Cancer Survival Group, Department of Non-Communicable Disease Epidemiology, London School of Hygiene and Tropical Medicine, London, UK.
Division of Pediatric Oncology, Department of Pediatrics, Cantonal Hospital of Aarau, Aarau, Switzerland.
Pediatr Blood Cancer. 2018 Dec;65(12):e27422. doi: 10.1002/pbc.27422. Epub 2018 Aug 31.
Survival probability in children, adolescents, and young adults with chronic myeloid leukemia (CML) has dramatically improved during recent years. Tyrosine kinase inhibitors (TKI), targeted drugs developed for patients with CML, were introduced in 2001 in England. We here quantify the trends in the "cure" proportion according to the year of diagnosis.
We included all children, adolescents, and young patients with CML (0 to 24 years) diagnosed in England during 1980 to 2005. We fitted mixture cure models to estimate the "cure" proportion and the median survival time among the "uncured" patients according to the year of diagnosis, adjusted for age at diagnosis.
The "cure" proportion increased dramatically between 1980 and 2005, from under 10% to over 80%, while conversely, the median survival time of "uncured" patients decreased slightly between 1980 and 1999, with the trend from 2000 being uncertain.
The striking improvement of the "cure" fraction in young patients with CML since the early 1980s is concomitant with improvement of treatment, especially the allogeneic hematopoietic stem-cell transplant and, later, the introduction of TKI. The trends over the last years (2000-2005) remain, however, uncertain and would benefit from further studies with more recent data and updated follow-up.
近年来，儿童、青少年和年轻的慢性髓性白血病（CML）患者的生存概率显著提高。酪氨酸激酶抑制剂（TKI）是为 CML 患者开发的靶向药物，于 2001 年在英国推出。我们在此根据诊断年份量化了“治愈”比例的趋势。
我们纳入了 1980 年至 2005 年期间在英国诊断的所有儿童、青少年和年轻 CML（0 至 24 岁）患者。我们拟合混合治愈模型，根据诊断年份，调整诊断时的年龄，估计“治愈”比例和“未治愈”患者的中位生存时间。
1980 年至 2005 年间，“治愈”比例从不足 10%急剧上升至 80%以上，而相反，“未治愈”患者的中位生存时间在 1980 年至 1999 年间略有下降，2000 年以后的趋势不确定。
自 20 世纪 80 年代初以来，年轻 CML 患者“治愈”比例的显著提高与治疗的改善，尤其是异基因造血干细胞移植以及后来 TKI 的引入有关。然而，过去几年（2000-2005 年）的趋势仍不确定，需要进一步研究，以获取更近期的数据和更新的随访结果。
