Development and validation of a prediction model (AHC) for early identification of refractory thrombotic thrombocytopenic purpura using nationally representative data
- 1 October 2020
- journal article
- research article
- Published by Wiley in British Journal of Haematology
- Vol. 191 (2), 269-281
- https://doi.org/10.1111/bjh.16767
Abstract
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.Keywords
Funding Information
- Beijing Municipal Science and Technology Commission (Z171100001017084)
- National Natural Science Foundation of China (81970113, 81670116, 81730004)
- Natural Science Foundation of Beijing Municipality (H2018206423, 7171013)
This publication has 52 references indexed in Scilit:
- Measurement of anti‐ADAMTS13 neutralizing autoantibodies: a comparison between CBA and FRET assaysJournal of Thrombosis and Haemostasis, 2012
- Development and validation of a predictive model for death in acquired severe ADAMTS13 deficiency-associated idiopathic thrombotic thrombocytopenic purpura: the French TMA Reference Center experienceHaematologica, 2012
- AKI in the ICU: definition, epidemiology, risk stratification, and outcomesKidney International, 2012
- Pathogenesis and treatment of acquired idiopathic thrombotic thrombocytopenic purpuraHaematologica, 2010
- The utility of patient characteristics in predicting severe ADAMTS13 deficiency and response to plasma exchangeTransfusion, 2010
- Thrombotic thrombocytopenic purpura at the Johns Hopkins Hospital from 1992 to 2008: clinical outcomes and risk factors for relapseTransfusion, 2010
- An inquiry into the relationship between ABO blood group and thrombotic thrombocytopenic purpuraVox Sanguinis, 2009
- Thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS): a 24-year clinical experience with 178 patientsJournal of Hematology & Oncology, 2008
- Remission in acute refractory and relapsing thrombotic thrombocytopenic purpura following rituximab is associated with a reduction in IgG antibodies to ADAMTS‐13British Journal of Haematology, 2006
- Thrombotic thrombocytopenic purpura/haemolytic uraemic syndrome: a new index predicting response to plasma exchangeBritish Journal of Haematology, 2005