Department of Hematology, Yokohama Municipal Citizen's Hospital, Yokohama, Japan.
Department of Medicine, Division of Hematology and Rheumatology, Nihon University School of Medicine, Tokyo, Japan.
Ann Med. 2022 Dec;54(1):1244-1254. doi: 10.1080/07853890.2022.2069280.
Few studies have reported the outcomes of adolescents and young adults (AYAs) with chronic-phase chronic myeloid leukaemia (CML-CP) on tyrosine kinase inhibitors (TKIs).
We retrospectively analysed the clinical features, treatment response, and long-term outcomes of 42 AYA patients, in comparison to older patients. The initial therapies of AYA patients between 2001 and 2016 included imatinib ( = 24), dasatinib ( = 13) and nilotinib ( = 5).
In AYA patients, the peripheral blood (PB) white blood cell count and percentage of blasts at the diagnosis were significantly higher, haemoglobin levels were lower and the spleen size was larger. The major molecular response (MMR), event-free survival (EFS) and overall survival (OS) rates were comparable. A sub-analysis comparing imatinib to second-generation TKIs as the initial therapy also showed that their prognosis was comparable.
In conclusion, the tumour burden at the diagnosis of CML-CP is higher in AYA patients; however, their prognosis was not worse in comparison to older patients treated with TKIs. KEY MESSAGESFew studies have reported the outcomes of adolescents and young adults (AYAs) with chronic-phase chronic myeloid leukaemia (CML-CP) on tyrosine kinase inhibitors (TKIs). This study showed the tumour burden at the diagnosis of CML-CP is higher in AYA pa tients; however, their prognosis was not worse in comparison to older patients treated with TKIs. Understanding the biological and non-biological features of AYA patients with CML-CP on TKI therapy is essential for better management and to improve the outcomes.
很少有研究报告过接受酪氨酸激酶抑制剂（TKI）治疗的慢性期慢性髓系白血病（CML-CP）的青少年和年轻成人（AYA）的结局。
我们回顾性分析了 42 例 AYA 患者的临床特征、治疗反应和长期结局，并与老年患者进行了比较。2001 年至 2016 年间，AYA 患者的初始治疗包括伊马替尼（ = 24）、达沙替尼（ = 13）和尼洛替尼（ = 5）。
在 AYA 患者中，外周血（PB）白细胞计数和原始细胞百分比在诊断时明显较高，血红蛋白水平较低，脾脏较大。主要分子反应（MMR）、无事件生存（EFS）和总生存（OS）率相当。亚分析比较伊马替尼与第二代 TKI 作为初始治疗也表明，其预后相当。
总之，CML-CP 诊断时 AYA 患者的肿瘤负荷较高；然而，与接受 TKI 治疗的老年患者相比，其预后并不差。
很少有研究报告过接受酪氨酸激酶抑制剂（TKI）治疗的青少年和年轻成人（AYA）慢性期慢性髓系白血病（CML-CP）的结局。本研究表明，CML-CP 诊断时 AYA 患者的肿瘤负荷较高；然而，与接受 TKI 治疗的老年患者相比，其预后并不差。了解接受 TKI 治疗的 CML-CP AYA 患者的生物学和非生物学特征对于更好的管理和改善结局至关重要。
