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Nilotinib Therapy Safe, Effective for Certain Patients With CML
Results from a phase 2 study demonstrate the efficacy and manageable safety of nilotinib for the treatment of pediatric patients with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase (Ph-positive CML-CP; Blood. 2019;134[23]:2036-2045).
“When we initiated this study with nilotinib, imatinib was the only tyrosine kinase inhibitor indicated for pediatric patients with…[Ph-positive CML-CP]; alternative treatment options were needed, particularly for patients who developed resistance or intolerance…to imatinib,” explained Nobuko Hijiya, MD, Department of Pediatrics, Columbia University Medical Center, New York, and colleagues.
The phase 2 study included 58 patients with newly diagnosed Ph-positive CML-CP (n = 25) or Ph-positive CML-CP resistant or intolerant to imatinib or dasatinib (n = 33). Patients received imatinib 230 mg/m2 twice daily for 28-day cycles.
The primary efficacy end point was the major molecular response (MMR) rate by 6 cycles in patients with Ph-positive CML-CP resistant or intolerant to imatinib or dasatinib, and MMR rate by 12 cycles and complete cytogenetic response (CCyR) rate by 12 cycles in patients with newly diagnosed Ph-positive CML-CP.
The MMR rate at cycle 6 in the cohort of patients resistant or intolerant to imatinib or dasatinib was 39.4%. Additionally, 57.6% of patients achieved or maintained MMR and 81.8% achieved or maintained CCyR by 24 cycles.
The rates of MMR and CCyR by cycle 12 in the cohort of newly diagnosed patients were 64% each; the cumulative MMR and CCyR rates by cycle 24 were 68% and 84%, respectively.
“[A]lthough the size of this study was limited by the rarity of CML in children, these results demonstrate the efficacy of nilotinib in pediatric patients at the recommended 230-mg/m2 twice-per-day dose, as well as a manageable safety profile comparable with the known safety profile of nilotinib in adults with [Ph-positive CML-CP],” Dr Hijiya and colleagues concluded.—Janelle Bradley
