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Amyloidosis extended survival

H Landau, M Smith, C Landry, J F Chou, S M Devlin, H Hassoun, C Bello, S Giralt, R L Comenzo
Stem cell transplantation (SCT), an effective therapy for amyloid light chain (AL) amyloidosis patients, is associated with low treatment-related mortality (TRM) with appropriate patient selection and risk-adapted dosing of melphalan (RA-SCT). Consolidation after SCT increases hematologic complete response (CR) rates and may improve overall survival (OS) for patients with <CR. We retrospectively analyzed outcomes for 143 patients who underwent RA-SCT with or without consolidation. Melphalan was administered at 100 (14%), 140 (52%) and 200 (34%) mg/m(2)...
January 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
S Y Lee, R S Meehan, D C Seldin, J M Sloan, K Quillen, A Shelton, D Brauneis, V Sanchorawala
High-dose melphalan with stem cell transplantation (HDM/SCT) extends survival and induces hematologic and clinical responses in patients with light chain (AL) amyloidosis. Eighty percent of melphalan is bound to plasma proteins (60% albumin-bound). We hypothesized that patients with profound hypoalbuminemia have a greater free melphalan fraction and more toxicity. Patients with AL amyloidosis treated with HDM/SCT between 2011 and 2014 with severe hypoalbuminemia (SH), defined as serum albumin ⩽2 g/dL were studied retrospectively...
October 2016: Bone Marrow Transplantation
Bouke P C Hazenberg, Alexandra Croockewit, Bronno van der Holt, Sonja Zweegman, Gerard M J Bos, Michel Delforge, Reinier A P Raymakers, Pieter Sonneveld, Edo Vellenga, Pierre W Wijermans, Peter A von dem Borne, Marinus H van Oers, Okke de Weerdt, Fokje M Spoelstra, Henk M Lokhorst
In a prospective multicenter phase II study, we evaluated the effect of three courses of vincristine, doxorubicin and dexamethasone followed by high-dose melphalan and autologous stem cell transplantation on an intention-to-treat basis. Sixty-nine newly diagnosed patients with amyloid light chain amyloidosis were included between November 2000 and January 2006: 37 men and 32 women with a median age of 56 years, including 46% of patients with cardiac and 22% of patients with involvement of 3 or 4 organs. Initial results presented in 2008 showed a 4-year overall survival rate of 62% among all the patients, while the 4-year survival rate after transplantation was 78%...
May 2015: Haematologica
Grzegorz Charliński, Artur Jurczyszyn, Wiesław Wiktor-Jedrzejczak
Immunoglobulin (Ig) light chain amyloidosis is a clonal, nonproliferative plasma cell disorder in which fragments of Ig light chain are deposited in tissues. Clinical features depend on organs involved but can include restrictive cardiomyopathy, nephrotic syndrome, hepatic failure, peripheral/ autonomic neuropathy. The diagnosis can be challenging, requiring a biopsy and often specialized testing to confirm the subtype of systemic disease. The goal of treatment is eradication of the monoclonal plasma cell population and suppression of the pathologic light chains which can result in organ improvement and extend patient survival...
2014: Przegla̧d Lekarski
Giampaolo Merlini, Raymond L Comenzo, David C Seldin, Ashutosh Wechalekar, Morie A Gertz
Primary light chain amyloidosis is the most common form of systemic amyloidosis and is caused by misfolded light chains that cause proteotoxicity and rapid decline of vital organ function. Early diagnosis is essential in order to deliver effective therapy and prevent irreversible organ damage. Accurate diagnosis requires clinical skills and advanced technologies. The disease can be halted and the function of target organs preserved by the prompt reduction and elimination of the plasma cell clone producing the toxic light chains in the bone marrow...
February 2014: Expert Review of Hematology
Giovanni Palladini, Paolo Milani, Andrea Foli, Laura Obici, Francesca Lavatelli, Mario Nuvolone, Riccardo Caccialanza, Stefano Perlini, Giampaolo Merlini
The combination of oral melphalan and dexamethasone is considered standard therapy for patients with light-chain amyloidosis ineligible for autologous stem cell transplantation. However, previous trials reported different rates of response and survival, mainly because of the different proportions of high-risk patients. In the present study, including a total of 259 subjects, we treated 119 patients with full-dose melphalan and dexamethasone (dexamethasone 40 mg days 1-4), and 140 patients with advanced cardiac disease with an attenuated dexamethasone schedule (20 mg)...
April 2014: Haematologica
T Pika, P Lochman, J Vymětal, R Metelka, J Minařík, P Látalová, J Zapletalová, J Bačovský, V Ščudla
BACKGROUND: Symptomatic cardiac involvement is the most important prognostic factor in AL amyloidosis patients. Longterm survival is limited not only by cardiac involvement condition, but also by limited choice of treatment with unsatisfactory results. The aim of the present report is to assess the effect of achieved treatment response on survival of AL amyloidosis patients with symptomatic cardiac involvement under conventional treatment. MATERIAL AND METHODS: The monitored patient set consisted of 19 patients with systemic AL amyloidosis and symptomatic cardiac involvement, treated and monitored at the III...
2013: Klinická Onkologie: Casopis Ceské a Slovenské Onkologické Spolecnosti
Yan Zhang, Yi-Hong Ren, Geng Qian, Chao-Fei Zhou, Qing-Kao Zhang, Peng-Cheng Zhao, Guo-Shu Liu
OBJECTIVE: To analyze the clinical characteristics, diagnosis, treatment and outcome of patients with cardiac amyloidosis (CA). METHODS: Clinical data from 18 patients diagnosed as CA by endomyocardial biopsy (EMB) from 1995 to 2005 were retrospectively analyzed. RESULTS: Among the 18 patients with CA, all patients had reduced diastolic dysfunction; 12 had mitral valve early diastolic blood flow peak velocity/late diastolic blood flow peak velocity (E/A) > 2...
April 2013: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
Bernard Canaud, Sudhir K Bowry
Online hemodiafiltration (OL-HDF), first described in 1985, is today a widely prescribed treatment modality for end-stage chronic kidney disease (CKD) patients. Other than in the United States, prescription of the treatment modality is widespread with a steady increase since its inception. Indeed, in Western Europe, more CKD patients receive OL-HDF than peritoneal dialysis, hitherto the second most prescribed therapy after conventional hemodialysis. The rise and success of OL-HDF can be attributed to diverse clinical advantages that have been documented over the last two decades...
2013: Blood Purification
Taimur Sher, Suzanne R Hayman, Morie A Gertz
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...
October 2012: Clinical Advances in Hematology & Oncology: H&O
M Rosenzweig, S Giralt, H Landau
Light-chain amyloidosis is a plasma cell dyscrasia characterized by the production of fibrillar proteins comprised of monoclonal light chains, which deposit in tissues causing multiorgan dysfunction and death. The diagnosis is challenging and requires a biopsy and often specialized testing to confirm the subtype of systemic disease. The goal of treatment is eradication of the monoclonal plasma cell population and suppression of the pathologic light chains, which improve organ function and extend survival. Standard treatment approaches have included high-dose melphalan followed by autologous hematopoietic SCT or oral melphalan with dexamethasone...
August 2013: Bone Marrow Transplantation
Mizuki Ogura, Rieko Sekine, Sayuri Nishiyama, Yu Abe, Hiromitsu Iizuka, Sayaka Kusaka, Yasuhiro Nakagawa, Kenshi Suzuki
Long-term survival remains poor for patients with cardiac amyloidosis. High-dose melphalan (MEL) with stem cell transplantation (HDM/SCT) is an effective treatment for AL amyloidosis, but patients with cardiac involvement are ineligible because of high therapy-related mortality. Here we report detailed HDM/SCT outcomes of 9 patients with cardiac failure. Their median age was 56 years (range, 45∼66). After a median follow-up of 15 months (range 9∼32), three died of multiorgan failure within the early phase after HDM/SCT, and the other six including poor risk patients are alive at present...
July 2012: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Michael Rosenzweig, Heather Landau
Light chain (AL) amyloidosis is a plasma cell dyscrasia characterized by the pathologic production of fibrillar proteins comprised of monoclonal light chains which deposit in tissues and cause organ dysfunction. The diagnosis can be challenging, requiring a biopsy and often specialized testing to confirm the subtype of systemic disease. The goal of treatment is eradication of the monoclonal plasma cell population and suppression of the pathologic light chains which can result in organ improvement and extend patient survival...
November 18, 2011: Journal of Hematology & Oncology
Michael Michael, Efstathios Kastritis, Sossana Delimpassi, Evridiki Michalis, Panagiotis Repoussis, Marie-Christine Kyrtsonis, Eirini Katodritou, Nicolaos Anagnostopoulos, Konstantinos Zervas, Meletios A Dimopoulos
BACKGROUND: Primary systemic light-chain (AL) amyloidosis is characterized by the deposition of immunoglobulin light chain-derived amyloid fibrils in various tissues leading to multiorgan dysfunction. PATIENTS AND METHODS: In order to define characteristics, treatment, and outcome of Greek patients with AL amyloidosis, we analyzed 112 unselected patients with AL from several hospitals. RESULTS: The heart was involved in 59% of patients and kidneys in 71%...
February 2010: Clinical Lymphoma, Myeloma & Leukemia
M Rosenzweig, D C Seldin, D G Remick, M Skinner, K Quillen, B Oran, K T Finn, V Sanchorawala
Aggressive treatment with high-dose i.v. melphalan followed by auto-SCT (HDM/SCT) is effective in inducing hematological and clinical remissions, and in extending survival in AL amyloidosis. Tandem cycles of HDM/SCT have been shown to increase hematologic complete response rates in patients with AL amyloidosis. Between April 1994 and July 2008, 57 patients with AL amyloidosis at the Boston University Medical Center were treated with a second cycle of HDM/SCT after failing to achieve a complete response after a first transplantation...
January 2010: Bone Marrow Transplantation
Nikhil C Munshi
Recent advances in genomics and proteomics have advanced our understanding of myeloma pathogenesis, recognized novel mediators of disease process, and identified new therapeutic targets. These developments have provided newer diagnostic tools for myeloma, improved monitoring of the disease status and allowed for molecular classification of the disease. The recent advances in investigative techniques that have helped refine the diagnostic work up in myeloma includes use of serum free light chains, especially in oligosecretory myeloma, patients with renal disease and with amyloidosis; use of MRI and PET scan in diagnosis and managing bone disease; and use of cytogenetics and fluorescent in situ hybridization (FISH) technique to determine prognosis...
2008: Hematology—the Education Program of the American Society of Hematology
Xibin Liang, Qian Wang, Ju Shi, Ludmila Lokteva, Richard M Breyer, Thomas J Montine, Katrin Andreasson
OBJECTIVE: Inflammation has emerged as an important factor in disease progression in human and transgenic models of amyotrophic lateral sclerosis (ALS). Recent studies demonstrate that the prostaglandin E(2) EP2 receptor is a major regulator of inflammatory oxidative injury in innate immunity. We tested whether EP2 signaling participated in disease pathogenesis in the G93A superoxide dismutase (SOD) model of familial ALS. METHODS: We examined the phenotype of G93A SOD mice lacking the EP2 receptor and performed immunocytochemistry, quantitative reverse transcriptase polymerase chain reaction, and Western analyses to determine the mechanism of EP2 toxicity in this model...
September 2008: Annals of Neurology
D Lebovic, J Hoffman, B M Levine, H Hassoun, H Landau, Y Goldsmith, M S Maurer, R M Steingart, A D Cohen, R L Comenzo
The treatment of systemic light-chain (AL) amyloidosis with symptomatic cardiac involvement at diagnosis remains a challenge. We report the results of 40 consecutive newly diagnosed AL cardiac patients who were not candidates for stem cell transplant and therefore received monthly oral melphalan and dexamethasone. Median survival was 10.5 months and baseline predictors of survival included gender, troponin I and interventricular septal thickness. The most significant predictor of survival was response to therapy...
November 2008: British Journal of Haematology
Ping Zhou, Julie Teruya-Feldstein, Ping Lu, Martin Fleisher, Adam Olshen, Raymond L Comenzo
In high doses with stem-cell transplantation, melphalan is an effective but toxic therapy for patients with systemic light-chain (AL-) amyloidosis, a protein deposition and monoclonal plasma cell disease. Melphalan can eliminate the indolent clonal plasma cells that cause the disease, an achievement called a complete response. Such a response is usually associated with extended survival, while no response (a less than 50% reduction) is not. Gene-expression studies and a stringently supervised analysis identified calreticulin as having significantly higher expression in the pretreatment plasma cells of patients with systemic AL-amyloidosis who then had a complete response to high-dose melphalan...
January 15, 2008: Blood
Mathew S Maurer, Amresh Raina, Charles Hesdorffer, Rachel Bijou, Paolo Colombo, Mario Deng, Ronald Drusin, Jennifer Haythe, Evelyn Horn, Sun Hi Lee, Charles Marboe, Yoshifumi Naka, Larry Schulman, Brian Scully, Peter Shapiro, Kenneth Prager, Jai Radhakrishnan, Susan Restaino, Donna Mancini
BACKGROUND: Systemic amyloidosis complicated by heart failure is associated with high cardiovascular morbidity and mortality. Heart transplantation for patients with systemic amyloidosis is controversial due to recurrence of disease in the transplanted organ or progression of disease in other organs. METHODS: All patients with systemic amyloidosis and heart failure referred for heart transplant evaluation from 1997 to 2004 were included in this retrospective cohort analysis...
March 15, 2007: Transplantation
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