de la Fuente Josu, Baruchel André, Biondi Andrea, de Bont Eveline, Dresse Marie-Françoise, Suttorp Meinolf, Millot Frédéric
Paediatrics Department, Saint Mary Hospital, London, UK.
Br J Haematol. 2014 Oct;167(1):33-47. doi: 10.1111/bjh.12977. Epub 2014 Jun 30.
Chronic myeloid leukaemia in children and young people is a relatively rare form of leukaemia that shows increased incidence with age and some evidence suggests that the molecular basis differs from that in adults. Significant advances in targeted therapy with the development and use in children of tyrosine kinase inhibitors and the ability to monitor and understand the prognostic significance of minimal residual disease by standardized molecular techniques has shifted the management of this condition from bone marrow transplantation as the main therapeutic modality to individualized treatment for each patient based on achieving specific milestones. The physiological changes occurring during childhood, particularly those affecting growth and development and the long-term use of treatment, pose specific challenges in this age group, which we are only beginning to understand.
儿童和青少年慢性髓性白血病是一种相对罕见的白血病形式，其发病率随年龄增长而增加，并且一些证据表明其分子基础与成人不同。随着酪氨酸激酶抑制剂在儿童中的研发和应用以及通过标准化分子技术监测和理解微小残留病预后意义的能力取得重大进展，这种疾病的管理已从以骨髓移植作为主要治疗方式转变为基于实现特定目标为每位患者提供个体化治疗。儿童期发生的生理变化，尤其是那些影响生长发育和长期治疗使用的变化，给这个年龄组带来了特殊挑战，而我们才刚刚开始了解这些挑战。
