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. 2020 Aug 14;41(8):637-642.
doi: 10.3760/cma.j.issn.0253-2727.2020.08.004.

[Evaluation of the efficacy of cyclosporin A combined with recombined human thrombopoietin for treating patients with non-severe aplastic anemia]

[Article in Chinese]
M L Zhang  1 , W S Chen  1 , B Han  1
Affiliations

[Evaluation of the efficacy of cyclosporin A combined with recombined human thrombopoietin for treating patients with non-severe aplastic anemia]

[Article in Chinese]
M L Zhang et al. Zhonghua Xue Ye Xue Za Zhi. .

Abstract
in English, Chinese

Objectives: To compare the efficacy of cyclosporin A (CsA) alone and CsA combined with recombined human thrombopoietin (rhTPO) in patients with non-severe aplastic anemia (NSAA) . Methods: Data from 83 patients with NSAA between August 2014 and February 2019 were collected retrospectively. The study population included 35 men and 48 women, with a median age of 45 years (14-85 years) . Among them, 57 had been treated with CsA + rhTPO, TPO was administered at 15 000 U QD for 7 days, once a month for 3 months, and the other 26 patients with compatible baseline characters were treated with CsA alone. All the enrolled patients had been treated with CsA for at least 6 months and were followed up for at least 1 year. The efficacy and outcome were compared between the two groups. Results: Total 23 men and 34 women, with a median age of 46 years (14-85 years) were treated with CsA + rhTPO. The median duration of CsA treatment was 17 (8-28) months, and the patients were followed up for a median of 27 (12-45) months. Total 12 men and 14 women, with a median age of 40 years (20-64) were treated with CsA alone. The median duration of CsA treatment was 19 months (9-30 months) , and the median follow-up duration was 29 months (16-66 months) . There was no significant difference in the baseline characteristics of the two groups (P>0.05) . There was no significant difference in the CR and OR rates of the two groups at 1, 3, 6, 12, and 24 months of treatment (P>0.05) . The change in the platelet level for the CsA + rhTPO treated group after 1 month[8 (-12-86) ×10(9)/L vs. 3 (16-57) ×10(9)/L, P=0.029) , 3 months[24 (-6-102) ×10(9)/L vs. 7 (-9-76) ×10(9)/L, P=0.006], and 6 months[33.5 (-4-123) ×10(9)/L vs. 12.5 (-14-109) ×10(9)/L, P=0.048] of treatment was higher than that in the CsA alone group, while no significant difference was found between the two groups at other time points. There was no significant difference in the change in the megakaryocyte level between the two groups[3 (0-4) vs. 2 (0-5) , z=-0.868, P=0.385] after 6 months of treatment. Apart from 10.5% (6/57) of the patients in the CsA + rhTPO treated group who reported soreness at the injection site, there was no other significant difference between the two groups in terms of adverse effects. During the follow-up period, there were two cases of increasing paroxysmal nocturnal hemoglobinuria clone to over 10%, one in the CsA + rhTPO treated group, the other in the CsA alone group; and there was one case of progression to SAA in the CsA + rhTPO treated group; while no case of death or thromboembolic event (TEE) , fibrosis or reticulin proliferation, progression to myelodysplastic syndrome (MDS) , or acute myeloid leukemia was observed in either group. There was one case of progression to SAA in the CsA + rhTPO treated group but none in the CsA alone group. Conclusion: Compared to CsA alone, CsA + rhTPO treatment can accelerate the recovery of the platelet level with acceptable adverse effects.

目的： 比较环孢素A（CsA）联合重组人血小板生成素（rhTPO）和单用CsA治疗非重型再生障碍性贫血（NSAA）的疗效。 方法： 回顾2014年8月至2019年2月83例初治NSAA患者临床资料，男35例，女48例，中位年龄45（14~85）岁。其中57例使用CsA+rhTPO治疗（TPO治疗组），TPO 15 000 U，皮下注射，每日1次，共7 d，28 d为1个疗程，最多治疗3个疗程；26例为同期基线相匹配的患者，仅单用CsA治疗（对照组）。入选患者CsA使用至少6个月，并随访至少1年。比较两组的疗效及转归。 结果： TPO治疗组患者男23例，女34例；中位年龄46（14~85）岁；CsA中位应用时间为17（8~28）个月；中位随访时间为27（12~45）个月。对照组26例，男12例，女14例；中位年龄40（20~64）岁；CsA中位应用时间为19（9~30）个月；中位随访时间为29（16~66）个月。两组基线水平差异均无统计学意义（P值均>0.05）。两组1、3、6、12和24个月的CR率及OR率差异均无统计学意义（P值均>0.05）。TPO治疗组1个月[8（-12~86）×10(9)/L对3（16~57）×10(9)/L，P=0.029]、3个月[24（-6~102）×10(9)/L对7（-9~76）×10(9)/L，P=0.006]和6个月[33.5（-4~123）×10(9)/L对12.5（-14~109）×10(9)/L，P=0.048]血小板升高水平均显著高于对照组，但12个月及24个月与对照组差异无统计学意义（P值均>0.05）。两组6个月时骨髓巨核细胞变化水平差异无统计学意义[3（0~4）个对2（0~5）个，z=-0.868，P=0.385]。TPO治疗组10.5%（6/57）出现注射部位疼痛，除此之外，两组不良反应无明显差异。随访期间，两组患者各有1例阵发性睡眠性血红蛋白尿（PNH）克隆>10%；TPO治疗组1例进展为SAA；均无死亡、无血栓事件发生，未见网状纤维及胶原纤维增生，未见骨髓增生异常综合征和急性髓系白血病转化。 结论： CsA联合TPO治疗相比CsA单药治疗可较快提升NSAA患者血小板水平，而不增加明显的不良反应。.

Keywords: Adverse effects; Aplastic anemia; Cyclosporin A; Recombined human thrombopoietin; Treatment outcome.

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