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首页> 外文期刊>Haematologica >Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
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Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

机译：小儿再生障碍性贫血的免疫抑制治疗：一项北美小儿再生障碍性贫血联盟的研究

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Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and outcomes for 314 children treated from 2002 to 2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin (hATG) plus cyclosporine (CyA) with a median 61 months follow up. Following hATG/CyA, 71.2% (95%CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response achieved in pediatric patients was high, with 59.8% (95%CI: 53.7,65.8) complete response and 68.2% (95%CI: 62.2,73.8) achieving at least a very good partial response with a platelet count ≥50×109L. At five years post-hATG/CyA, overall survival was 93% (95%CI: 89,96), but event-free survival without subsequent treatment was only 64% (95%CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis after a median 25.2 months (range: 4.3-71 months) post treatment. Myelodysplastic syndrome or leukemia developed in 6 of 314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95%CI: 0.08,0.47; P=0.0003). This study highlights the need for improved therapies to achieve sustained high-quality remission for children with severe aplastic anemia.
机译：小儿严重再生障碍性贫血对免疫抑制治疗的反应质量和长期预后仍不完全明确。用于小儿患者复发或难治的严重再生障碍性贫血的治疗的当代证据也很有限。回顾性分析了北美小儿再生障碍性贫血联合会的25个机构对2002年至2014年用免疫抑制疗法治疗获得性严重再生障碍性贫血的314例儿童的临床特征和结局。大多数受试者（n = 264）接受了马抗胸腺细胞球蛋白（hATG）加环孢素（CyA）的治疗，中位随访时间为61个月。在hATG / CyA之后，有71.2％（95％CI：65.3,76.6）达到了客观反应。与成人研究相比，小儿患者的反应质量很高，完全缓解的比例为59.8％（95％CI：53.7,65.8），至少达到很好的局部反应的比例为68.2％（95％CI：62.2,73.8）血小板计数≥50×109L时有反应。在hATG / CyA后五年，总体生存率为93％（95％CI：89,96），而没有随后治疗的无事件生存率仅为64％（95％CI：57,69），而没有平台期。在171位可评估患者中，有12位（占7％）在诊断后平均中位25.2个月（范围：4.3-71个月）后获得了克隆异常。 314人中有6人发展为骨髓增生异常综合症或白血病（1.9％）。对于复发/难治性疾病，在多因素分析中，与第二次免疫抑制治疗相比，造血干细胞移植治疗具有更好的无事件生存率（HR = 0.19，95％CI：0.08,0.47; P = 0.0003）。这项研究强调，需要针对严重再生障碍性贫血的儿童改善治疗方法，以实现持续的高质量缓解。

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