Successful heart transplantation following melphalan plus dexamethasone therapy in systemic AL amyloidosis.
Recurrence in the allograft and progression in other organs increase mortality after cardiac transplantation in AL amyloidosis. Survival may be improved after suppression of monoclonal light chain (LC) production following high dose melphalan and autologous stem cell transplantation (HDM/ASCT). However, because of high treatment related mortality, this tandem approach is restricted to few patients without significant extra-cardiac involvement. A diagnosis of systemic AL amyloidosis was established in a 45-year old patient with congestive heart failure related to restrictive cardiomyopathy, nephrotic syndrome, peripheral neuropathy, postural hypotension, macroglossia, and lambda LC monoclonal gammopathy. After melphalan and dexamethasone (M-Dex) therapy, which resulted in 80% reduction of serum free lambda LC, he underwent orthotopic cardiac transplantation. Two years later, he remains in a sustained hematologic remission, with no evidence of allograft or extra-cardiac amyloid accumulation. M-Dex should be considered as an alternative therapy in AL amyloid heart transplant recipients ineligible for HDM/ASCT.
Mignot
Aude
A
Department of Clinical Hematology, CHU Limoges, 1 avenue Martin Luther King, 87000 Limoges, France. arnaud.jaccard@chu-limoges.fr
Bridoux
Frank
F
Thierry
Antoine
A
Varnous
Shaida
S
Pujo
Myriam
M
Delcourt
Annick
A
Gombert
Jean Marc
JM
Goujon
Jean-Michel
JM
Favreau
Fréderic
F
Touchard
Guy
G
Herpin
Daniel
D
Jaccard
Arnaud
A
D000686:Amyloidosis / Q000209:etiology / Q000601:surgery*; D003907:Dexamethasone / Q000008:administration & dosage / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D006084:Graft Rejection / Q000517:prevention & control; D006333:Heart Failure / Q000209:etiology / Q000601:surgery*; D016027:Heart Transplantation; D006801:Humans; D007145:Immunoglobulin kappa-Chains / Q000032:analysis; D007146:Immunoglobulin lambda-Chains / Q000032:analysis; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D009404:Nephrotic Syndrome / Q000209:etiology; D010265:Paraproteinemias / Q000150:complications*; D010523:Peripheral Nervous System Diseases / Q000209:etiology; D055502:Secondary Prevention
D007145:Immunoglobulin kappa-Chains; D007146:Immunoglobulin lambda-Chains; D003907:Dexamethasone; D008558:Melphalan