The second-generation immunomodulatory drug pomalidomide (Pomalyst) “has shown impressive results in patients with multiple myeloma who are refractory to lenalidomide [Revlimid] and bortezomib [Velcade],” according to a review of clinical data leading to the drug’s approval by the FDA. In February, the FDA approved pomalidomide for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and bortezomib and have demonstrated disease progression on or within 60 days of completion of the last therapy.
“Pomalidomide is well tolerated, with minimal toxicity, features important for a drug that is targeted for heavily treated, multiple relapsed patients,” reported the authors of the review, published online by Blood.
The review also provided advice for physicians who are prescribing the drug for patients, including information about dosing, toxicities, and special populations. “Available data suggests that efficacy and toxicity are similar at the 2 mg and 4 mg dose levels,” the authors noted.
Toxicity Data
“Fatigue is the most commonly reported adverse effect, with 62% of patients experiencing any fatigue and 8% experiencing fatigue of grade 3 or higher,” the authors noted. Grade 3/4 neutropenia occurred in 26% to 66% of patients. Less common hematologic toxicities were anemia, with grade 3/4 in 17% of patients, and thrombocytopenia, with grade 3/4 in 13% of patients.
The incidence of thromboembolic events in the studies reviewed was low, 2% to 3%, and similar to patients treated with other immunomodulatory drugs. “Prophylactic treatment with aspirin is a reasonable strategy to prevent thromboembolic complications in these patients and has been successfully used in pomalidomide clinical trials to date,” the authors stated.
A large multicenter randomized phase III trial “established that pomalidomide “is safe and well tolerated in myeloma patients with moderate renal failure (clearance < 60 mL/min), but no data is available for patients with severe renal failure,” the authors wrote. A trial currently accruing patients is expected to address this issue. Data are not available regarding the use of pomalidomide in pediatric patients, or dosing, safety, or efficacy in patients with liver failure.
Ongoing studies are testing pomalidomide in combination with other drugs in patients with relapsed or refractory multiple myeloma. These studies include a phase I/II trial of pomalidomide, dexamethasone, and pegylated liposomal doxorubicin (Doxil), and a phase III trial comparing bortezomib and dexamethasone with and without pomalidomide. ■
Lacy MQ, McCurdy AR: Blood. August 23, 2013 (early release online).
