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Alternative TKI Therapy
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 Recurrent or Refractory Childhood CML
In children who develop a hematologic or cytogenetic relapse during treatment with imatinib or who have an inadequate initial response to their initial TKI agents, determination of BCR::ABL1 kinase domain variant status should be considered to help guide subsequent therapy. Depending on the patient’s variant status, alternative TKIs such as dasatinib, nilotinib, or bosutinib can be considered on the basis of the adult and pediatric experience with these agents.[1-6]
Evidence (dasatinib in children with resistant or intolerant CML):
In a study of 14 children with resistant or intolerant CML, the following results were observed:[6]
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In a study of 14 children with resistant or intolerant CML, the following results were observed:[6]
76% of patients were in complete cytogenetic remission, and 41% of patients had a major molecular response (MMR) after 12 months of dasatinib therapy.
In a study of 14 children with resistant or intolerant CML, the following results were observed:[6]
The progression-free survival (PFS) rate was 78% at 48 months.
Evidence (nilotinib in children with resistant or intolerant CML):
In a study of 44 children with CML who were resistant or intolerant to imatinib or dasatinib, the following results were observed:[7]
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In a study of 44 children with CML who were resistant or intolerant to imatinib or dasatinib, the following results were observed:[7]
40.7% of patients achieved an MMR after 12 months of nilotinib therapy.
In a study of 44 children with CML who were resistant or intolerant to imatinib or dasatinib, the following results were observed:[7]
After a median of 11.3 months, no patients had experienced disease progression.
Dasatinib and nilotinib are active against many BCR::ABL1 variants that confer resistance to imatinib, although the agents are ineffective in patients with the T315I variant. In the presence of the T315I variant, which is resistant to all U.S. Food and Drug Administration (FDA)–approved TKIs, an allogeneic HSCT should be considered. Ponatinib, the BCR::ABL1 inhibitor effective against the T315I variant in adults, has not been prospectively studied in the pediatric population.
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Preserved source evidence · Not medical advice