Fouladi M, Herman R, Rolland-Grinton M, Jones-Wallace D, Blanchette V, Calderwood S, Doyle J, Halperin D, Leaker M, Saunders E F, Zipursky A, Freedman M H
The Hospital for Sick Children, and Department of Pediatrics, University of Toronto Faculty of Medicine, Ontario, Canada.
Bone Marrow Transplant. 2000 Dec;26(11):1149-56. doi: 10.1038/sj.bmt.1702699.
Multi-agent immunosuppressive therapy has produced improved survival for severe acquired aplastic anemia in children. Recently, some investigators have suggested that immunosuppressive therapy may replace bone marrow transplantation as first-line therapy for this disorder. To assess its validity, we compared the outcomes of bone marrow transplantation vs immunosuppressive therapy in one institution from 1987 to 1997. We studied 46 consecutive patients less than 18 years of age who presented between January 1987 and April 1997. Inherited marrow failure syndromes and myelodysplastic syndromes were excluded. Patients received immunosuppressive therapy vs bone marrow transplantation based on availability of HLA-matched donors. The main outcome measures were survival, complete marrow and hematological remission, or partial remission but achieving independence from transfusional support. Twenty patients received multi-agent immunosuppressive therapy (cyclosporine, antithymocyte globulin and methylprednisolone); 11 attained complete remission and three partial remission for a transfusion-independent survival of 70%. Six patients died of infectious and hemorrhagic complications. Twenty-six patients were transplanted and 24 (93%) achieved complete remission; one achieved a PR, 25 remain transfusion independent with a median follow-up of 5.9 years or 70 months. One patient developed AML 34 months after successful transplant and one patient died due to graft failure and complications of transplant. There has been a striking improvement in survival for pediatric patients treated with multi-agent immunosuppression in the last decade. However, transplantation results have also improved and this remains the definitive first-line therapy for severe acquired aplastic anemia in this age group.
多药联合免疫抑制疗法已提高了儿童重型获得性再生障碍性贫血的生存率。最近，一些研究人员提出免疫抑制疗法可能会取代骨髓移植成为这种疾病的一线治疗方法。为评估其有效性，我们比较了1987年至1997年在一家机构中骨髓移植与免疫抑制疗法的治疗结果。我们研究了1987年1月至1997年4月间连续收治的46例年龄小于18岁的患者。排除遗传性骨髓衰竭综合征和骨髓增生异常综合征。根据是否有HLA匹配的供体，患者接受免疫抑制疗法或骨髓移植。主要观察指标为生存率、骨髓完全缓解和血液学缓解，或部分缓解但不再依赖输血支持。20例患者接受了多药联合免疫抑制疗法（环孢素、抗胸腺细胞球蛋白和甲泼尼龙）；11例达到完全缓解，3例部分缓解，不依赖输血生存的比例为70%。6例患者死于感染和出血并发症。26例患者接受了移植，24例（93%）达到完全缓解；1例达到部分缓解，25例不再依赖输血，中位随访时间为5.9年或70个月。1例患者在成功移植34个月后发生急性髓系白血病，1例患者因移植失败和移植并发症死亡。在过去十年中，接受多药联合免疫抑制治疗的儿科患者的生存率有了显著提高。然而，移植结果也有所改善，对于这个年龄组的重型获得性再生障碍性贫血，移植仍然是明确的一线治疗方法。
