Blood stem cell transplantation as therapy for primary systemic amyloidosis (AL)

This study investigated the response rate and toxicity of blood cell transp lantation as treatment for primary amyloidosis (AL), Twenty-three patients had stem cells collected between November 1995 and September 1998, Conditio ning included melphalan and total body irradiation in 16 and melphalan alon e in 4, Three patients did not undergo stem cell infusion because of poor p erformance status. Two died of progressive amyloid at 1 and 3 months. One p atient is alive on hemodialysis, Fourteen males and six females (median age , 57 years) underwent transplantation. Renal, cardiac (by echocardiography) , peripheral neuropathy or liver amyloidosis occurred in 14, 12, 3, and 1, respectively. Echocardiography demonstrated an interventricular septal thic kness greater than or equal to 15 mm in six patients, five of whom died pos t transplantation. Three patients died of progressive amyloidosis at 7, 7, and 21 months. Thirteen patients are alive with a follow-up of 3 to 26 mont hs. Twelve (60%) fulfilled the criteria of a hematologic or organ response. Severe gastrointestinal tract toxicity was seen in five (25%), We conclude that blood cell transplantation for amyloidosis had a much higher morbidit y and mortality compared with transplantation for myeloma, The best results appear to occur in patients with nephrotic syndrome as the only manifestat ion of their disease.