Does one model fit all? Predicting non-relapse mortality after allogeneic hematopoietic cell transplantation
Does one model fit all? Predicting non-relapse mortality after allogeneic hematopoietic cell transplantation
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一种型号适合所有型号吗？
DOI：
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发表时间：
2021
影响因子：
4.8
通讯作者：
H. Nakasone
中科院分区：
文献类型：
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作者：
M. Yanada;N. Uchida;T. Ichinohe;T. Fukuda;J. Kanda;Y. Kanda;Y. Atsuta;H. Nakasone
异基因造血细胞移植（HCT）是一种成熟的治疗方法，可以使血液病患者长期生存;然而，非复发死亡率（NRM）的风险很大，这使得精确预测NRM具有临床重要性。评估异基因HCT中NRM风险的现有模型包括欧洲血液和骨髓移植学会（EBMT）评分[1]和HCT特异性合并症指数（HCT-CI）[2]，但其预测准确性不佳。我们最近开发了一种综合系统，用于预测急性髓系白血病（AML）首次完全缓解（CR 1）期间同种异基因HCT后的NRM [3]。在将2344例患者随机分为训练集或验证集后，我们首先确定并评分了五个参数-年龄，性别，体能状态（PS），HCT-CI和供体类型-基于它们对训练集中患者NRM的影响。新的评分系统被命名为“NRM-J指数”，使用指定评分的总和将患者分为四个不同的风险组。NRM-J指数对验证集中患者的应用具有比EBMT评分和HCT-CI更好的区分能力[3]。尽管NRM-J指数是专门为CR 1期AML患者开发的，但它仅基于患者和移植相关因素，不包括疾病相关因素。这种情况促使我们评估NRM-J指数是否有效地适用于其他疾病，在这里，我们提出的分析结果。在这项回顾性研究中，我们使用了由日本造血细胞移植学会和日本造血细胞移植数据中心赞助的移植登记统一管理计划收集的登记数据。该注册计划目前几乎覆盖了全国300多家移植中心，普及率超过99% [4]。有资格参加这项研究的患者是那些在2008年至2017年期间从匹配的同胞供体，匹配的无关供体或脐带血（UCB）单位接受首次异基因HCT的血液病患者。UCB单位必须是含有总有核细胞剂量至少为2.0 × 10/kg受体体重的单个单位，在HLA-A、-B和-DR抗原水平上至少有4/6匹配，这与日本的临床实践一致[5]。未纳入接受无关供体外周血干细胞移植的患者，因为研究期间日本很少进行该手术[6]。我们还排除了计算NRM-J指数所需信息缺失的患者。该研究由爱知癌症中心的机构审查委员会批准，并根据赫尔辛基宣言进行。获得每例患者的知情同意书。* Yanada Masamitsu myanada@aichi-cc.jp
Allogeneic hematopoietic cell transplantation (HCT) is an established treatment that enables long-term survival for patients with hematological diseases; however, the risk of non-relapse mortality (NRM) is substantial, which makes precise prediction of NRM a matter of clinical importance. Existing models for assessing the risk of NRM in allogeneic HCT include the European Society for Blood and Marrow Transplantation (EBMT) score [1] and the HCT-specific comorbidity index (HCT-CI) [2], but their predictive accuracy is suboptimal. We recently developed a comprehensive system for predicting NRM after allogeneic HCT during first complete remission (CR1) of acute myeloid leukemia (AML) [3]. After dividing 2344 patients randomly into a training set or a validation set, we first identified and scored five parameters—age, sex, performance status (PS), HCT-CI, and donor type—on the basis of their impact on NRM in patients in the training set. The new scoring system which was named “NRM-J index”, used the sum of the assigned scores to stratify patients into four distinct risk groups. The application of the NRM-J index to patients in the validation set had better discriminative capacity than did those of the EBMT score and the HCT-CI [3]. Despite the fact that the NRM-J index has been developed exclusively for patients with AML in CR1, it is based solely on patientand transplantation-related factors, and does not include diseaserelated factors. This situation prompted us to evaluate whether the NRM-J index is effectively applicable to other diseases, and here we present the analytic results. In this retrospective study, we used registry data collected through the Transplant Registry Unified Management Program, sponsored by the Japanese Society for Hematopoietic Cell Transplantation and the Japanese Data Center for Hematopoietic Cell Transplantation. This registration program currently covers nearly all of the more than 300 transplantation centers nationwide, with a penetration rate exceeding 99% [4]. Patients eligible for this study were those with hematological diseases who had undergone their first allogeneic HCT between 2008 and 2017 from a matched sibling donor, a matched unrelated donor, or an umbilical cord blood (UCB) unit. The UCB unit had to be a single unit containing a total nucleated cell dose at least 2.0 × 10/kg of the recipient’s body weight with at least 4/6match at the antigen level for HLA-A, -B, and -DR, which is in accordance with clinical practice in Japan [5]. Patients undergoing peripheral blood stem cell transplantation from an unrelated donor were not included because this procedure was rarely performed in Japan during the study period [6]. We also excluded patients for whom information needed for calculation of the NRM-J index was missing. The study was approved by the institutional review board of the Aichi Cancer Center, and was conducted in accordance with the Declaration of Helsinki. Informed consent was obtained from each patient. * Masamitsu Yanada myanada@aichi-cc.jp