Symptomatic burden in myelofibrosis (MF): Prospective international assessment in 128 MF patients.

Abstract

6610 Background: The symptomatic burden in patients (pts) with myeloproliferative neoplasm myelofibrosis (MF) is significant and being evaluated as a major target of experimental therapies. We conducted an international prospective assessment of symptomatic burden in MF. Methods: Pts with MF (either primary myelofibrosis [PMF] or from antecedent polycythemia vera [PVMF] or essential thrombocythemia [ETMF]) were enrolled in Argentina, France, Italy, Puerto Rico, Spain, Sweden, Uruguay, and USA. Pts completed translated versions of Myeloproliferative Symptom Assessment Form (MPN-SAF) and EORTC-QLQ-C30. Results: Data from 128 MF pts was combined for analysis (PMF [59%], ETMF [22%], and PVMF [19%]). Age (mean 65, range 34-89) and gender (46% F) were common for this disease. Pts were an average of 5.5yrs from MF diagnosis. Many pts had anemia (58% Hob<11 g/dL), leukopenia (10% WBC<3.5 x 109/L), thrombocytopenia (28% platelets <150 x 109/L). The majority of pts were symptomatic for most symptoms surveyed (Table). 43% of pts endorsed clinically deficient QOL (MPN-SAF score >4/10). 67% of pts with deficient QOL had at least one lab abnormality. Worse QOL correlated with higher BFI score (r=0.68, p<0.001), inactivity (r=0.54, p<0.001), and concentration problems (r=0.54, p<0.001). In EORTC scales, worse QOL was associated with fatigue (r=0.57, p<0.001) and reduced physical (r=0.61, p<0.001), role (r=0.62, p<0.001), and social functioning (r=0.57, p<0.001). EORTC scores indicated symptom severity in MF was similar to metastatic cancer, particularly for decreased QOL (62 vs 56), physical functioning (75 vs 76), and fatigue (42 vs 42). Conclusions: Overall, our data indicate that MF adversely effects physical and emotional functioning and QOL. This severe profile justifies the search for MF therapies that seek to improve symptoms. Prevalence of common MF symptoms (n=128). Patient symptom Prevalence Fatigue 99% QOL 92% Inactivity 79% Abdominal discomfort 73% Early satiety 73% Insomnia 72% Concentration 70% Sad mood 69% Sexual problems 69% Night Sweats 64% Abdominal pain 62% Dizziness 60% Numbness 59% Cough 55% Bone pain 54% Itching 51% Weight loss 48% Headache 44% Fevers 25%