Tyrosine kinase inhibitorFDA-approvedSecond-line

Ponatinib

How it works

Blocks the BCR-ABL tyrosine kinase enzyme, which is overactive in CML cells, and also inhibits other tyrosine kinases involved in cancer cell growth.

Cancer types

Leukemia— BCR-ABL-positive

Efficacy

In clinical trials, around 30% of patients with BCR-ABL-positive CML achieved a major cytogenetic response, with a median progression-free survival of approximately 1 year.

Side effects

Severe

This treatment carries a higher risk of serious side effects. Close medical monitoring is required throughout treatment.

Evidence from research

StudyCancer typeStageEfficacy
Testing Ponatinib for Pediatric Cancer TreatmentLeukemiaphase-1—Source →
Testing Treatments for Pancreatic Cancer in Lab ModelsPancreatic Cancerphase-2—Source →
Study of Decitabine, Venetoclax, and Ponatinib for Leukemia TreatmentLeukemiaphase-2—Source →
Testing a Combination of Medications for Acute Lymphoblastic LeukemiaLeukemiaphase-2—Source →
Asciminib Study for CML Patients Resistant to PonatinibLeukemiaphase-2—Source →
Combination Chemotherapy and Ponatinib Hydrochloride in Treating Acute Lymphoblastic LeukemiaLeukemiaphase-2—Source →
Testing Combination Therapy for CD22-Positive B-Lineage Acute Lymphoblastic LeukemiaLeukemiaphase-2—Source →
Evaluating Ponatinib Hydrochloride for Chronic Myeloid LeukemiaLeukemiaphase-2—Source →
Combining Ponatinib and Vitamin K2 May Help Treat Acute Myeloid LeukemiaLeukemialab-study—Source →
Choosing the Right Second-Line Treatment for Chronic Myeloid LeukemiaLeukemiareview—Source →
Ponatinib Improves Quality of Life for Leukemia PatientsLeukemiaphase-3—Source →
Ponatinib-based therapy shows promise for CML in myeloid blast phaseLeukemiaphase-2The rate of morphological remission with or without count recovery (i.e. overall response rate [ORR]) was 49%.Source →
Combining Two Leukemia Treatments Shows Promise in Lab ExperimentsLeukemialab-study—Source →
Combining Ponatinib and Blinatumomab in Leukemia TreatmentLeukemiaphase-2The complete molecular response rate was 83% and the rate of measurable residual disease negativity was 98%.Source →
BCR::ABL1 Inhibitors Affect Blood Vessel Growth DifferentlyLeukemialab-study—Source →
New Study Compares Treatments for a Specific Type of LeukaemiaLeukemiaphase-3—Source →
Comparing Ponatinib Doses for Chronic Myeloid LeukemiaLeukemiameta-analysis45 mg ponatinib was superior to other doses of ponatinib and other TKIs in CCyR, MCyR and CHR.Source →
Ponatinib's Long-Term Effects in Chronic Myeloid Leukemia PatientsLeukemiaobservationalThe estimated 2-year progression-free survival was 84%.Source →
CAR-T Cells and Ponatinib Help Leukemia PatientLeukemiaobservational—Source →
Ponatinib Shows Promise as Third-Line Treatment for Chronic Myeloid LeukemiaLeukemiaobservationalThe 3-year progression-free survival was 59% with 2G-TKI and 83% with ponatinib.Source →
Ponatinib and Dasatinib Compared in Leukemia Patients with Brain RelapseLeukemiaobservationalThe total CR (complete remission) rate was 90.5%.Source →
Safer Version of Cancer Drug Ponatinib DevelopedLeukemialab-studyThe new compounds showed a larger therapeutic window in vivo, leading to regression of human T315I mutant CML xenografts without cardiotoxicity.Source →
Real-World Study of Ponatinib Treatment for Chronic Myeloid LeukaemiaLeukemiaobservationalA molecular response was reported for 59% of patients.Source →
Ponatinib Effective in Some Pediatric Leukemia PatientsLeukemiaobservationalSix patients with Ph+ ALL and two with CML responded to ponatinib.Source →
Combining Herbal Extract with Leukemia Drug Shows PromiseLeukemialab-studyOSM reduced the ponatinib dose ∼5-fold.Source →
Ponatinib Affects Platelet Function in Leukemia PatientsLeukemialab-study—Source →
Ponatinib May Improve Outcomes for Leukemia PatientsLeukemiaobservationalThe 5-year progression-free survival and overall survival rates were 68% and 72%, respectively.Source →
Similar Heart Risks for Two Leukemia TreatmentsLeukemiaobservationalPonatinib and bosutinib was associated with similar incidence and risk of CV events, including MACEs (HR: 1.02; 95% CI: 0.35, 3.01), AOEs (HR: 0.90; 95% CI: 0.43, 1.85) and VOEs (HR: 0.92; 95% CI: 0.44, 1.94).Source →
Ponatinib vs Imatinib for Leukemia Treatment: Cost ComparisonLeukemiaphase-3Ponatinib offered an approximate gain of 3 months of life and 6 months of quality-adjusted life.Source →

This information is provided for general education only and is not medical advice. Always consult a qualified healthcare professional before making treatment decisions.