Klingebiel T, Creutzig U, Dopfer R, Ehninger G, Schmidt H, Ritter J, Niethammer D
Children's Hospital, Department of Pediatric Hematology and Oncology, Tübingen, FRG.
Bone Marrow Transplant. 1990 May;5(5):317-20.
From 1983 to 1988 14 patients under 16 years of age with adult type chronic myelogenous leukemia (CML) in chronic or blastic phase were treated by allogeneic bone marrow transplantation (BMT) in our center. These comprise 54% of all patients under 16 years of age grafted for this disease in FRG. These BMT patients were compared with 24 similar patients treated conventionally with busulfan and/or hydroxyurea in various centers. The probability of an event-free survival 5 1/2 years after BMT was 0.61 (SD 0.16); the estimated probability of survival for 3-8 years after diagnosis in the group treated by GMT was 0.78 (SD 0.14) vs 0.55 (SD 0.12) for the non-BMT group. The difference is not significant. In the BMT group only two patients died of transplant-related complications. The non-BMT patient with the longest survival period died recently 10 years after diagnosis. For children with adult type CML, BMT is a safe and effective treatment, and should be recommended if there is an HLA compatible sibling donor or even a fully compatible unrelated donor. However, for a more conclusive comparison between bone marrow transplantation and conventional treatment a longer observation period and larger patient numbers are necessary.
1983年至1988年，我们中心对14例16岁以下处于慢性期或急变期的成人型慢性粒细胞白血病（CML）患者进行了异基因骨髓移植（BMT）治疗。这些患者占联邦德国所有16岁以下因该病接受移植患者的54%。将这些接受BMT的患者与其他各中心24例接受白消安和/或羟基脲常规治疗的类似患者进行比较。BMT后5年半无事件生存的概率为0.61（标准差0.16）；在接受常规治疗的组中，诊断后3至8年的估计生存概率为0.78（标准差0.14），而非BMT组为0.55（标准差0.12）。差异不显著。在BMT组中，仅2例患者死于移植相关并发症。非BMT组中生存时间最长的患者在诊断后10年最近死亡。对于成人型CML儿童患者，BMT是一种安全有效的治疗方法，若有HLA相合的同胞供者甚至完全相合的无关供者，应予以推荐。然而，为了更确切地比较骨髓移植和传统治疗，需要更长的观察期和更多的患者数量。
