| Jiaming Li,Sujiang Zhang,Yubao Chen,Zeying Yan,Ying Wang,Zhiyin Liu,Haimin Sun,Yu Chen. Efficacy and safety of combined decitabine and ruxolitinib in the treatment of chronic myelomonocytic leukemia. Oncol Transl Med, 2019, 5: 237-241. |
| 地西他滨联合芦可替尼治疗慢性粒单核细胞白血病 |
| Efficacy and safety of combined decitabine and ruxolitinib in the treatment of chronic myelomonocytic leukemia |
| Received:April 08, 2019 Revised:November 14, 2019 |
| DOI：10.1007/s10330-019-0349-9 |
| 中文关键词: 地西他滨; 芦可替尼; 慢性粒单核细胞白血病；突变频率 |
| 英文关键词: decitabine (DEC); ruxolitinib (RUX); chronic myelomonocytic leukemia (CMML) |
| 基金项目: |
| Author Name | Affiliation | E-mail | | Jiaming Li | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | lijiaming007007@126.com | | Sujiang Zhang* | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | zhangsjb1181@126.com | | Yubao Chen | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | | | Zeying Yan | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | | | Ying Wang | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | | | Zhiyin Liu | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | | | Haimin Sun | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | | | Yu Chen | Ruijin Hospital North Affiliated with Shanghai Jiao Tong University School of Medicine | |
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| 中文摘要: |
| 目的 分析地西他滨联合芦可替尼治疗慢性粒单核细胞白血病 ( CMML) 的临床疗效，并探索基因突变对疗效及预后的影响。方法 回顾性分析地西他滨联合芦可替尼治疗5 例CMML患者的临床特征及疗效，并采用二代测序监测治疗前后骨髓标本中基因突变频率的改变。 结果 5例患者对地西他滨联合芦可替尼治疗的总应答率为60%， 其中例2获得CR, 例3获得HI,例5获得mCR。所有患者脾脏较治疗前均有不同程度的缩小。通过动态监测骨髓样本中的基因突变频率（VAF）可知，JAK2-p.V617F基因VAF从治疗前的17 %降低至VAF<1%。SRSF2_p.P95L, P5.TET2_p.R550X，TET2_p.Q705X，CBL_p.R420Q和CBL_p.C396R 下降显著； ASXL1_p.G710fs, TET2_p.C1298Y 和CBL_p.W408S 几乎无变化。 随着疾病发展，CBL_p.H398P，TET2_p.L1721W，P1.TET2_p.Q273fs 和ASXL1_p.G642fs呈递增趋势。 结论 地西他滨联合芦可替尼治疗CMML，可有效改善患者临床症状，提高生存质量。 基于单基因突变频率评价临床疗效仍存在很大程度的变异性。 |
| 英文摘要: |
| Objective The aim of the study was to evaluate the clinical efficacy of decitabine (DEC) combined with
ruxolitinib (RUX) in the treatment of chronic myelomonocytic leukemia (CMML).
Methods The clinical characteristics of 12 patients with CMML were analyzed retrospectively and
subsequent target sequencing was performed to investigate the efficacy of the combined treatment with
DEC and RUX and the molecular signatures therein.
Results Among the 12 cases, clinical improvement was observed in all patients (100%), spleen reduction
was observed in six patients (67%), and hematologic improvement was observed in four patients (33%).
In the CMML-1 group, the overall response was 50% (3/6), one case achieved complete response, one
achieved bone marrow remission, and one achieved hematological improvement. In the CMML-2 group, the
overall response was 17% (1/6), one case achieved complete response, four showed disease progression
(PD), and one exhibited no response. As expected, ASXL1 mutation was predictive for the outcome of
CMML (hazard ratio of 2.97, 95% confidence interval of 1.21–7.06; P = 0.02).
Conclusion The use of DEC combined with RUX in the treatment of CMML effectively improved the
clinical response and quality of life, especially for CMML-1 patients. Ongoing clinical trials will further
evaluate the safety and efficacy of this novel therapeutic approach. |
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