Associations between gender, disease features and symptom burden in the MPN population : An analysis by the MPN QOL International Working Group

The myeloproliferative neoplasms including polycythemia vera, essential thrombocythemia and myelofibrosis, are distinguished by their debilitating symptom profiles, life-threatening complications and profound impact on quality of life. The role gender plays in myeloproliferative neoplasm symptomatology remains under-investigated. In this study, we evaluated how gender relates to patient characteristics, disease complications and overall symptom expression. A total of 2006 patients (polycythemia vera=711, essential thrombocythemia=830, myelofibrosis=460, unknown=5) were prospectively evaluated with patients completing the MPN-SAF and Brief Fatigue Inventory Patient Reported Outcome tools. Information on individual patient characteristics, disease complications and laboratory data was collected. Consistent with known literature, most female patients were more likely to have essential thrombocythemia (48.6% vs. 33.0%; p<0.001) and most male patients were more likely to have polycythemia vera (41.8% vs. 30.3%; p<0.001). Males demonstrated higher rates of thrombocytopenia (13.9% vs. 8.2%; p<0.001) and had higher red-blood cell transfusion requirements (7.3% vs. 4.9%; p=0.02) with lower mean disease durations (6.4 vs. 7.2 years, p=0.03). Despite no statistical differences in risk scores, receipt of most therapies or prior complications (hemorrhage, thrombosis), females had more severe and more frequent symptoms for most individual symptoms, along with overall total symptom score (22.8 vs. 20.3; p<0.001). Females demonstrated particularly burdensome scores for abdominal-related symptoms (abdominal pain/discomfort) and microvascular symptoms (headache, fatigue, insomnia, concentration difficulties, dizziness; all p<0.01). Despite vocalizing more severe symptom burdens, females documented similar quality of life scores to males. The results of this study suggest that gender contributes to myeloproliferative neoplasm heterogeneity by influencing phenotypic profiles and symptom expression.