Khouri I F, Kantarjian H M, Talpaz M, Giralt S, Rios M O, Hester J P, Champlin R E, Deisseroth A B
Department of Hematology, University of Texas MD Anderson Cancer Center, Houston 77030, USA.
Bone Marrow Transplant. 1996 May;17(5):775-9.
The purpose of this study was to evaluate the effectiveness of unpurged autologous stem cell transplantation (ASCT) for chronic myelogenous leukemia (CML) and its impact on the survival of patients in first and late chronic phase (CML-CP) including those resistant to or unable to tolerate interferon alfa (IFN-alpha) therapy. Between 1982 and 1993, 73 patients with CML who underwent ASCT were evaluated. Twenty-eight patients had signs of transformation, 20 were in second or subsequent CP, 22 had CML-CP and had shown resistance to or were unable to tolerate IFN-alpha therapy, and there had Philadelphia (Ph) chromosome-negative CML. Survival of patients in CML-CP who underwent ASCT was compared to controls who were in first CP receiving INF-a therapy. Patients and controls were matched for age, decade of therapy, response to IFN-alpha therapy (resistance vs toxicity) and the time to ASCT (study group) vs time to resistance (control group). Nine 12% patients failed to achieve hematologic recovery, and five (7%) had early death secondary to toxicity. Twenty-seven (58%) patients who received transplants in advanced-stage CML and 18 (82%) transplanted in CML-CP achieved complete hematologic remission (CHR). The incidence of complete cytogenetic response was 10 and 14%, respectively. The median survival of these two groups of patients was 5 and 34 months, respectively (P < 0.001). However, the survival of patients in CML-CP was not significantly different from controls (34 vs 49 months; P = 0.17). We conclude that unpurged ASCT does not prolong the survival of patients in CML-CP who are resistant to IFN-alpha therapy. Progress in autotransplantation in CML might require innovative approaches to eradicate the leukemic cells from the autologous stem cells prior to transplants.
本研究的目的是评估未净化的自体干细胞移植（ASCT）治疗慢性粒细胞白血病（CML）的有效性及其对处于慢性期早期和晚期（CML-CP）患者生存的影响，包括那些对干扰素α（IFN-α）治疗耐药或不耐受的患者。1982年至1993年期间，对73例行ASCT的CML患者进行了评估。28例患者有疾病转化迹象，20例处于第二次或后续慢性期，22例为CML-CP且对IFN-α治疗耐药或不耐受，还有费城（Ph）染色体阴性的CML患者。将接受ASCT的CML-CP患者的生存率与接受INF-α治疗处于慢性期早期的对照组进行比较。患者和对照组在年龄、治疗年代、对IFN-α治疗的反应（耐药与毒性）以及ASCT时间（研究组）与出现耐药时间（对照组）方面进行了匹配。9例（12%）患者未实现血液学恢复，5例（7%）因毒性过早死亡。27例（58%）晚期CML患者接受移植，18例（82%）CML-CP患者接受移植后实现了完全血液学缓解（CHR）。完全细胞遗传学反应的发生率分别为10%和14%。这两组患者的中位生存期分别为5个月和34个月（P<0.001）。然而，CML-CP患者的生存率与对照组无显著差异（34个月对49个月；P=0.17）。我们得出结论，未净化的ASCT不能延长对IFN-α治疗耐药的CML-CP患者的生存期。CML自体移植的进展可能需要创新方法，以便在移植前从自体干细胞中清除白血病细胞。
