Zheng Jing, Zhou Yuping, Zhao Yanmin, Luo Yi, Yu Jian, Lai Xiaoyu, Wang Jinuo, Ye Yishan, Liu Lizhen, Fu Huarui, Yang Luxin, Wu Yibo, Sun Jie, Zheng Weiyan, He Jingsong, Zhao Yi, Wu Wenjun, Cai Zhen, Wei Guoqing, Huang He, Li Weiming, Shi Jimin
Bone Marrow Transplantation Center, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, 310000, China.
Departments of Hematology, The First College of Clinical Medical Science, China Three Gorges University, Yichang Central People's Hospital, Yichang, 443000, China.
Ann Hematol. 2025 Mar;104(3):1867-1876. doi: 10.1007/s00277-025-06202-7. Epub 2025 Jan 23.
The aim of this study was to develop and validate a nomogram predicting progression-free survival (PFS) for adult patients with positive acute lymphoblastic leukemia(Ph + ALL) who have undergone allogeneic hematopoietic stem cell transplantation(allo-HSCT) and tyrosine kinase inhibitor(TKI) treatment. Data were retrospectively collected from 176 adult patients diagnosed with Ph + ALL and treated with allo-HSCT and TKIs at The First Affiliated Hospital, Zhejiang University School of Medicine, between January 2015 and May 2023. 70% of the patients were randomly assigned to the training group(n = 124) and 30% of the patients were assigned to the validation group(n = 52). Univariate Cox regression analysis and Akaike Information Criterion(AIC) were utilized to identify significant predictive factors, leading to the development of a nomogram designed to forecast the probability of PFS at 6, 9, and 12 months post-transplantation. The final nomogram incorporated three key variables: presence of complex additional cytogenetic abnormalities (ACAs), minimal residual disease (MRD) status prior to allo-HSCT, and IKZF1 gene deletions. The calibration curves showed excellent consistency between the nomogram prediction and actual observation for 6-, 9- and 12-month PFS in the training set and validation set. The C-index of the training set was 0.726(95%CI: 0.635-0.816), which was no significantly different from the validation set(C-index = 0.774, 95%CI: 0.674-0.875, P > 0.05). This study may provide a simple and efficient prediction model for patients with Ph + ALL undergoing allo-HSCT and TKIs, which can accurately predict PFS subsequent to transplantation. This tool could potentially aid clinicians in decision-making processes and improve patient outcomes.
本研究的目的是开发并验证一种列线图，用于预测接受异基因造血干细胞移植（allo-HSCT）和酪氨酸激酶抑制剂（TKI）治疗的成人阳性急性淋巴细胞白血病（Ph+ALL）患者的无进展生存期（PFS）。回顾性收集了2015年1月至2023年5月期间在浙江大学医学院附属第一医院诊断为Ph+ALL并接受allo-HSCT和TKI治疗的176例成年患者的数据。70%的患者被随机分配到训练组（n = 124），30%的患者被分配到验证组（n = 52）。采用单因素Cox回归分析和赤池信息准则（AIC）来识别显著的预测因素，从而开发出一种列线图，用于预测移植后6、9和12个月的PFS概率。最终的列线图纳入了三个关键变量：复杂额外细胞遗传学异常（ACA）的存在、allo-HSCT前的微小残留病（MRD）状态以及IKZF1基因缺失。校准曲线显示，训练集和验证集中列线图预测与6个月、9个月和12个月PFS实际观察之间具有良好的一致性。训练集的C指数为0.726（95%CI：0.635 - 0.816），与验证集（C指数 = 0.774，95%CI：0.674 - 0.875，P > 0.05）无显著差异。本研究可能为接受allo-HSCT和TKI治疗的Ph+ALL患者提供一种简单有效的预测模型，该模型可以准确预测移植后的PFS。这个工具可能有助于临床医生进行决策过程并改善患者预后。
