Kennedy-Nasser Alana A, Bollard Catherine M, Myers G Doug, Leung Kathryn S, Gottschalk Stephen, Zhang Yiqun, Liu Hao, Heslop Helen E, Brenner Malcolm K, Krance Robert A
Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas 77030, USA.
Biol Blood Marrow Transplant. 2008 Nov;14(11):1245-52. doi: 10.1016/j.bbmt.2008.08.010.
HLA-matched sibling donor (MSD) stem cell transplantation can cure>60% of pediatric patients with acute lymphoblastic leukemia (ALL), but <30% of patients will have a sibling donor. Alternative donor (AD) transplantation can be curative but has a higher risk of graft-versus-host disease (GVHD). The addition of alemtuzumab (Campath 1-H) to AD transplants produces in vivo T cell depletion, which may reduce the risk for GVHD. We now report the outcome for 83 children with ALL (41 MSD, 42 AD) undergoing stem cell transplantation in first or second complete remission. All patients received myeloablative conditioning, including cyclophosphamide, cytarabine arabinoside, and total-body irradiation, with alemtuzumab administered to AD recipients. GVHD prophylaxis consisted of a calcineurin inhibitor with either short-course methotrexate or prednisone. Disease-free survival (DFS) for MSD recipients was 72.3% (95% confidence interval [CI], 55.4%-83.6%) versus 62.4% (95% CI, 45.2%-75.4%) for AD recipients. The 100-day mortality was 7.1% in the AD group and 2.4% in the MSD group. Relapse rates were identical (24%). Treatment-related mortality, principally viral infection, explained the difference in survival. For children undergoing stem cell transplantation (SCT) from alternative donors, alemtuzumab with a myeloablative conditioning regimen resulted in DFS comparable to MSD.
人类白细胞抗原（HLA）匹配的同胞供者（MSD）干细胞移植可治愈60%以上的儿童急性淋巴细胞白血病（ALL）患者，但只有不到30%的患者能找到同胞供者。替代供者（AD）移植也可治愈疾病，但移植物抗宿主病（GVHD）风险更高。在AD移植中加入阿仑单抗（Campath 1-H）可在体内使T细胞耗竭，这可能降低GVHD风险。我们现在报告83例处于首次或第二次完全缓解期接受干细胞移植的ALL患儿（41例MSD，42例AD）的治疗结果。所有患者均接受了清髓性预处理，包括环磷酰胺、阿糖胞苷和全身照射，AD受者还接受了阿仑单抗治疗。GVHD预防采用钙调神经磷酸酶抑制剂联合短疗程甲氨蝶呤或泼尼松。MSD受者的无病生存率（DFS）为72.3%（95%置信区间[CI]，55.4%-83.6%）；AD受者为62.4%（95%CI，45.2%-75.4%）。AD组100天死亡率为7.1%，MSD组为2.4%。复发率相同（24%）。与治疗相关的死亡率，主要是病毒感染，解释了生存率的差异。对于接受替代供者干细胞移植（SCT）的儿童，阿仑单抗联合清髓性预处理方案的DFS与MSD相当。
