Real-world efficacy and safety of mezigdomide-dexamethasone in heavily pre-treatred multiple myeloma patients: An Italian case series
Abstract
Introduction Heavily pre-treated multiple myeloma (MM) patients have very poor outcomes and limited survival. Mezigdomide, a novel oral cereblon E3 ligase modulator (CELMoD), has demonstrated promising efficacy and safety in combination with dexamethasone (Mez-D) for relapsed/refractory MM (RRMM), as documented in the phase I–II trial (NCT03374085). Due to its recent approval, especially in Europe, real-world evidence remains limited. In this retrospective, observational, multicenter, real-world Italian case series, we collected clinical data on RRMM patients treated with Mez-D outside clinical trials, to assess efficacy and safety of this novel combination.
Methods A total of 10 RRMM patients treated with Mez-D through expanded access programs (EAP) outside clinical trials were enrolled from August 2024 to July 2025 across seven Italian Hematology Units. High-risk genetic abnormalities and prior treatment exposure/refractoriness were defined according to the International Myeloma Working Group (IMWG) criteria.
Results The median age was 71 years (range 61–85), and 30% were males. Half of the patients had an ECOG performance status of 0–1, while the remaining ≥2. The M-protein subtype was IgG in 40% of cases, IgA in 30%, light chain only in 20%, and biclonal IgG-IgA in 10% of subjects. Kappa light chain was more frequent (60%) compared to lambda (40%).
High genetic risk was observed in 40% of patients, and 30% met the definition of high-risk based on updated criteria…
How strong is the evidence?
Evidence Hub reads this as a case series, done in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health. Reported size: 10 participants or records.
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