A retrospective study on effectiveness of combined recombinant human interferon-α-1b, interleukin-2, and thalidomide for the treatment of acute myeloid leukemia in various disease states.

Background: Interferon-α-1b, interleukin-2 combined with thalidomide (ITI) improved the outcome and prognosis of some acute myeloid leukemia (AML) patients, but the cases was insufficient. This study observed the efficacy and safety of this regimen in the treatment of numbers of AML patients in various disease states.

Methods: Starting in January 2014, patients with AML (n=188) were treated with ITI regimen, including 60 refractory/relapses patients in group A, 40 patients in group B remained minimal residual disease-positive (MRD) or changed from negative to positive again after consolidation therapy, and 88 patients in group C with initial complete remission of AML received the ITI treatment after routine consolidation therapy. Bone marrow, fusion gene and MRD were detected to judge the curative effect and the adverse reactions were observed. The remission rate, MRD status and long-term survival of three groups were analyzed. An AML mouse model was constructed to observe the anti-leukemia effect of the three drugs .

Results: Sixty patients with primary AML who were unable to receive chemotherapy, or with relapsed/refractory AML, showed a total response rate of 28.3% (17/60) after receiving the ITI regimen. Forty patients with morphologically complete remission and MRD-positive achieved a response rate of 77.5% (31/40); the MRD converted to negative in 19 patients and was mitigated in 12 patients. Among 88 patients with initial complete remission, 11 failed to maintain the negative MRD, and the relapse rate was 12.5%, which was significantly lower than that of the non-maintenance treatment group (54.3%). In the mouse model, interferon, interleukin-2, and thalidomide exerted an anti-leukemia effect, prolonged the survival time of the mice, and the anti-leukemia effect was further enhanced after administration of the combination ITI regimen.

Conclusions: For suitable patients, hematopoietic stem cell transplantation is still strong recommended. The ITI regimen may be an effective option for patients with AML who cannot tolerate conventional chemotherapy, including those with relapsed/refractory disease, those with a complete remission status but are MRD-positive, or those who require maintenance treatment after consolidation therapy. However, a rigorous clinical randomized controlled trial and more in-depth mechanism exploration are still needed to verify this conclusion.