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Optimizing Pomalidomide-Based Therapy for Relapsed or Refractory Multiple Myeloma

By: Julia Fiederlein Cipriano, MS
Posted: Tuesday, March 12, 2024

Asian patients with relapsed or refractory multiple myeloma who had previous exposure to a proteasome inhibitor (bortezomib, carfilzomib, or ixazomib) and lenalidomide demonstrated a significantly prolonged median duration of progression-free survival, as well as an improved overall response rate and median duration of response, after treatment with pomalidomide plus dexamethasone with vs without cyclophosphamide, according to Wee-Joo Chng, MBBS, PhD, FRCPath, FRCP, of the National University Cancer Institute, Singapore, and colleagues. The results of this multicenter, multinational phase III trial, which were presented during the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition (Abstract 1009), also seem to support a manageable safety profile for this combination.

“Pomalidomide is shown to improve survival in [patients who relapse or are refractory to lenalidomide and bortezomib],” the investigators remarked. “[Prior to this study,] the optimal drugs to combine pomalidomide with were not known.”

A total of 124 patients were randomly assigned in a 1:1 ratio to receive pomalidomide plus dexamethasone with or without cyclophosphamide. Of this population, 122 were treated with at least one dose and were thus included in the evaluable modified intention-to-treat cohort.

At a median follow-up of 13.5 months, the median duration of progression-free survival was found to be significantly prolonged with vs without cyclophosphamide (10.9 vs 5.8 months; hazard ratio [HR] = 0.43; P < .001). The overall response rate was 55.4% with cyclophosphamide vs 32.0% without it (P = .007). The median durations of response were 12.0 and 5.7 months, respectively (HR = 0.41). Patients treated with cyclophosphamide demonstrated a numerically longer median duration of overall survival (41.5 vs 27.5 months).

The adverse event rates appeared to be similar between the arms. Neutropenia, anemia, and pneumonia were the most frequently observed grade 3 and 4 treatment-emergent adverse events in both arms. The investigators reported 34 deaths (with and without cyclophosphamide: both n = 17), of which 3 were deemed treatment-related (n = 1 and 2, respectively).

Disclosure: For full disclosures of the study authors, visit

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