Nilotinib 300 Mg Twice Daily Is Effective and Well Tolerated as First Line Treatment of Ph-Positive Chronic Myeloid Leukemia in Chronic Phase: Preliminary Results of the ICORG 0802 Phase 2 Study.

Abstract 3294

Poster Board III-1

Imatinib (IM) 400 mg daily is currently the standard treatment for early chronic phase (ECP) CML. Nilotinib, a more potent, second generation Abl inhibitor, is highly effective in IM resistant patients and two recent phase II trials have demonstrated impressive activity of nilotinib 400mg BID in ECP. Given the relatively favourable prognosis of this population with IM 400mg alone, any significant increase in toxicity compared to IM could be problematic. Thus, while initial results with nilotinib 400mg BID have been very encouraging, we wished to explore a lower dose schedule, 300mg BID, believing this could be equally effective but with the potential for fewer adverse events, such as lipase and bilirubin elevations and QT prolongation. To investigate the safety and efficacy of nilotinib 300 mg BID in untreated, ECP, Ph-pos CML patients, ICORG, the All-Ireland Cooperative Oncology Research Group is conducting an open-label, single stage, multicenter, phase II study (ClinicalTrials.gov NCT00809211). The primary endpoint is the CCyR rate at 6 months; secondary endpoints include the kinetics of molecular response, determined by Q-PCR at baseline and 3 monthly from start of treatment. To date, 15 patients have been enrolled on the trial. The median age is 56 (range 26 –77); 47% have low risk Sokal score, 20% intermediate and 33% high risk. Median follow up is currently 70 days (range 10–250).