Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2025;148(4):419-426.
doi: 10.1159/000541594. Epub 2024 Oct 2.

Belantamab Mafodotin in Relapsed/Refractory AL Amyloidosis: Real-World Multi-Center Experience and Review of the Literature

Affiliations
Review

Belantamab Mafodotin in Relapsed/Refractory AL Amyloidosis: Real-World Multi-Center Experience and Review of the Literature

Eyal Lebel et al. Acta Haematol. 2025.

Abstract

Introduction: Treatment for relapsed/refractory AL amyloidosis (AL) is an unmet need. The safety and efficacy of belantamab mafodotin (BLM) in multiple myeloma are known, whereas in AL data are limited.

Methods: We report a multi-center cohort of AL patients receiving BLM, and review all previous data on BLM therapy in AL.

Results: Twelve patients with a median of 3 (range 2-9) prior lines of therapy were included. The overall hematological response rate (ORR) was 75% (9/12), including 5 complete responses. Six of the 10 evaluable patients had organ responses. The median event-free survivals/overall survivals were 22.3 and 28.8 months, respectively. Grade 3 toxicities were mostly infections and keratopathy, occurring in 7/12 (58%). Hematological toxicities were rare. No grade 4/5 toxicities occurred. The review of the previous series reveals BLM provides an ORR of 60-83% with similar rates of corneal toxicity.

Conclusion: BLM, being an off-the-shelf therapy, with acceptable toxicity even in frail patients, may be a valuable option in AL, with a high ORR, and a signal for durable responses and high-quality organ responses.

Keywords: AL amyloidosis; Antibody drug conjugate; BCMA; Belantamab mafodotin; Real-world experience.

PubMed Disclaimer

Conflict of interest statement

Paolo Milani and Giovanni Palladini report participation in advisory boards for Alexion, Argobio, Janssen, and Protego; honoraria from Alexion, Argobio, Janssen, Protego, The Binding Site, Pfizer, Prothena, Sebia, and Siemens; and research funding from Gate Bioscience and The Binding Site. All the other authors declare no conflict of interest.

Figures

Fig. 1.
Fig. 1.
BLM in AL amyloidosis-hematological responses. NR, no response; PR, partial response; VGPR, very good partial response; CR, complete response. Event = hematologic progression or therapy change for inadequate response or death. → = Ongoing therapy at last follow-up.

References

    1. Palladini G, Merlini G. How I treat AL amyloidosis. Blood. 2022;139(19):2918–30. - PubMed
    1. Gertz MA. Immunoglobulin light chain amyloidosis: 2022 update on diagnosis, prognosis, and treatment. Am J Hematol. 2022;97(6):818–29. - PubMed
    1. Minnema MC, Nasserinejad K, Hazenberg B, Hegenbart U, Vlummens P, Ypma PF, et al. Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial. Hematologica. 2019;104(11):2274–82. - PMC - PubMed
    1. Kastritis E, Palladini G, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, et al. Daratumumab-based treatment for immunoglobulin light-chain amyloidosis. N Engl J Med. 2021;385(1):46–58. - PubMed
    1. Mahmood S, Venner CP, Sachchithanantham S, Lane T, Rannigan L, Foard D, et al. Lenalidomide and dexamethasone for systemic AL amyloidosis following prior treatment with thalidomide or bortezomib regimens. Br J Haematol. 2014;166(6):842–8. - PubMed

MeSH terms

Substances