A long term follow-up of a randomized trial comparing interferon-α with busulfan for chronic myelogenous leukemia

To evaluate the long-term effectiveness of interferon-α (IFN-α) therapy in patients with chronic myelogenous leukemia (CML) in chronic phase, we examined the updated outcomes of 159 patients who had been enrolled between 1988 and 1991 into a randomized trial comparing IFN-α with busulfan. At a median follow-up of 73 months, the median survival was 71 months in the IFN-α group and 55 months in the busulfan group (P = 0.0563), and the median time of remaining in chronic phase was 58 months in the IFN-α group and 39 months in the busulfan group (P = 0.4676). Landmark analysis showed a significant advantage in survival (P = 0.009) and duration of chronic phase (P = 0.0001) in patients with any cytogenetic response among the IFN-α group. About half patients were discontinued IFN-α administration in spite of cytogenetic response in this study. It appears that continuation of IFN-α might possibly confer a survival advantage. Pretreatment factors associated with cytogenetic response included high hemoglobin level, low percentage of peripheral basophils and low leukocyte counts. Multivariate analysis identified lower percentage of bone marrow basophilia (P = 0.007) for survival advantage. If a group with a very good prognosis is predicted by a new prognostic model, it might be an option to wait for bone marrow transplantation.