低剂量和标准剂量马抗胸腺细胞球蛋白治疗重型再生障碍性贫血的疗效相当。

IF 2.3 Q2 HEMATOLOGY
Arihant Jain, Aditya Jandial, Thenmozhi Mani, Kamal Kishore, Charanpreet Singh, Deepesh Lad, Gaurav Prakash, Alka Khadwal, Reena Das, Neelam Varma, Subhash Varma, Pankaj Malhotra
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引用次数: 0

摘要

背景:治疗再生障碍性贫血(AA)的马抗胸腺细胞球蛋白(hATG)ATGAM(辉瑞公司,美国)或其生物类似物胸腺球蛋白(巴拉特血清公司,印度)的标准剂量(SD)为 40 毫克/千克/天,与环孢素联合使用 4 天。有关 hATG 剂量对长期疗效影响的数据十分有限。在此,我们介绍了使用25毫克/千克/天(低剂量[LD])hATG与SD治疗再生障碍性贫血4天的比较经验:我们对接受过两种剂量 hATG 联合环孢素治疗的 AA 患者(年龄大于 12 岁)进行了回顾性研究。在93例接受hATG治疗的AA患者中,分别有62例(66.7%)和31例(33.3%)接受了LD和SD hATG联合环孢素治疗。其中,17 例(18.2%)患者在接受 hATG 和环孢素治疗的同时还接受了艾曲波帕治疗。LD 组和 SD hATG 组在 3 个月(50% vs. 48.4%;P = 0.88)、6 个月(63.8% vs. 71.4%;P = 0.67)和 12 个月(69.6% vs. 79.2%;P = 0.167)的总反应率[完全反应(CR)和部分反应(PR)]相当。在研究人群中,5 年总生存率和无事件生存率的 Kaplan-Meier 估计平均值(标准差)分别为 82.1 (4.6)% 和 70.9 (5.5)%。接受LD hATG剂量与SD hATG剂量的5年Kaplan-Meier估计总生存率和无事件生存率的平均值(标准差)分别为82.9(5-3)%对74.8(10-3)%(P = 0-439),75.2(6.2)%对61.4(11.2)%(P = 0-441):我们的研究表明,在真实世界中,AA 和 LD 患者对 hATG 联合环孢素的反应率与 SD 患者相似。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Comparable outcomes with low-dose and standard-dose horse anti-thymocyte globulin in the treatment of severe aplastic anemia.

Background: The standard dose (SD) of horse anti-thymocyte globulin (hATG) ATGAM (Pfizer, USA) or its biosimilar thymogam (Bharat Serum, India) for the treatment of Aplastic Anemia (AA) is 40 mg/kg/day for 4 days in combination with cyclosporine. Data on the impact of hATG dose on long-term outcomes are limited. Here, we describe our comparative experience using 25 mg/kg/day (low-dose [LD]) hATG for 4 days with SD for the treatment of AA.

Methods: We retrospectively studied patients with AA (age > 12 years) who received two doses of hATG combined with cyclosporine. Among 93 AA patients who received hATG, 62 (66.7%) and 31 (33.3%) patients received LD and SD hATG with cyclosporine, respectively. Among these,seventeen(18.2%) patients also received eltrombopag with hATG and cyclosporine. Overall response rates [complete response (CR) and partial response (PR)] of LD and SD hATG groups at 3 months (50% vs. 48.4%; p = 0.88), 6 months (63.8% vs. 71.4%; p = 0.67), and 12 months (69.6% vs. 79.2%; p = 0.167) were comparable. The mean (Standard Deviation) 5-year Kaplan-Meier estimate of overall survival and event-free survival was 82.1 (4.6)% and 70.9 (5.5)% for the study population. The mean (standard deviation) 5-year Kaplan-Meier estimate of overall survival and event-free survival of those who received LD hATG versus SD hATG dose was 82.9 (5·3)% versus 74.8 (10·3)% (P = 0·439), and 75.2 (6.2)% versus 61.4(11.2)% (P = 0·441).

Conclusion: Our study revealed that the response rates of patients with AA and LD were similar to those of patients with SD to hATG combined with cyclosporine in a real-world setting.

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来源期刊
Blood Research
Blood Research HEMATOLOGY-
CiteScore
3.70
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