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Validation of the revised International Prognostic Score of Thrombosis for Essential Thrombocythemia (IPSET-thrombosis) in 585 Mayo Clinic patients.

The primary objective of treatment in essential thrombocythemia (ET) is to prevent thromboembolic complications. In this regard, advanced age and thrombosis history have long distinguished "low" from "high" risk patients. More recently, JAK2V617F and cardiovascular (CV) risk factors were identified as additional modifiers, leading to the development of a 3-tiered International Prognostic Score of Thrombosis for ET (IPSET-thrombosis): "low," "intermediate," and "high". The international data set used to develop IPSET-thrombosis was recently re-analyzed in order to quantify the additional pro-thrombotic effect of JAK2V617F and CV risk factors in specific risk subcategories. The revised IPSET-thrombosis identified four risk categories based on three adverse variables (thrombosis history, age >60 years and JAK2V617F): very low (no adverse features), low (presence of JAK2V617F), intermediate (age >60 years) and high (presence of thrombosis history or presence of both advanced age and JAK2V617F). In this study of 585 patients with ET (median age 68 years; 61% female), we validated the revised IPSET-thrombosis by confirming significant differences in thrombosis risk between "very low" and "low" (HR 2.4, 95% CI 1.1 - 5.3) and between "intermediate" and "high" (HR 2.3, 95% CI 1.1 - 5.2) risk patients. Furthermore, in multivariable analysis, only JAK2V617F (HR=1.8, CI= 1.07 - 2.94) and history of thrombosis (HR=2.1, CI= 1.20 - 3.58) were independently predictive of future thrombotic events. The revised IPSET-thrombosis needs confirmation in prospective studies, especially in terms of risk-adapted therapy that includes the need for aspirin therapy in very low risk, twice-daily aspirin therapy for low risk and cytoreductive therapy for low or intermediate risk patients.

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