Zaimoku et al reported factors associated with response to therapy for aplastic anemia. These can help to identify a patient who is more likely to benefit from non-transplant therapy. The authors are from the National Heart, Lung and Blood Institute at NIH.
Patient selection: severe aplastic anemia
Therapy: immunosuppression with or without eltrombopag (Promacta, a thrombopoietin receptor stimulant)
Predictors of response to immunosuppression:
(1) pretreatment absolute reticulocyte count >= 25 * 10^9/L
Predictors of response to immunosuppression plus eltrombopag:
(1) pretreatment absolute reticulocyte count 10-30 * 10^9/L
(2) higher pretreatment neutrophil count (> 0.25 * 10^9/L)
(3) reduced thrombopoietin concentrations
These predictors reflect residual hematopoiesis.
Predictors of complete response to immunosuppressive therapy with or without eltrombopag:
(1) age >= 10 years AND absolute lymphocyte count > 1.3 * 10^9/L
