Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemia
Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemia
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DOI：
10.1182/blood-2011-02-334953
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发表时间：
2011-08-04
期刊：
影响因子：
20.3
通讯作者：
Storb, Rainer F.
中科院分区：
文献类型：
--
作者：
Sanders, Jean E.;Woolfrey, Ann E.;Storb, Rainer F.
再生障碍性贫血（AA）是一种潜在的致命性疾病，可以通过骨髓移植治愈。儿科患者在移植后早期的生存率非常好，但有关晚期影响的数据有限。本研究评估了152例患者的晚期效应，随访时间为1-38年（中位数为21.8年）。移植准备方案主要是环磷酰胺与或不与抗胸腺细胞球蛋白。获得性AA患者的30年生存率为82%，范可尼贫血患者为58%（P = 0.01）。多变量分析表明慢性GVHD（P = .02）和范可尼贫血（P = .03）对生存率有负面影响。2例Fanconi患者和18例获得性AA患者发生恶性肿瘤，其中4例死亡。在接受全身照射的患者中，甲状腺功能检查异常的发生率增加。环磷酰胺受体表现出正常的生长，基本正常的发育，怀孕与大多数正常的后代。对该儿科移植队列的成年存活者进行的生活质量研究表明，除了健康和人寿保险方面的困难外，患者与对照组患者相当。这些数据表明，大多数长期存活移植后的AA在童年时期可以有一个正常的生产生活。（血。2011; 118（5）：1421-1428）
Aplastic anemia (AA), a potentially fatal disease, may be cured with marrow transplantation. Survival in pediatric patients has been excellent early after transplantation, but only limited data are available regarding late effects. This study evaluates late effects among 152 patients followed 1-38 years (median, 21.8 years). Transplantation-preparative regimes were mostly cyclophosphamide with or without antithymocyte globulin. Survival at 30 years for the acquired AA patients is 82%, and for the Fanconi anemia patients it is 58% (P = .01). Multivariate analysis demonstrated that chronic GVHD (P = .02) and Fanconi anemia (P = .03) negatively impacted survival. Two Fanconi patients and 18 acquired AA patients developed a malignancy that was fatal for 4. There was an increased incidence of thyroid function test abnormalities among those who received total body irradiation. Cyclophosphamide recipients demonstrated normal growth, basically normal development, and pregnancies with mostly normal offspring. Quality-of-life studies in adult survivors of this pediatric transplantation cohort indicated that patients were comparable with control patients except for difficulty with health and life insurance. These data indicate that the majority of long-term survivors after transplantation for AA during childhood can have a normal productive life. (Blood. 2011; 118(5): 1421-1428)