Luo C J, Chen J, Wang J M, Qin X, Zhang B H, Zhu H, Wang X N, Cai J Y, Luo C Y
Shanghai Jiao Tong University, School of Medicine, Shanghai Children's Medical Center, Shanghai 200217, China.
Zhonghua Xue Ye Xue Za Zhi. 2020 Feb 14;41(2):128-131. doi: 10.3760/cma.j.issn.0253-2727.2020.02.008.
To investigate the efficacy of alternative donor (AD) in the treatment of aplastic anemia (AA) in children. The clinical data of AA children who received AD HSCT in our center from Apr. 2010 to Dec. 2016 were retrospectively analyzed. The overall survival (OS) rate, implant success rate, incidence of acute and chronic graft-versus-host disease (GVHD) were statistically analyzed. A total of 109 children with acquired AA, including 64 severe AA (SAA) , 32 very severe AA (VSAA) and 13 transfusion dependent non-severe AA (NSAA) , were recruited in this retrospective AD HSCT study, the median age was 6 (0.8-18) years old. Of them, 44 patients with 10/10 matched unrelated donor (MUD) , 44 patients with mismatched unrelated donor (MMUD) and 21 patients with mismatched related donor (MMRD) . All patients did not receive ATG before HSCT and the active infection was excluded. Except 3 patients suffered from a second graft failure (2 of them rescued by second HSCT) , 106/109 (97.2%) were engrafted with neutrophil and platelet recovery occurring at a median of 13 days (range, 9-19) and 16 days (range, 10-81) post-transplant. Until day 100 post transplantation, the incidence was 74.3% (81/109) for acute GVHD (aGVHD) and 39.4% (43/109) for grade Ⅱ-Ⅳ aGVHD, 30.7% (31/101) and 9.9% (10/101) for overall chronic GVHD (cGVHD) and moderate cGVHD, respectively, and nobody developed an extend cGVHD. After median follow up of 39 (0.7-103) months for all patients, 13 of 109 patients died. The estimated 5-year overall survival (OS) of the entire cohort was 88.1% (95% 81.1%-91.4%) with no difference among the MUD, MMUD and MMRD cohort (93.2%, 84.1% and 85.7%, respectively, =0.361) . These excellent outcomes suggest that unmanipulated AD PBSC is a good HSCT source for children with SAA. It's reasonable to consider AD HSCT as first line therapy for SAA children without matched sibling donor. Better strategies are required to prevent GVHD.
探讨替代供者（AD）治疗儿童再生障碍性贫血（AA）的疗效。回顾性分析2010年4月至2016年12月在本中心接受AD造血干细胞移植（HSCT）的AA患儿的临床资料。对总生存率（OS）、植入成功率、急性和慢性移植物抗宿主病（GVHD）的发生率进行统计学分析。本项回顾性AD HSCT研究共纳入109例获得性AA患儿，其中64例重型AA（SAA），32例极重型AA（VSAA），13例输血依赖型非重型AA（NSAA），中位年龄为6（0.8 - 18）岁。其中，44例为10/10匹配无关供者（MUD），44例为不匹配无关供者（MMUD），21例为不匹配相关供者（MMRD）。所有患者在HSCT前均未接受抗胸腺细胞球蛋白（ATG）治疗且排除活动性感染。除3例发生二次移植失败（其中2例通过二次HSCT挽救）外，106/109（97.2%）例患者中性粒细胞植入，血小板恢复中位时间为移植后13天（范围9 - 19天）和16天（范围10 - 81天）。至移植后100天，急性GVHD（aGVHD）发生率为74.3%（81/109），Ⅱ - Ⅳ级aGVHD发生率为39.4%（43/109），慢性GVHD（cGVHD）总发生率和中度cGVHD发生率分别为30.7%（31/101）和9.9%（10/101），无患者发生广泛性cGVHD。所有患者中位随访39（0.7 - 103）个月后，109例患者中有13例死亡。整个队列的估计5年总生存率为88.1%（95% 81.1% - 91.4%），MUD、MMUD和MMRD队列之间无差异（分别为93.2%、84.1%和85.7%，P = 0.361）。这些优异的结果表明，未处理的AD外周血干细胞是SAA患儿良好的HSCT来源。对于没有匹配同胞供者的SAA患儿，将AD HSCT作为一线治疗是合理的。需要更好的策略来预防GVHD。
