Through the Registries of the Italian Medicines Agency (AIFA), real-world data on routine practice management, treatment adjustments, and outcome of a sizable cohort of chronic myeloid leukemia (CML) patients treated with ponatinib were gathered.

From February 2015 to December 2020, 666 CML patients were registered in the ponatinib registry and qualified for analysis: 515 in the chronic phase (CP), 50 in the accelerated phase (AP), and 101 in blast crises (BC). Male individuals made up the majority (57.1%), with a median age of 58.7 years at baseline. The median amount of time from diagnosis to ponatinib start was 2.35 years; 259 (38.9%) participants had had two or more prior lines of therapy, 260 (39.0%) had gotten three or more, and 147 (22.1%) had received four or more.

Out of the 593 individuals analyzed, 59% of patients had a molecular response, defined as a significant molecular reaction (MMR), to a score of less or around 0.01% on the international reporting scale (IS). With a median follow-up of 14.4 months, 136 individuals (20.4%) needed at least one dosage reduction because of adverse events (AEs), whereas 309 patients (46.4%) needed dose reductions even though there was no sign of side effects. About 261 individuals (39%) stopped receiving treatment.

The real-life investigation demonstrated that dosage reductions were mainly made out of prudence rather than because adverse responses occurred.

Reference: onlinelibrary.wiley.com/doi/10.1111/bjh.18359