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儿科适应风险指数预测儿童 HSCT 后 2 年移植相关死亡率。

Pediatric adapted risk index to predict 2-year transplant-related mortality post-HSCT in children.

机构信息

Immunology Department, Great Ormond Street Hospital, London, United Kingdom.

Infection, Immunity, and Inflammation Department, GOS Hospital for Children NHS Foundation Trust, University College London GOS Institute of Child Health, and NIHR GOSH BRC, London, United Kingdom.

出版信息

Blood Adv. 2024 Nov 26;8(22):5838-5852. doi: 10.1182/bloodadvances.2024013484.

Abstract

Several attempts have been made to optimize pretransplant risk assessment to improve hematopoietic stem cell transplantation (HSCT) decision-making and to predict post-HSCT outcomes. However, the relevance of pretransplant risk assessment to the pediatric population remains unclear. We report the results of revalidation of the hematopoietic cell transplantation comorbidity index (HCT-CI) in 874 children who received 944 HSCTs for malignant or nonmalignant diseases at a single center. After finding the HCT-CI invalid in our patient population, we proposed a modified pediatric adapted scoring system that captures risk factors (RFs) and comorbidities (CoMs) relevant to pediatrics. Each RF/CoM was assigned an integer weight based on its hazard ratio (HR) for transplant-related mortality (TRM): 0 (HR < 1.2), 1 (1.2 ≥ HR < 1.75), 2 (1.75 ≥ HR < 2.5), and 3 (HR ≥ 2.5). Using these weights, the pediatric adapted risk index (PARI) for HSCT was devised, and patients were divided into 4 risk groups (group 1: without RF/CoM; group 2: score 1-2; group 3: score 3-4; and group 4: score ≥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 have better performance than HCT-CI in predicting 2-year TRM in children, with Akaike and Schwarz Bayesian information criteria values 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 counseling and decision-making for pediatric patients with HSCT.

摘要

已经有几种尝试对移植前风险评估进行优化,以改善造血干细胞移植(HSCT)的决策,并预测移植后的结果。然而,移植前风险评估与儿科人群的相关性仍不清楚。我们报告了在单一中心对 874 名儿童进行的 944 例恶性或非恶性疾病的 HSCT 中,对造血细胞移植合并症指数(HCT-CI)进行重新验证的结果。在我们的患者群体中发现 HCT-CI 无效后,我们提出了一种改良的儿科适应评分系统,该系统可捕获与儿科相关的风险因素(RFs)和合并症(CoMs)。根据其与移植相关死亡率(TRM)的危害比(HR),每个 RF/CoM 被赋予一个整数权重:0(HR<1.2),1(1.2<HR<1.75),2(1.75<HR<2.5)和 3(HR≥2.5)。使用这些权重,制定了 HSCT 的儿科适应风险指数(PARI),并将患者分为 4 个风险组(组 1:无 RF/CoM;组 2:评分 1-2;组 3:评分 3-4;组 4:评分≥5)。从组 1 到组 4,2 年 TRM 呈线性增加(TRM,组 1 为 6.2%,组 4 为 50.9%)。PARI 在儿科患者的内部和外部队列中均成功验证。使用 C 统计量比较模型,发现 PARI 在预测儿童 2 年 TRM 方面的表现优于 HCT-CI,Akaike 和 Schwarz Bayesian 信息准则值分别为 1069.245 和 1073.269,而使用 HCT-CI 的则分别为 1223.158 和 1227.051。我们相信,PARI 将成为一种有价值的工具,使接受 HSCT 的儿科患者能够得到更好的咨询和决策。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/192c/11609366/5b6321ca8864/BLOODA_ADV-2024-013484-ga1.jpg

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