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造血干细胞移植后闭塞性细支气管炎综合征患者的临床危险因素和预后模型。

Clinical risk factors and prognostic model for patients with bronchiolitis obliterans syndrome after hematopoietic stem cell transplantation.

机构信息

Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.

National Clinical Research Center for Hematologic Disease, Beijing, China.

出版信息

Bone Marrow Transplant. 2024 Feb;59(2):239-246. doi: 10.1038/s41409-023-02151-9. Epub 2023 Nov 27.

Abstract

Bronchiolitis obliterans syndrome (BOS) is a common and potentially devastating noninfectious pulmonary complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Currently, predictive tools for BOS are not available. We aimed to identify the clinical risk factors and establish a prognostic model for BOS in patients who undergo allo-HSCT. We retrospectively identified a cohort comprising 195 BOS patients from 6100 consecutive patients who were allografted between 2008 and 2022. The entire cohort was divided into a derivation cohort and a validation cohort based on the time of transplantation. Via multivariable Cox regression methods, declining forced expiratory volume at 1 s (FEV1) to <40%, pneumonia, cGVHD except lung, and respiratory failure were found to be independent risk factors for the 3-year mortality of BOS. A risk score called FACT was constructed based on the regression coefficients. The FACT model had an AUC of 0.863 (95% CI: 0.797-0.928) in internal validation and 0.749 (95% CI: 0.621-0.876) in external validation. The calibration curves showed good agreement between the FACT-predicted probabilities and actual observations. The FACT risk score will help to identify patients at high risk and facilitate future research on developing novel, effective interventions to personalize treatment.

摘要

闭塞性细支气管炎综合征(BOS)是异基因造血干细胞移植(allo-HSCT)后常见且潜在破坏性的非感染性肺部并发症。目前,尚无预测 BOS 的工具。我们旨在确定 BOS 的临床危险因素,并为接受 allo-HSCT 的患者建立 BOS 的预后模型。我们回顾性地从 2008 年至 2022 年接受 allo-HSCT 的 6100 例连续患者中确定了一个包含 195 例 BOS 患者的队列。整个队列根据移植时间分为推导队列和验证队列。通过多变量 Cox 回归方法,我们发现 1 秒用力呼气量(FEV1)下降至<40%、肺炎、肺外 cGVHD 和呼吸衰竭是 BOS 3 年死亡率的独立危险因素。基于回归系数构建了一个名为 FACT 的风险评分。FACT 模型在内部验证中的 AUC 为 0.863(95%CI:0.797-0.928),在外部验证中的 AUC 为 0.749(95%CI:0.621-0.876)。校准曲线表明,FACT 预测概率与实际观察结果之间具有良好的一致性。FACT 风险评分将有助于识别高危患者,并为开发针对特定患者的新型有效干预措施以进行个体化治疗提供帮助。

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