Barriga C Francisco, Wietstruck P Angélica, Becker K Anna, Zúñiga C Pamela, Besa De C Pelayo, Alvarez Z Manuel, Ocqueteau T Mauricio, Bertín C Pablo
Departamento de Pediatría, Pontificia Universidad Católica de Chile, Santiago, Chile.
Rev Med Chil. 2007 Nov;135(11):1421-8. Epub 2008 Jan 16.
Severe acquired aplastic anemia (SAA) is an uncommon disease of childhood. Patients with SAA receive supportive care with transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be allogenic stem cell transplantation (SCT) from a matched sibling or immunosuppressive therapy (IT).
To report the experience in the management of SAA.
Twenty five children with acquired SAA were treated from July 1992 to September 2005. Patients with full matched sibling donors received allogenic SCT after conditioning with a cyclophosphamide containing regimen. The other patients received immune suppression with cyclosporine plus methylprednisolone (n= 18) plus ATG (n=17). All received supportive care until recovery of hematopoietic function. Those who had severe opportunistic infections at diagnosis or did not respond to two cycles of ATG were evaluated for unrelated donor SCT.
Seven patients received sibling donor SCT and 18 IT, which was repeated in six. Three patients received mismatched related (1) or unrelated (2) SCT. Nineteen patients survived with a median follow up time of 4 years, 14 with full hematologic recovery. Six patients died: four due to infections after IT or SCT, one due to intracranial hemorrhage and one with secondary myelodysplasia 12 years after IT.
Most children with SAA can be treated successfully with sibling donor SCT or IT. Patients without a histocompatible sibling who fail to respond to IS have a worse prognosis.
严重获得性再生障碍性贫血（SAA）是一种儿童期罕见疾病。SAA患者接受输血支持治疗以及及时治疗机会性感染，同时接受特定治疗，可能是来自匹配同胞的异基因干细胞移植（SCT）或免疫抑制治疗（IT）。
报告SAA的治疗经验。
1992年7月至2005年9月期间，对25例获得性SAA患儿进行了治疗。有完全匹配同胞供者的患者在接受含环磷酰胺方案预处理后接受异基因SCT。其他患者接受环孢素加甲泼尼龙（n = 18）加抗胸腺细胞球蛋白（ATG，n = 17）的免疫抑制治疗。所有患者均接受支持治疗直至造血功能恢复。对诊断时有严重机会性感染或对两个周期ATG无反应的患者评估无关供者SCT。
7例患者接受了同胞供者SCT，18例接受了IT治疗，其中6例进行了重复治疗。3例患者接受了不匹配的相关（1例）或无关（2例）SCT。19例患者存活，中位随访时间为4年，14例获得完全血液学缓解。6例患者死亡：4例死于IT或SCT后的感染，1例死于颅内出血，1例在IT治疗12年后发生继发性骨髓增生异常综合征。
大多数SAA患儿可通过同胞供者SCT或IT成功治疗。没有组织相容性同胞且对免疫抑制治疗无反应的患者预后较差。
