title: Treatment of primary systemic amyloidosis (AL): role of intensive and standard therapy.
abstract: Immunoglobulin light-chain (AL) amyloidosis is a clonal plasma cell dyscrasia. Delay in diagnosis is the major hurdle in improving the outcomes of AL patients. Almost all patients with systemic AL need cytotoxic therapy. Treatment can improve symptoms and quality of life, as well as extend survival. Supportive care is an integral part of the treatment plan. Severity of cardiac involvement is an important determinant of prognosis and influences the choice of therapy. Cardiac biomarkers and serum free light chain assay are important tools for assessing prognosis and monitoring treatment response. Myeloablative chemotherapy with melphalan and autologous stem cell rescue appears to offer survival benefit; however, it is an option for only a quarter of AL patients. Standard-dose combination chemotherapy with steroids and alkylating agents is a safe and effective treatment strategy that can result in improvement of organ function in many patients. Newer agents such as bortezomib and lenalidomide have shown promising activity and are being evaluated as part of combination regimens in clinical trials.
authors.affiliation: Division of Hematology and Medical Oncology, Mayo Clinic, Jacksonville, Florida 32246, USA. sher.taimur@mayo.edu
authors.forename: Taimur
authors.initials: T
authors.lastname: Sher
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
mesh_terms: D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D015415:Biomarkers / Q000032:analysis; D001897:Boronic Acids / Q000627:therapeutic use; D000069286:Bortezomib; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000032:analysis; D000075363:Immunoglobulin Light-chain Amyloidosis; D000077269:Lenalidomide; D008558:Melphalan / Q000627:therapeutic use; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D009206:Myocardium / Q000473:pathology; D011719:Pyrazines / Q000627:therapeutic use; D013256:Steroids / Q000627:therapeutic use; D016019:Survival Analysis; D013792:Thalidomide / Q000031:analogs & derivatives / Q000627:therapeutic use; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers; D001897:Boronic Acids; D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D011719:Pyrazines; D013256:Steroids; D013792:Thalidomide; D000069286:Bortezomib; D000077269:Lenalidomide; D008558:Melphalan