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Ponatinib
Source: Chronic Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 13, 2025 · Captured 2026-09-09.
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Context: Treatment of Relapsed CML / Treatment Options for Relapsed CML / Targeted therapy with TKIs
Ponatinib is an oral TKI that has activity in patients with T315I variants or in patients for whom another TKI failed.[17-19] Multiple phase II studies concluded that the optimal response (≤1% BCR::ABL1) and least toxicity occurred at a 45 mg starting dose, with a decrease to 15 mg upon achieving the response.[20,21][Level of evidence C3] Ponatinib is associated with increased cardiovascular adverse events. Patients with significant cardiovascular disease, hypertension, or diabetes mellitus have been excluded from clinical trials.[20,21]
Evidence (ponatinib):
Ponatinib has been studied in multiple phase II studies involving 799 patients.[17,21][Level of evidence C3]
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Ponatinib has been studied in multiple phase II studies involving 799 patients.[17,21][Level of evidence C3]
Of the 799 patients with the T315I variant or resistance to two or more prior TKIs, 46% to 68% had an optimal response (≤1% BCR::ABL1) to ponatinib.
In a retrospective review of 184 patients with recurrent chronic CML and the T315I variant, the following was reported:[18][Level of evidence C3]
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In a retrospective review of 184 patients with recurrent chronic CML and the T315I variant, the following was reported:[18][Level of evidence C3]
Patients treated with ponatinib had a higher 4-year overall survival (OS) rate than did patients treated with allogeneic SCT (73% vs. 56%; hazard ratio [HR], 0.37; 95% confidence interval [CI], 0.16−0.84; P = .017).
In a retrospective review of 184 patients with recurrent chronic CML and the T315I variant, the following was reported:[18][Level of evidence C3]
For patients with accelerated-phase CML, survival was equivalent; however, for patients with blast crisis-phase CML, OS was worse for those who received ponatinib (HR, 2.29; 95% CI, 1.08−4.82; P = .030).
In a retrospective review, patients with a T315I variant and CML that did not respond to ponatinib had a poor prognosis, with a median survival of 16 months. The outcomes for these patients were best after allogeneic SCT, but this could have resulted from selection bias.[22][Level of evidence C3]
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A phase II trial of 282 patients was conducted to determine the lowest efficacious dose of ponatinib, because higher doses are correlated with arterial occlusive events.[20]
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A phase II trial of 282 patients was conducted to determine the lowest efficacious dose of ponatinib, because higher doses are correlated with arterial occlusive events.[20]
The optimal dose was found to be an initial 45 mg dose given once daily, then lowered to 15 mg upon achievement of a response (≤1% BCR::ABL1).[20]
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Preserved source evidence · Not medical advice