The Long-term Efficacy and Safety of Nilotinib in Pediatric Patients With CML: a 5-Year Update of the DIALOG Study.

The efficacy and safety of nilotinib in pediatric patients with imatinib/dasatinib resistant/intolerant (R/I) or newly diagnosed (ND) Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia in chronic phase (CML-CP) was demonstrated in the phase II, open-label DIALOG study. In this final analysis, long-term efficacy and safety are presented for patients who completed 66 cycles (of 28 days) of treatment with nilotinib (230 mg/m2 twice daily) or discontinued early. Fifty-nine patients were enrolled and 58 were treated (R/I, n=33; ND n=25; median time on treatment: 60.5 and 51.9 months, respectively). In the R/I cohort, the cumulative major molecular response (MMR) rate was 60.6% and no patients had a confirmed loss of MMR. Among ND patients, the best overall MMR rate was 76.0%; three patients had a confirmed loss of MMR. The cumulative molecular response MR4 and MR4.5 (BCR::ABL1IS ≤0.01% and ≤0.0032%, respectively) rates by 66 cycles were 27.3% and 12.1% in the R/I cohort, and 56.0% and 44.0% in the ND cohort, respectively. The safety profile of nilotinib was consistent with earlier reports. No on-treatment deaths occurred. These long-term (up to approximately 5 years) data support the efficacy and safety of nilotinib in pediatric patients with Ph+ CML-CP. www.clinicaltrials.gov.uk #NCT01844765.