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AL amyloidosis treatment

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https://www.readbyqxmd.com/read/28434927/impact-of-autologous-stem-cell-transplantation-on-the-incidence-and-outcome-of-oligoclonal-bands-in-patients-with-light-chain-amyloidosis
#1
Luis Gerardo Rodríguez-Lobato, Carlos Fernández de Larrea, M Teresa Cibeira, Natalia Tovar, Juan I Aróstegui, Laura Rosiñol, Tania Díaz, Ester Lozano, Montserrat Elena, Jordi Yagüe, Joan Bladé
The emergence of oligoclonal bands (OB) in patients with multiple myeloma achieving a complete remission (CR) after autologous stem cell transplantation (ASCT) and the use of novel agents is a well-recognized event. The presence of OB is associated with favorable outcome. However, the emergence of OB in light-chain (AL) amyloidosis has never been investigated. The aim of the study was to determine the incidence, natural history and prognostic impact of OB in 50 patients with AL amyloidosis who achieved at least a partial response (PR) either after up-front ASCT (20 patients [40%]) or after conventional treatment in patients ineligible for transplant (30 patients [60%])...
April 18, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28429761/a-specific-nanobody-prevents-amyloidogenesis-of-d76n-%C3%AE-2-microglobulin-in-vitro-and-modifies-its-tissue-distribution-in-vivo
#2
Sara Raimondi, Riccardo Porcari, P Patrizia Mangione, Guglielmo Verona, Julien Marcoux, Sofia Giorgetti, Graham W Taylor, Stephan Ellmerich, Maurizio Ballico, Stefano Zanini, Els Pardon, Raya Al-Shawi, J Paul Simons, Alessandra Corazza, Federico Fogolari, Manuela Leri, Massimo Stefani, Monica Bucciantini, Julian D Gillmore, Philip N Hawkins, Maurizia Valli, Monica Stoppini, Carol V Robinson, Jan Steyaert, Gennaro Esposito, Vittorio Bellotti
Systemic amyloidosis is caused by misfolding and aggregation of globular proteins in vivo for which effective treatments are urgently needed. Inhibition of protein self-aggregation represents an attractive therapeutic strategy. Studies on the amyloidogenic variant of β2-microglobulin, D76N, causing hereditary systemic amyloidosis, have become particularly relevant since fibrils are formed in vitro in physiologically relevant conditions. Here we compare the potency of two previously described inhibitors of wild type β2-microglobulin fibrillogenesis, doxycycline and single domain antibodies (nanobodies)...
April 21, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28394369/revisiting-conditioning-dose-in-newly-diagnosed-light-chain-amyloidosis-undergoing-frontline-autologous-stem-cell-transplant-impact-on-response-and-survival
#3
N Tandon, E Muchtar, S Sidana, A Dispenzieri, M Q Lacy, D Dingli, F K Buadi, S R Hayman, R Chakraborty, W J Hogan, W Gonsalves, R Warsame, T V Kourelis, N Leung, P Kapoor, S K Kumar, M A Gertz
Autologous stem cell transplantation (ASCT) is an important treatment modality in light chain (AL) amyloidosis. Use of reduced-dose melphalan conditioning is common, given the associated organ and functional decline. The impact of full-intensity melphalan conditioning (n=314) was compared to reduced-dose conditioning (n=143). Patients in the full-intensity group were younger, with better performance status, fewer involved organs, lower tumor burden and lower Mayo stage. Full-dose conditioning was associated with higher rate of very good partial response or better (79% vs 62%; P<0...
April 10, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28370245/renal-outcomes-in-patients-with-al-amyloidosis-prognostic-factors-renal-response-and-the-impact-of-therapy
#4
Efstathios Kastritis, Maria Gavriatopoulou, Maria Roussou, Magdalini Migkou, Despina Fotiou, Dimitrios C Ziogas, Nikos Kanellias, Evangelos Eleutherakis-Papaiakovou, Ioannis Panagiotidis, Stavroula Giannouli, Erasmia Psimenou, Smaragdi Marinaki, Theofanis Apostolou, Hariklia Gakiopoulou, Anna Tasidou, Ioannis Papassotiriou, Evangelos Terpos, Meletios A Dimopoulos
A staging system for patients with renal AL amyloidosis, based on eGFR (<50 ml/min/1.73 m(2) ) and proteinuria (≥5gr/day) at diagnosis, as well as criteria for renal progression (≥25% eGFR reduction) and response (≥30% reduction of proteinuria without renal progression) were recently proposed. We validated these criteria in a cohort of 125 patients with renal AL amyloidosis, mostly treated with bortezomib or lenalidomide. We confirmed the prognostic value of the renal staging system but also identified the limitations of renal progression criteria which are based only on eGFR reduction...
March 28, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28352011/biopsy-of-small-kidneys-a-safe-and-a-useful-guide-to-potentially-treatable-kidney-disease
#5
Kamel El-Reshaid, Wael El-Reshaid, Dalal Al-Bader, Jozsef Varro, John Madda, Hosameldin Tawfik Sallam
Over the past four years, all patients with unexplained rapid progression of their renal disease were subjected to kidney biopsy, despite their small size (<9 cm), to define its etiology. Children, pregnant women, morbidly obese patients, and those with an unstable cardiovascular state, septicemia, bleeding diathesis as well as those kidney size with size <6 cm were excluded from the study. Doppler ultrasound was used to exclude renovascular/ischemic nephropathy. The procedure was performed by an interventional radiologist using a biopsy gun technique and under ultrasound guidance...
March 2017: Saudi Journal of Kidney Diseases and Transplantation
https://www.readbyqxmd.com/read/28329248/clinical-characteristics-of-wild-type-transthyretin-cardiac-amyloidosis-disproving-myths
#6
Esther González-López, Christian Gagliardi, Fernando Dominguez, Cristina Candida Quarta, F Javier de Haro-Del Moral, Agnese Milandri, Clara Salas, Mario Cinelli, Marta Cobo-Marcos, Massimiliano Lorenzini, Enrique Lara-Pezzi, Serena Foffi, Luis Alonso-Pulpon, Claudio Rapezzi, Pablo Garcia-Pavia
Aims: Wild-type transthyretin amyloidosis (ATTRwt) is mostly considered a disease predominantly of elderly male, characterized by concentric LV hypertrophy, preserved LVEF, and low QRS voltages. We sought to describe the characteristics of a large cohort of ATTRwt patients to better define the disease. Methods and results: Clinical findings of consecutive ATTRwt patients diagnosed at 2 centres were reviewed. ATTRwt was diagnosed histologically or non-invasively (LV hypertrophy ≥12 mm, intense cardiac uptake at 99mTc-DPD scintigraphy and AL exclusion)...
March 1, 2017: European Heart Journal
https://www.readbyqxmd.com/read/28314084/treatment-patterns-and-outcome-following-initial-relapse-or-refractory-disease-in-patients-with-systemic-light-chain-amyloidosis
#7
Nidhi Tandon, Surbhi Sidana, Morie A Gertz, Angela Dispenzieri, Martha Q Lacy, Francis K Buadi, David Dingli, Amie L Fonder, Miriam A Hobbs, Suzanne R Hayman, Wilson I Gonsalves, Yi Lisa Hwa, Prashant Kapoor, Robert A Kyle, Nelson Leung, Ronald S Go, John A Lust, Stephen J Russell, Steven R Zeldenrust, S Vincent Rajkumar, Shaji K Kumar
We analyzed the outcomes following initial relapse or refractory disease in systemic light chain amyloidosis (AL) and the impact of type of therapy employed.A total of 1327 patients with AL seen at Mayo Clinic within 90 days of diagnosis, between 2006 and 2015, were reviewed. The study included 366 patients experiencing a documented hematological or organ relapse or refractory disease requiring start of second line therapy. Overall survival (OS) and time to next treatment (TTNT) were calculated from start of second line treatment...
March 17, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28290973/total-artificial-heart-implantation-as-a-bridge-to-heart-transplantation-in-an-active-duty-service-member-with-amyloid-cardiomyopathy
#8
Michael S Scully, Dylan E Wessman, James M McKee, Gregory M Francisco, Keshav R Nayak, Jon A Kobashigawa
INTRODUCTION: Cardiac involvement by light-chain (AL) amyloid occurs in up to 50% of patients with primary AL amyloidosis. The prognosis of amyloid heart disease is poor with 1-year survival rates of 35 to 40%. Historically, heart transplantation was considered controversial for patients with AL amyloid cardiomyopathy (CM) given the systemic nature of the disease and poor survival. We present a case report of an active duty service member diagnosed with advanced cardiac amyloid who underwent total artificial heart transplant as a bridge to heart transplant and eventual autologous stem cell transplant...
March 2017: Military Medicine
https://www.readbyqxmd.com/read/28281017/heart-transplantation-in-cardiac-amyloidosis
#9
REVIEW
Matthew Sousa, Gregory Monohan, Navin Rajagopalan, Alla Grigorian, Maya Guglin
"Cardiac amyloidosis" is the term commonly used to reflect the deposition of abnormal protein amyloid in the heart. This process can result from several different forms, most commonly from light-chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis, which in turn can represent wild-type (ATTRwt) or genetic form. Regardless of the origin, cardiac involvement is usually associated with poor prognosis, especially in AL amyloidosis. Although several treatment options, including chemotherapy, exist for different forms of the disease, cardiac transplantation is increasingly considered...
March 9, 2017: Heart Failure Reviews
https://www.readbyqxmd.com/read/28278302/neuropathy-and-efficacy-of-once-weekly-subcutaneous-bortezomib-in-multiple-myeloma-and-light-chain-al-amyloidosis
#10
Surbhi Sidana, Mayur Narkhede, Paul Elson, Debbie Hastings, Beth Faiman, Jason Valent, Christy Samaras, Kimberly Hamilton, Hien K Liu, Mitchell R Smith, Frederic J Reu
INTRODUCTION: Randomized studies have shown that bortezomib (BTZ) can be given weekly via intravenous (IV) route or twice weekly via subcutaneous (SC) route with lower neuropathy risk and no loss of anti-myeloma efficacy compared to original standard IV twice weekly schedule. Weekly SC should therefore yield the best therapeutic index and is widely used but has not been compared to established administration schedules in the context of a clinical trial. METHODS: Comprehensive electronic medical record review was done for disease control and neuropathy symptoms of 344 consecutive patients who received their first BTZ-containing regimen for myeloma or AL amyloidosis before or after we changed to SC weekly in December 2010...
2017: PloS One
https://www.readbyqxmd.com/read/28277869/disease-burden-of-systemic-light-chain-al-amyloidosis-a-systematic-literature-review
#11
Huamao Mark Lin, Xin Gao, Catherine E Cooke, Deborah Berg, Richard Labotka, Douglas V Faller, Brian Seal, Parameswaran Hari
A systematic literature review on AL amyloidosis was conducted in order to understand the disease burden, and identify unmet medical needs and knowledge gaps. MEDLINE, EMBASE and COCHRANE databases were searched for English language studies published in the last 10 years using search terms that focused on the clinical, economic, and patient-reported outcome (PRO) aspects of AL amyloidosis. There was a low yield of articles in the economic and PRO categories and additional searches were conducted in clinical conference proceedings, and using Google and Google Scholar...
February 21, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28176254/-18-fluorine-sodium-fluoride-positron-emission-tomography-a-potential-biomarker-of-transthyretin-cardiac-amyloidosis
#12
Rachelle Morgenstern, Randy Yeh, Adam Castano, Mathew S Maurer, Sabahat Bokhari
BACKGROUND: Non-invasive imaging to diagnose and quantify amyloid load, progression, and response to treatment are central for the care of patients with cardiac amyloidosis. (18)Fluorine-labeled sodium fluoride ((18)F-NaF) is a widely available radioisotope and PET imaging allows for absolute quantification of tracer uptake. METHODS: Patients with biopsy-proven transthyretin (ATTR-CA) and light-chain cardiac amyloidosis (AL) (3 ATTRwt, 2 ATTRV122I, 2 AL) and controls (n = 5), underwent (18)F-NaF PET imaging...
February 7, 2017: Journal of Nuclear Cardiology: Official Publication of the American Society of Nuclear Cardiology
https://www.readbyqxmd.com/read/28153807/amyloidosis-a-cancer-derived-paraproteinemia-and-kidney-involvement
#13
REVIEW
Jolanta Małyszko, Klaudia Kozłowska, Jacek Stanisław Małyszko
Amyloidosis is the general term describing the extracellular tissue deposition of fibrils composed of low molecular weight subunits of a variety of proteins. There are multiple different human protein precursors of amyloid fibrils. Amyloid deposits are stained using Congo Red and show typical apple-green birefringence in polarized microscopy. Nowadays, a novel technique LMD/MS technique or laser microdissection combined with mass spectrometry help to diagnose amyloidosis. Amyloidosis of the kidney is typically classified as being either one of two types: AL or AA...
January 30, 2017: Advances in Medical Sciences
https://www.readbyqxmd.com/read/28145535/amyloid-cardiomyopathy
#14
Lucie Karafiatova, Tomas Pika
Amyloidosis is a heterogeneous group of diseases characterized by the deposition of amyloid. It is caused by extracellular deposition of insoluble fibrils with beta-pleated sheet configuration. The protein misfolding abnormalities result in amyloid fibrils and may manifest as primary, secondary, or familial amyloidosis. Amyloid deposition can occur in multiple organs (eg, heart, liver, kidney, skin, eyes, lungs, nervous system) resulting in a variety of clinical manifestations. Cardiac involvement can occur as part of a systemic disease or as a localized phenomenon...
February 1, 2017: Biomedical Papers of the Medical Faculty of the University Palacký, Olomouc, Czechoslovakia
https://www.readbyqxmd.com/read/28134587/amyloidosis-of-the-lung
#15
REVIEW
Andras Khoor, Thomas V Colby
CONTEXT: -Amyloidosis is a heterogeneous group of diseases characterized by the deposition of congophilic amyloid fibrils in the extracellular matrix of tissues and organs. To date, 31 fibril proteins have been identified in humans, and it is now recommended that amyloidoses be named after these fibril proteins. Based on this classification scheme, the most common forms of amyloidosis include systemic AL (formerly primary), systemic AA (formerly secondary), systemic wild-type ATTR (formerly age-related or senile systemic), and systemic hereditary ATTR amyloidosis (formerly familial amyloid polyneuropathy)...
February 2017: Archives of Pathology & Laboratory Medicine
https://www.readbyqxmd.com/read/28130212/a-phase-ii-trial-of-pomalidomide-and-dexamethasone-rescue-treatment-in-patients-with-al-amyloidosis
#16
Giovanni Palladini, Paolo Milani, Andrea Foli, Marco Basset, Francesca Russo, Stefano Perlini, Giampaolo Merlini
Immunomodulatory drugs are active agents in AL amyloidosis. However, previous studies could not show a survival advantage for patients with AL amyloidosis responding to salvage treatment with pomalidomide. In this phase II trial, we assessed safety and efficacy of pomalidomide and dexamethasone in patients with AL amyloidosis previously exposed to bortezomib, alkylators, and other immunomodulatory drugs. Twenty-eight patients were enrolled. Three patients received pomalidomide 2 mg/day with no dose-limiting toxicity...
January 27, 2017: Blood
https://www.readbyqxmd.com/read/28110969/-acquired-non-amyloid-related-factor%C3%A2-x-deficiency-a-first-case-associated-with-atypical-chronic-lymphocytic-leukemia-and-literature-review
#17
L Boudin, M Patient, E Roméo, J-S Bladé, O Gisserot, J-P de Jauréguiberry
INTRODUCTION: Acquired factor X deficiency is in most cases associated with AL amyloidosis. Acquired non-amyloid related factor X deficiency (DNAA-FX) has been exceptionally reported in the literature. CASE REPORT: We report the first case of acquired, non-amyloid related factor X deficiency associated with atypical chronic lymphoid leukemia in a 66-year-old patient with spontaneous hematomas. After therapeutic failure with polyclonal intravenous immunoglobulins, specific lymphoid malignancy treatment allowed symptoms and coagulation disorder resolution...
January 19, 2017: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/28103898/the-burden-of-amyloid-light-chain-amyloidosis-on-health-related-quality-of-life
#18
Martha Bayliss, Kristen L McCausland, Spencer D Guthrie, Michelle K White
BACKGROUND: Light chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs, and little is known about the burden of AL amyloidosis on health-related quality of life. This study aimed to quantify the burden of AL amyloidosis in terms of health-related quality of life in a diverse, community-based sample of AL amyloidosis patients. RESULTS: The SF-36v2® Health Survey (SF-36v2), a widely used generic measure of health-related quality of life (using physical and mental summary scales and subscales assessing eight aspects of functioning and well-being), was administered as an online survey of AL amyloidosis patients with AL amyloidosis (ClinicalTrials...
January 19, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28103719/pegylated-nanoliposomal-clusterin-for-amyloidogenic-light-chain-induced-endothelial-dysfunction
#19
Diana Guzman-Villanueva, Raymond Q Migrino, Seth Truran, Nina Karamanova, Daniel A Franco, Camelia Burciu, Subhadip Senapati, Dobrin Nedelkov, Parameswaran Hari, Volkmar Weissig
Light chain (AL) amyloidosis is a disease associated with significant morbidity and mortality arising from multi-organ injury induced by amyloidogenic light chain proteins (LC). There is no available treatment to reverse the toxicity of LC. We previously showed that chaperone glycoprotein clusterin (CLU) and nanoliposomes (NL), separately, restore human microvascular endothelial function impaired by LC. In this work, we aim to prepare PEGylated-nanoliposomal clusterin (NL-CLU) formulations that could allow combined benefit against LC while potentially enabling efficient delivery to microvascular tissue, and test efficacy on human arteriole endothelial function...
January 20, 2017: Journal of Liposome Research
https://www.readbyqxmd.com/read/28087478/renal-involvement-in-lysinuric-protein-intolerance-contribution-of-pathology-to-assessment-of-heterogeneity-of-renal-lesions
#20
Emmanuel Estève, Pauline Krug, Aurélie Hummel, Jean-Baptiste Arnoux, Olivia Boyer, Anais Brassier, Pascale de Lonlay, Vincent Vuiblet, Stéphanie Gobin, Rémi Salomon, Christine Piètrement, Jean-Paul Bonnefont, Aude Servais, Louise Galmiche
Lysinuric protein intolerance (LPI) is a rare autosomal recessive disease caused by mutations in the SLC7A7 gene encoding the light subunit of a cationic amino acid transporter. Symptoms mimic primary urea cycle defects but dysimmune symptoms are also described. Renal involvement in LPI was first described in the 1980s. In 2007, it appeared that it could concern as much as 75% of LPI patients and could lead to end-stage renal disease. The most common feature is proximal tubular dysfunction and nephrocalcinosis but glomerular lesions are also reported...
April 2017: Human Pathology
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