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.