Clinical and Molecular Heterogeneity of Moderate Aplastic Anemia

Based on clinical Camitta criteria, acquired aplastic anemia is categorized according to the degree of blood count depression as severe (sAA) or moderate (mAA). In some cases mAA is a precursor of sAA; in others it is a pathophysiologically distinct chronic non-progressive entity (chronic mAA (cmAA)). It is also possible that some seemingly mAA cases represent entities ranging from congenital bone marrow failure syndromes or hypocellular MDS to misdiagnosed systemic conditions with secondary aplasias. Discriminating true cmAA from these may be important for clinical management and requires prolonged observation.We have been able to accurately dx and treat patients (pts) with acute sAA. In contrast, pts with cmAA can have mildly depressed counts, which remain relatively stable for yrs without the need for treatment (tx).Tx delay, though, may lead to unopposed destruction of stem cells.At our institution we have evaluated and managed 308 AA pts from 1998-2018. Of these pts, we identified 88 who met the Camitta criteria for mAA and of these, 2 progressed to sAA within 3 mos, 1 had clinically significant PNH at dx, and 85 were truly cmAA. Focusing on this true cmAA cohort our goals were to identify its distinct clinical features and response to therapies.The median f/u for the cohort was 45 mos, with median Hgb 10.1 g/dl, ANC 1.36 k/uL, Plts 47 k/uL, and absolute retic count of 0.053 M/uL at dx. The median age for cmAA was 43 yrs (range 6-88), with 55% (47/85) females (vs 48% (10...

Source: Blood - November 21, 2018 Category: Hematology Authors: Patel, B. J., Barot, S. V., Kuzmanovic, T., Przychodzen, B. P., Hirsch, C. M., Awada, H., Hasipek, M., Thota, S., Radivoyevitch, T., Advani, A. S., Kalaycio, M., Sekeres, M. A., Carraway, H. E., Maciejewski, J. P. Tags: 508. Bone Marrow Failure: Poster II Source Type: research