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Light chain amyloidosis 2012: a new era
Summary AL amyloidosis patients with multi‐organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high‐risk patients, but responding patients have prol...
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Published in: | British journal of haematology 2013-03, Vol.160 (5), p.582-598 |
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container_title | British journal of haematology |
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creator | Gatt, Moshe E. Palladini, Giovanni |
description | Summary
AL amyloidosis patients with multi‐organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high‐risk patients, but responding patients have prolonged overall survival. Toxicities can be decreased by careful patient selection, but this reduces the applicability of this treatment modality to a limited number of patients. Efforts are therefore needed to design novel more effective regimens, with the use of new medications, such as thalidomide, lenalidomide and bortezomib, next generation immunomodulatory drugs and proteasome inhibitors. Their combination with dexamethasone and alkylating agents show promising results, allowing a high percentage of remission and subsequent event‐free and overall survival, even in a significant proportion of high risk, poor prognosis populations. This review includes the state‐of‐the‐art treatment for AL amyloidosis patients as of 2012, in light of the progress in management of this disease during recent years. |
doi_str_mv | 10.1111/bjh.12191 |
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AL amyloidosis patients with multi‐organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high‐risk patients, but responding patients have prolonged overall survival. Toxicities can be decreased by careful patient selection, but this reduces the applicability of this treatment modality to a limited number of patients. Efforts are therefore needed to design novel more effective regimens, with the use of new medications, such as thalidomide, lenalidomide and bortezomib, next generation immunomodulatory drugs and proteasome inhibitors. Their combination with dexamethasone and alkylating agents show promising results, allowing a high percentage of remission and subsequent event‐free and overall survival, even in a significant proportion of high risk, poor prognosis populations. This review includes the state‐of‐the‐art treatment for AL amyloidosis patients as of 2012, in light of the progress in management of this disease during recent years.</description><identifier>ISSN: 0007-1048</identifier><identifier>EISSN: 1365-2141</identifier><identifier>DOI: 10.1111/bjh.12191</identifier><identifier>PMID: 23294331</identifier><identifier>CODEN: BJHEAL</identifier><language>eng</language><publisher>Oxford: Blackwell</publisher><subject>Alkylating Agents - therapeutic use ; Amyloid - metabolism ; amyloidosis ; Amyloidosis - complications ; Amyloidosis - drug therapy ; Amyloidosis - metabolism ; Amyloidosis - pathology ; Amyloidosis - surgery ; Biological and medical sciences ; Biomarkers ; Boronic Acids - therapeutic use ; Bortezomib ; Cardiomyopathies - blood ; Cardiomyopathies - etiology ; Cardiomyopathies - pathology ; Clinical Trials as Topic ; Combined Modality Therapy ; Dexamethasone - therapeutic use ; Forecasting ; Hematologic and hematopoietic diseases ; Humans ; Immunoglobulin Light Chains - metabolism ; Immunologic Factors - therapeutic use ; Medical sciences ; Natriuretic Peptide, Brain - blood ; novel agents ; Peptide Fragments - blood ; Prognosis ; Pyrazines - therapeutic use ; Risk ; Severity of Illness Index ; Stem Cell Transplantation ; therapy ; Troponin C - blood ; Troponin T - blood ; Tumors</subject><ispartof>British journal of haematology, 2013-03, Vol.160 (5), p.582-598</ispartof><rights>2013 Blackwell Publishing Ltd</rights><rights>2014 INIST-CNRS</rights><rights>2013 Blackwell Publishing Ltd.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4891-351bd4fc7e36df19c240f054ad64a81eb5d852a6fa9ff133cc59a6f15ae74d243</citedby><cites>FETCH-LOGICAL-c4891-351bd4fc7e36df19c240f054ad64a81eb5d852a6fa9ff133cc59a6f15ae74d243</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=27099916$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/23294331$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Gatt, Moshe E.</creatorcontrib><creatorcontrib>Palladini, Giovanni</creatorcontrib><title>Light chain amyloidosis 2012: a new era</title><title>British journal of haematology</title><addtitle>Br J Haematol</addtitle><description>Summary
AL amyloidosis patients with multi‐organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high‐risk patients, but responding patients have prolonged overall survival. Toxicities can be decreased by careful patient selection, but this reduces the applicability of this treatment modality to a limited number of patients. Efforts are therefore needed to design novel more effective regimens, with the use of new medications, such as thalidomide, lenalidomide and bortezomib, next generation immunomodulatory drugs and proteasome inhibitors. Their combination with dexamethasone and alkylating agents show promising results, allowing a high percentage of remission and subsequent event‐free and overall survival, even in a significant proportion of high risk, poor prognosis populations. This review includes the state‐of‐the‐art treatment for AL amyloidosis patients as of 2012, in light of the progress in management of this disease during recent years.</description><subject>Alkylating Agents - therapeutic use</subject><subject>Amyloid - metabolism</subject><subject>amyloidosis</subject><subject>Amyloidosis - complications</subject><subject>Amyloidosis - drug therapy</subject><subject>Amyloidosis - metabolism</subject><subject>Amyloidosis - pathology</subject><subject>Amyloidosis - surgery</subject><subject>Biological and medical sciences</subject><subject>Biomarkers</subject><subject>Boronic Acids - therapeutic use</subject><subject>Bortezomib</subject><subject>Cardiomyopathies - blood</subject><subject>Cardiomyopathies - etiology</subject><subject>Cardiomyopathies - pathology</subject><subject>Clinical Trials as Topic</subject><subject>Combined Modality Therapy</subject><subject>Dexamethasone - therapeutic use</subject><subject>Forecasting</subject><subject>Hematologic and hematopoietic diseases</subject><subject>Humans</subject><subject>Immunoglobulin Light Chains - metabolism</subject><subject>Immunologic Factors - therapeutic use</subject><subject>Medical sciences</subject><subject>Natriuretic Peptide, Brain - blood</subject><subject>novel agents</subject><subject>Peptide Fragments - blood</subject><subject>Prognosis</subject><subject>Pyrazines - therapeutic use</subject><subject>Risk</subject><subject>Severity of Illness Index</subject><subject>Stem Cell Transplantation</subject><subject>therapy</subject><subject>Troponin C - blood</subject><subject>Troponin T - blood</subject><subject>Tumors</subject><issn>0007-1048</issn><issn>1365-2141</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2013</creationdate><recordtype>article</recordtype><recordid>eNp10E1Lw0AQBuBFFFurB_-A5CLqIe3OfiRZb1rUKgUvel4mm127JWlqtqX035uaqifnMgw8zAsvIedAh9DOKJ_PhsBAwQHpA09kzEDAIelTStMYqMh65CSEOaXAqYRj0mOcKcE59MnV1H_MVpGZoV9EWG3L2hd18CFiFNhthNHCbiLb4Ck5clgGe7bfA_L--PA2nsTT16fn8d00NiJTEHMJeSGcSS1PCgfKMEEdlQKLRGAGNpdFJhkmDpVzwLkxUrUXSLSpKJjgA3Ld_V029efahpWufDC2LHFh63XQkEouZJJkSUtvOmqaOoTGOr1sfIXNVgPVu15024v-7qW1F_u367yyxa_8KaIFl3uAwWDpGlwYH_5cSpVSsAsddW7jS7v9P1Hfv0y66C921HX2</recordid><startdate>201303</startdate><enddate>201303</enddate><creator>Gatt, Moshe E.</creator><creator>Palladini, Giovanni</creator><general>Blackwell</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>H94</scope></search><sort><creationdate>201303</creationdate><title>Light chain amyloidosis 2012: a new era</title><author>Gatt, Moshe E. ; Palladini, Giovanni</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4891-351bd4fc7e36df19c240f054ad64a81eb5d852a6fa9ff133cc59a6f15ae74d243</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2013</creationdate><topic>Alkylating Agents - therapeutic use</topic><topic>Amyloid - metabolism</topic><topic>amyloidosis</topic><topic>Amyloidosis - complications</topic><topic>Amyloidosis - drug therapy</topic><topic>Amyloidosis - metabolism</topic><topic>Amyloidosis - pathology</topic><topic>Amyloidosis - surgery</topic><topic>Biological and medical sciences</topic><topic>Biomarkers</topic><topic>Boronic Acids - therapeutic use</topic><topic>Bortezomib</topic><topic>Cardiomyopathies - blood</topic><topic>Cardiomyopathies - etiology</topic><topic>Cardiomyopathies - pathology</topic><topic>Clinical Trials as Topic</topic><topic>Combined Modality Therapy</topic><topic>Dexamethasone - therapeutic use</topic><topic>Forecasting</topic><topic>Hematologic and hematopoietic diseases</topic><topic>Humans</topic><topic>Immunoglobulin Light Chains - metabolism</topic><topic>Immunologic Factors - therapeutic use</topic><topic>Medical sciences</topic><topic>Natriuretic Peptide, Brain - blood</topic><topic>novel agents</topic><topic>Peptide Fragments - blood</topic><topic>Prognosis</topic><topic>Pyrazines - therapeutic use</topic><topic>Risk</topic><topic>Severity of Illness Index</topic><topic>Stem Cell Transplantation</topic><topic>therapy</topic><topic>Troponin C - blood</topic><topic>Troponin T - blood</topic><topic>Tumors</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Gatt, Moshe E.</creatorcontrib><creatorcontrib>Palladini, Giovanni</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><jtitle>British journal of haematology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Gatt, Moshe E.</au><au>Palladini, Giovanni</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Light chain amyloidosis 2012: a new era</atitle><jtitle>British journal of haematology</jtitle><addtitle>Br J Haematol</addtitle><date>2013-03</date><risdate>2013</risdate><volume>160</volume><issue>5</issue><spage>582</spage><epage>598</epage><pages>582-598</pages><issn>0007-1048</issn><eissn>1365-2141</eissn><coden>BJHEAL</coden><abstract>Summary
AL amyloidosis patients with multi‐organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high‐risk patients, but responding patients have prolonged overall survival. Toxicities can be decreased by careful patient selection, but this reduces the applicability of this treatment modality to a limited number of patients. Efforts are therefore needed to design novel more effective regimens, with the use of new medications, such as thalidomide, lenalidomide and bortezomib, next generation immunomodulatory drugs and proteasome inhibitors. Their combination with dexamethasone and alkylating agents show promising results, allowing a high percentage of remission and subsequent event‐free and overall survival, even in a significant proportion of high risk, poor prognosis populations. This review includes the state‐of‐the‐art treatment for AL amyloidosis patients as of 2012, in light of the progress in management of this disease during recent years.</abstract><cop>Oxford</cop><pub>Blackwell</pub><pmid>23294331</pmid><doi>10.1111/bjh.12191</doi><tpages>17</tpages><oa>free_for_read</oa></addata></record> |
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subjects | Alkylating Agents - therapeutic use Amyloid - metabolism amyloidosis Amyloidosis - complications Amyloidosis - drug therapy Amyloidosis - metabolism Amyloidosis - pathology Amyloidosis - surgery Biological and medical sciences Biomarkers Boronic Acids - therapeutic use Bortezomib Cardiomyopathies - blood Cardiomyopathies - etiology Cardiomyopathies - pathology Clinical Trials as Topic Combined Modality Therapy Dexamethasone - therapeutic use Forecasting Hematologic and hematopoietic diseases Humans Immunoglobulin Light Chains - metabolism Immunologic Factors - therapeutic use Medical sciences Natriuretic Peptide, Brain - blood novel agents Peptide Fragments - blood Prognosis Pyrazines - therapeutic use Risk Severity of Illness Index Stem Cell Transplantation therapy Troponin C - blood Troponin T - blood Tumors |
title | Light chain amyloidosis 2012: a new era |
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