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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
Main Authors: Gatt, Moshe E., Palladini, Giovanni
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Language:English
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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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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. 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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. 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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. 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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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