与化疗相比，对处于第二次缓解期的急性淋巴细胞白血病患儿进行来自 HLA 相同同胞的骨髓移植。

Bone marrow transplants from HLA-identical siblings as compared with chemotherapy for children with acute lymphoblastic leukemia in a second remission.

作者信息

Barrett A J, Horowitz M M, Pollock B H, Zhang M J, Bortin M M, Buchanan G R, Camitta B M, Ochs J, Graham-Pole J, Rowlings P A

机构信息

National Heart, Lung, and Blood Institute, Bethesda, Md.

出版信息

N Engl J Med. 1994 Nov 10;331(19):1253-8. doi: 10.1056/NEJM199411103311902.

DOI:10.1056/NEJM199411103311902

PMID:7935682

Abstract

BACKGROUND

It is unclear how best to treat children with acute lymphoblastic leukemia who are in a second remission after a bone marrow relapse. For those with HLA-identical siblings, the question of whether to perform a bone marrow transplantation or to continue chemotherapy has not been answered.

METHODS

We compared the results of treatment with marrow transplants from HLA-identical siblings in 376 children, as reported to the International Bone Marrow Transplant Registry, with the results of chemotherapy in 540 children treated by the Pediatric Oncology Group. A preliminary analysis identified variables associated with treatment failure in both groups. We selected cohorts by matching these variables. A possible bias associated with differences in the interval between remission and treatment was controlled for by choosing matched pairs in which the duration of the second remission in the chemotherapy recipient was at least as long as the time between the second remission and transplantation in the transplant recipient. A total of 255 matched pairs were studied.

RESULTS

The mean (+/- SE) probability of a relapse at five years was significantly lower among the transplant recipients than among the chemotherapy recipients (45 +/- 4 percent vs. 80 +/- 3 percent, P < 0.001). At five years the probability of leukemia-free survival was higher after transplantation than after chemotherapy (40 +/- 3 percent vs. 17 +/- 3 percent, P < 0.001). The relative benefit of transplantation as compared with chemotherapy was similar in children with prognostic factors indicating a high or low risk of relapse (the duration of the first remission, age, leukocyte count at the time of the diagnosis, and phenotype of the leukemic cells).

CONCLUSIONS

For children with acute lymphoblastic leukemia in a second remission, bone marrow transplants from HLA-identical siblings result in fewer relapses and longer leukemia-free survival than does chemotherapy.

摘要

背景

对于急性淋巴细胞白血病患儿在骨髓复发后处于第二次缓解期时，如何进行最佳治疗尚不清楚。对于那些有 HLA 匹配同胞的患儿，是进行骨髓移植还是继续化疗的问题仍未得到解答。

方法

我们将国际骨髓移植登记处报告的 376 名接受 HLA 匹配同胞骨髓移植治疗的患儿结果，与儿科肿瘤学组治疗的 540 名接受化疗的患儿结果进行了比较。初步分析确定了两组中与治疗失败相关的变量。我们通过匹配这些变量来选择队列。通过选择化疗接受者第二次缓解期的持续时间至少与移植接受者第二次缓解期和移植之间的时间一样长的匹配对，控制了与缓解期和治疗间隔差异相关的可能偏差。共研究了 255 对匹配对。

结果

移植接受者五年时复发的平均（±标准误）概率显著低于化疗接受者（45±4%对 80±3%，P<0.001）。五年时移植后无白血病生存的概率高于化疗后（40±3%对 17±3%，P<0.001）。在有预后因素表明复发风险高或低的患儿中（首次缓解期的持续时间、年龄、诊断时的白细胞计数以及白血病细胞的表型），移植与化疗相比的相对益处相似。