Pioglitazone Plus Imatinib Leads to Molecular Response in Patients With Chronic-Phase Chronic Myeloid Leukemia

For patients with chronic-phase chronic myeloid leukemia (CML) treated with imatinib, adding the diabetes 2 medication pioglitazone to treatment could deepen molecular response, according to results from a phase II, proof-of-concept study published in Cancer. The regimen was also well tolerated, the authors reported.

Philippe Rousselot MD, PhD, from the Department of Hematology and Oncology at the Centre Hospitalier de Versailles in France, and authors enrolled 24 patients with CML (age range = 24-79 years) who were treated with imatinib for at least two years at a stable daily dose and had achieved a major MR (MMR), but not MR4.5 (defined by BCR-ABL1/ABL1IS RNA levels ≤0.0032%).

Patients received add-on therapy with pioglitazone 30 mg to 45 mg.

By 12 months, patients experienced a cumulative incidence of molecular response 4.5 (MR4.5) of 56 percent (95% CI 37-76), despite a wide range of therapy duration (1.9-15.5 months), the authors noted. By 48 months, 88 percent of 17 evaluable patients who were still receiving imatinib achieved MR4.5.

The most common treatment-related adverse event was weight gain (n=12). One patient reported grade 3 hypokalemia, and one patient discontinued treatment due to grade 2 edema.

The researchers recommend these findings be confirmed in a future randomized trial.

Source: Rousselot P, Prost S, Guilhot J, et al. Pioglitazone together with imatinib in chronic myeloid leukemia: A proof of concept study. Cancer. 2016 December 27. [Epub ahead of print]