Evidence map›Paper›PMID 42192911›Full record

ReviewCancers2026

Melflufen in Multiple Myeloma: Clinical Limitations, Biological Rationale, and Future Perspectives.

Matteo Garibotto, Debora Soncini, Roberto Massimo Lemoli, Antonia Cagnetta, Michele Cea

Abstract readReview
In one paragraph

Review in Cancers, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Matteo GaribottoClinic of Hematology, IRCCS Azienda Ospedaliera Metropolitana, Ospedale Policlinico San Martino, 16132 Genoa, Italy.ORCID 0009-0004-3477-7958
Debora SonciniClinic of Hematology, Department of Internal Medicine and Medical Specialties (DiMI), University of Genoa, 16132 Genoa, Italy.
Roberto Massimo LemoliClinic of Hematology, IRCCS Azienda Ospedaliera Metropolitana, Ospedale Policlinico San Martino, 16132 Genoa, Italy.
Antonia CagnettaClinic of Hematology, IRCCS Azienda Ospedaliera Metropolitana, Ospedale Policlinico San Martino, 16132 Genoa, Italy.
Michele CeaClinic of Hematology, IRCCS Azienda Ospedaliera Metropolitana, Ospedale Policlinico San Martino, 16132 Genoa, Italy.ORCID 0000-0002-1530-6729

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Despite transformative therapeutic advances, multiple myeloma (MM) remains an incurable malignancy characterized by sequential relapses and progressive treatment resistance. Patients with heavily pretreated relapsed or refractory MM continue to face limited therapeutic options and poor outcomes. Melflufen (melphalan flufenamide) is a peptide-drug conjugate that enhances intracellular delivery of alkylating agents via aminopeptidase-mediated activation. Early clinical studies demonstrated encouraging activity in advanced MM, leading to accelerated approval by the U.S. Food and Drug Administration in 2021. However, results from the phase III OCEAN trial raised concerns regarding overall survival, ultimately resulting in withdrawal of the drug from the U.S. market. In this review, we examine the biological rationale, clinical development, and regulatory trajectory of melflufen, and critically reassess its role within the evolving therapeutic landscape of MM. While the negative survival findings observed in the phase III OCEAN trial substantially limited the clinical development of melflufen, the biological principles underlying enzyme-targeted intracellular drug delivery may still provide relevant insights for biomarker-driven therapeutic strategies and future peptide-drug conjugate development in MM.

Indexed as

aminopeptidasesmelflufenmultiple myelomapeptide–drug conjugateprecision medicine

Identifiers

PMID42192911
PMCPMC13204213

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.