Yoshimi Ai, Kato Keisuke, Hosaka Sho, Suzuki Ryoko, Fukushima Hiroko, Nakao Tomohei, Kobayashi Chie, Fukushima Takashi, Koike Kazutoshi, Sumazaki Ryo, Tsuchida Masahiro
Division of Pediatric Hematology and Oncology, Ibaraki Children's Hospital, Mito, Japan.
Department of Pediatrics, Institute of Clinical Medicine, University of Tsukuba, Tsukuba, Japan.
Pediatr Transplant. 2017 Jun;21(4). doi: 10.1111/petr.12918. Epub 2017 Mar 22.
We present two infants with KMT2A(MLL)-gene-R-associated BCP-ALL, who received HLA haploidentical PBSCT after RIC. The patients developed ALL at age 6 months and 3 months, respectively. Case 1 underwent PBSCT at the second CR with detectable KMT2A-AFF1(MLL-AF4) fusion gene transcript at 11 months of age, and Case 2 at the first CR without KMT2A-MLLT1(MLL-ENL) fusion gene transcript at 8 months of age. Both patients received G-CSF-mobilized unmanipulated peripheral blood mononuclear cells from their HLA haploidentical mothers after administration of FLU, MEL, and ATG. Tacrolimus, methotrexate, and mPSL were administered as prophylaxis against GVHD. Engraftment was rapidly obtained with complete chimerism in both patients. Acute adverse events included acute GVHD in Case 1 and bacterial sepsis in Case 2. At last clinical check at age 5 years and 4 years, respectively, both patients were recurrence-free and attained normal growth and development. We conclude that PBSCT from an HLA haploidentical mother with non-TBI and non-BU regimen seems feasible and efficacious, offering favorable life quality for infants.
我们报告了两名患有KMT2A（MLL）基因相关BCP-ALL的婴儿，他们在减低预处理强度后接受了HLA单倍体相合外周血干细胞移植（PBSCT）。这两名患者分别在6个月和3个月大时患上ALL。病例1在11个月大的第二次完全缓解期接受PBSCT，当时可检测到KMT2A-AFF1（MLL-AF4）融合基因转录本；病例2在8个月大的第一次完全缓解期接受PBSCT，当时未检测到KMT2A-MLLT1（MLL-ENL）融合基因转录本。两名患者在接受氟达拉滨、美法仑和抗胸腺细胞球蛋白治疗后，均接受了来自其HLA单倍体相合母亲的粒细胞集落刺激因子动员的未处理外周血单个核细胞。给予他克莫司、甲氨蝶呤和甲泼尼龙预防移植物抗宿主病（GVHD）。两名患者均迅速实现植入，且完全嵌合。急性不良事件包括病例1发生急性GVHD，病例2发生细菌性败血症。在分别为5岁和4岁的最后一次临床检查时，两名患者均无复发，生长发育正常。我们得出结论，采用非全身照射（TBI）和非白消安方案，从HLA单倍体相合母亲进行PBSCT似乎可行且有效，可为婴儿提供良好的生活质量。
