Discontinuation of imatinib in children with chronic myeloid leukaemia in sustained deep molecular remission: results of the STOP IMAPED study
Discontinuation of imatinib in children with chronic myeloid leukaemia in sustained deep molecular remission: results of the STOP IMAPED study
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DOI：
10.1111/bjh.15826
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发表时间：
2019-05-01
影响因子：
6.5
通讯作者：
de Bont, Eveline S. J. M.
中科院分区：
文献类型：
--
作者：
de Bruijn, Clara M. A.;Millot, Frederic;de Bont, Eveline S. J. M.
这项国际研究旨在评估儿童慢性粒细胞白血病(CML)深分子缓解(DMR)后停用伊马替尼至少2年的效果。这项分析的主要终点是通过非参数Kaplan-Meier方法估计的分子无复发生存率。主要终点是6个月内无分子复发患者的估计率。14例患者入选；4例患者维持DMR，随访时间分别为24个月(2例)、34个月和66个月，10例患者复发。所有分子复发均发生在停用伊马替尼后6个月内(中位数3个月，范围1-6个月)。在6个月内维持DMR的总体概率为28中心点6%。目前尚不能确定与分子复发相关的参数。考虑到儿童慢性粒细胞白血病的罕见，这是导致队列规模较小的原因，我们的发现表明，在持续DMR后，伊马替尼停药仅在有限数量的患者中成功，而在成人患者中报告的发生率要高得多。需要进一步的研究来扩大儿科慢性粒细胞白血病患者的队列，这些患者可能实现无治疗缓解，理想的前提是预测伊马替尼停药后L分子复发的发生。
This international study aimed to assess the effect of imatinib discontinuation in paediatric patients with chronic myeloid leukaemia (CML) after deep molecular remission (DMR) had been achieved and maintained for at least 2 years. The primary endpoint of this analysis was the molecular relapse-free survival, estimated by the non-parametric Kaplan-Meier method. Major endpoint was the estimated rate of patients without molecular relapse at 6 months. Fourteen patients were enrolled; 4 patients maintained DMR with a follow-up of 24 (two patients), 34 and 66 months, respectively, whereas 10 patients relapsed. All molecular relapses occurred within 6 months (median 3 months, range 1-6) after imatinib discontinuation. The overall probability of maintaining DMR at 6 months was 28 center dot 6%. No parameters associated with molecular relapse could be identified. Keeping in mind the rarity of paediatric CML, which contributed to the small size of the cohort, our findings illustrate that imatinib cessation after sustained DMR is successful in only limited numbers of patients, whereas much higher rates are reported in adult patients. Further research is needed to extend the cohort of paediatric CML patients who might achieve treatment-free remission with an ideal prerequisite of predicting the occurrence of molecular relapse l after imatinib cessation.