Blood:Midostaurin可明显延长携带FLT3-ITD的急性髓系白血病患者的无进展存活期

2019-01-09 MedSci MedSci原创

携带FLT3内部串联重复序列(ITD)的急性髓系白血病(AML)患者预后差、易复发。Richard F. Schlenk等人开展一II期的假设生成试验,探究强化化疗联合多靶向激酶抑制剂midostaurin(米哚妥林)治疗,随后再予以异基因造血干细胞移植(alloHCT)和单药维持治疗12个月是否可行,对无进展存活期(EFS)是否有积极影响。研究人员共招募了284位18-70岁新确诊的携带FLT3

携带FLT3内部串联重复序列(ITD)的急性髓系白血病(AML)患者预后差、易复发。Richard F. Schlenk等人开展一II期的假设生成试验,探究强化化疗联合多靶向激酶抑制剂midostaurin(米哚妥林)治疗,随后再予以异基因造血干细胞移植(alloHCT)和单药维持治疗12个月是否可行,对无进展存活期(EFS)是否有积极影响。

研究人员共招募了284位18-70岁新确诊的携带FLT3-ITD的AML患者进行治疗,其中198位为年轻人(18-60岁),86位老年人(61-70岁)。诱导化疗后的完全缓解(CR)率(包括CR伴部分血液学恢复[CRi])达76.4%(年轻人75.8%、老年人77.9%)。大部分CR/CRi的患者(72.4%)进一步接受alloHCT治疗。97位(34%)患者进行维持治疗,其中75位是在alloHCT后,22位是在采用高剂量阿糖胞苷(HiDAC)巩固治疗后;维持治疗的中位时间分别是9个月(alloHCT)和10.5个月(HiDAC);提前终止大多是非复发因素(肠毒性和感染)。

年轻患者的2年的EFS和总体存活率分别是39%(95% CI 33-47%)和34%(95% CI 24-47%),而老年患者的分别是53%(95% CI 46-61%)和46%(35-59%)。

将EFS与5个前瞻性试验中的415位历史对照进行对比分析显示,米哚妥林治疗可明显提高患者的EFS(风险比[HR]0.58;95% CI 0.48-0.70,p<0.001)。


原始出处:

Richard F. Schlenk, et al. Midostaurin added to chemotherapy and continued single agent maintenance therapy in acute myeloid leukemia with FLT3-ITD. Blood 2018 :blood-2018-08-869453; doi: https://doi.org/10.1182/blood-2018-08-869453 

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    2019-12-04 tongyongming
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    2019-01-10 szhvet
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    2019-01-10 kord1986
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    2019-01-10 fengyi816
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