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目的:观察亲溶酶体药物IHW2体外净化急性髓细胞白血病骨髓(BM)进行骨髓移植(ABMT)的临床疗效。方法:用IHW2体外净化ABMT患者的BM,冷冻保存后回输给患者。结果:3例患者输用净化处理的BM后均成功地恢复造血功能。2例患者移植后至今已分别持续缓解超过36、34个月。其中1例患者移植前染色体为46XY,del(16)(q22),移植后2个月检测为46XY正常染色体核型。1例患者于移植后19个月复发。结论:初步研究显示亲溶酶体药物IHW2,用于临床移植患者是安全可行的,并且对白血病骨髓有一定的净化作用
OBJECTIVE: To observe the clinical efficacy of the in vitro purification of acute myeloid leukemia (BM) using the lysosomal drug IHW2 in bone marrow transplantation (ABMT). Methods: The BM of ABMT patients was purified with IHW2 in vitro and cryopreserved and then transfused to patients. Results: All three patients successfully restored hematopoiesis after decontamination with BM. Two patients have been continuously relieved over 36,34 months after transplantation. One patient had 46XY, del (16) (q22) before transplantation and 46XY normal karyotype at 2 months after transplantation. One patient relapsed 19 months after transplantation. CONCLUSIONS: Preliminary studies have shown that the prodrug lysate IHW2 is safe and feasible for use in clinical transplant recipients and has a decontamination effect on leukemic bone marrow