Scott Bart L, Storer Barry, Loken Michael R, Storb Rainer, Appelbaum Frederick R, Deeg H Joachim
Fred Hutchinson Cancer Research Center, Seattle, Washington 98109-1024, USA.
Biol Blood Marrow Transplant. 2005 Jan;11(1):65-73. doi: 10.1016/j.bbmt.2004.10.001.
Hematopoietic cell transplantation is the only curative therapy for patients with myelodysplastic syndrome (MDS). However, treatment-related toxicity and, in patients with advanced MDS (refractory anemia with excess blasts [RAEB]; RAEB in transformation [RAEB-T]) or transformation to acute myeloid leukemia with multilineage dysplasia (tAML), posttransplantation relapse continue to be prevalent. Induction chemotherapy (IC) has been used in an attempt to decrease the risk of posttransplantation relapse, but the benefit for posttransplantation long-term survival is uncertain. We reviewed results in 125 patients with advanced MDS and tAML who received transplants from HLA-identical related or unrelated donors after preparation with myeloablative conditioning regimens. Thirty-three patients (3 with RAEB, 6 with RAEB-T, and 24 with tAML) received IC before transplantation, and 92 patients (62 with RAEB, 22 with RAEB-T, and 8 with tAML) did not. Seventy-six patients were conditioned with oral busulfan 16 mg/kg, which was adjusted to achieve steady-state plasma concentrations of 800 to 900 ng/mL, plus cyclophosphamide 2 x 60 mg/kg, and 49 patients received busulfan 7 mg/kg (without dose adjustment) and total body irradiation 6 x 200 cGy given over 3 days. There was no evidence of a benefit in posttransplantation outcome associated with prior IC, either for patients with RAEB/RAEB-T or those with tAML, with either conditioning regimen. There was a correlation of the severity of pretransplantation flow cytometric aberrancies on marrow cells and posttransplantation relapse. Further studies that randomize patients to IC versus no IC need to appropriately address the possible beneficial effect of IC.
造血细胞移植是骨髓增生异常综合征（MDS）患者唯一的治愈性疗法。然而，治疗相关毒性以及在晚期MDS患者（伴有过多原始细胞的难治性贫血[RAEB]；转化中的RAEB[RAEB-T]）或转化为伴有多系发育异常的急性髓系白血病（tAML）患者中，移植后复发仍然很普遍。诱导化疗（IC）已被用于试图降低移植后复发的风险，但对移植后长期生存的益处尚不确定。我们回顾了125例晚期MDS和tAML患者的结果，这些患者在接受清髓性预处理方案后，从 HLA 相同的相关或无关供体接受了移植。33例患者（3例RAEB、6例RAEB-T和24例tAML）在移植前接受了IC，92例患者（62例RAEB、22例RAEB-T和8例tAML）未接受。76例患者接受口服白消安16mg/kg，并进行调整以达到稳态血浆浓度800至900ng/mL，加环磷酰胺2×60mg/kg，49例患者接受白消安7mg/kg（未调整剂量）和3天内给予的全身照射6×200cGy。无论是对于RAEB/RAEB-T患者还是tAML患者，无论采用哪种预处理方案，均没有证据表明移植前IC对移植后结局有好处。移植前骨髓细胞流式细胞术异常的严重程度与移植后复发存在相关性。将患者随机分为接受IC组与不接受IC组的进一步研究需要适当考虑IC可能的有益作用。
