Dini G, Boni L, Abla O, Uderzo C, Polchi P, Locatelli F, Di Bartolomeo P, Arcese W, Iori A P, Rossetti F
Department of Pediatric Hematology/Oncology-Bone Marrow Transplant Unit, Istituto Giannina Gaslini, Genova, Italy.
Bone Marrow Transplant. 1994 Jun;13(6):771-6.
Fifty-nine children, aged 1-15 years, with acute myelogenous leukemia (AML) received a bone marrow transplant (BMT) from an HLA-identical sibling (n = 57) or from an identical twin (n = 2), while in first remission (CR). These children represent, to the best of our knowledge, all children grafted in first CR in 11 Italian centers between 1980 and 1990. Patients were prepared with total body irradiation (TBI) plus cyclophosphamide (CY) (n = 50) or melphalan (n = 2) or with busulfan plus CY (n = 7). GVHD prophylaxis consisted of cyclosporin A (n = 48), methotrexate (n = 7) or cyclosporin and methotrexate (n = 2). Survivors have been followed for 21-137 months (median 59 months). Actuarial relapse-free survival was 58% at 66-137 months (95% confidence interval (CI) 44-72). Actuarial risk of relapse was 23% at 48 months (95% CI 10.9-34.8). Risk of non-relapse deaths was 33% in the period 1980-87 and 4% in the period 1988-90 (p = 0.02). In multivariate analysis patients with a blood cell count > 14 x 10(9)/l at diagnosis showed a lower relapse-free survival compared with patients with counts < 14 x 10(9)/l (p = 0.006). We could not detect an effect of FAB subtype, patient age, time to achieve remission or transplant-related variables, including year of BMT, on relapse-free survival. In conclusion, allogeneic marrow transplantation can achieve long-term relapse-free survival in over 50% of children with AML and should be considered as consolidation therapy if a matched sibling is available.(ABSTRACT TRUNCATED AT 250 WORDS)
59名年龄在1至15岁之间的急性髓系白血病(AML)患儿在首次缓解期(CR)接受了来自人类白细胞抗原(HLA)匹配同胞(n = 57)或同卵双胞胎(n = 2)的骨髓移植(BMT)。据我们所知,这些患儿代表了1980年至1990年间在11个意大利中心接受首次CR期移植的所有患儿。患者接受全身照射(TBI)加环磷酰胺(CY)(n = 50)或美法仑(n = 2)或白消安加CY(n = 7)进行预处理。移植物抗宿主病(GVHD)预防措施包括环孢素A(n = 48)、甲氨蝶呤(n = 7)或环孢素与甲氨蝶呤联合使用(n = 2)。对幸存者进行了21至137个月(中位时间59个月)的随访。66至137个月时的无复发生存率为58%(95%置信区间(CI)44 - 72)。48个月时的复发风险为23%(95% CI 10.9 - 34.8)。1980 - 1987年期间非复发死亡风险为33%,1988 - 1990年期间为4%(p = 0.02)。多因素分析显示,诊断时血细胞计数>14×10⁹/L的患者与计数<14×10⁹/L的患者相比,无复发生存率较低(p = 0.006)。我们未发现FAB亚型、患者年龄、达到缓解的时间或与移植相关的变量(包括BMT年份)对无复发生存率有影响。总之,异基因骨髓移植可使超过50%的AML患儿实现长期无复发生存,如果有匹配的同胞供者,应将其视为巩固治疗方法。(摘要截短至250字)