Development and validation of a prediction model (AHC) for early identification of refractory thrombotic thrombocytopenic purpura using nationally representative data
单位：[1]Peking University People’s Hospital, Peking University Institute of Hematology,[2]National Clinical Research Center for Hematologic Disease,[3]Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,[4]Collaborative Innovation Center of Hematology, Peking University,[5]Department of Clinical Epidemiology, Peking University People’s Hospital,[6]Department of Hematology, Navy General Hospital, ,[7]Second Hospital of Shanxi Medical University, Taiyuan,[8]The Second Hospital of Hebei Medical University, Shijiazhuang,[9]Qingpu Branch of Zhongshan Hospital,Fudan University, Shanghai,[10]Shanxi Dayi Hospital, Taiyuan,[11]Department of Hematology, People’s Hospital of Xinjiang, Xinjiang,[12]Zhongshan Hospital Affiliated to Dalian University, Dalian,[13]Department of Hematology, Beijing Hospital,[14]Department of Hematology, Fu Xing Hospital, Capital Medical University,[15]Department of Hematology, Beijing Friendship Hospital, Capital Medical University,临床科室血液科血液科首都医科大学附属北京友谊医院[16]Department of Hematology, Beijing LuHe Hospital, Capital Medical University,[17]Department of Hematology, ShunDe Hospital of GuangZhou University of Chinese Medicine, Guangzhou,[18]Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou,[19]Department of Hematology, He Ping Central Hospital of the Changzhi Medical College, Changzhi,[20]State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin,[21]The First Affiliated Hospital of Harbin Medical University, Harbin,[22]CAS Key Laboratory of Genomic and Precision Medicine, Collaborative Innovation Center of Genetics and Development, Beijing Institute of Genomics, Chinese Academy of Sciences,[23]China National Center for Bioinformation[24]University of Chinese Academy of Sciences, Beijing, China
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening haematological emergency. Although therapeutic plasma exchange together with corticosteroids achieve successful outcomes, a considerable number of patients remain refractory to this treatment and require early initiation of intensive therapy. However, a method for the early identification of refractory iTTP is not available. To develop and validate a model for predicting the probability of refractory iTTP, a cohort of 265 consecutive iTTP patients from 17 large medical centres was retrospectively identified. The derivation cohort included 94 patients from 11 medical centres. For the validation cohort, we included 40 patients from the other six medical centres using geographical validation. An easy-to-use risk score system was generated, and its performance was assessed using internal and external validation cohorts. In the multivariable logistic analysis of the derivation cohort, three candidate predictors were entered into the final prediction model: age, haemoglobin and creatinine. The prediction model had an area under the curve of 0 center dot 886 (95% CI: 0 center dot 679-0 center dot 974) in the internal validation cohort and 0 center dot 862 (95% CI: 0 center dot 625-0 center dot 999) in the external validation cohort. The calibration plots showed a high agreement between the predicted and observed outcomes. In conclusion, we developed and validated a highly accurate prediction model for the early identification of refractory iTTP. It has the potential to guide tailored therapy and is a step towards more personalized medicine.
基金：
Beijing Municipal Science and Technology CommissionBeijing Municipal Science & Technology Commission [Z171100001017084]; National Natural Science Foundation of ChinaNational Natural Science Foundation of China (NSFC) [81970113, 81730004, 81670116]; Beijing Natural Science FoundationBeijing Natural Science Foundation [H2018206423, 7171013]; National Key Research and Development Program of China [2017YFA0105500, 2017YFA0105503]; Foundation for Innovative Research Groups of the National Natural Science Foundation of ChinaNational Natural Science Foundation of China (NSFC) [81621001]
第一作者单位：[1]Peking University People’s Hospital, Peking University Institute of Hematology,[2]National Clinical Research Center for Hematologic Disease,[3]Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,[4]Collaborative Innovation Center of Hematology, Peking University,
共同第一作者：
通讯作者：
通讯机构：[1]Peking University People’s Hospital, Peking University Institute of Hematology,[2]National Clinical Research Center for Hematologic Disease,[3]Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,[4]Collaborative Innovation Center of Hematology, Peking University,[*1]Peking University People’s Hospital, Peking University Institute of Hematology, No. 11 Xizhimen South Street, Xicheng District, Beijing, China.
推荐引用方式(GB/T 7714)：
Gui Ruo-Yun,Huang Qiu-Sha,Cai Xuan,et al.Development and validation of a prediction model (AHC) for early identification of refractory thrombotic thrombocytopenic purpura using nationally representative data[J].BRITISH JOURNAL of HAEMATOLOGY.2020,191(2):269-281.doi:10.1111/bjh.16767.
APA：
Gui, Ruo-Yun,Huang, Qiu-Sha,Cai, Xuan,Wu, Jin,Liu, Hui-Xin...&Zhang, Xiao-Hui.(2020).Development and validation of a prediction model (AHC) for early identification of refractory thrombotic thrombocytopenic purpura using nationally representative data.BRITISH JOURNAL of HAEMATOLOGY,191,(2)
MLA：
Gui, Ruo-Yun,et al."Development and validation of a prediction model (AHC) for early identification of refractory thrombotic thrombocytopenic purpura using nationally representative data".BRITISH JOURNAL of HAEMATOLOGY 191..2(2020):269-281
