儿科适应风险指数 (PARI) 用于预测儿童 HSCT 后 2 年移植相关死亡率。
Pediatric Adapted Risk index (PARI) to predict 2-year transplant related mortality post-HSCT in children.
发表日期:2024 Aug 02
作者:
Reem Elfeky, Natalia Builes, Rachel M Pearce, Soumya P Kania, Zohreh Nademi, Giovanna Lucchini, Robert Chiesa, Persis J Amrolia, Mohamed L Sorror, Paul Veys, Kanchan Rao
来源:
Blood Advances
摘要:
已经进行了多种尝试来优化移植前风险评估,以改善造血干细胞移植(HSCT)决策并预测 HSCT 后的结果。然而,其与儿科人群的相关性仍不清楚。我们报告了 874 名儿童的 HCT-CI 重新验证结果,这些儿童在单一中心接受了 944 例恶性或非恶性疾病的 HSCT。在发现 HCT-CI 在我们的患者群体中无效后;我们提出了一种改进的儿科适应评分系统,该系统捕获与儿科相关的风险因素(RF)和合并症(CoM)。每个 RF/CoM 根据 TRM 的风险比 (HR) 分配一个整数权重; 0 (HR <1.2), 1 (1.2 ≥HR <1.75), 2 (1.75 ≥HR <2.5), 3 (HR ≥2.5) 。使用这些权重,设计了儿科适应 HSCT-RI (PARI),并让患者分为4个风险组;第 1 组无 RF/CoM,第 2 组:分数 1-2,第 3 组:分数 3-4,第 4 组:分数 ≥5。从第 1 组到第 4 组,2 年 TRM 呈线性增加(第 1 组 TRM= 6.2%,第 4 组为 50.9%)。 PARI 已在内部和外部儿科患者队列中成功得到验证。比较使用 c 统计量的模型,发现 PARI 模型在预测 Akaike 和 Schwarz 贝叶斯信息标准(AIC 和 BIC)分别为 1069.245 和 1073.269 的儿童 2 年 TRM 方面比 HCT-CI 更好;分别使用 PARI 与 1223.158 和 1227.051;分别使用HCT-CI。我们相信 PARI 将成为一个有价值的工具,为儿科 HSCT 患者提供更好的咨询和决策。版权所有 © 2024 美国血液学会。
Several attempts have been made to optimize pre-transplant risk assessment to improve hematopoietic stem cell transplantation (HSCT) decision-making and to predict outcome post- HSCT. However, its relevance to the pediatric population remains unclear. We report the results of revalidation of the HCT-CI in 874 children who received 944 HSCTs for malignant or non-malignant diseases at a single centre. After finding the HCT-CI invalid in our patient population; we proposed a modified pediatric adapted scoring system that captures risk factors (RF) and comorbidities (CoM) relevant to pediatrics. Each RF/CoM was assigned an integer weight based on its hazard ratio (HR) for TRM; 0 (HR <1.2), 1 (1.2 ≥HR <1.75), 2 (1.75 ≥HR <2.5), 3 (HR ≥2.5) .Using these weights, the pediatric adapted HSCT-RI (PARI) was devised, and patients were divided into 4 risk groups; group 1 without RF/CoM, group 2: scores 1-2, group 3: scores 3-4, group 4: scores ≥5. There was a linear increase in 2-year TRM from group 1 to 4 (TRM= 6.2% in group 1, 50.9% in group 4). PARI was successfully validated on an internal and external cohort of pediatric patients. Comparing models using c-statistics, PARI was found to be a better model than HCT-CI in predicting 2-year TRM in children with Akaike's and Schwarz's Bayesian information criteria (AIC and BIC) of 1069.245 and 1073.269; respectively using PARI vs 1223.158 and 1227.051; respectively using HCT-CI. We believe that PARI will be a valuable tool enabling better counselling and decision making for pediatric HSCT patients.Copyright © 2024 American Society of Hematology.