Leahey A M, Friedman D L, Bunin N J
Department of Pediatrics, The Children's Hospital of Philadelphia and the University of Pennsylvania, USA.
Bone Marrow Transplant. 1999 Jan;23(1):21-5. doi: 10.1038/sj.bmt.1701517.
Eleven children underwent BMT for therapy-related MDS or leukemia, four from HLA-identical siblings and seven from unrelated donors. Ten of the 11 were conditioned with busulfan and cyclophosphamide as the majority had received prior irradiation to the chest and/or abdomen. All patients engrafted. Regimen-related toxicity was more common when compared to historical controls. Eight patients developed acute GVHD and four of eight who survived 100 days post transplant developed extensive chronic GVHD. Non-relapse related mortality occurred in three patients. Five patients developed recurrent malignancy: one died from recurrence of osteosarcoma, three died of recurrent leukemia or MDS and another developed two subsequent malignancies (duodenal carcinoma and anaplastic astrocytoma). Three survive disease-free at 14+, 22+ and 43+ months for a 2 year actuarial cancer-free survival of 24% (95% confidence interval = 5-53%). Although allogeneic BMT can be curative, regimen-related toxicity is frequent and recurrent malignancy remains the major obstacle.
11名儿童因治疗相关的骨髓增生异常综合征或白血病接受了骨髓移植，其中4名来自人类白细胞抗原（HLA）匹配的同胞，7名来自无关供者。11名患者中有10名接受了白消安和环磷酰胺预处理，因为大多数患者之前接受过胸部和/或腹部放疗。所有患者均成功植入。与历史对照相比，方案相关毒性更为常见。8名患者发生了急性移植物抗宿主病（GVHD），移植后存活100天的8名患者中有4名发生了广泛的慢性GVHD。3名患者发生了非复发相关死亡。5名患者出现了复发恶性肿瘤：1名死于骨肉瘤复发，3名死于白血病或骨髓增生异常综合征复发，另1名随后发生了两种恶性肿瘤（十二指肠癌和间变性星形细胞瘤）。3名患者分别在14+、22+和43+个月时无病存活，2年无癌生存率为24%（95%置信区间=5-53%）。尽管异基因骨髓移植可能治愈疾病，但方案相关毒性很常见，复发恶性肿瘤仍然是主要障碍。
