Liu Xiaoming, Zou Yao, Wang Shuchun, Zhang Li, Yang Wenyu, Zhang Jiayuan, Liu Fang, Liu Tianfeng, Chen Xiaojuan, Ruan Min, Zhou Jianfeng, Cai Xiaojin, Qi Benquan, Chang Lixian, An Wenbin, Guo Ye, Chen Yumei, Zhu Xiaofan
Department of Pediatric Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China.
Department of Pediatric Hematology,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China.
Zhonghua Er Ke Za Zhi. 2014 Feb;52(2):84-9.
To compare the efficacy and safety of four different regimens for pediatric severe aplastic anemia (SAA) with immuno-suppressive therapy (IST) with or without combined human granulocyte colony-stimulating factor (G-CSF).
The authors retrospectively analyzed 105 children with SAA treated with IST with or without G-CSF in the hospital from February 2000 to September 2010. Regimen A, without G-CSF in the whole treatment, was used to treat Group A patients, n = 27; Regimen B, G-CSF, was initiated in Group B, n = 24, before the IST until hematologic recovery; Regimen C, G-CSF, was used together with the IST for Group C patients, n = 24, until hematologic recovery; Regimen D,G-CSF was used for Group D, n = 30, after the end of IST until hematologic recovery. The response rate, relapse rate, mortality, infection rate, infection-related death rate, risk of evolving into MDS/AML, survival rate, factors affecting the time of event-free survival and so on.
(1) The response (CR+PR) rates 4, 6, 12 and 24 months after IST of the whole series of 105 SAA children were 50.5% (7.6%+42.9%) , 60.0% (21.9%+38.1%) , 67.6% (38.1%+29.5%) and 69.5% (40.0%+29.5%) respectively. The 2-year survival rate was 90.5%; the follow-up of the patients for 13 years showed that the whole survival rate was 87.6%. (2) The differences of the response rates 4, 6, 12 and 24 months after IST of the 4 groups were not significant (P > 0.05). (3) No significant differences were found in the mortalities 4, 6, 12 and 24 months among the 4 groups (P > 0.05). (4) Of the 105 patients, 4 children had relapsed disease in the period of time from 6 to 24 months after IST. All the four patients belonged to the groups with G-CSF. (5) The use of G-CSF could not decrease the infection period before IST (day) (P = 0.273), and it had no impact on the infection rate after IST (P = 0.066). It did not reduce the rates of septicemia and infectious shock. And to the infection-related death rate no significant conclusion can be made. (6) Follow up of the patients for 13 years, showed that 2 had the evolution to MDS/AML in the 105 patients and the two children belonged to the groups with G-CSF. (7) Kaplan-meier curve analysis did not show any differences in the survival rates of the four groups. (8) Cox regression analysis showed that the use of G-CSF had no benefit to the patients' long term survival. While the age of diagnosis and the infection history before IST were significantly related to the patients' long term survival.
The use of G-CSF did not contribute to the early response and could not reduce the infection rate, infection-related death rate and the patients' long term survival. There were no significant differences in the survival rates of the four groups. Attention should be paid to the risk of the evolution to MDS/AML.
比较四种不同方案治疗小儿重型再生障碍性贫血（SAA）行免疫抑制治疗（IST）联合或不联合人粒细胞集落刺激因子（G-CSF）的疗效和安全性。
回顾性分析2000年2月至2010年9月在本院接受IST联合或不联合G-CSF治疗的105例SAA患儿。A组（n = 27）采用全程不使用G-CSF的方案A治疗；B组（n = 24）在IST前开始使用G-CSF直至血液学恢复；C组（n = 24）在IST期间同时使用G-CSF直至血液学恢复；D组（n = 30）在IST结束后使用G-CSF直至血液学恢复。观察缓解率、复发率、死亡率、感染率、感染相关死亡率、演变为骨髓增生异常综合征/急性髓系白血病（MDS/AML）的风险、生存率、影响无事件生存时间的因素等。
（1）105例SAA患儿IST后4、6、12和24个月的缓解（CR + PR）率分别为50.5%（7.6% + 42.9%）、60.0%（21.9% + 38.1%）、67.6%（38.1% + 29.5%）和69.5%（40.0% + 29.5%）。2年生存率为90.5%；对患者随访13年，总体生存率为87.6%。（2）4组IST后4、6、12和24个月的缓解率差异无统计学意义（P > 0.05）。（3）4组IST后4、6、12和24个月的死亡率差异无统计学意义（P > 0.05）。（4）105例患者中，4例在IST后6至24个月期间复发，4例均属于使用G-CSF的组。（5）使用G-CSF不能缩短IST前的感染期（天）（P = 0.273），对IST后的感染率无影响（P = 0.066），未降低败血症和感染性休克的发生率，对感染相关死亡率未得出显著结论。（6）对患者随访13年，105例中有2例演变为MDS/AML，2例患儿均属于使用G-CSF的组。（7）Kaplan-meier曲线分析显示4组生存率无差异。（8）Cox回归分析显示使用G-CSF对患者长期生存无益处，而诊断时年龄和IST前感染史与患者长期生存显著相关。
使用G-CSF无助于早期缓解，不能降低感染率、感染相关死亡率和患者长期生存率。4组生存率无显著差异。应注意演变为MDS/AML的风险。
