Low-dose cytarabine maintenance therapy vs observation after remission induction in advanced acute myeloid leukemia: an Eastern Cooperative Oncology Group Trial (E5483)

The Eastern Cooperative Oncology Group (ECOG) conducted a prospective phase III study in patients with relapsed/refractory acute myeloid leukemia (AML ) to evaluate whether administration of repeated courses of low-dose cytara bine (LDAC) maintenance therapy after induction of complete remission in ad vanced AML would improve disease-free and overall survival, Patients with A ML in second/later relapse or refractory disease were first treated with a combination of high-dose cytarabine and amsacrine. Those who achieved compl ete remission were then randomized to observation or to receive LDAC, 10 mg /m(2) subcutaneously twice a day x2 21 days every 2 months until relapse oc curred. Of 86 patients eligible for randomization, 41 patients were assigne d to receive LDAC and 45 patients to observation, The median disease-free s urvival was 7.4 months for patients assigned to LDAC compared to 3.3 months for patients receiving no additional therapy, P=0.084. The median survival from randomization was 10.9 months and 7.0 months for patients receiving L DAC maintenance chemotherapy and observation, respectively (P = 0.615). The data from this study suggest that LDAC maintenance therapy given to patien ts with advanced AML who achieve complete remission can increase disease-fr ee survival compared to observation, but does not improve overall survival. Nevertheless, because of the ineffectiveness and toxicity of intensive pos t-remission chemotherapy in this circumstance, LDAC maintenance therapy, a tolerable outpatient regimen, offers the potential for improved quality of life.