Jacobsohn David A, Hewlett Brad, Morgan Elaine, Tse William, Duerst Reggie E, Kletzel Morris
Northwestern University, The Feinberg School of Medicine, Children's Memorial Hospital, Chicago, Illinois 60614, USA.
Biol Blood Marrow Transplant. 2005 Dec;11(12):999-1005. doi: 10.1016/j.bbmt.2005.08.031.
Infants with acute lymphoblastic leukemia (ALL) have a poor prognosis when treated with standard chemotherapy. A subset of these infants, particularly those with mixed-lineage leukemia (MLL) rearrangements, has a high likelihood of relapse. Hematopoietic stem cell transplantation (HSCT) performed early in first remission may improve outcome. We present the results of 16 patients with infant ALL who were treated with HSCT in first remission. Six patients were < or =6 months of age at diagnosis, 11 had an initial white blood cell count of >50000/microL, and all patients with determinable cytogenetics had a high-risk karyotype [t(4:11) abnormality or other MLL rearrangement]. All patients received 150 cGy of total body irradiation for 8 doses (1200 cGy). Fifteen of 16 patients received etoposide at 1000 mg/m(2) as a continuous infusion over 24 hours and cyclophosphamide at 60 mg/kg/d for 3 days. Eight patients received HSCT from an HLA-identical sibling, and 8, from unrelated cord blood. Twelve (75%) patients remain long-term survivors (median follow-up, 4.7 years). Two patients, 1 of whom had minimal residual disease at HSCT, died after relapse following HSCT. Two patients died of transplant-related causes. The HSCT was well tolerated; 15 patients achieved neutrophil engraftment at a median of 16 days. Acute and chronic graft-versus-host disease were minimal in these patients. These results support the use of HSCT in the treatment of infant ALL, especially when used as consolidation in first remission. The risk of relapse seems to be decreased with this approach. Further work is being performed to determine the long-term effects from this therapy.
急性淋巴细胞白血病（ALL）婴儿接受标准化疗时预后较差。这些婴儿中的一部分，尤其是那些有混合谱系白血病（MLL）重排的婴儿，复发可能性很高。首次缓解期早期进行造血干细胞移植（HSCT）可能改善预后。我们报告了16例首次缓解期接受HSCT治疗的婴儿ALL患者的结果。6例患者诊断时年龄≤6个月，11例初始白细胞计数>50000/μL，所有可确定细胞遗传学的患者均有高危核型[t(4;11)异常或其他MLL重排]。所有患者接受8次全身照射，总剂量150 cGy（1200 cGy）。16例患者中的15例接受依托泊苷1000 mg/m²持续24小时静脉输注，环磷酰胺60 mg/kg/d共3天。8例患者接受来自HLA相同同胞的HSCT，8例接受无关脐血HSCT。12例（75%）患者为长期存活者（中位随访4.7年）。2例患者，其中1例HSCT时存在微小残留病，HSCT后复发死亡。2例患者死于移植相关原因。HSCT耐受性良好；15例患者中性粒细胞中位16天植入。这些患者急慢性移植物抗宿主病均很轻微。这些结果支持HSCT用于婴儿ALL的治疗，尤其是作为首次缓解期的巩固治疗。采用这种方法似乎可降低复发风险。正在进一步开展工作以确定该治疗的长期影响。
