In non-severe aplastic anemia, the presence of a paroxysmal nocturnal hemoglobinuria (PNH) clone was positively associated with survival, while age was negatively associated with survival, according to results published in Leukemia. Austin Kulasekararaj, MD, and colleagues examined clinical and molecular features, treatment, evolution, and survival in 259 patients with non-severe aplastic anemia. Most were treated with cyclosporine (CyA) alone (n=84) or in combination with anti-thymocyte globulin (ATG; n=44) or eltrombopag (n=20). The researchers reported a 6-month overall response rate of 73% for CyA, 74% for ATG plus CyA, 68% for CyA plus eltrombopag, and 87% for eltrombopag. In addition, 39% of patients treated primarily with CyA plus eltrombopag achieved a trilineage response (P=0.02). Progression to myeloid neoplasms was limited (8%) and unrelated to mutational status. Hemolytic PNH occurred in 10% of cases and was predicted by identifying small clones at diagnosis. Age, male gender, and the need for platelet/erythrocyte transfusion were among the factors negatively associated with survival. Factors that positively impacted survival included higher neutrophil counts at diagnosis, the presence of PNH clones, and trilineage response at 6 and 12 months.
