Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis
Systemic amyloid light-chain (AL) amyloidosis is a protein conformation disorder caused by clonal plasma cell dyscrasias. Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends most...
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| description | Systemic amyloid light-chain (AL) amyloidosis is a protein conformation disorder caused by clonal plasma cell dyscrasias. Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends mostly on the severity of cardiac involvement. The treatment is derived from the therapy of multiple myeloma with the main goal being to reach a complete hematological remission (CR). High-dose melphalan (HDM) and autologous hematopoietic cell transplantation can induce CR rates in about 40%. The main concern was the high transplant-related mortality of up to 40% due to organ dysfunction, which could be reduced to 50% of patients in CR survive longer than 10 years, suggesting that HDM has the potential to change the natural course of the disease. As there is evidence that ‘graft-versus-plasma-cell-dyscrasia’ effects are active in AL amyloidosis, allogeneic hematopoietic cell transplantation might be an option for younger patients with preserved organ functions who have relapsed after HDM. |
| doi_str_mv | 10.1038/bmt.2011.152 |
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Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends mostly on the severity of cardiac involvement. The treatment is derived from the therapy of multiple myeloma with the main goal being to reach a complete hematological remission (CR). High-dose melphalan (HDM) and autologous hematopoietic cell transplantation can induce CR rates in about 40%. The main concern was the high transplant-related mortality of up to 40% due to organ dysfunction, which could be reduced to <12% by careful patient selection in experienced centers. However, >50% of patients in CR survive longer than 10 years, suggesting that HDM has the potential to change the natural course of the disease. As there is evidence that ‘graft-versus-plasma-cell-dyscrasia’ effects are active in AL amyloidosis, allogeneic hematopoietic cell transplantation might be an option for younger patients with preserved organ functions who have relapsed after HDM.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/bmt.2011.152</identifier><identifier>PMID: 21785469</identifier><identifier>CODEN: BMTRE9</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>Amyloid ; Amyloidosis ; Amyloidosis - drug therapy ; Amyloidosis - surgery ; Amyloidosis - therapy ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Autografts ; Biological and medical sciences ; Bone marrow ; Bone marrow transplantation ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Care and treatment ; Cell Biology ; Chains ; Deposits ; Diagnosis ; Dosage ; Dose-Response Relationship, Drug ; Health aspects ; Heart ; Hematology ; Hematopoietic Stem Cell Transplantation - methods ; Hematopoietic stem cells ; Hemopoiesis ; Humans ; Internal Medicine ; Medical sciences ; Medicine ; Medicine & Public Health ; Melphalan ; Melphalan - administration & dosage ; Monoclonal gammopathy ; Mortality ; Multiple myeloma ; Paraproteinemia ; Patient outcomes ; Patients ; Prognosis ; Protein structure ; Public Health ; Remission ; review ; Signs and symptoms ; Stem cell transplantation ; Stem Cells ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Transplantation ; Transplants & implants</subject><ispartof>Bone marrow transplantation (Basingstoke), 2012-07, Vol.47 (7), p.895-905</ispartof><rights>Macmillan Publishers Limited 2012</rights><rights>2015 INIST-CNRS</rights><rights>COPYRIGHT 2012 Nature Publishing Group</rights><rights>Copyright Nature Publishing Group Jul 2012</rights><rights>Macmillan Publishers Limited 2012.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c584t-18223faa519128dba0301441de07cb350fe27d8d4ac02982990b42800dd593b53</citedby><cites>FETCH-LOGICAL-c584t-18223faa519128dba0301441de07cb350fe27d8d4ac02982990b42800dd593b53</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1038/bmt.2011.152$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1038/bmt.2011.152$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=26078874$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/21785469$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Schönland, S O</creatorcontrib><creatorcontrib>Dreger, P</creatorcontrib><creatorcontrib>de Witte, T</creatorcontrib><creatorcontrib>Hegenbart, U</creatorcontrib><title>Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><addtitle>Bone Marrow Transplant</addtitle><description>Systemic amyloid light-chain (AL) amyloidosis is a protein conformation disorder caused by clonal plasma cell dyscrasias. Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends mostly on the severity of cardiac involvement. The treatment is derived from the therapy of multiple myeloma with the main goal being to reach a complete hematological remission (CR). High-dose melphalan (HDM) and autologous hematopoietic cell transplantation can induce CR rates in about 40%. The main concern was the high transplant-related mortality of up to 40% due to organ dysfunction, which could be reduced to <12% by careful patient selection in experienced centers. However, >50% of patients in CR survive longer than 10 years, suggesting that HDM has the potential to change the natural course of the disease. As there is evidence that ‘graft-versus-plasma-cell-dyscrasia’ effects are active in AL amyloidosis, allogeneic hematopoietic cell transplantation might be an option for younger patients with preserved organ functions who have relapsed after HDM.</description><subject>Amyloid</subject><subject>Amyloidosis</subject><subject>Amyloidosis - drug therapy</subject><subject>Amyloidosis - surgery</subject><subject>Amyloidosis - therapy</subject><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Autografts</subject><subject>Biological and medical sciences</subject><subject>Bone marrow</subject><subject>Bone marrow transplantation</subject><subject>Bone marrow, stem cells transplantation. Graft versus host reaction</subject><subject>Care and treatment</subject><subject>Cell Biology</subject><subject>Chains</subject><subject>Deposits</subject><subject>Diagnosis</subject><subject>Dosage</subject><subject>Dose-Response Relationship, Drug</subject><subject>Health aspects</subject><subject>Heart</subject><subject>Hematology</subject><subject>Hematopoietic Stem Cell Transplantation - methods</subject><subject>Hematopoietic stem cells</subject><subject>Hemopoiesis</subject><subject>Humans</subject><subject>Internal Medicine</subject><subject>Medical sciences</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Melphalan</subject><subject>Melphalan - administration & dosage</subject><subject>Monoclonal gammopathy</subject><subject>Mortality</subject><subject>Multiple myeloma</subject><subject>Paraproteinemia</subject><subject>Patient outcomes</subject><subject>Patients</subject><subject>Prognosis</subject><subject>Protein structure</subject><subject>Public Health</subject><subject>Remission</subject><subject>review</subject><subject>Signs and symptoms</subject><subject>Stem cell transplantation</subject><subject>Stem Cells</subject><subject>Transfusions. Complications. Transfusion reactions. 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Intensive care medicine. Transfusions. Cell therapy and gene therapy</topic><topic>Autografts</topic><topic>Biological and medical sciences</topic><topic>Bone marrow</topic><topic>Bone marrow transplantation</topic><topic>Bone marrow, stem cells transplantation. Graft versus host reaction</topic><topic>Care and treatment</topic><topic>Cell Biology</topic><topic>Chains</topic><topic>Deposits</topic><topic>Diagnosis</topic><topic>Dosage</topic><topic>Dose-Response Relationship, Drug</topic><topic>Health aspects</topic><topic>Heart</topic><topic>Hematology</topic><topic>Hematopoietic Stem Cell Transplantation - methods</topic><topic>Hematopoietic stem cells</topic><topic>Hemopoiesis</topic><topic>Humans</topic><topic>Internal Medicine</topic><topic>Medical sciences</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Melphalan</topic><topic>Melphalan - administration & dosage</topic><topic>Monoclonal gammopathy</topic><topic>Mortality</topic><topic>Multiple myeloma</topic><topic>Paraproteinemia</topic><topic>Patient outcomes</topic><topic>Patients</topic><topic>Prognosis</topic><topic>Protein structure</topic><topic>Public Health</topic><topic>Remission</topic><topic>review</topic><topic>Signs and symptoms</topic><topic>Stem cell transplantation</topic><topic>Stem Cells</topic><topic>Transfusions. Complications. Transfusion reactions. 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Symptoms result from fibrillar extracellular deposits in various tissues. The deposits disrupt organ function and ultimately lead to death. The prognosis is poor and depends mostly on the severity of cardiac involvement. The treatment is derived from the therapy of multiple myeloma with the main goal being to reach a complete hematological remission (CR). High-dose melphalan (HDM) and autologous hematopoietic cell transplantation can induce CR rates in about 40%. The main concern was the high transplant-related mortality of up to 40% due to organ dysfunction, which could be reduced to <12% by careful patient selection in experienced centers. However, >50% of patients in CR survive longer than 10 years, suggesting that HDM has the potential to change the natural course of the disease. As there is evidence that ‘graft-versus-plasma-cell-dyscrasia’ effects are active in AL amyloidosis, allogeneic hematopoietic cell transplantation might be an option for younger patients with preserved organ functions who have relapsed after HDM.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>21785469</pmid><doi>10.1038/bmt.2011.152</doi><tpages>11</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Amyloid Amyloidosis Amyloidosis - drug therapy Amyloidosis - surgery Amyloidosis - therapy Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Autografts Biological and medical sciences Bone marrow Bone marrow transplantation Bone marrow, stem cells transplantation. Graft versus host reaction Care and treatment Cell Biology Chains Deposits Diagnosis Dosage Dose-Response Relationship, Drug Health aspects Heart Hematology Hematopoietic Stem Cell Transplantation - methods Hematopoietic stem cells Hemopoiesis Humans Internal Medicine Medical sciences Medicine Medicine & Public Health Melphalan Melphalan - administration & dosage Monoclonal gammopathy Mortality Multiple myeloma Paraproteinemia Patient outcomes Patients Prognosis Protein structure Public Health Remission review Signs and symptoms Stem cell transplantation Stem Cells Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transplantation Transplants & implants |
| title | Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis |
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