Long-Term Outcomes of Imatinib Treatment for Chronic Myeloid Leukemia.
Level 2 - randomized trial
Randomized controlled trial with long-term (median 10.9 years) follow-up
PubMed 28273028 · doi:10.1056/NEJMoa1609324
What was done
In an open-label, multicenter trial with a crossover design, patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML) were randomly assigned to receive either imatinib or interferon alfa plus cytarabine. Due to high crossover (65.6%) among patients assigned to interferon alfa plus cytarabine after a median of 0.8 years, long-term analyses focused on the group initially assigned to imatinib. Evaluated endpoints included overall survival, treatment response, and serious adverse events over a median follow-up of 10.9 years.
What was found
Among patients assigned to imatinib, the estimated 10-year overall survival rate was 83.3%. Overall, 48.3% of patients completed study treatment with imatinib, and 82.8% achieved a complete cytogenetic response. Investigator-assessed serious adverse events related to imatinib were uncommon, occurred most frequently during the first year of treatment, and showed no evidence of unacceptable cumulative or late toxic effects.
Why it matters
This study provides definitive decade-long outcome data demonstrating that frontline imatinib provides durable efficacy without late cumulative toxicities in chronic-phase CML.
Limits
The total sample size (n) was not reported in the abstract. Extensive crossover (65.6%) and short control-arm treatment duration (median 0.8 years) precluded long-term randomized comparative analysis against interferon alfa plus cytarabine. The study was open-label and industry-funded.
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