儿童和青少年慢性髓性白血病 (CML) - 临床病理学发现
Louis Nevejan1, Veerle Labarque2,3, Nancy Boeckx1,4
1Department of Laboratory Medicine, University Hospitals Leuven, Leuven, Belgium.
European journal of haematology
|November 20, 2023
概括
氨酸激酶抑制剂 (TKI) 显著改善了儿科慢性髓性白血病 (CML) 的治疗结果. 然而,确保患者坚持TKI治疗仍然是长期疾病管理的关键挑战.
科学领域:
- 血液学 血液学 血液学
- 儿科瘤学 儿科瘤学
背景情况:
- 慢性髓性白血病 (CML) 是儿童和青少年的一种罕见恶性瘤.
- 在比利时,每年发生率约为每百万人中2人.
研究的目的:
- 研究儿科CML的诊断特征,临床特征和治疗结果.
- 评估不断变化的治疗策略对患者预后的影响.
主要方法:
- 在1986年至2021年间在卢旺大学医院诊断的儿科CML患者 (年龄≤17岁) 病历的回顾性审查.
- 对治疗方案,反应率和疾病进展的分析.
主要成果:
- 分析了14名患者,所有患者都在慢性阶段被诊断出来.
- 在2003年之前的治疗方法 (基尿素,干扰素-α, allo-Tx) 显示出有限的疗效,有一些进展.
- 2003年后的氨酸激酶抑制剂 (TKI) 治疗导致高血,细胞遗传和分子反应率,没有观察到进展或死亡率.
- 药物合规性成为影响治疗成功的重要因素.
结论:
- 氨酸激酶抑制剂 (TKI) 显著改善了儿科CML患者的预后.
- 坚持TKI治疗对于实现和维持最佳治疗结果至关重要,并提出了相当大的挑战.
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