Systemic light chain amyloidosis myopathy responsive to daratumumab monotherapy

• Published in: European journal of neurology Vol. 30; no. 3; pp. 745 - 748
• Main Authors: Chitimus, Diana Maria, Berling, Edouard, Garderet, Laurent, Venturelli, Nadia, Malfatti, Edoardo, Authier, François Jérôme, Nicolas, Guillaume, Laforêt, Pascal, Lefeuvre, Claire
• Format: Journal Article
• Language: English
• Published: England John Wiley & Sons, Inc 01.03.2023; Wiley; John Wiley and Sons Inc
• Subjects: Aged; amyloid myopathy; Amyloidosis; Amyloidosis - complications; Amyloidosis - diagnosis; Amyloidosis - drug therapy; Biopsy; Bortezomib; Bortezomib - therapeutic use; Case reports; Chains; daratumumab; Diagnosis; Hematology; Humans; Immunoglobulin A; Immunoglobulin Light-chain Amyloidosis - complications; Immunoglobulin Light-chain Amyloidosis - diagnosis; Immunoglobulin Light-chain Amyloidosis - drug therapy; Immunotherapy; Life Sciences; light chain amyloidosis; Male; Monoclonal antibodies; Monoclonal gammopathy; Muscle and NMJ disorders; Muscle Weakness; Muscles; Muscular Diseases - complications; Muscular Diseases - diagnosis; Muscular Diseases - drug therapy; Myopathy; Neurons and Cognition; Patients; Short Communication; systemic AL amyloidosis; light chain amyloidosis; amyloid myopathy; daratumumab; systemic AL amyloidosis
• ISSN: 1351-5101; 1468-1331
• DOI: 10.1111/ene.15640

Background and purpose Amyloid myopathy is a rare and severe manifestation of systemic light chain (AL) amyloidosis. Early diagnosis and staging are mandatory for optimal therapy, given the rapid progression of muscle weakness. Despite the efficacy of bortezomib‐based treatment regimens, there is a lack of therapeutic alternatives in non‐responsive patients. Method The case report of a patient with systemic AL amyloidosis myopathy treated with daratumumab is presented. Results A 70‐year‐old man displayed severe proximal muscle weakness which had developed over a 10‐month period. Blood tests revealed an immunoglobulin A lambda monoclonal gammopathy, whilst muscle biopsy showed amyloid deposits within the arteriolar walls, confirming the diagnosis of amyloid myopathy associated with AL amyloidosis. Initial treatment with a bortezomib‐based regimen showed no clinical or hematological improvement. After switching to daratumumab monotherapy, our patient achieved a favorable evolution with respect to functional muscle scoring and a complete hematological response. Conclusion To our knowledge, this is the first case report of an amyloid myopathy showing a remarkable clinical improvement in response to daratumumab monotherapy. It thereby highlights the potential of daratumumab as a monotherapeutical approach to the treatment of amyloid myopathy complicating AL amyloidosis.