Posted: Friday, September 29, 2023
For patients with multiple myeloma with an extensive treatment history, combination oral therapy with the cereblon E3 ubiquitin ligase modulator mezigdomide and the steroid dexamethasone may prove to be an efficacious treatment strategy, according to a study published in The New England Journal of Medicine. However, patients did experience myelosuppression and infection attributable to this treatment regimen, explained Paul G. Richardson, MD, of the Dana-Farber Cancer Institute, Boston, and colleagues. These preliminary results are being evaluated as part of larger, ongoing phase III clinical trials.
“The responses are especially promising because the new agent, mezigdomide, is a pill that can be taken at home once daily, unlike many other immunotherapies that must be custom-made for each patient, are administered intravenously, and require sometimes lengthy hospital stays,” expressed Dr. Richardson in a Dana-Farber press release.
This study was conducted in two phases, with 77 patients enrolled in stage 1 and 101 patients enrolled in stage 2. All patients had multiple myeloma with an extensive treatment history. During phase I, patients were given a combination therapy of mezigdomide and dexamethasone to determine the safe dose to be administered in phase II. In phase II, the safety, response, and efficacy of this combination therapy were assessed.
Based on results from the phase I investigative efforts, the recommended dose of mezigdomide was identified as 1 mg combined with dexamethasone for a total of 21 consecutive days, followed by 7 days of no treatment. After treatment during phase II, 41% of patients demonstrated an overall response to therapy. Moreover, a 4.4-month median progression-free survival period was identified in these patients. As for toxicity, treatment-related adverse events included neutropenia (77%) and infection (65%).
Disclosure: For full disclosures of the study authors, visit nejm.org.