Site Editors

Shaji K. Kumar, MD

Prashant Kapoor, MD, FACP


OCEAN and HORIZON Trials: More Benefit Than Risk With Melflufen Plus Dexamethasone in Myeloma?

By: Julia Fiederlein Cipriano, MS
Posted: Wednesday, November 29, 2023

The primary results of a phase III trial revealed significantly longer progression-free survival—but not overall survival—among patients with relapsed or refractory multiple myeloma who received dexamethasone plus the alkylating peptide drug-conjugate melphalan flufenamide (melflufen) vs pomalidomide. Pieter Sonneveld, MD, PhD, of Erasmus MC Cancer Institute, Rotterdam, the Netherlands, and colleagues thus conducted a post-hoc analysis of both the pivotal phase II HORIZON and aforementioned confirmatory OCEAN study to further assess the benefit-risk profile of this combination. Their findings, which were published in the journal Clinical Lymphoma, Myeloma & Leukemia, may also offer a perspective on the U.S. Food and Drug Administration’s safety alert for melflufen.

“[Our findings] demonstrate a consistent benefit for melflufen and dexamethasone in patients…who have not received an autologous stem cell transplantation [ASCT] or [whose disease] progressed more than 36 months after receiving an ASCT,” the investigators commented.

The OCEAN study identified age (P = .011; < 65 favoring pomalidomide) and having no prior ASCT or a remission duration of more than 36 months after the procedure (P = .001; favored melflufen) as prognostic factors for overall survival. A total of 49% of the randomly assigned patients underwent prior ASCT; of this population, 82% experienced disease progression within 36 months. After excluding the patients treated with melflufen (n = 145) and pomalidomide (n = 148) who experienced disease progression within 36 months of undergoing ASCT, the median durations of overall survival were 23.6 and 19.8 months, respectively (hazard ratio = 0.83; P = .22).

Among patients from the HORIZON study with triple-class–refractory disease, those who experienced disease progression within 36 months of undergoing ASCT (n = 58) were found to have a lower response rate, as well as shorter durations of response and progression-free survival, than the remainder of the population (n = 52). The safety profile of melflufen plus dexamethasone appeared to be congruent with previous reports.

Disclosure: For full disclosures of the study authors, visit

By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.