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Nilotinib
Source: Childhood Chronic Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: December 10, 2024 · Captured 2026-09-09.
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Context: Treatment of Childhood CML / TKI Therapy
Nilotinib is a TKI that is approved by the FDA for the treatment of children with CML.
Evidence (nilotinib in children):
The FDA approved nilotinib in March 2018 for the treatment of children with CML based on two sponsored trials.[3,17]
An initial study (NCT01077544 [CAMN107A2120]) of 11 patients evaluated pharmacokinetic, safety, and preliminary efficacy data.
Source links and citations
A second study (NCT01844765 [CAMN107A2203; AAML1321]) of 58 patients evaluated efficacy and safety.[3] Data from both studies were combined for a pooled-data analysis of 69 patients, which included 25 patients with newly diagnosed CML and 44 patients with resistant or intolerant CML. Both studies used a dose of 230 mg/m2 given twice daily (rounded to the nearest 50 mg; maximum single dose, 400 mg).[3,17]
Source links and citations
A second study (NCT01844765 [CAMN107A2203; AAML1321]) of 58 patients evaluated efficacy and safety.[3] Data from both studies were combined for a pooled-data analysis of 69 patients, which included 25 patients with newly diagnosed CML and 44 patients with resistant or intolerant CML. Both studies used a dose of 230 mg/m2 given twice daily (rounded to the nearest 50 mg; maximum single dose, 400 mg).[3,17]
In the phase II trial, 64% of patients with newly diagnosed CML achieved an MMR at 1 year.
A second study (NCT01844765 [CAMN107A2203; AAML1321]) of 58 patients evaluated efficacy and safety.[3] Data from both studies were combined for a pooled-data analysis of 69 patients, which included 25 patients with newly diagnosed CML and 44 patients with resistant or intolerant CML. Both studies used a dose of 230 mg/m2 given twice daily (rounded to the nearest 50 mg; maximum single dose, 400 mg).[3,17]
The tolerability of nilotinib in children was similar to that observed in adults. Primary side effects affecting more than 30% of children included headache, fever, and hyperbilirubinemia.
A second study (NCT01844765 [CAMN107A2203; AAML1321]) of 58 patients evaluated efficacy and safety.[3] Data from both studies were combined for a pooled-data analysis of 69 patients, which included 25 patients with newly diagnosed CML and 44 patients with resistant or intolerant CML. Both studies used a dose of 230 mg/m2 given twice daily (rounded to the nearest 50 mg; maximum single dose, 400 mg).[3,17]
Prolongation of QTc interval (defined in this trial as an increase of >30 msec over baseline) is a recognized side effect of nilotinib, and it was observed in 25% of children in these trials. The investigators recommended obtaining an electrocardiogram at baseline, 1 week, periodically afterward, and after dose adjustments.
Studies cited in this source section
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Preserved source evidence · Not medical advice