查看更多>>摘要:目的 研究美泊利珠单抗用于治疗嗜酸性肉芽肿性多血管炎(EGPA)患者的疗效及安全性。 方法 本研究为非随机对照实验,采用目的抽样法收集2021年8月至2023年8月在广州医科大学附属第一医院接受美泊利珠单抗治疗的20例EGPA患者资料,通过比较治疗前后的伯明翰血管炎活动度评分(BVAS)、血嗜酸性粒细胞计数及百分比、第1秒用力呼气容积占预估值百分比(FEV1%pred)、口服皮质类固醇激素(OCS)维持用量等指标的差异,评估美泊利珠单抗治疗EGPA的疗效,并观察患者用药期间的安全性。 结果 20例EGPA患者女性17例85.0%(17/20),年龄42.0(30.8,52.5)岁。治疗4个月后总应答率为85.0%,血嗜酸性粒细胞计数由0.7(0.5,1.5)×109/L下降至0.1(0,0.1)×109/L(Z=3.88,P<0.001),FEV1%pred由70.4%(49.7%,85.2%)上升至79.9%(72.0%,101.5%)(Z=2.24,P=0.025),FeNO由80.0(35.0,111.5)ppb下降至51.6(28.3,65.4)ppb,(Z=2.54,P=0.011),OCS由25.0(16.3,30.0) mg/d减量为5.9(5.0,7.5)mg/d(Z=3.83,P<0.001)。5例患者治疗12个月后上述临床指标较治疗前仍有明显改善。进一步比较5例美泊利单抗标准剂量治疗组(300 mg组)和15例非标准剂量组(100 mg组)治疗前后临床指标变化,治疗4个月后,2组在降低BVAS评分及血嗜酸性粒细胞计数、改善肺功能指标(FEV1%pred)、减少OCS用量方面差异无统计学意义(均P>0.05)。20例患者共接受125次注射治疗,仅有1例次在注射美泊利珠单抗后出现皮疹,可耐受。 结论 美泊利珠单抗对于治疗EGPA疗效较好,可减少OCS维持治疗剂量,改善肺功能,且安全性良好,未观察到严重不良事件,标准剂量组与非标准剂量组都可以改善EGPA的症状并减少OCS用量。 Objective To investigate the efficacy and safety of mepolizumab in the treatment of patients with eosinophilic granulomatosis with polyangiitis (EGPA). Methods This was a non-randomized controlled trials study.Purposive sampling was used to collect data of 20 EGPA patients treated with mepolizumab at the First Affiliated Hospital of Guangzhou Medical University from August 2021 to December 2023.The study evaluated the differences in Birmingham Vasculitis Activity Score (BVAS), blood eosinophil count and percentage, forced expiratory volume in one second (FEV1) as a percentage of predicted value (FEV1%pred), and oral corticosteroid (OCS) maintenance dose before and after treatment to assess the efficacy of mepolizumab and observe the safety of patients during treatment. Results Among the 20 patients with EGPA, there were 17 females (85.0%), with an average age of 42.0 (30.8, 52.5)years.After 4 months of treatment, the total response rate was 85.0%.The blood eosinophil count decreased from 0.7 (0.5, 1.5)×109/L to 0.1 (0, 0.1)×109/L (Z=3.88, P<0.001). The FEV1%pred increased from 70.4% (49.7%, 85.2%) to 79.9% (72.0%, 101.5%)(Z=2.24, P=0.025). The fractional exhaled nitric oxide (FeNO) level decreased from 80.0 (35.0, 111.5) ppb to 51.6 (28.3, 65.4) ppb (Z=2.54, P=0.011). The oral corticosteroid (OCS) dosage was reduced from 25.0 (16.3, 30.0) mg/d to 5.9 (5.0, 7.5) mg/d (Z=3.83, P<0.001). After 12 months of treatment, 5 patients still show significant improvement inthe above clinical indicators compared to those before treatment.The clinical indicator changes before and after treatment between the standard dose group (300 mg/4 weeks) and the non-standard dose group (100 mg/4 weeks) were compared.The results showed no significant statistical difference in reducing BVAS scores, decreasing blood eosinophil counts, improving lung function indicators (FEV1%pred), and reducing OCS dosage after 4 months of treatment (P>0.05). Among the 20 patients, a total of 125 injections were administered, with only one instance of rash occurring post-injection of mepolizumab, which was tolerable. Conclusions Mepolizumab has shown good efficacy in the treatment of EGPA, canreduce the maintenance dose of OCS andimprove lung function, and demonstrates good safety with no observed serious adverse events.Both the standard dose group and the non-standard dose group can effectively manage the symptoms and reduce the OCS usage in EGPA.