HEALTH PROFESSIONAL · SOURCE READING

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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An increasing number of targeted agents are now approved for use in adults with CML. The use of these agents in pediatric patients is slow because there are not many studies that include children. Table 1 and the following narratives describe findings from specific trials where pediatric data are available.

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Table 1

Table 1. Targeted Therapies and Outcomes Reported in Pediatric Clinical Trials
Target and Agents		Prospective Pediatric Outcomes				Reference
		CHR	CCyR	MMR	PFS
ATP = adenosine triphosphate; CHR = complete hematologic response; CCyR = complete cytogenetic response; MMR = major molecular response; PFS = progression-free survival.
aAt 36 months.
bAt 12 months.
cAt 48 months.
dFor available active clinical trials using this agent, see the Treatment Options Under Clinical Evaluation section.
BCR::ABL1 kinase domain ATP-binding pocket:
	Imatinib 260 mg/m2	98%a	61%	31%b	98%	Giona et al.[1]
	Imatinib 340 mg/m2		91.5%	66.6%b		Giona et al.[1]
	Dasatinib 60–72 mg/m2		92%b	52%b	93%c	Gore et al.[2]
	Nilotinib 230 mg/m2			64%b		Hijiya et al.[3]
	Bosutinib 400 mg/m2	Phase I study only				Brivio et al.[4]
	Ponatinibd	Anecdotal data only
BCR::ABL1 kinase domain myristoyl-binding pocket:
	Asciminibd	No prospective pediatric data

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Imatinib →Dasatinib →Nilotinib →Other TKIs →

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Preserved source evidence · Not medical advice