自身免疫性疾病中无干细胞救援的大剂量环磷酰胺:系统综述

IF 1.3 Q4 RHEUMATOLOGY European journal of rheumatology Pub Date : 2024-09-05 DOI:10.5152/eurjrheum.2024.23104
Jozélio Freire de Carvalho, Thelma L Skare
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引用次数: 0

摘要

大剂量环磷酰胺无干细胞救援是治疗难治性自身免疫性疾病的根本疗法。本研究旨在回顾大剂量环磷酰胺治疗自身免疫性疾病的结果。我们在PubMed、Scielo和Embase数据库中系统检索了1966年至2022年9月期间有关大剂量环磷酰胺(不含干细胞救援)治疗自身免疫性疾病的文章。共发现29项研究,包括404名患者。研究最多的疾病是系统性红斑狼疮(113例)、多发性硬化症(99例)、再生障碍性贫血(86例)和重症肌无力(33例)。大多数作者都采用了环磷酰胺 50 毫克/千克/天,连续四天的治疗方案,同时使用 Mesna、预防性抗生素、G-CSF(粒细胞集落刺激因子)、红细胞支持和血小板输注。最常见的副作用是发热性中性粒细胞减少症、脱发和胃肠道不适。在治疗效果方面,大多数研究表明,潜在的自身免疫性疾病得到了改善,其中一些持续时间较长,但也发现了复发和治疗失败的病例。总之,不使用干细胞救援的大剂量环磷酰胺是治疗严重自身免疫疾病的有效选择。如果采取适当的支持护理措施,这种方法相对安全。
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High-dose Cyclophosphamide Without Stem Cell Rescue in Autoimmune Diseases: A Systematic Review.

High-dose cyclophosphamide without stem cell rescue is a radical therapy for refractory autoimmune diseases. The objective was to review the results of high-dose cyclophosphamide without stem cell rescue in autoimmune diseases. PubMed, Scielo, and Embase databases were systematically searched for articles on high-dose cyclophosphamide without stem cell rescue treating autoimmune diseases between 1966 and September 2022. Twenty-nine studies were identified, including a total of 404 patients. The diseases most studied were systemic lupus erythematosus (n=113), multiple sclerosis (n=99), aplastic anemia (n=86), and myasthenia gravis (n=33). Most authors used the posology of 50mg/kg/day over four days of cyclophosphamide associated with Mesna, prophylactic antibiotics, G-CSF (granulocyte colony-stimulating factor), and support of red blood cells, and platelet transfusion. The most common side effects were febrile neutropenia, alopecia, and gastrointestinal complaints. Regarding outcomes, most of the studies demonstrated improvement of the underlying autoimmune disease, some long-lived, but relapses and failures were also identified. In conclusion, high-dose cyclophosphamide without stem cell rescue is an effective option for treating severe autoimmune diseases. This procedure is relatively safe when the appropriate supportive care measures are taken.

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