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儿童慢性髓性白血病一线酪氨酸激酶抑制剂:疗效与安全性研究

Front-Line Tyrosine Kinase Inhibitors in Pediatric Chronic Myeloid Leukemia: A Study on Efficacy and Safety.

作者信息

Yoo Jae Won, Jo Suejung, Ahn Moon Bae, Kim Seongkoo, Lee Jae Wook, Kim Myungshin, Cho Bin, Chung Nack-Gyun

机构信息

Department of Pediatrics, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul 06591, Republic of Korea.

Catholic Genetic Laboratory Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul 06591, Republic of Korea.

出版信息

Cancers (Basel). 2023 Jul 29;15(15):3862. doi: 10.3390/cancers15153862.

DOI:10.3390/cancers15153862
PMID:37568679
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10416896/
Abstract

We conducted a retrospective study on 51 pediatric patients with newly diagnosed chronic myeloid leukemia chronic phase or accelerated phase. The patients were classified into the IMA group (N = 33), treated with imatinib, and the DSA group (N = 18), treated with dasatinib, as front-line tyrosine kinase inhibitors (TKIs). At 12 months, the rates of complete cytogenetic response were similar between the IMA group (92.3%) and DSA group (100%) ( = 0.305). However, the rate of early molecular response was higher in the DSA group than in the IMA group (100.0% vs. 80.0%, = 0.043). By 12 and 24 months, the DSA group showed faster and higher cumulative rates of both major (DSA group: 72.2% and 100%, respectively; IMA group: 41.2% and 68.7%, respectively; = 0.002) and deep molecular responses (DSA group: 26.0% and 43.6%, respectively; IMA group: 13.8% and 17.5%, respectively; = 0.004). Both TKIs were well tolerated. Although the height standard deviation scores decreased in both groups, the height decline was greater in the DSA group between one and two years from the start of TKI therapy. In this study, dasatinib achieved faster and higher molecular responses with an acceptable safety profile. Further follow-up is necessary to assess the long-term outcomes of TKI treatment in children.

摘要

我们对51例新诊断为慢性髓性白血病慢性期或加速期的儿科患者进行了一项回顾性研究。患者被分为伊马替尼组(N = 33),接受伊马替尼治疗,以及达沙替尼组(N = 18),接受达沙替尼治疗,作为一线酪氨酸激酶抑制剂(TKIs)。在12个月时,伊马替尼组(92.3%)和达沙替尼组(100%)的完全细胞遗传学缓解率相似(P = 0.305)。然而,达沙替尼组的早期分子反应率高于伊马替尼组(100.0%对80.0%,P = 0.043)。到12个月和24个月时,达沙替尼组在主要分子反应(达沙替尼组分别为72.2%和100%;伊马替尼组分别为41.2%和68.7%;P = 0.002)和深度分子反应(达沙替尼组分别为26.0%和43.6%;伊马替尼组分别为13.8%和17.5%;P = 0.004)方面均显示出更快且更高的累积率。两种TKIs耐受性均良好。虽然两组的身高标准差分数均下降,但从TKI治疗开始的1至2年期间,达沙替尼组的身高下降幅度更大。在本研究中,达沙替尼实现了更快且更高的分子反应,安全性可接受。有必要进行进一步随访以评估儿童TKI治疗的长期结局。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/149eb050cd9d/cancers-15-03862-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/bdd2150a1f67/cancers-15-03862-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/f4a311f0da2c/cancers-15-03862-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/149eb050cd9d/cancers-15-03862-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/bdd2150a1f67/cancers-15-03862-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/f4a311f0da2c/cancers-15-03862-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04bd/10416896/149eb050cd9d/cancers-15-03862-g003.jpg

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Front Oncol. 2023 Mar 23;13:1122714. doi: 10.3389/fonc.2023.1122714. eCollection 2023.
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Front Pediatr. 2022 Jul 27;10:928136. doi: 10.3389/fped.2022.928136. eCollection 2022.
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