Growth factors

Colony-stimulating factors: Granulocyte colony–stimulating factor and granulocyte-macrophage colony–stimulating factor have been studied in an effort to shorten the period of granulocytopenia associated with leukemia treatment.[12] If used, these agents are administered after administration of chemotherapy. Although the use of growth factors decreases the time to neutrophil recovery by 2 to 5 days, and decreases rates of febrile neutropenia and duration of hospitalization, randomized clinical trials have not shown an impact of growth factors on overall survival and their cost-effectiveness has been inconsistently reported.[13,14] Use of growth factors is not routinely recommended in the remission induction setting.

Erythropoiesis-stimulating agents: Anemia associated with the diagnosis of AML and induction chemotherapy is managed primarily with red blood transfusions. Unlike myelodysplastic syndromes, there is no role for the use of erythropoiesis stimulating agents (e.g., epoetin alfa and darbepoetin) during the treatment of AML.

Thrombopoietin mimetics: Clinical trials have assessed the use of thrombopoietin mimetics in the treatment of AML. Eltrombopag as a single agent was tested in a multicenter, randomized, placebo-controlled, double-blind, phase I/II trial of 98 patients with platelet counts less than 30 × 109/L as a result of AML or MDS. No significant improvements in platelet counts were recorded. Significant hemorrhage was reported in ten (16%) patients given eltrombopag and nine (26%) patients given placebo. No difference in disease progression or overall survival was observed.[15]Eltrombopag appeared to hasten platelet recovery and reduce the number of platelet transfusions needed when added in an unblinded fashion to induction chemotherapy in older FLT3-negative AML patients.[16] However, in a separate, randomized double-blind study of 148 patients, eltrombopag or placebo was added to high-dose induction chemotherapy.[17] The results of this study did not indicate any clinical benefit of eltrombopag over placebo. Given the minimal efficacy signal at this point, eltrombopag is not routinely recommended in the supportive care or remission induction setting.