de Carvalho Jozélio Freire, Skare Thelma L
Núcleo de Pesquisa Em Doenças Crônicas Não Transmissíveis (NUPEN), School of Nutrition from the Federal University of Bahia, Salvador, Bahia, Brazil.
Rheumatology Unit, Hospital Evangélico Mackenzie, Curitiba, PR, Brazil.
Eur J Rheumatol. 2024 Sep 5;11(2):53-63. doi: 10.5152/eurjrheum.2024.23104.
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.
无干细胞救援的大剂量环磷酰胺是治疗难治性自身免疫性疾病的一种根治性疗法。目的是回顾无干细胞救援的大剂量环磷酰胺治疗自身免疫性疾病的结果。系统检索了PubMed、Scielo和Embase数据库中1966年至2022年9月期间关于无干细胞救援的大剂量环磷酰胺治疗自身免疫性疾病的文章。共鉴定出29项研究，包括404例患者。研究最多的疾病是系统性红斑狼疮（n = 113）、多发性硬化症（n = 99）、再生障碍性贫血（n = 86）和重症肌无力（n = 33）。大多数作者采用环磷酰胺50mg/kg/天，共4天的剂量方案，联合美司钠、预防性抗生素、粒细胞集落刺激因子（G-CSF）以及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、脱发和胃肠道不适。关于治疗结果，大多数研究表明潜在的自身免疫性疾病有所改善，有些改善持续时间较长，但也发现了复发和治疗失败的情况。总之，无干细胞救援的大剂量环磷酰胺是治疗严重自身免疫性疾病的有效选择。采取适当的支持性护理措施时，该治疗方法相对安全。
