Objective: To compare the efficacy of the Ph⁺ acute lymphoblastic leukemia (ALL)patients treated with combination of tyrosine kinase inhibitors (TKI)and chemotherapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) and Ph⁻ ALL patients with allo-HSCT.
Methods: A total of 19 Ph⁺ALL patients were matched with 19 Ph⁻ALL patients from 55 B-ALL patients receiving allo-HSCT in our hospital between January 2003 and August 2014 and were analyzed retrospectively.
Results: Gender, median age, number of patients with blood white count more than 30 × 10⁹/L, number of patients with meningeal leukemia, disease status before allo-HSCT, period of allo-HSCT, the source of stem cell from donors, HLA disparities between donor and recipient, conditioning regimens and number of infused mononuclear cells and CD34⁺ cells were comparable between two groups of Ph⁺ and 19 Ph⁻ALL patients. The median time of engraftment of neutrophil cells was 12 days versus 13 days (P= 0.284) and that of platelet 14 days versus 17 days (P=0.246), which were comparable between two groups. The estimated 3-year overall survival (OS) in Ph⁺ and Ph⁻ALL groups was (67.5 ± 12.4)% versus (74.3 ± 11.4)% (P=0.434) and 3-year disease free survival (DFS)was (67.8 ± 12.4)% versus (74.3 ± 11.4)% (P= 0.456), respectively. The cumulative incidence of degree Ⅱ-Ⅳ acute graft-versus-host disease (aGVHD)in Ph⁺ and Ph⁻ ALL group was (15.8±8.4)% versus (21.1 ± 9.4)% (P=0.665)and that of degree Ⅲ-Ⅳ aGVHD was (5.6 ± 5.4)% versus (11.5 ± 7.6)% (P=0.541), respectively. The cumulative incidence of cGVHD was (44.1 ± 14.0)% in Ph⁺ALL group versus (44.1 ± 13.0)% in Ph⁻ALL group (P=0.835) and that of extensive cGVHD was (13.1 ± 8.7)% versus (6.2 ± 6.1)% (P=0.379), respectively. The cumulative relapse rate and the cumulative non-relapse rate in both group also have no statistical difference [(10.8 ± 7.2)% versus (20.0 ± 10.7)% (P=0.957) and (23.9 ± 12.4)% versus (7.1±6.9)% (P=0.224), respectively].
Conclusion: The efficacy of Ph⁺ALL treated with combination of chemotherapy and TKIs and followed by allo-HSCT is comparable to that of Ph⁻ALL with allo-HSCT.
目的: 比较Ph染色体阳性（Ph+）急性淋巴细胞白血病（ALL）患者应用化疗联合酪氨酸激酶抑制剂（TKI）后行异基因造血干细胞移植（allo-HSCT）和Ph染色体阴性（Ph−）ALL患者化疗后进行allo-HSCT的疗效和安全性。
方法: 2003年1月至2014年8月行allo-HSCT的55例B-ALL患者中配对选取19例Ph−ALL患者（Ph−ALL组）与19例TKI联合allo-HSCT的Ph+ALL患者进行回顾性分析。
结果: Ph+ALL组和Ph−ALL组在性别、中位年龄、发病时外周血WBC >30×109/L的例数、合并中枢神经系统白血病的例数、移植前疾病缓解状态、移植时间、干细胞来源、供受者HLA相合情况、预处理方案、输入单个核细胞数和CD34+细胞数等方面基本匹配。Ph+ALL组和Ph−ALL组白细胞和血小板植活时间相当（12 d和13 d，P=0.284; 14 d和17 d，P=0.246）。Ph+ALL组和Ph−ALL组3年总生存率和无病生存率分别为（67.5±12.4）％和（74.3±11.4）%（P=0.434）、（67.8±12.4）％和（74.3±11.4）%（P=0.456），差异均无统计学意义。Ph+ALL组和Ph−ALL组Ⅱ~Ⅳ度急性移植物抗宿主病（aGVHD）的累积发生率分别为（15.8±8.4）％和（21.1±9.4）%（P=0.665），其中Ⅲ~Ⅳ度aGVHD的累积发生率分别为（5.6±5.4）％和（11.5±7.6）%（P=0.541）。慢性移植物抗宿主病（cGVHD）的累积发生率分别为（44.1±14.0）％和（44.1±13.0）%（P=0.835），其中广泛型cGVHD的累积发生率分别为（13.1±8.7）％和（6.2±6.1）%（P=0.379）。Ph+ALL组和Ph−ALL组累积复发率和累积非复发死亡率差异亦无统计学意义[分别为（10.8±7.2）％对（20.0±10.7）%（P=0.957）和（23.9±12.4）％对（7.1±6.9）%（P=0.224）]。
结论: Ph+ALL患者化疗联合TKI后行allo-HSCT与Ph−ALL患者行allo-HSCT的疗效相当。