Pongtanakul Bunchoo, Das Prabodh Kumar, Charpentier Karen, Dror Yigal
Cell Biology Program, Research Institute, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children, The University of Toronto, Toronto, Ontario, Canada.
Pediatr Blood Cancer. 2008 Jan;50(1):52-7. doi: 10.1002/pbc.21377.
Immunosuppressive therapy (IST) is the alternative treatment in children with aplastic anemia (AA) who do not have an HLA-matched sibling. The aim of this study is to evaluate the outcome of children with AA treated with IST.
We retrospectively reviewed the hospital records of children with AA from 1984 to 2004, treated at our institution with antithymocyte globulin (ATG), cyclosporine (CS), and short course of prednisone.
Forty-two patients were treated with IST (24 boys, 18 girls); of whom 26% received G-CSF. The median age at diagnosis was 8.5 years. Sixty-nine, 19, and 12% were diagnosed with severe, very severe, and moderate AA, respectively. Twenty-one percent had hepatitis-associated AA. Median follow-up time was 53.3 months. Sixty-two percent had complete response; 19% had partial response. Two patients relapsed and received a second course of ATG; both had a partial response. The actuarial 5 years survival rate was 67.5%. Two patients developed myelodysplastic syndrome (MDS); both received long-term G-CSF and had partial response after two courses of IST. Fifteen percent of survivors had significant hypertension which persisted after CS was discontinued.
This study shows promising response in children with AA treated with IST; however, the outcome was inferior to our institutional results with hematopoietic stem cell transplantation from a sibling donor. Hypertension and MDS are late complications. Longer follow-up, larger cohorts, and prospective studies are warranted to evaluate late complications and risk factors.
免疫抑制治疗（IST）是无人类白细胞抗原（HLA）相匹配同胞供者的再生障碍性贫血（AA）患儿的替代治疗方法。本研究旨在评估接受IST治疗的AA患儿的治疗结果。
我们回顾性分析了1984年至2004年在我院接受抗胸腺细胞球蛋白（ATG）、环孢素（CS）和短疗程泼尼松治疗的AA患儿的住院病历。
42例患儿接受了IST治疗（24例男孩，18例女孩）；其中26%接受了粒细胞集落刺激因子（G-CSF）治疗。诊断时的中位年龄为8.5岁。分别有69%、19%和12%的患儿被诊断为重度、极重度和中度AA。21%的患儿患有肝炎相关性AA。中位随访时间为53.3个月。62%的患儿获得完全缓解；19%的患儿获得部分缓解。2例患儿复发并接受了第二疗程的ATG治疗；二者均获得部分缓解。5年实际生存率为67.5%。2例患儿发生骨髓增生异常综合征（MDS）；二者均接受了长期G-CSF治疗，并在两疗程IST治疗后获得部分缓解。15%的存活患儿患有严重高血压，在停用CS后仍持续存在。
本研究显示IST治疗的AA患儿有良好的反应；然而，其治疗结果不如我院同胞供者造血干细胞移植的结果。高血压和MDS是晚期并发症。需要更长时间的随访、更大的队列研究和前瞻性研究来评估晚期并发症和危险因素。
