Blood Research

Summary of pediatric CML patients with the T315I mutation treated with ponatinib.

Gender Age (yr) Disease status at start of ponatinib KD mutations Initial ponatinib dose (/day) Ponatinib treatment duration (m) Response to ponatinib Reference
M 10 Disease progression T315I 45 mg (25 mg/m2) 66 UD [2, 6]
M 16 Blast phase T315I
E225K
30 mg (19 mg/m2) 3 MR4a) [6]
M 14.4 Blast phase T315I
E255K
2.5 None [7]
M 9.3 Blast phase T315I 1.5 MR4 [7]
M 10.8 Blast phase T315I 5 MMR [7]
M 8.6 MR4.5b) T315I 1.5 None [7]

a)MR4 defined as BCR-ABL1 transcript level ≤0.01% according to International Scale. b)MR4.5 defined as BCR-ABL1 transcript level ≤0.0032% according to International Scale.

Abbreviations: KD, kinase domain; MMR, major molecular response; UD, undetectable.

Blood Res 2021;56:342~345 https://doi.org/10.5045/br.2021.2021104
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