Rubie H, Attal M, Demur C, Brousset P, Duchayne E, Rigal-Huguet F, Dastugue N, Robert A
Bone Marrow Transplant Unit, CHU Purpan, Toulouse, France.
Bone Marrow Transplant. 1994 Jun;13(6):759-62.
Four consecutive children with myelodysplastic syndromes (MDS) underwent matched allogeneic bone marrow transplantation (BMT). Ages ranged from 3.2 to 6.3 years. Diagnosis was assessed according to FAB classification: refractory anemia-RA (n = 1), RA with excess of blasts (RAEB) (n = 1), and juvenile chronic myelogenous leukemia (JCML) (n = 2). Initial treatment included transfusions for all of them, splenectomy (n = 2) and chemotherapy (n = 1). Patients were all prepared with busulfan 21 mg/kg (480 mg/m2), cytosine arabinoside 24,000 mg/m2, melphalan 140 mg/m2. Graft-versus-host disease (GVHD) prophylaxis associated cyclosporine-methotrexate. Engraftment was prompt and complete in all children. Toxicity included severe mucositis (n = 3), moderate veno-occlusive disease (n = 2), acute GVHD (n = 3), chronic GVHD (n = 1). Sequelae have not yet been seen. All patients are alive and disease-free with a follow-up ranging from 7 to 35 months, with a Karnofsky score of 90-100%. Combined busulphan conditioning can offer an alternative to total body irradiation-based regimens in order to avoid late side-effects in children.
四名患有骨髓增生异常综合征（MDS）的儿童接受了匹配的异基因骨髓移植（BMT）。年龄范围为3.2至6.3岁。根据FAB分类进行诊断：难治性贫血-RA（n = 1）、伴有原始细胞增多的难治性贫血（RAEB）（n = 1）和青少年慢性粒细胞白血病（JCML）（n = 2）。初始治疗包括对所有患儿进行输血、脾切除术（n = 2）和化疗（n = 1）。患者均接受白消安21 mg/kg（480 mg/m²）、阿糖胞苷24,000 mg/m²、美法仑140 mg/m²预处理。移植物抗宿主病（GVHD）预防采用环孢素-甲氨蝶呤联合方案。所有患儿均迅速且完全植入。毒性反应包括严重黏膜炎（n = 3）、中度静脉闭塞性疾病（n = 2）、急性GVHD（n = 3）、慢性GVHD（n = 1）。尚未观察到后遗症。所有患者均存活且无疾病，随访时间为7至35个月，卡诺夫斯基评分90 - 100%。联合白消安预处理方案可为基于全身照射的方案提供替代选择，以避免儿童出现晚期副作用。
