Molecular response (MR) in patients with chronic myeloid leukemia (CML) who were not eligible for treatment discontinuation was maintained with a long-term tyrosine kinase inhibitor (TKI) regimen of 1 month on/1 month off. These findings, from an interim analysis of a phase III, randomized study, were published in Cancer Medicine.

Many patients receiving TKIs for CML who do not achieve a stable deep molecular response and are not eligible for treatment discontinuation may be candidates for intermittent treatment. The OPTkIMA study sought to evaluate if a progressive de-escalation of TKI therapy would maintain molecular response and improve health-related quality of life (HRQOL).

For this study, 185 patients with CML (age, median 71 years [range, 60 to 89]) were recruited for the OPTkIMA study at 26 centers in Italy in 2015. A continuous 1 month on/off TKI treatment plan for 3 years (fixed plan; 99 patients) was compared with a progressive plan comprised of 1 month on/off for 1 year; 1 month on, 2 months off for 1 year; and 1 month on, 3 months off for 1 year (86 patients). Patients were assessed for molecular response (MR) and by health-related quality of life (HRQOL).

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After the first year of 1 month on/off TKI therapy, molecular response was maintained in 81% of patients. Patients who had been in MR for more than 3 years were less likely to lose MR (hazard ratio [HR], 0.23; 95% CI, 0.10-0.61; P =.0025).

A total of 119 patients continued therapy into year 2 (59 in the fixed group; 46 in the progressive group). Molecular response was maintained through the second year.

Symptoms of diarrhea (P =.022) and nausea (P =.006) improved during the first months of TKI therapy, but fatigue was exacerbated over time (P =.022). Women had lower HRQOL scores compared with men (P =.001).

This study was limited by its low sample sizes and retention rates.

This intermittent analysis suggested 1 month on/off TKI therapy was effective at maintaining molecular response among most patients with CML who were ineligible for treatment discontinuation.

Disclosure: Multiple authors declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original article for a full list of disclosures.


Malagola M, Iurlo A, Abruzzese E, et al. Molecular response and quality of life in chronic myeloid leukemia patients treated with intermittent TKIs: First interim analysis of OPTkIMA study. Cancer Med. Published online February 16, 2021. doi:10.1002/cam4.3778.