Pomalidomide for the management of refractory multiple myeloma

The pharmacology, pharmacokinetics, clinical efficacy, safety, dosage and administration, and place in therapy of pomalidomide for the management of refractory multiple myeloma are reviewed.

Pomalidomide is a second-generation immunomodulatory agent that has been approved by the Food and Drug Administration (FDA) for the management of multiple myeloma refractory to both lenalidomide and bortezomib, with or without the addition of dexamethasone. The overarching mechanism of action is thought to be antiproliferative and directly cytotoxic to malignant plasma cells in the bone marrow. Clinical trials have demonstrated both safety and efficacy with the 4-mg dose given orally on days 1-21 of a 28-day cycle with the possible addition of dexamethasone 40 mg weekly. The most common nonhematologic toxicities found in clinical trials were fatigue, pneumonia, and deep vein thrombosis. The most common hematologic toxicity was neutropenia, which was the only dose-limiting factor of pomalidomide. In order to be able to prescribe and dispense pomalidomide, physicians, patients, and pharmacies must enroll in an FDA-mandated risk evaluation and mitigation strategy program due to the drug's teratogenic effects. Future studies will evaluate the use of pomalidomide with other oncolytic agents, as well as combination regimens with proteasome inhibitors, such as bortezomib, for the management of multiple myeloma.

Pomalidomide when administered with weekly low-dose dexamethasone appears to be both safe and effective for the treatment of relapsed or refractory multiple myeloma in patients who have had disease progression after completing treatment with bortezomib, lenalidomide, or both.