Phase II Study of Bortezomib as a Single Agent in Patients with Previously Untreated or RelapsedRefractory Acute Myeloid Leukemia Ineligible for Intensive Therapy

Abstract EN

We explored the safety and efficacy of bortezomib given as single agent in patients with untreated or relapsed/refractory acute myeloid leukemia (AML), unfit for conventional chemotherapy.

Fourteen patients were treated with bortezomib 1.5 mg/m2 administered twice weekly for two weeks, every 3 weeks.

Median age was 70 years (range 60–81) and the median number of cycles delivered was 2 (range 1–4).

Of 13 evaluable patients, in 8 (61%), the administration of bortezomib resulted in an antileukemic effect as demonstrated by peripheral blood and/or bone marrow blast reduction.

In 4 (50%) of these 8, a decrease by 37% of transfusion requirement was also observed (P = 0.009).

Overall median survival was 4 months (range 0.25–10).

Neurotoxicity was the most frequent adverse event with 7 of 13 (54%) patients experiencing grades 3-4 peripheral neuropathy.

Neurotoxicity led to treatment discontinuation in 4 (57%) of 7.

In conclusion, the observed anti-leukemic activity of bortezomib indicates that there is room for designing additional studies in which combination with other chemotherapeutic agents should be considered.

Clinical registration no.: EUDRACT 2006-006923-38.