Nilotinib is now approved for use by children aged 1 year and older with Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML) in the chronic phase.
The Food and Drug Administration expanded the drug’s indication to include use as first- and second-line treatment in children.
Adverse events in the pediatric studies were similar to those observed in adults. However, children experienced hyperbilirubinemia (grade 3/4: 13%) and transaminase elevation (AST grade 3/4: 1%; ALT grade 3/4: 9%). Additionally, one previously treated pediatric patient progressed with advance phase/blast crisis after about 10 months of treatment.
Nilotinib (Tasigna) was already approved in adults with newly diagnosed Ph+ CML in the chronic phase and adults with chronic phase and accelerated phase Ph+ CML resistant or intolerant to prior therapy.