Individualized Approach to Management of Light Chain Amyloidosis

Authors:
Giovanni PalladiniDepartment of Molecular Medicine, University of Pavia, Italy
Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

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 MD, PhD
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Paolo MilaniDepartment of Molecular Medicine, University of Pavia, Italy
Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

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Systemic light chain (AL) amyloidosis is caused by a B-cell (most commonly plasma cell) clone that produces a toxic light chain that forms amyloid fibrils in tissues and causes severe, progressive organ dysfunction. The clinical presentation is protean, and patients are usually extremely frail, thus requiring careful adaptation of the treatment approach. However, the severity of organ involvement can be accurately assessed with biomarkers that allow a sharp prognostic stratification and precise tailoring of the treatment strategy. Moreover, the availability of biomarker-based response criteria also allows adjustment of the treatment approach over time. The recent completion of 3 large randomized clinical trials has offered new evidence for designing appropriate treatments. All this information has recently been integrated in the joint guidelines of the International Society of Amyloidosis and the European Hematology Association for the treatment of AL amyloidosis. Other clinical trials are underway testing new agents directed against the amyloid clone and the amyloid deposits. Our understanding of the peculiarities of the amyloid clone, as well as our ability to detect residual clonal disease and improve organ dysfunction, are also being refined and will result in more precise personalization of the treatment approach.

Submitted July 17, 2022; final revision received October 22, 2022; accepted for publication October 24, 2022.

Disclosures: Dr. Palladini has disclosed receiving honoraria from Janssen, Protego, Zentalis, Pfizer, Sebia, Siemens, and The Binding Site; and serving on the advisory board for Janssen, Protego, and Zentalis. Dr. Milani has disclosed receiving honoraria from Janssen and Pfizer, and serving on the advisory board for Janssen.

Correspondence: Giovanni Palladini, MD, PhD, Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Viale Golgi, 19, 27100 Pavia, Italy. Email: giovanni.palladini@unipv.it
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