Luo Xiu, Lu Huina, Xiu Bing, Wu Hao, Li Bing, Li Ping, Chen Yuhua, Zhou Lili, Zhang Wenjun, Dong Yan, Liang Aibin, Ding Yi
Department of Hematology, Tongji Hospital, Tongji University School of Medicine, Shanghai 200065, P.R. China.
Exp Ther Med. 2018 Feb;15(2):1966-1974. doi: 10.3892/etm.2017.5616. Epub 2017 Dec 12.
The present study aimed to evaluate the efficacy and safety of combined immunosuppressive therapy (IST) plus umbilical cord blood infusion (UCBI) in severe aplastic anemia (SAA) patients. A total of 68 patients with SAA were enrolled in the current prospective cohort study and divided into the IST (n=35; positive control) and IST+UCBI (n=33; experimental) groups according to the treatment conditions. Patients in the IST group were treated with rabbit antithymocyte globulin (r-ATG) at a dose of 2.5 mg/kg through intravenous infusion once a day for five days. This was combined with oral cyclosporine A (CsA) at a dose of 3-5 mg/kg twice a day for 2 years. Patients in the IST+UBCI group were treated with r-ATG and CsA at the same doses and frequencies as the IST group plus one UCBI 1 day after the final treatment with r-ATG. At 6 months post treatment, the complete response and overall response rate (ORR) of the IST+UCBI group were markedly higher compared with those in the IST group. Furthermore, patients in the IST+UCBI group achieved absolute neutrophil count (ANC) and platelet count responses more rapidly as compared with the IST group. However, no difference in the hemoglobin (Hb) response was identified between the two groups. In addition, SAA patients achieved responses in the ANC and platelet count more rapidly in comparison with very severe aplastic anemia (VSAA) patients, while the number of days to Hb responses were similar in the SAA and VSAA patients. Multivariate logistic regression analysis also revealed that IST+UCBI treatment was an independent predicting factor for patients achieving complete response or partial response, whereas VSAA was an independent predictor of a worse ORR. Platelet and reticulocyte were also independent predicting factors. Finally, the survival of patients was similar between the groups, and no difference in the safety of the treatment was observed. In conclusion, combined IST plus UCBI treatment may be applied as an effective and safe therapy for SAA patients.
本研究旨在评估联合免疫抑制治疗（IST）加脐血输注（UCBI）对重型再生障碍性贫血（SAA）患者的疗效和安全性。共有68例SAA患者纳入当前的前瞻性队列研究，并根据治疗情况分为IST组（n = 35；阳性对照）和IST + UCBI组（n = 33；试验组）。IST组患者接受兔抗胸腺细胞球蛋白（r-ATG）治疗，剂量为2.5 mg/kg，通过静脉输注，每天1次，共5天。这与口服环孢素A（CsA）联合使用，剂量为3 - 5 mg/kg，每天2次，持续2年。IST + UBCI组患者接受与IST组相同剂量和频率的r-ATG和CsA治疗，外加在最后一次r-ATG治疗后1天进行1次UCBI。治疗后6个月，IST + UCBI组的完全缓解率和总缓解率（ORR）显著高于IST组。此外，与IST组相比，IST + UCBI组患者的绝对中性粒细胞计数（ANC）和血小板计数反应更快。然而，两组之间血红蛋白（Hb）反应无差异。此外，与极重型再生障碍性贫血（VSAA）患者相比，SAA患者的ANC和血小板计数反应更快，而SAA和VSAA患者达到Hb反应的天数相似。多因素逻辑回归分析还显示，IST + UCBI治疗是患者实现完全缓解或部分缓解的独立预测因素，而VSAA是ORR较差的独立预测因素。血小板和网织红细胞也是独立预测因素。最后，两组患者的生存率相似，且未观察到治疗安全性方面的差异。总之，联合IST加UCBI治疗可作为SAA患者有效且安全的治疗方法。
