By Cecilia Brown - Last Updated: March 23, 2023
A new study suggests mutations in NPM1 have a “minimal impact” on prognosis in patients with relapsed or refractory acute myeloid leukemia (AML).
Ghayas C. Issa, MD, of the MD Anderson Cancer Center, and colleagues conducted the research and published their findings in Blood Advances.
It was important to conduct the study because mutations in NPM1 are “associated with a favorable prognosis” in patients with newly diagnosed AML, but their “prognostic impact in relapsed/refractory settings are unknown,” Dr. Issa and colleagues wrote.
They conducted a retrospective analysis of 1,722 patients with AML. Of those patients, 12% (206) had mutated NPM1, while the remaining 88% (1,516) of patients had wild-type NPM1.
After each line of therapy, mutated NPM1 was associated with a higher rate of complete remission (CR) or CR with incomplete count recovery than wild-type NPM1.
Following the first line of salvage therapy, 56% of patients with mutated NPM1 had a CR or CR with incomplete count recovery, while 37% of those with wild-type NPM1 had a CR or CR with incomplete count recovery (P<.0001). After the second line of salvage therapy, 33% of patients with mutated NPM1 had a CR or CR with incomplete count recovery, while 22% of those with wild-type NPM1 had a CR or CR with incomplete count recovery (P=.02). Following the third line of salvage therapy, 24% of patients with mutated NPM1 had a CR or CR with incomplete count recovery, while 14% of those with wild-type NPM1 had a CR or CR with incomplete count recovery (P=.02).
While NPM1 mutations impacted CR rates, they had “no impact” on relapse-free survival (RFS) and overall survival (OS) after each line of salvage therapy, Dr. Issa and colleagues wrote.
After one salvage therapy, the median OS was 7.8 months in patients with mutated NPM1, while it was six months in those with wild-type NPM1. Following two salvage therapies, the median OS was 5.3 months in patients with mutated NPM1, while it was 4.1 months in those with wild-type NPM1. After a third salvage therapy, the median OS was 5.3 months in patients with mutated NPM1, while it was 4.1 months in those with wild-type NPM1.
However, when venetoclax was added to salvage regimens, patients with mutated NPM1 had a significantly longer median RFS (15.8 vs 4.6 months; P=.05) and OS (14.7 vs 5.9 months; P=.02) than those with wild-type NPM1.
“In conclusion, NPM1 mutational status has a minimal impact on prognosis in relapsed or refractory AML; therefore, novel treatment strategies are required to improve outcomes in this entity,” Dr. Issa and colleagues concluded.
Reference
Issa GC, Bidikian A, Venugopal S, et al. Clinical outcomes associated with NPM1 mutations in patients with relapsed or refractory AML. Blood Adv. 2023;7(6):933-942. doi:10.1182/bloodadvances.2022008316
Original Source: NPM1 Mutational Status has ‘Minimal Impact’ on Prognosis in Relapsed, Refractory AML | Blood Cancers Today