The study, authored by researchers at the Dana-Farber Cancer Institute in Boston, Massachusetts, USA found that melflufen demonstrated clinically meaningful efficacy, with an overall response rate (ORR) of 55%, including complete responses in 27% of patients. The safety profile was consistent with previous clinical trials, with manageable hematologic toxicities. The findings indicate that melflufen is an effective treatment option for patients with heavily pretreated RRMM in the real-world setting.

The article, titled “Outcomes of Melflufen Treatment in Patients with Relapsed/Refractory Multiple Myeloma,” evaluates real-world data from patients treated at the Dana-Farber Cancer Institute. The study provides additional clinical insights into the efficacy and safety of melflufen outside of the controlled clinical trial setting, reinforcing its potential role in the treatment of RRMM.

“Our real-world experience has strengthened our understanding of melflufen’s important role in treating relapsed and refractory multiple myeloma, as reflected by our data,” says Paul G. Richardson, MD, Clinical Program Leader and Director of Clinical Research at Dana-Farber Cancer Institute and senior author of the article. “The results indicate that melflufen’s efficacy and safety profile observed in clinical trials have favorably translated into real-world practice. In my view, this offers hope to our patients who may have limited remaining treatment options, and in particular need therapeutic strategies that circumvent immune exhaustion with a novel mechanism of action, as well as having the advantage of outpatient, “off the shelf” administration and low rates of infection.” 

The full article can be accessed here. 

About the Study
The retrospective study analyzed 12 patients with RRMM treated with melflufen plus dexamethasone at the Dana-Farber Cancer Institute. The cohort had a median of 5.5 prior lines of therapy, with a significant proportion exhibiting high-risk disease characteristics. Key findings include:

• 55% ORR, with 27% achieving complete response (CR)
• 21.3-week median duration of response
• Manageable safety profile, with primarily hematologic adverse events
• No new safety signals compared to clinical trial data

Reasons for treatment discontinuation included disease progression (42%), drug withdrawal from the US market (33%), adverse events (17%), and one unrelated sudden death (8%).
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