Wang Liyu, Liu Huilan, Zheng Changcheng, Tang Baolin, Zhu Xiaoyu, Yao Wen, Zhang Lei, Sun Zimin
Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei 230001, China.
Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei 230001, China. Email:
Zhonghua Yi Xue Za Zhi. 2014 Nov 4;94(40):3150-3.
To explore the therapeutic efficacy of allo-geneic hematopoietic stem cell transplantation (allo-HSCT) for chronic myelogenous leukemia (CML) patients with T315I mutation.
Retrospective analyses were conducted for 4 patients with T315I mutation of CML undergoing allo-HSCT from June 2012 to January 2014, including 2 cases in acceleration phase and 2 in chronic phase. There were 2 males and 2 females with ages from 26 to 45 years. Two patients received HLA-matched sibling allo-geneic peripheral blood stem cell transplantation (allo-PBSCT) while another 2 unrelated cord blood stem cell transplantation (UCBT). No splenomegaly was found before transplantation. One case had F317L mutation. All of them were treated with imatinib before transplantation. And the time from medication to T315I mutation was 20-35 months. All of them were conditioned with myeloablative regimen and received a combination of cyclosporine A (CsA) and mycophenolate mofetil (MMF) for preventing graft-versus-host disease (GVHD).
Myeloid implantation was achieved in all of them. The time of absolute neutrophil count (ANC) ≥ 0.5×10(9)/L were 10-28 days. One patient whose platelet was not implanted died from severe pulmonary infection at Day 88 post-transplantation. For 3 patients, platelet ≥ 20×10(9)/L were 15-33 days. But the marrow short tandem repeat (STR)-PCR was 100% donor type at the time of 30 days post-transplantation in all patients. One case of UCBT developed pre-implantation immune response syndrome (PES) and one acute GVHD of gradeI at Day 12 after allo-PBSCT. However both were controlled after treatment with methylprednisolone. And 1/3 evaluatable patients developed chronic GVHD. BCR/ABL transcript was detected by qualitative PCR after transplantation. And all BCR/ABL fusion genes turned negative after 30 days of transplantation. Up to the follow-up endpoint, there was no relapse except for one mortality. And the time of disease-free survival was 133, 248 and 704 days respectively.
Allo-HSCT is currently the optimal treatment for T315I mutation of CML. And umbilical cord blood is an ideal donor for those patients without HLA-matched sibling donor.
探讨异基因造血干细胞移植（allo-HSCT）治疗T315I突变慢性髓性白血病（CML）患者的疗效。
对2012年6月至2014年1月接受allo-HSCT的4例CML T315I突变患者进行回顾性分析，其中加速期2例，慢性期2例。男性2例，女性2例，年龄26～45岁。2例患者接受HLA匹配的同胞异基因外周血干细胞移植（allo-PBSCT），另外2例接受非血缘脐血干细胞移植（UCBT）。移植前未发现脾肿大。1例有F317L突变。所有患者移植前均接受伊马替尼治疗，用药至T315I突变时间为20～35个月。所有患者均采用清髓性预处理方案，并接受环孢素A（CsA）和霉酚酸酯（MMF）联合预防移植物抗宿主病（GVHD）。
所有患者均实现髓系植入。中性粒细胞绝对计数（ANC）≥0.5×10⁹/L的时间为10～28天。1例血小板未植入的患者在移植后第88天死于严重肺部感染。3例患者血小板≥20×10⁹/L的时间为15～33天。但所有患者移植后30天时骨髓短串联重复序列（STR）-PCR检测均为100%供者型。1例UCBT患者发生植入前免疫反应综合征（PES），1例allo-PBSCT患者在移植后第12天发生Ⅰ级急性GVHD。但经甲泼尼龙治疗后均得到控制。1/3可评估患者发生慢性GVHD。移植后通过定性PCR检测BCR/ABL转录本，移植30天后所有BCR/ABL融合基因均转阴。至随访终点，除1例死亡外无复发。无病生存时间分别为133天、248天和704天。
allo-HSCT是目前治疗CML T315I突变的最佳方法。对于没有HLA匹配同胞供者的患者，脐血是理想的供者。
Zotero 插件