Heparin-induced thrombocytopenia leading to a diagnosis of essential thrombocythemia

International audienceEssential thrombocythemia (ET) belongs to the “BCR-ABL-negative” subcategory of myeloproliferative neoplasms (MPN) along with polycythemia vera (PV) and primary myelofibrosis (PM). Various recurrent molecular alterations have been described in classical MPN, such as JAK2 V617F, MPL W515L/K mutations, and deletion or insertions in the calreticulin (CALR) gene.1 MPN are known for high incidence of thrombotic complications, with a predominance of arterial rather than venous events (16.2% vs 6.2%). Indeed, the prevalence of overall thrombosis has been described in 28.6%, 20.7%, and 9.5% of patients with PV, ET, and PM, respectively.2 In addition to the traditional risk factors of thrombosis, blood cells count, mutational profile, chronic inflammation, and abnormal cell adhesion appear to be specific risk factors of thrombosis in MPN-patients.3 The classical initial treatment of these thrombotic complications includes unfractionated heparin (UFH)

A Novel 8-Predictors Signature to Predict Complicated Disease Course in Pediatric-onset Crohn’s Disease: A Population-based Study

International audienceBackground The identification of patients at high risk of a disabling disease course would be invaluable in guiding initial therapy in Crohn’s disease (CD). Our objective was to evaluate a combination of clinical, serological, and genetic factors to predict complicated disease course in pediatric-onset CD. Methods Data for pediatric-onset CD patients, diagnosed before 17 years of age between 1988 and 2004 and followed more than 5 years, were extracted from the population-based EPIMAD registry. The main outcome was defined by the occurrence of complicated behavior (stricturing or penetrating) and/or intestinal resection within the 5 years following diagnosis. Lasso logistic regression models were used to build a predictive model based on clinical data at diagnosis, serological data (ASCA, pANCA, anti-OmpC, anti-Cbir1, anti-Fla2, anti-Flax), and 369 candidate single nucleotide polymorphisms. Results In total, 156 children with an inflammatory (B1) disease at diagnosis were included. Among them, 35% (n = 54) progressed to a complicated behavior or an intestinal resection within the 5 years following diagnosis. The best predictive model (PREDICT-EPIMAD) included the location at diagnosis, pANCA, and 6 single nucleotide polymorphisms. This model showed good discrimination and good calibration, with an area under the curve of 0.80 after correction for optimism bias (sensitivity, 79%, specificity, 74%, positive predictive value, 61%, negative predictive value, 87%). Decision curve analysis confirmed the clinical utility of the model. Conclusions A combination of clinical, serotypic, and genotypic variables can predict disease progression in this population-based pediatric-onset CD cohort. Independent validation is needed before it can be used in clinical practice