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门诊自体造血细胞移植治疗自身免疫性疾病患者的早期病死率:1700例患者的经验

Early morbimortality in autologous hematopoietic cell transplantation performed on outpatient basis in patients with autoimmune diseases: experience in 1700 patients.

作者信息

Lira-Lara Olivia, Robles-Nasta Max, Olivares-Gazca Juan Carlos, Kharfan-Dabaja Mohamed, Rivera-Álvarez Montserrat, García-Vélez Danae, Lavoignet-Cisneros Michelle, Salgado-Cabrera Mónica D, Gomez-Almaguer David, Gómez-De-León Andrés, Ruiz-Delgado Guillermo J, Ruiz-Argüelles Guillermo J

机构信息

Centro de Hematología y Medicina Interna, Clínica Ruiz, Puebla, Mexico.

Universidad Veracruzana, Veracruz, Veracruz, Mexico.

出版信息

Bone Marrow Transplant. 2025 May;60(5):640-644. doi: 10.1038/s41409-025-02544-y. Epub 2025 Mar 11.

DOI:10.1038/s41409-025-02544-y
PMID:40069376
Abstract

Autologous hematopoietic cell transplantation (aHSCT) is a viable therapeutic approach in patients with autoimmune diseases. Since June 2015, we have autografted on an outpatient basis 1700 aHSCT patients. The objective was to analyze the salient features of early post-aHSCT complications when performed in the outpatient setting. The primary endpoints were early morbi-mortality-free survival (MFS) and overall survival (OS), whereas secondary endpoints entailed hospital admissions, neutropenic fever, Multiple Sclerosis (MS) flare-up, pneumothorax, hyponatremia and myocarditis. Following the "Mexican Method"; 1700 consecutive aHSCT recipients were analyzed: 1667 with MS, 29 with CIDP and 4 with other autoimmune diseases. A total of 1643 (96.6%) grafts were fully completed in the outpatient setting. The 30-day MFS and 30-day OS were 87.7% and 99.8%, respectively. The 30-day MFS has increased from 94.9% in the first 5 years to 98.2% in the last 5 years (p = 0.0002). The 28-day mortality was 0.17%, whereas the 28-day morbidity was 3.3%. The rate of early complications decreased over time, most likely reflecting a learning curve effect. These data support that employing our method is safe in the short term; as this has been done in a 'trial' setting, further research is needed.

摘要

自体造血细胞移植(aHSCT)是治疗自身免疫性疾病患者的一种可行治疗方法。自2015年6月以来,我们已在门诊为1700例患者进行了自体造血细胞移植。目的是分析在门诊进行aHSCT后早期并发症的显著特征。主要终点是早期无病死亡率生存(MFS)和总生存(OS),而次要终点包括住院、中性粒细胞减少性发热、多发性硬化(MS)复发、气胸、低钠血症和心肌炎。按照“墨西哥方法”,对1700例连续的aHSCT受者进行了分析:1667例患有MS,29例患有慢性炎症性脱髓鞘性多发性神经病(CIDP),4例患有其他自身免疫性疾病。共有1643例(96.6%)移植在门诊完成。30天MFS和30天OS分别为87.7%和99.8%。30天MFS从最初5年的94.9%增加到最近5年的98.2%(p = 0.0002)。28天死亡率为0.17%,而28天发病率为3.3%。早期并发症发生率随时间下降,很可能反映了学习曲线效应。这些数据支持在短期内采用我们的方法是安全的;由于这是在“试验”环境中进行的,还需要进一步研究。

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本文引用的文献

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BEAM or cyclophosphamide in autologous haematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis.在自体造血干细胞移植治疗复发缓解型多发性硬化症中,使用硼替佐米或环磷酰胺。
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Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE).自体造血干细胞移植及其他细胞疗法在多发性硬化症和免疫介导的神经疾病中的应用:欧洲血液与骨髓移植学会自身免疫性疾病工作组(ADWP)以及欧洲血液与骨髓移植学会和国际细胞治疗学会联合认证委员会(JACIE)的更新指南和建议
Bone Marrow Transplant. 2020 Feb;55(2):283-306. doi: 10.1038/s41409-019-0684-0. Epub 2019 Sep 26.
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A Feasibility Study of the Full Outpatient Conduction of Hematopoietic Transplants in Persons with Multiple Sclerosis Employing Autologous Non-Cryopreserved Peripheral Blood Stem Cells.对使用自体非冷冻保存外周血干细胞的多发性硬化症患者进行全门诊造血移植的可行性研究。
Acta Haematol. 2017;137(4):214-219. doi: 10.1159/000469655. Epub 2017 May 18.
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