Gender and survival in essential thrombocythemia: A two‐center study of 1,494 patients

Abstract

Based on suggestive information from recent epidemiologic data and earlier retrospective studies, we revisited the effect of gender on survival in 1,494 patients with essential thrombocythemia (ET). The primary study population included 904 patients from the Mayo Clinic (median age 58 years; 65% females); risk distribution, according to the international prognostic score for ET (IPSET), was 23% high, 42% intermediate and 35% low. Multivariable analysis that included IPSET-relevant risk factors identified male sex (HR 1.6, 95% CI 1.3-2.0), age ≥60 years (HR 4.3, 95% CI 3.4-5.4) and leukocyte count ≥11 × 10(9)/L (HR 1.5, 95% CI 1.3-1.9) as independent predictors of shortened survival. These findings were confirmed by analysis of a separate cohort of 590 ET patients (65% females) from the University of Florence, Italy, with corresponding HRs (95% CI) of 1.6 (1.1-2.5), 4.6 (2.2-9.5) and 1.8 (1.1-2.8). The independent prognostic effect of gender was further corroborated by a separate multivariable analysis against IPSET risk categories; HR (95% CI) for the Mayo Clinic/Florence cohorts were 1.5/1.6 (1.2/1.1-1.8/2.5) for male sex, 6.8/7.5 (5.0/3.1-9.3/18.3) for IPSET high risk and 2.8/4.1 (2.1/1.8-3.8/9.5) for IPSET intermediate risk. Furthermore, the survival disadvantage in men was most apparent in IPSET high risk category and in patients older than 60 years. In both patient cohorts, thrombosis history garnered significance in univariate, but not in multivariable analysis. The observations from the current study suggest that women with ET live longer than their male counterparts and that gender might supersede thrombosis history as a risk variable for overall survival.

Polycythemia vera and essential thrombocythemia: 2019 update on diagnosis, risk‐stratification and management

“…28 Furthermore, the above-mentioned collaboration between the Mayo Clinic, Rochester, Minnesota and University of Florence, Florence, 28 Furthermore, the above-mentioned collaboration between the Mayo Clinic, Rochester, Minnesota and University of Florence, Florence,…”

“…In a study of over 1500 patients with PV, risk factors for survival included advanced age, leukocytosis, venous thrombosis and abnormal In an even more recent report, we have identified male sex as an independent risk factor for overall survival in ET but not in PV. 28 and high risk (thrombosis history or age > 60 years with JAK2 mutation).…”

20+ Years and alive with primary myelofibrosis: Phenotypic signature of very long‐lived patients

“…The practical interpretation of the observations from this study include that genetic risk factors in PMF are most important in predicting short‐term survival while very long survival might be predicted by considering a limited number of clinical risk factors, including age, sex, constitutional symptoms, hemoglobin level, platelet count, leukocyte count, and circulating blast percentage. The survival benefit of female patients has been recently recognized in the setting of other MPN . In other words, young asymptomatic patients with PMF can expect 20+ years of survival, in the absence of thrombocytopenia (platelets <100 × 10 9 /L), anemia (hemoglobin <10 g/dL in women and < 11 g/dL in men), marked leukocytosis (leukocyte count <25 × 10 9 /L) and circulating blasts ≥2%; PMF patients with such clinical phenotype are unlikely to display high VHR karyotype or HMR mutations, thus providing an explanation for the limited role of genetic risk factors in determining long‐term survival.…”

Decreased survival and increased rate of fibrotic progression in essential thrombocythemia chronicled after the FDA approval date of anagrelide

“…[1][2][3][4] To date, drug therapy has not been shown to improve survival in ET and treatment is primarily indicated in order to prevent thrombotic complications. [1][2][3][4] To date, drug therapy has not been shown to improve survival in ET and treatment is primarily indicated in order to prevent thrombotic complications.…”

“…Life-expectancy in essential thrombocythemia (ET) is shorter than expected from the age-and sex-matched control population but is significantly better than that of other myeloproliferative neoplasms (MPN), including polycythemia vera (PV) and myelofibrosis (MF), with median survival for patients younger than 60 years of age approaching 33 years. [1][2][3][4] To date, drug therapy has not been shown to improve survival in ET and treatment is primarily indicated in order to prevent thrombotic complications. Accordingly, lower risk patients are managed by aspirin therapy or observation alone while cytoreductive therapy is reserved for high risk disease.…”