P = 0.021), and the time to onset of TA-TMA (P = 0.002) were independent risk factors for overall survival with TA- TMA. (3) Probable TA-TMA had the highest survival rate, but the Jodele criteria are expected to diagnose earlier and provide the greatest benefit to patients. CONCLUSIONS PE is an effective treatment for TA-TMA especially in cases where complement blockers are not available. In addition, probable TA-TMA improved prognostic survival through early detection of patients with TA-TMA. There is a need for further large prospective trials to identify the population more suitable for PE treatment of TA-TMA and more valid diagnostic criteria.

摘要

背景

移植相关血栓性微血管病(TA-TMA)是造血干细胞移植(HSCT)的严重并发症。血浆置换(PE)治疗 TA-TMA 的疗效和生存情况尚未完全明确。此外,TA-TMA 的诊断标准也缺乏共识。

材料与方法

我们回顾性分析了 2018 年 1 月至 2022 年 2 月在解放军总医院第一医学中心根据不同诊断标准诊断为 TA-TMA 的 32 例患者。

结果

(1)本研究中接受 PE 治疗的 TA-TMA 患者缓解率为 42.8%,100 天 OS 为 47.6%,6 个月 OS 为 38.1%。唯一影响 PE 治疗反应的因素是 PE 次数(P = 0.047)。(2)TA-TMA 诊断前 III-IV 度急性移植物抗宿主病(aGVHD,P = 0.002)、肾功能或神经系统功能障碍(P = 0.021)以及 TA-TMA 发病时间(P = 0.002)是 TA-TMA 总生存的独立危险因素。(3)可能的 TA-TMA 具有最高的生存率,但 Jodele 标准有望更早诊断,并为患者带来最大益处。

结论

PE 是治疗 TA-TMA 的有效方法,特别是在补体阻滞剂不可用时。此外,通过早期发现 TA-TMA 患者,可能的 TA-TMA 提高了预后生存率。需要进一步的大型前瞻性试验来确定更适合 TA-TMA 的 PE 治疗人群和更有效的诊断标准。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/cf8870968d64/anntransplant-28-e939890-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/acf2e11e1e1c/anntransplant-28-e939890-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/2d0d7f25c25c/anntransplant-28-e939890-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/faf7be11ad04/anntransplant-28-e939890-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/cf8870968d64/anntransplant-28-e939890-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/acf2e11e1e1c/anntransplant-28-e939890-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/2d0d7f25c25c/anntransplant-28-e939890-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/faf7be11ad04/anntransplant-28-e939890-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ac61/10290434/cf8870968d64/anntransplant-28-e939890-g004.jpg

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本文引用的文献

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The Role of Complement in HSCT-TMA: Basic Science to Clinical Practice.补体在 HSCT-TMA 中的作用:基础科学到临床实践。
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Risk factors and appropriate therapeutic strategies for thrombotic microangiopathy after allogeneic HSCT.异基因造血干细胞移植后血栓性微血管病的危险因素及适宜的治疗策略。
Blood Adv. 2020 Jul 14;4(13):3169-3179. doi: 10.1182/bloodadvances.2020002007.
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Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab.补体阻断治疗 TA-TMA:依库珠单抗治疗的大型儿科队列中获得的经验教训。
Blood. 2020 Mar 26;135(13):1049-1057. doi: 10.1182/blood.2019004218.
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Distribution of Transplantation-Associated Thrombotic Microangiopathy (TA-TMA) and Comparison between Renal TA-TMA and Intestinal TA-TMA: Autopsy Study.移植相关血栓性微血管病(TA-TMA)的分布及肾 TA-TMA 与肠 TA-TMA 的比较:尸检研究。
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