Department of Pediatrics, School of Medicine, Nihon University, 30-1 Ohyaguchi-Kamicho, Itabashi-ku, 173-8610, Tokyo, Japan,
Ann Hematol. 2014 May;93(5):747-52. doi: 10.1007/s00277-013-1984-x. Epub 2013 Dec 14.
Patients with severe aplastic anemia (SAA) and an absolute neutrophil count (ANC) of 0 typically have fatal outcomes. We defined fulminant AA (FAA) as ANC = 0 for at least 2 weeks prior to and after immunosuppressive therapy (IST). We analyzed the outcomes of 35 children with FAA among 288 children who enrolled in a prospective study for AA (AA-97 study). AA was classified as FAA (n = 35), very SAA (vSAA; n = 129), or SAA (n = 124). All of the children received the IST with horse anti-thymocyte globulin (ATG) and cyclosporine (CsA). A significantly lower response rate at 6 months was seen in children with FAA when compared to those with vSAA or SAA (40.0, 63.6, and 63.7 %, respectively; p = 0.027). Of 20 nonresponder patients in the FAA group, 11 were rescued by alternative donor transplantation, and 5 patients showed a late response after 6 months. Consequently, no significant difference was noted in overall survival when comparing the FAA, vSAA, and SAA groups (88.5, 95.8, and 96.8 %). These findings indicate that IST with ATG and CsA is justified as a first-line treatment for children with FAA who lack a human leukocyte antigen-matched sibling donor.
患有严重再生障碍性贫血（SAA）且中性粒细胞绝对计数（ANC）为 0 的患者通常具有致命结局。我们将暴发性再生障碍性贫血（FAA）定义为免疫抑制治疗（IST）前后至少 2 周 ANC = 0。我们分析了 288 名接受 AA 前瞻性研究（AA-97 研究）的儿童中 35 名 FAA 患儿的结局。AA 分为 FAA（n = 35）、非常 SAA（vSAA；n = 129）或 SAA（n = 124）。所有儿童均接受马抗胸腺细胞球蛋白（ATG）和环孢素（CsA）的 IST。与 vSAA 或 SAA 患儿相比，FAA 患儿在 6 个月时的反应率显著较低（分别为 40.0%、63.6%和 63.7%；p = 0.027）。在 FAA 组的 20 名无反应患者中，11 名通过替代供体移植获救，5 名患者在 6 个月后出现迟发反应。因此，FAA、vSAA 和 SAA 组之间的总生存率无显著差异（分别为 88.5%、95.8%和 96.8%）。这些发现表明，对于缺乏人类白细胞抗原匹配同胞供体的 FAA 患儿，ATG 和 CsA 的 IST 可作为一线治疗方法。
