Department of Hematology, Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Science, Beijing, China.
Ann Med. 2023;55(2):2282180. doi: 10.1080/07853890.2023.2282180. Epub 2023 Nov 15.
Some patients with warm autoimmune haemolytic anaemia (wAIHA) or Evans syndrome (ES) have no response to glucocorticoid or relapse. Recent studies found that sirolimus was effective in autoimmune cytopenia with a low relapse rate.
Data from patients with refractory/relapsed wAIHA and ES in Peking Union Medical College Hospital from July 2016 to May 2022 who had been treated with sirolimus for at least 6 months and followed up for at least 12 months were collected retrospectively. Baseline and follow-up clinical data were recorded and the rate of complete response (CR), partial response (PR) at different time points, adverse events, relapse, outcomes, and factors that may affect the efficacy and relapse were analyzed.
There were 44 patients enrolled, with 9 (20.5%) males and a median age of 44 (range: 18-86) years. 37 (84.1%) patients were diagnosed as wAIHA, and 7 (15.9%) as ES. Patients were treated with sirolimus for a median of 23 (range: 6-80) months and followed up for a median of 25 (range: 12-80) months. 35 (79.5%) patients responded to sirolimus, and 25 (56.8%) patients achieved an optimal response of CR. Mucositis (11.4%), infection (9.1%), and alanine aminotransferase elevation (9.1%) were the most common adverse events. 5/35 patients (14.3%) relapsed at a median of 19 (range: 15-50) months. Patients with a higher sirolimus plasma trough concentration had a higher overall response (OR) and CR rate ( 0.009, 0.011, respectively). At the time of enrolment, patients were divided into two subgroups that relapsed or refractory to glucocorticoid, and the former had poorer relapse-free survival ( 0.032) than the other group.
Sirolimus is effective for patients with primary refractory/relapsed wAIHA and ES, with a low relapse rate and mild side effects. Patients with a higher sirolimus plasma trough concentration had a higher OR and CR rate, and patients who relapsed to glucocorticoid treatment had poorer relapse-free survival than those who were refractory.
一些温抗体自身免疫性溶血性贫血（wAIHA）或 Evans 综合征（ES）患者对糖皮质激素无反应或复发。最近的研究发现，西罗莫司在自身免疫性血细胞减少症中有效，且复发率低。
回顾性收集 2016 年 7 月至 2022 年 5 月在我院接受西罗莫司治疗至少 6 个月、随访至少 12 个月的难治性/复发性 wAIHA 和 ES 患者的临床资料。记录患者的基线和随访临床资料，并分析不同时间点完全缓解（CR）、部分缓解（PR）的比例、不良反应、复发、转归以及可能影响疗效和复发的因素。
共纳入 44 例患者，男 9 例（20.5%），中位年龄 44 岁（范围：18-86 岁）。37 例（84.1%）患者诊断为 wAIHA，7 例（15.9%）为 ES。患者接受西罗莫司治疗的中位时间为 23 个月（范围：6-80 个月），中位随访时间为 25 个月（范围：12-80 个月）。35 例（79.5%）患者对西罗莫司有反应，25 例（56.8%）患者达到 CR 的最佳缓解。最常见的不良反应是黏膜炎（11.4%）、感染（9.1%）和丙氨酸氨基转移酶升高（9.1%）。5/35 例（14.3%）患者在中位 19 个月（范围：15-50 个月）时复发。西罗莫司血药谷浓度较高的患者总缓解（OR）和 CR 率较高（ 0.009，0.011）。入组时，患者分为对糖皮质激素复发或难治的两组，前者无复发生存率较差（ 0.032）。
西罗莫司治疗原发性难治性/复发性 wAIHA 和 ES 有效，复发率低，副作用轻微。西罗莫司血药谷浓度较高的患者 OR 和 CR 率较高，对糖皮质激素治疗复发的患者无复发生存率较难治的患者差。
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