Clinical studyStem cell transplantation for the management of primary systemic amyloidosis☆
Section snippets
Methods
Amyloidosis was confirmed with a Congo red–stained tissue biopsy specimen in all patients. Patients whose disease was limited to only cutaneous involvement, purpura, or carpal tunnel syndrome were excluded. The diagnosis of light-chain amyloid required a serum or urine monoclonal light chain or the presence of clonal plasma cells in the bone marrow (5). Baseline evaluation of patients included immunofixation of serum and urine and an echocardiogram. For the purpose of counting organ
Results
We studied 66 patients who underwent transplantation between March 8, 1996, and January 17, 2001 (Table 1). All except 5 patients had evidence of an M protein in urine or serum (Table 2 ); 23 (35%) had κ light chains and 43 (65%) had λ light chains. At the time of diagnosis, signs or symptoms of amyloid were seen in the kidney in 45 patients (68%), the heart in 32 patients (48%), peripheral nerves in 11 patients (17%), the liver in 11 patients (17%), and the autonomic nervous system in 4
Discussion
Melphalan and prednisone have been used to treat amyloidosis since 1971 (10), but only a minority of patients respond, and the median survival does not exceed 2 years 11, 12. Because stem cell transplantation benefits patients with multiple myeloma (13), its use in the management of amyloidosis is logical (14). An initial report showed a clinical response in 5 patients who underwent autologous stem cell transplantation (15). With greater experience, the hematologic response rate was 62% and the
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Recent developments in the treatment of amyloidosis
2022, Drug Delivery Systems for Metabolic DisordersHematopoietic cell transplantation for light-chain amyloidosis
2019, Hematopoietic Cell Transplantation for Malignant ConditionsTreatment With Bortezomib-based Therapy, Followed by Autologous Stem Cell Transplantation, Improves Outcomes in Light Chain Amyloidosis: A Retrospective Study
2018, Clinical Lymphoma, Myeloma and LeukemiaAdverse Prognostic Factors for Morbidity and Mortality During Peripheral Blood Stem Cell Mobilization in Patients with Light Chain Amyloidosis
2018, Biology of Blood and Marrow TransplantationPrimary Amyloidosis With Renal Involvement: Outcomes in 77 Consecutive Patients at a Single Center
2017, Clinical Lymphoma, Myeloma and LeukemiaStem cell transplantation for immunoglobulin light chain amyloidosis
2017, Current Problems in Cancer
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This study was supported in part by the Mayo Hematology Malignancies Program, Rochester, Minnesota.