In a nutshell
This study aimed to investigate if asciminib was effective in patients with chronic myeloid leukemia who had resistance or unacceptable side effects to other tyrosine kinase inhibitors. This study concluded that asciminib showed promise in these patients.
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 other leukemia treatments such as tyrosine kinase inhibitors (TKIs).
The safety and effectiveness of asciminib in patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) are unknown.
Methods & findings
This study involved 141 patients with chronic-phase and 9 patients with accelerated-phase CML. All patients involved had resistance or unacceptable side effects from at least 2 previous TKIs. 70% of patients involved had received at least three TKIs previous to this study. All patients received asciminib either once or twice daily. Patients were followed for an average of 59 weeks.
In patients with chronic-phase CML, 92% of those without a hematologic response after previous therapies achieved a complete hematologic response (CHR). A CHR is when there are no signs of CML and blood counts and spleen size have returned to normal.
In patients with chronic-phase CML, 54% of those without a complete cytogenic response at the beginning (after previous therapies) achieved a complete cytogenic response (CCyR). A CCyr is when no Philadelphia chromosomes are found in the bone marrow.
At 12 months, 48% of 91 patients evaluated achieved a major molecular response (MMR). A MMR is when there is a very low level of BCR ABL genes in the blood. MMR was maintained in 40 of 44 patients.
Among the 9 patients with accelerated-phase CML, 8 patients (88%) had a CHR and 1 (11%) had a MMR. These responses were maintained for at least 11 weeks.
Side effects that let patients to reduce the dose or stop treatment included pancreatitis. Common side effects included tiredness, headache, joint pain, high blood pressure, and low blood platelets.
The bottom line
This study concluded that asciminib was active in patients with CML who had resistance or unacceptable side effects from other TKIs.
The fine print
This study was funded by Novartis, the manufacturer of asciminib.
Published By :
The New England Journal of Medicine
Dec 12, 2019