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移植相关性血栓性微血管病的预后模型(BATAP)及其外部验证。

A prognostic model (BATAP) with external validation for patients with transplant-associated thrombotic microangiopathy.

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

Blood Adv. 2021 Dec 28;5(24):5479-5489. doi: 10.1182/bloodadvances.2021004530.

Abstract

Transplant-associated thrombotic microangiopathy (TA-TMA) is a potentially life-threatening complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Information on markers for early prognostication remains limited, and no predictive tools for TA-TMA are available. We attempted to develop and validate a prognostic model for TA-TMA. A total of 507 patients who developed TA-TMA following allo-HSCT were retrospectively identified and separated into a derivation cohort and a validation cohort, according to the time of transplantation, to perform external temporal validation. Patient age (odds ratio [OR], 2.371; 95% confidence interval [CI], 1.264-4.445), anemia (OR, 2.836; 95% CI, 1.566-5.138), severe thrombocytopenia (OR, 3.871; 95% CI, 2.156-6.950), elevated total bilirubin (OR, 2.716; 95% CI, 1.489-4.955), and proteinuria (OR, 2.289; 95% CI, 1.257-4.168) were identified as independent prognostic factors for the 6-month outcome of TA-TMA. A risk score model termed BATAP (Bilirubin, Age, Thrombocytopenia, Anemia, Proteinuria) was constructed according to the regression coefficients. The validated c-statistic was 0.816 (95%, CI, 0.766-0.867) and 0.756 (95% CI, 0.696-0.817) for the internal and external validation, respectively. Calibration plots indicated that the model-predicted probabilities correlated well with the actual observed frequencies. This predictive model may facilitate the prognostication of TA-TMA and contribute to the early identification of high-risk patients.

摘要

移植相关性血栓性微血管病(TA-TMA)是异基因造血干细胞移植(allo-HSCT)后一种潜在危及生命的并发症。关于早期预后标志物的信息仍然有限,目前尚无 TA-TMA 的预测工具。我们试图开发和验证 TA-TMA 的预后模型。回顾性分析了 507 例 allo-HSCT 后发生 TA-TMA 的患者,根据移植时间将其分为推导队列和验证队列,进行外部时间验证。患者年龄(比值比 [OR],2.371;95%置信区间 [CI],1.264-4.445)、贫血(OR,2.836;95% CI,1.566-5.138)、严重血小板减少症(OR,3.871;95% CI,2.156-6.950)、总胆红素升高(OR,2.716;95% CI,1.489-4.955)和蛋白尿(OR,2.289;95% CI,1.257-4.168)被确定为 TA-TMA 6 个月结局的独立预后因素。根据回归系数构建了一个风险评分模型,命名为 BATAP(胆红素、年龄、血小板减少症、贫血、蛋白尿)。内部验证的验证 c 统计量为 0.816(95%CI,0.766-0.867),外部验证的验证 c 统计量为 0.756(95%CI,0.696-0.817)。校准图表明,模型预测的概率与实际观察到的频率密切相关。该预测模型有助于预测 TA-TMA,并有助于早期识别高危患者。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cee0/8714708/612a3c501739/advancesADV2021004530absf1.jpg

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