ASH 2014: Combination Therapy Shown to Be Effective for Patients With Myelodysplastic Syndrome and AML


Key Points

  • Azacitidine/lenalidomide was effective in patients with high-risk AML and myelodysplastic syndrome with up to 30% blasts.
  • The treatment was well tolerated.
  • Responses were durable and rapid, with a median of two cycles for the drugs to be effective.

A phase II study investigating the potential of the drugs azacitidine and lenalidomide (Revlimid) demonstrated that the two therapies in combination may be an effective frontline treatment regimen for patients with higher-risk forms of myelodysplastic syndrome and acute myeloid leukemia (AML). The study shed new light on effective dosage schedule and amounts for the drugs, something previously unknown. The combination therapy was well tolerated in the study of 88 patients.

The findings were presented by Courtney Dinardo, MD, of The University of Texas MD Anderson Cancer Center, at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract 164).

Myelodysplastic syndrome is a type of cancer in which the bone marrow does not make enough healthy blood cells, resulting in blast cells in the blood and/or bone marrow. Higher-risk patients experience an unusually large percentage of blasts in their blood. Patients often develop infections, anemia, or excessive bleeding. Acute myeloid leukemia is the most common acute leukemia affecting adults, with incidences increasing with age.

Study Details

“Hypomethylating agents such as azacitidine and lenalidomide are currently the front line of therapeutic choice for patients with higher-risk myelodysplastic syndrome, and also frequently employed in elderly AML patients not otherwise eligible for standard intensive therapy,” said Dr. DiNardo. “A number of combination strategies are under development to improve the results of hypomethylating agent therapy. Given what we know about the effectiveness of azacitidine and lenalidomide in patients with myelodysplastic syndrome and AML, a scientific rationale existed to explore this therapeutic combination strategy.”

Dr. DiNardo's team evaluated the administration of azacitidine and lenalidomide on days 6 to 10 of a 28-day cycle of treatment. The combination therapy appeared to be effective in patients presenting with as high as 30% blasts or abnormal blood/bone marrow cells.

“The responses were rapid with a median of two cycles for the drugs to be effective. Treatment with this dosage and schedule was well tolerated,” said Dr. DiNardo.

The study was funded by the National Institutes of Health, Celgene Corporation, the Edward P. Evans Foundation, and Fundación Ramón Areces.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.