Worth L, Tran H, Petropoulos D, Culbert S, Mullen C, Roberts W, Przepiorka D, Chan K
Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Bone Marrow Transplant. 1999 Nov;24(9):947-52. doi: 10.1038/sj.bmt.1702016.
Seventeen children with advanced myeloid malignancies (induction failure, relapse, myelodysplasia, secondary AML, or CR >1) received thiotepa 750 mg/m2 i.v., busulfan 12 mg/kg or 640 mg/m2 p.o., and cyclophosphamide 120 mg/kg i.v. as a preparative regimen for allogeneic or autologous hematopoietic stem cell (HSC) transplantation. Of the 15 allogeneic transplants, eight were from matched siblings, one was from a mismatched sibling, and six were from unrelated donors. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine or tacrolimus and methotrexate. Regimen-related toxicity was common but tolerable, affecting mainly the skin and gastrointestinal tract. Three patients died early and were not evaluable for engraftment; engraftment occurred in the remaining patients. Nine patients with active disease at the time of transplant were evaluable for response; all achieved remission. With a median follow-up of 40 months (range, 10-71 months), nine patients are alive and disease-free. The 3-year actuarial event-free survival was 51% (95% confidence interval (CI) 27-76%). Seven patients died of transplant-related complications: infection (n = 4), chronic GVHD (n = 1), veno-occlusive disease, VOD, (n= 1) and pulmonary alveolar hemorrhage (n = 1). Only one patient had leukemia relapse and died. We conclude that the use of high-dose thiotepa, busulfan and cyclophosphamide is an effective conditioning regimen for childhood myeloid malignancies and may be tested in patients with less advanced disease (eg CR1).
17名患有晚期髓系恶性肿瘤（诱导失败、复发、骨髓增生异常综合征、继发性急性髓系白血病或完全缓解>1年）的儿童接受了硫替派750mg/m²静脉注射、白消安12mg/kg或640mg/m²口服以及环磷酰胺120mg/kg静脉注射，作为异基因或自体造血干细胞（HSC）移植的预处理方案。在15例异基因移植中，8例来自匹配的同胞，1例来自不匹配的同胞，6例来自无关供者。移植物抗宿主病（GVHD）预防包括环孢素或他克莫司以及甲氨蝶呤。方案相关毒性常见但可耐受，主要影响皮肤和胃肠道。3例患者早期死亡，无法评估植入情况；其余患者实现植入。9例移植时患有活动性疾病的患者可评估反应；所有患者均实现缓解。中位随访40个月（范围10 - 71个月），9例患者存活且无疾病。3年无事件生存率为51%（95%置信区间（CI）27 - 76%）。7例患者死于移植相关并发症：感染（n = 4）、慢性GVHD（n = 1）、静脉闭塞性疾病（VOD，n = 1）和肺泡出血（n = 1）。只有1例患者白血病复发死亡。我们得出结论，使用高剂量硫替派、白消安和环磷酰胺是儿童髓系恶性肿瘤的一种有效预处理方案，可在疾病程度较轻（如完全缓解1期）的患者中进行测试。
