Muñoz A, Bureo E, Ortega J J, Richard C, Olivé T, Maldonado M S, Madero L, Díaz M A
Department of Pediatrics, Ramón y Cajal Hospital, Madrid, Spain.
Haematologica. 1998 Nov;83(11):981-4.
To compare the estimated survival and disease-free survival between children with Ph1-positive chronic myeloid leukemia treated with allogeneic bone marrow transplantation or conventional chemotherapy.
In this retrospective study we compared the results obtained in a group of 14 children who received allogeneic bone marrow transplantation (BMT) between 1983 and 1993, and another group of 27 children treated with busulfan, hydroxyurea or alpha-interferon during the same time period. Patients were transplanted at a median of 7 months from diagnosis and all except one were in their first chronic phase. Conditioning consisted in total body irradiation and cyclophosphamide in 12 cases, and busulfan was added in two.
Of the 14 patients treated with BMT, two died of transplant-related complications and two relapsed 18 and 48 months after the BMT. Ten children remain alive and disease free at a median follow up of 60 months. The probability of DFS at 5 years is 70%. Of the 27 patients treated with chemotherapy, 22 have died at a median of 36 months from diagnosis. The probability of survival at 5 years is 5% versus 83% for the BMT group (p = 0.001).
Allogeneic BMT is a safe and very effective treatment for Ph-positive CML in children. Patients who have an HLA-identical sibling donor must receive a transplant as soon as possible after being diagnosed.
比较接受异基因骨髓移植或传统化疗的Ph1阳性慢性髓性白血病患儿的预计生存率和无病生存率。
在这项回顾性研究中，我们比较了1983年至1993年间接受异基因骨髓移植（BMT）的14名儿童和同期接受白消安、羟基脲或α干扰素治疗的27名儿童的结果。患者自诊断起中位7个月时接受移植，除1例患者外，其余均处于慢性期。预处理方案中，12例采用全身照射和环磷酰胺，2例加用白消安。
接受BMT治疗的14例患者中，2例死于移植相关并发症，2例分别于BMT后18个月和48个月复发。10名儿童在中位随访60个月时仍存活且无病。5年无病生存率为70%。接受化疗的27例患者中，22例自诊断起中位36个月时死亡。5年生存率为5%，而BMT组为83%（p = 0.001）。
异基因BMT是治疗儿童Ph阳性慢性髓性白血病的一种安全且非常有效的方法。有HLA匹配同胞供者的患者确诊后必须尽快接受移植。
