CAJ | 학술논문

目的总结以抗胸腺细胞球蛋白(ATG)为主的免疫抑制疗法治疗儿童再生障碍性贫血时,ATG的实施方法、不良反应防治和长期随访措施等与远期疗效的相关性.方法儿童再生障碍性贫血35例,其中极重型再生障碍性贫血(VSAA)6例,急性再生障碍性贫血(SAA)11例,慢性重型SAA8例,中型再生障碍性贫血(MAA)10例.ATG治疗期间措施包括:治疗前清除感染灶;积极防治过敏反应;密切观察和处理因ATG相关血小板减少所致严重出血和免疫抑制所致严重感染;积极防治血清病;重视长期随访中的治疗措施与质量.结果 ATG治疗后,所有病例均出现60%以上的淋巴细胞绝对计数下降.平均随访28个月,总有效率为77.14%(27/35),显效率为57.14%(20/35).VSAA、SAA和MAA间疗效无明显差异.ATG不良反应观察结果:①48.6%出现轻度类过敏反应;②血清病发生率42.9%,平均病程3.6 d;③9例(25.7%)外周血小板(BPC)计数＜10×109/L;④8例(22.9%)在ATG治疗后1个月内发生感染;⑤未发生ATG治疗相关死亡.Genzyme和Fresenius两种不同ATG制剂,在疗效和ATG相关不良反应发生率方面差异均无统计学意义.结论 ATG治疗儿童SAA和MAA疗效显著,但需积极预防和控制ATG不良反应,避免治疗相关死亡.长期辅助治疗和提高随访质量,也是确保疗效的重要环节.
목적 총결이항흉선세포구단백(ATG)위주적면역억제요법치료인동재생장애성빈혈시,ATG적실시방법、불량반응방치화장기수방조시등여원기료효적상관성.방법 인동재생장애성빈혈35례,기중겁중형재생장애성빈혈(VSAA)6례,급성재생장애성빈혈(SAA)11례,만성중형SAA8례,중형재생장애성빈혈(MAA)10례.ATG치료기간조시포괄:치료전청제감염조;적겁방치과민반응;밀절관찰화처리인ATG상관혈소판감소소치엄중출혈화면역억제소치엄중감염;적겁방치혈청병;중시장기수방중적치료조시여질량.결과 ATG치료후,소유병례균출현60%이상적림파세포절대계수하강.평균수방28개월,총유효솔위77.14%(27/35),현효솔위57.14%(20/35).VSAA、SAA화MAA간료효무명현차이.ATG불량반응관찰결과:①48.6%출현경도류과민반응;②혈청병발생솔42.9%,평균병정3.6 d;③9례(25.7%)외주혈소판(BPC)계수＜10×109/L;④8례(22.9%)재ATG치료후1개월내발생감염;⑤미발생ATG치료상관사망.Genzyme화Fresenius량충불동ATG제제,재료효화ATG상관불량반응발생솔방면차이균무통계학의의.결론 ATG치료인동SAA화MAA료효현저,단수적겁예방화공제ATG불량반응,피면치료상관사망.장기보조치료화제고수방질량,야시학보료효적중요배절.
Objective To evaluate the efficacy of antithymocyte globulin (ATG) based immunosupression therapy for childhood aplastic anemia, to reduce the adverse effects and to observe the long-term outcome. Method Thirty-five children with aplastic anemia (AA) were enrolled in this study.Six of the cases had very severe AA (VSAA), 11 had severe AA (SAA)-Ⅰ, 8 had SAA-Ⅱ and 10 had moderate AA (MAA). All these patients were treated with ATG plus Cyclosporin A (CSA). The following measures were taken during the ATG therapy: infection of the patients had been controlled before ATG treatment. Comprehensive anti-allergic measures were implemented. Close attention was paid to the hemorrhage related with platelet reduction caused by ATG and severe infection of the patients. Result Shortly after the ATG usage, all the patients had a significant decrease of absolute peripheral lymphoblast count by more than 60 percent. With a mean follow-up time of 28 months, the total effective rate was 77.14％ ( 27/35), significant response rate was 57.14％(20/35). There was no significant difference among VSAA, SAA and MAA groups in the response rate. Adverse reactions included the following:① 48.6％ (17/35) patients presented mild anaphylactoid reaction during the first day of ATG treatment; ②42.9％(15/35) cases presented serum sickness 5-11 days after the last dose of ATG with a mean duration of 3. 6 days, all the patients were cured effectively with methylprednisolone; ③25.7％ (9/35)patient's peripheral blood platelet count was reduced, might be caused by ATG, to below 10 × 109/L, but no patient had severe hemorrhagic complication after platelet transfusion was performed; ④22.9％ ( 8/35 ) of patients got infection within a month after ATG therapy, including 3 cases with clinical septicemia, all the 3 cases recovered after antibiotics treatment. There was no ATG treatment-related death in this series. Conclusion ATG is a very effective therapy for children with SAA and MAA. Comprehensive measures are needed to prevent and handle the side effects to avoid treatment-related death. Long-term supportive therapy and proper follow up contribute to the favourable outcomes of the patients.