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      AL Amyloidosis with Renal Involvement

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          Abstract

          Primary (AL amyloidosis) is a systemic disease characterized by an amyloid deposition process in many organs, with unsatisfactory survival of patients. The monoclonal light chains form the fibrils that deposit and accumulate in tissues. Renal involvement is very frequent in AL amyloidosis and could lead to development of nephrotic syndrome followed by the renal failure in many cases. Classic therapeutic combination melphalan and prednisone has been supplemented with drugs with different mechanisms of action in this group of patients: high-dose dexamethasone, high-dose dexamethasone with melphalan, combination of vincristine, doxorubicin, and dexamethasone or newly high-dose melphalan supported by peripheral blood stem cell transplantation. This progressive therapy leads to the better survival and prognosis in the majority of patients. Alternative therapeutic approaches include thalidomide (alone or in combination with cyclophosphamide), lenalidomide, iododoxorubicin, etanercept and rituximab. The development of immunotherapy is expected in the near future.

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          Most cited references 7

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          Association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis who are ineligible for stem cell transplantation.

          The most efficient therapeutic approach for immunoglobulin light chain amyloidosis (AL) is autologous stem cell transplantation (ASCT); however, the toxicity of ASCT limits its feasibility to a minority of patients. Patients ineligible for ASCT are usually treated with standard oral melphalan and prednisone, but the response rate to this regimen is unsatisfactory, and time to response is long. High-dose dexamethasone provides a rapid response time in patients with AL. We evaluated the combination of oral melphalan and high-dose dexamethasone (M-Dex) in 46 patients with AL ineligible for ASCT. Thirty-one (67%) achieved a hematologic response and 15 (33%) a complete remission. In 22 (48%) of the responsive patients functional improvement of the organs involved was observed. Five patients (11%) experienced severe adverse events, 3 required hospitalization, and no treatment-related deaths were observed. M-Dex represents a feasible and effective therapeutic option for patients with advanced AL who are ineligible for ASCT.
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            Treatment of amyloidosis.

            Amyloidosis is the extracellular deposition of normally soluble autologous protein in a characteristic abnormal fibrillar form. Systemic amyloidosis and some local forms are progressive, cause major morbidity, and are often fatal. No treatment specifically causes the resolution of amyloid deposits, but therapy that reduces the supply of amyloid fibril precursor proteins can improve survival and preserve organ function. Major regression of amyloid occurs in at least a proportion of such cases, suggesting that the clinical improvement reflects mobilization of amyloid. The clearest evidence for regression of amyloid has been obtained in juvenile rheumatoid arthritis patients with AA amyloidosis treated with chlorambucil. This drug suppresses the acute phase production of serum amyloid A protein, the precursor of AA amyloid fibrils, and is associated with remission of proteinuria and greatly improved survival. In many such patients, scintigraphy with serum amyloid P component shows major regression of amyloid over 12 to 36 months and frequently reveals a discrepancy between the local amyloid load and organ dysfunction. Measurement of target organ function is therefore not an adequate method for monitoring treatment aimed at promoting the resolution of amyloid. In monoclonal immunoglobulin light chain (AL) amyloidosis the aim of treatment is to suppress the underlying B-cell clone and, therefore, production of the amyloid fibril precursor protein. This can be difficult to achieve or sustain and, since the prognosis is so poor, many patients die before benefits of therapy are realized. A recent development has been the introduction of liver transplantation as treatment for familial amyloid polyneuropathy caused by transthyretin gene mutations. This leads to the disappearance of variant transthyretin from the plasma and halts progression of the neurologic disease. Features of autonomic neuropathy frequently ameliorate, and improvement in peripheral motor nerve function has been recently reported. Serum amyloid P component scans show regression of associated visceral amyloidosis. This surgical form of gene therapy holds much promise for patients with familial amyloid polyneuropathy and has been widely adopted. The only other form of amyloidosis in which the supply of the fibril precursor protein can be sharply reduced is beta 2M amyloidosis in long-term hemodialysis patients. Renal transplantation lowers the plasma concentration of beta 2M to normal levels and is associated with rapid improvement of the osteoarticular symptoms. Preliminary observations suggest that the beta 2M amyloid deposits also can regress in some patients.(ABSTRACT TRUNCATED AT 400 WORDS)
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              Serum amyloid P component scintigraphy for diagnosis and monitoring amyloidosis

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                Author and article information

                Journal
                KBR
                Kidney Blood Press Res
                10.1159/issn.1420-4096
                Kidney and Blood Pressure Research
                S. Karger AG
                1420-4096
                1423-0143
                2007
                November 2007
                11 September 2007
                : 30
                : 6
                : 359-364
                Affiliations
                Nephrology Clinic, 1st Faculty of Medicine, Charles University, Prague, Czech Republic
                Article
                107980 Kidney Blood Press Res 2007;30:359–364
                10.1159/000107980
                17851224
                © 2007 S. Karger AG, Basel

                Copyright: All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means, electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher. Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. Disclaimer: The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publishers and the editor(s). The appearance of advertisements or/and product references in the publication is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.

                Page count
                Figures: 3, Tables: 1, References: 20, Pages: 6
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