Evaluating the effectiveness and safety of asciminib in real-world patients with chronic myeloid leukemia previously treated with ponatinib.

This study evaluated the effectiveness and safety of asciminib (Scemblix) in real-world patients with chronic myeloid leukemia (CML) previously treated with ponatinib (Iclusig). The data showed that asciminib treatment had promising effectiveness in these patients.

CML is a type of cancer that starts in blood-forming cells of the bone marrow and enters the blood. Highly effective targeted therapies have been developed for CML as it can be challenging to treat. Tyrosine kinase inhibitor (TKI) such as ponatinib is a type of targeted therapy that works by blocking the growth of cancer cells.

Asciminib is a targeted therapy known as an ABL kinase inhibitor (AKI). It targets the BCR-ABL genes which are found in leukemia cells. It may be suitable for use in patients who have experienced treatment failure with TKIs. Asciminib has shown good effectiveness and safety profile in patients with CML who had resistance or unacceptable side effects from previous TKIs. However, in clinical trials, patients are carefully selected to be fit and without additional medical conditions. The effectiveness and safety of asciminib in real-world patients with CML previously treated with ponatinib are still known.

This study involved 52 patients with CML who received asciminib. 46 patients (88%) had received at least 3 previous TKIs. 34 patients (65%) switched to asciminib due to unacceptable side effects and 18 (35%) patients due to resistance to previous TKIs. Patients were divided into two groups. Group 1 included 20 patients who had been previously treated with ponatinib. Group 2 included 32 patients who were not previously treated with ponatinib. The average follow-up time was 30 months.

A complete cytogenic response (CCyR) was achieved or maintained in 53% of the patients in group 1 versus 74% of the patients in group 2. A CCyr is when no Philadelphia chromosomes are found in the bone marrow. The Philadelphia chromosome is a genetic abnormality found in patients with leukemia.

A major molecular response (MMR) was achieved in 32% of the patients in group 1 versus 65% of the patients in group 2. A MMR is when there is a very low level of BCR-ABL genes in the blood.

A complete molecular response (CMR) was achieved in 11% of the patients in group 1 versus 19% of the patients in group 2. A CMR is when there are no BCR-ABL genes in the blood.

Serious side effects occurred in 20% of the patients in group 1 versus 22% of the patients in group 2. The most common side effects were pancreatitis (inflammation of the pancreas) and low blood platelets (blood cells involved in clotting).

This study concluded that asciminib treatment had promising effectiveness in patients with CML previously treated with ponatinib.

The sample size was small. This study only included patients treated at institutions in Spain and looked back at medical records.