Background: phase III RCT included 846 patients with newly diagnosed CML patients with chronic phase Ph+ CML

Arm A: nilotinib 300mg BID
Arm B: nilotinib 400mg BID
Arm C: imatinib 400mg BID

Primary end point: MMR at 12mo (BCR-ABL transcript levels on the International Scale (BCR-ABL(IS)) of <=0.1%)

12-mo MMR: 44% vs 43% vs 22%, arm A vs B vs C respectively
12-mo CCyR: 80% vs 78% vs 65%, arm A vs B vs C respectively

UPDATE at 24mo, A Hagop M Kantarjian , Lancent Onc, 2011, PMID:21856226
Minimum follow-up: 24mo
24-mo MMR: 72% vs 67% and 44%, arm A vs B vs C respectively
24-mo CMR: 26% vs 21% vs 10%, arm A vs B vs C respectively

UPDATE at 10yr: Hagop M Kantarjian et al, Leukemia, PMID:33414482
10-yr MMR: 77.7% vs 79.7% vs 62.5%, arm A vs B vs C respectively
10-yr MR4.5: 61.0% vs 61.2 vs 39.2%, arm A vs B vs C respectively
10-yr OS: 87.9% (HR vs imatinib: 1.07, 95%CI 0.64–1.76) vs 90.3% (HR vs imatinib: 0.79, 95%CI 0.46–1.36) vs 88.3%, arm A vs B vs C respectively
10-yr PFS: 86.2% (HR vs imatinib: 1.08, 95%CI 0.67–1.74) vs 89.9% (HR vs imatinib: 0.74, 95%CI 0.44–1.25) vs 87.2%, arm A vs B vs C respectively

Main adverse events. Peripheral arterial occlusive disease, hyperglycemia, hypercholesterolemia, QT prolongation (black box warning needs EKG before therapy: 6.8% vs 7.9% vs 3.9%), hepatotoxicity (any grade: 48.4% vs 53.1% vs 17.5%), elevated amylase/lipase, elevated bilirubin. 10-yr cumulative cardiovascular events: 16.5% vs 23.5% vs 3.6%

Conclusions: nilotinib significantly improved MMR, MR and MR4.5 and lowered rates of progression to AP/BP and CML-related death comparing to patients receiving imatinib.

Summarized by Veli Bakalov, MD

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