Key result
Ponatinib fails to prevent disease progression and death in pulmonary tumor thrombotic microangiopathy.
Why the study?
Does oral ponatinib improve outcomes in adult patients with pulmonary tumor thrombotic microangiopathy?
Does oral ponatinib improve outcomes in adult patients with pulmonary tumor thrombotic microangiopathy?
In a small prospective study of 3 patients with pulmonary tumor thrombotic microangiopathy, ponatinib failed to demonstrate efficacy as all patients rapidly died from disease progression.
Answers are grounded in this paper and cite their sources.
No support for ponatinib in pulmonary tumor thrombotic microangiopathy; leaves open effective therapies for this rare condition.
Background: We evaluated the safety and efficacy of ponatinib, a multi-target tyrosine kinase inhibitor, for pulmonary tumor thrombotic microangiopathy (PTTM).
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Sato et al. (2026) studied Pulmonary tumor thrombotic microangiopathy (PTTM) (n=3). Ponatinib was evaluated on Composite of all-cause death and severe vascular occlusive events. All 3 patients with pulmonary tumor thrombotic microangiopathy treated with ponatinib died from disease progression, failing to demonstrate the drug's efficacy.
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