Andolina M, Maximova N, Rabusin M, Vujic D, Bunjevacki G, Vidali C, Beorchia A
Transplant Department, IRCCS Burlo Garofolo, 34100 Trieste, Italy.
Haematologica. 2000 Nov;85(11 Suppl):37-40.
From 1986 to June 2000, sixty children suffering from acute and chronic leukemia (n = 42, 33 of which in resistant relapse), genetic diseases (n = 11), aplastic anemia (n = 2, one of which with platelet refractoriness and bleeding), myelodysplasia (n = 5) received an haploidentical bone marrow, mismatched for 2-3 HLA loci. The donor's marrow was treated in vitro with vincristine and methylprednisolone to obtain a functional T depletion (MLC and CTL inhibition, functional blockade of Th1 and Th2). The prevalence of infectious complications and GVHD was similar to that recorded in matched unrelated donor (MUD) transplants. In situations of high risk of rejection (chronic leukemia, genetic diseases) we infused immediately one half of the harvest and then frozen aliquots from the second week. Of the 25 ALL and 8 AML in resistant relapse, 3 survived, disease-free at 14, 8 and 1 years respectively. Of the 3 ALL, transplanted during remission, 1 is surviving at 18 months. Of the 6 CML, 1 had fractionated bone marrow and is surviving at 3 years, and 5 had standard single dose infusion and died of progression of their disease after rejection of the graft (4) or blast crisis after complete engraftment (1). The 2 patients with aplastic anemia, those with myelodysplasia, and 6 of the 10 with genetic disorders died of transplant-related complications or disease progression. 4 patients with osteopetrosis (n = 2), MLD (n = 1), Wiskott Aldrich dis. (n = 1) survive at 8, 2, 5 and 1.5 years respectively. In patients transplanted with fractionated marrow GVHD > 2nd grade occurred in 15%. Only one patient rejected the graft. Compared with MUD transplantation, mismatched BMT whenever performed in patients in good conditions provides similar outcome and widens the donor availability.
1986年至2000年6月，60例患有急性和慢性白血病（n = 42，其中33例处于耐药复发期）、遗传性疾病（n = 11）、再生障碍性贫血（n = 2，其中1例伴有血小板难治性和出血）、骨髓增生异常综合征（n = 5）的儿童接受了单倍体相合骨髓移植，该骨髓与2 - 3个HLA位点不匹配。供体骨髓在体外接受长春新碱和甲基强的松龙处理，以实现功能性T细胞清除（混合淋巴细胞培养和细胞毒性T淋巴细胞抑制，Th1和Th2的功能阻断）。感染并发症和移植物抗宿主病的发生率与匹配无关供体（MUD）移植中记录的发生率相似。在排斥风险高的情况下（慢性白血病、遗传性疾病），我们立即输注一半采集的骨髓，然后从第二周开始输注冷冻的等分样本。25例耐药复发的急性淋巴细胞白血病和8例急性髓细胞白血病中，3例存活，分别在14年、8年和1年时无病生存。3例在缓解期移植的急性淋巴细胞白血病中，1例在18个月时存活。6例慢性粒细胞白血病中，1例接受了分次骨髓移植，在3年时存活，5例接受了标准单剂量输注，其中4例在移植物排斥后死于疾病进展，1例在完全植入后死于急变期。2例再生障碍性贫血患者、骨髓增生异常综合征患者以及10例遗传性疾病患者中的6例死于移植相关并发症或疾病进展。4例患有骨质石化症（n = 2）、粘多糖贮积症（n = 1）、威斯科特-奥尔德里奇综合征（n = 1）的患者分别在8年、2年、5年和1.5年时存活。在接受分次骨髓移植的患者中，2级以上移植物抗宿主病的发生率为15%。只有1例患者排斥了移植物。与MUD移植相比，只要在身体状况良好的患者中进行不匹配的骨髓移植，就能提供相似的结果，并扩大了供体来源。
