Ponatinib, as a third-generation BCR-ABL tyrosine kinase inhibitor, has significant therapeutic effects on chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL), especially in patients with T315I mutations and other drug-resistant mutations.

1. Overall efficacy data

(1) Chronic phase CML: 56% of patients achieve major cytogenetic remission, 46% achieve complete cytogenetic remission, and the median progression free survival is 12.9 months.

(2) During the accelerated phase of CML, 55% of patients achieved major hematological remission, and 39% achieved major cytogenetic remission.

(3) During the acute phase of CML/Ph+ALL, 31% of patients achieved major hematological remission, and 12% achieved major cytogenetic remission.

2 special mutation efficacy

(1) T315I mutation: 70% of chronic phase patients achieve major cytogenetic remission, with a median duration of remission of 32 months.

(2) Other drug-resistant mutations: showed activity against mutations such as V299L and F317L/V/I/C.

3. Duration of therapeutic effect

(1) Chronic phase CML: The median duration of treatment is 32.2 months, with some patients experiencing sustained remission for more than 5 years.

(2) Accelerated phase CML: median treatment duration of 15.2 months, median overall survival of 28.5 months.

4 Factors affecting therapeutic efficacy

(1) Baseline characteristics: The number of previous treatment lines and disease staging affect the efficacy.

(2) Mutation type: T315I mutation patients have better therapeutic effects than other mutations.

(3) Treatment compliance: Standardized medication can improve the remission rate.