Management of Chronic Myeloid Leukemia in Childhood
Management of Chronic Myeloid Leukemia in Childhood
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DOI：
10.1007/s11899-012-0113-6
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发表时间：
2012-06-01
影响因子：
2.9
通讯作者：
Millot, Frederic
中科院分区：
文献类型：
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作者：
Suttorp, Meinolf;Eckardt, Louise;Millot, Frederic
儿童慢性粒细胞白血病(CML)是一种罕见的恶性肿瘤，其最佳治疗经验非常有限。传统上，异基因造血干细胞移植被认为是唯一有效的治疗方法。伊马替尼是一种BCR-ABL酪氨酸激酶(TKI)的小分子抑制剂，已被证明在成人CML中非常成功，导致分子反应延长，药物毒性有限。这种药物现在也被纳入儿科CML的一线治疗，尽管对严重的晚期后遗症的合理担忧仍未解决。具体的儿科治疗指南尚未制定，大多数算法都是从成人CML的经验中得出的。这篇综述试图总结关于TKI治疗的剂量、持续时间、不良反应和指导标准等问题的儿科研究，使成人医学中制定的指南适用于儿科。最重要的是，接受TKI治疗的CML儿童患者应纳入正式试验。
Childhood chronic myelogenous leukemia (CML) is a rare malignancy, and experience with optimal treatment is very limited. Traditionally, allogeneic hematopoietic stem cell transplantation was considered the only curative treatment. Imatinib, a small-molecule inhibitor of the BCR-ABL tyrosine kinase (TKI), has been proven highly successful in adults with CML, resulting in prolonged molecular response with limited drug toxicity. This drug is now included as front-line therapy for CML in pediatrics as well, though valid concerns about serious late sequelae remain unresolved. Specific pediatric treatment guidelines have not yet been formulated, and most algorithms are derived from experience in adult CML. This overview attempts to summarize pediatric studies on issues such as dose, duration, adverse effects, and steering criteria for TKI treatment, adapting guidelines developed in adult medicine to pediatrics. Most importantly, pediatric patients with CML receiving TKI treatment should be enrolled into formal trials.