Prognostic factors in idiopathic (primary) osteomyelofibrosis

Abstract

BACKGROUND. Prognostic variables for idiopathic (primary) osteomyelofibrosis (IMF) are ill-defined because of the lack of large control studies based on uniform diagnostic criteria. METHODS. A retrospective clinicopathologic study was performed on 250 consecutively recruited patients (115 males and 135 females) with an established diagnosis of IMF. In contrast to previous studies, the current study cohort encompassed the full spectrum of initial to advanced stages of the disease process according to laboratory data and particularly histology. Because of the relatively high patient age on admission (median, 66.5 years), relative survival rates with corresponding life expectancies and disease specific life loss were calculated. Moreover, a classification and regression tree (CART) analysis was performed to segregate the study patients into subgroups with significantly different prognosis. RESULTS. Analysis of the life expectancy and the proportion of deaths attributable to IMF showed a global reduction in life expectancy of 31%. Further calculation disclosed a consistently greater impact of disease in older patients. Age, hemoglobin level on admission, and leukocyte and thrombocyte counts remained as the most relevant parameters for prognosis in multivariate consideration (CART analysis) and facilitated a clear-cut separation into three risk groups. The life expectancy of low risk patients was approximately 10 times higher than that of high risk patients (22.07 years vs. 2.25 years). CONCLUSIONS. These results are in keeping with the assumption that features signaling bone marrow insufficiency are associated with a worsening of survival. Generalization, indicated by myeloid metaplasia, can occur at every stage, even in so-called hypercellular phases of IMF. Conversely, myelofibrosis alone is not necessarily predictive of poor survival.