Shaw P J, Bergin M E, Burgess M A, Dalla Pozza L, Kellie S J, Rowell G, Stevens M M, Webster B H, Bradstock K F
Department of Oncology, Royal Alexandra Hospital for Children, Camperdown, Sydney, Australia.
J Clin Oncol. 1994 Oct;12(10):2138-45. doi: 10.1200/JCO.1994.12.10.2138.
To report the impact of bone marrow transplantation (BMT) with busulfan/cyclophosphamide (BuCy) as end consolidation in a cohort of consecutively diagnosed children with acute myeloid leukemia (AML).
Between May 1987 and November 1992, 43 patients were diagnosed with AML. Tissue typing at diagnosis determined whether patients would proceed to autologous or allogeneic BMT as end consolidation after six cycles of chemotherapy. Conditioning for BMT was with BuCy, followed by allogeneic or unpurged autologous marrow infusion.
Of 37 patients who received chemotherapy, 35 achieved remission (95%) after one to six courses of treatment and 34 (92%) were transplanted. Five relapsed before BMT, four were subsequently transplanted in second complete remission (CR2) (n = 3) or untreated first relapse (n = 1), and one failed to respond to further therapy. All other patients proceeded to BMT in first complete remission (CR1). Eleven patients received allografts: one relapsed and one died of graft-versus-host disease (GvHD), for a leukemia-free survival rate of 90% at a median of 41 months after BMT (range, 3 to 60). For 23 autografts, there were two toxic deaths and eight relapses, with a leukemia-free survival rate of 61% at a median of 11 months after BMT (range, 0 to 66). The high relapse rate following autologous BMT led us to escalate the dose of Bu from 16 mg/kg to 600 mg/m2 using a single daily dose of Bu.
With modern supportive therapy, most newly diagnosed children with AML will enter remission and are eligible for intensification therapy. BuCy is well tolerated in children, which allowed us to escalate the dose of Bu in recent patients. Further follow-up is needed to determine whether this has an impact on the relapse rate following autologous BMT.
报告采用白消安/环磷酰胺(BuCy)进行骨髓移植(BMT)作为巩固治疗对一组连续诊断的急性髓系白血病(AML)患儿的影响。
1987年5月至1992年11月期间,43例患者被诊断为AML。诊断时进行组织配型,以确定患者在接受六个周期化疗后是否进行自体或异基因BMT作为巩固治疗。BMT的预处理方案为BuCy,随后输注异基因或未净化的自体骨髓。
37例接受化疗的患者中,35例(95%)在接受1至6个疗程的治疗后达到缓解,34例(92%)接受了移植。5例在BMT前复发,4例随后在第二次完全缓解(CR2)(n = 3)或未治疗的首次复发(n = 1)时接受移植,1例对进一步治疗无反应。所有其他患者在首次完全缓解(CR1)时进行BMT。11例患者接受了同种异体移植:1例复发,1例死于移植物抗宿主病(GvHD),BMT后中位41个月(范围3至60个月)的无白血病生存率为90%。对于23例自体移植,有2例因毒性死亡,8例复发,BMT后中位11个月(范围0至66个月)的无白血病生存率为61%。自体BMT后较高的复发率促使我们将Bu的剂量从16 mg/kg提高到600 mg/m²,采用每日单次剂量的Bu。
采用现代支持治疗,大多数新诊断的AML患儿将进入缓解期并适合强化治疗。儿童对BuCy耐受性良好,这使我们能够在近期患者中提高Bu的剂量。需要进一步随访以确定这是否会对自体BMT后的复发率产生影响。