Pomalidomide is safe and effective for advanced multiple myeloma when combined with dexamethasone

This study examined the safety and effectiveness of pomalidomide (Pomalyst) for multiple myeloma patients who have received multiple prior therapies. Researchers reported improved treatment outcomes when pomalidomide was combined with the steroid drug dexamethasone (Decadron) compared to pomalidomide alone.

Standard treatment for multiple myeloma involves giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment with a stem cell transplant. This has significantly improved response and survival rates among patients. However, nearly all patients will relapse over time or will develop resistance to standard treatment (refractory disease). A treatment option for relapsed multiple myeloma is biologic therapy, such as pomalidomide, that uses the patient's immune system to fight cancer. Early studies have reported promising results with pomalidomide for previously treated multiple myeloma when it was combined with the steroid drug dexamethasone.

The aim of this study was to examine the safety and effectiveness of pomalidomide for relapsed or refractory multiple myeloma.

221 multiple myeloma patients were included in this study. All patients had relapsed or refractory disease and at least 2 prior therapies (average was 5). Patients were randomly assigned to receive either pomalidomide alone or in combination with dexamethasone. Treatment outcomes were examined for an average of 14.2 months.

The average time until disease progression was significantly longer with combination therapy (4.2 months) compared to pomalidomide alone (2.7 months). Overall survival (time from treatment until death from any cause) was similar between the two groups. It was 16.5 months with combination therapy and 13.6 months with pomalidomide alone.

Resistance to previous biologic therapy with lenalidomide (Revlimid) or bortezomib (Velcade) did not affect results. Outcomes with combination therapy were also not affected by age.

The most common serious side effect was low neutrophil levels (a type of white blood cell). This occurred in 41% of patients receiving combination therapy and in 48% of patients receiving pomalidomide alone. Low hemoglobin levels (a protein in red blood cells carrying oxygen to the body) and low platelet levels (cells involved in clotting) were similar in both groups and occurred in between 19 to 24% of patients. Common side effects not related to blood results included lung infection, fatigue, difficulty breathing, and back pain. These were also similar between groups, occurring in 8 to 22% of patients. 

Researchers concluded that combining pomalidomide with dexamethasone is a safe and effective treatment option for patients who have received multiple prior therapies.