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Discontinuation of treatment amyloidosis

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https://www.readbyqxmd.com/read/27802513/assessment-of-the-incremental-diagnostic-value-of-florbetapir-f-18-imaging-in-patients-with-cognitive-impairment-the-incremental-diagnostic-value-of-amyloid-pet-with-18f-florbetapir-india-fbp-study
#1
Marina Boccardi, Daniele Altomare, Clarissa Ferrari, Cristina Festari, Ugo Paolo Guerra, Barbara Paghera, Claudio Pizzocaro, Giulia Lussignoli, Cristina Geroldi, Orazio Zanetti, Maria Sofia Cotelli, Marinella Turla, Barbara Borroni, Luca Rozzini, Dario Mirabile, Carlo Defanti, Michele Gennuso, Alessandro Prelle, Simona Gentile, Alessandro Morandi, Stefano Vollaro, Giorgio Dalla Volta, Angelo Bianchetti, Marta Zaffira Conti, Melania Cappuccio, Pasqualina Carbone, Daniele Bellandi, Luciano Abruzzi, Luigi Bettoni, Daniele Villani, Maria Clara Raimondi, Alessia Lanari, Alfonso Ciccone, Emanuela Facchi, Ignazio Di Fazio, Renzo Rozzini, Stefano Boffelli, Laura Manzoni, Giovanni Pietro Salvi, Sabina Cavaliere, Gloria Belotti, Stefano Avanzi, Patrizio Pasqualetti, Cristina Muscio, Alessandro Padovani, Giovanni B Frisoni
Importance: Cerebral amyloidosis is a key abnormality in Alzheimer disease (AD) and can be detected in vivo with positron emission tomography (PET) ligands. Although amyloid PET has clearly demonstrated analytical validity, its clinical utility is debated. Objective: To evaluate the incremental diagnostic value of amyloid PET with florbetapir F 18 in addition to the routine clinical diagnostic assessment of patients evaluated for cognitive impairment. Design, Setting, and Participants: The Incremental Diagnostic Value of Amyloid PET With [18F]-Florbetapir (INDIA-FBP) Study is a multicenter study involving 18 AD evaluation units from eastern Lombardy, Northern Italy, 228 consecutive adults with cognitive impairment were evaluated for AD and other causes of cognitive decline, with a prescan diagnostic confidence of AD between 15% and 85%...
October 31, 2016: JAMA Neurology
https://www.readbyqxmd.com/read/26677890/a-patient-with-al-amyloidosis-with-negative-free-light-chain-results
#2
Paolo Milani, Veronica Valentini, Giovanni Ferraro, Marco Basset, Francesca Russo, Andrea Foli, Giovanni Palladini, Giampaolo Merlini
The detection and quantification of amyloidogenic monoclonal light chains are necessary for the diagnosis and evaluation of response to treatment in AL amyloidosis. However, the amyloid clone is often small and difficult to detect. We report the case of a 68-year-old man who was referred to our Center in April 2013 after syncope and the identification of left ventricular hypertrophy at echocardiography, suspected for amyloidosis. A commercial agarose gel electrophoresis immunofixation (IFE) did not reveal monoclonal components in serum and urine...
June 1, 2016: Clinical Chemistry and Laboratory Medicine: CCLM
https://www.readbyqxmd.com/read/26662359/a-review-of-tafamidis-for-the-treatment-of-transthyretin-related-amyloidosis
#3
Márcia Waddington Cruz, Merril D Benson
Transthyretin (TTR)-related amyloidosis (ATTR) is a devastating disease which affects a combination of organs including the heart and the peripheral nerves, and which has a fatal outcome if not treated within a average of 10 years. Tafamidis, or 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylic acid, selectively binds to TTR with negative cooperativity and kinetically stabilizes wild-type native TTR and mutant TTR; tafamidis therefore has the potential to halt the amyloidogenic cascade initiated by TTR tetramer dissociation, monomer misfolding, and aggregation...
December 2015: Neurology and Therapy
https://www.readbyqxmd.com/read/26239119/colchicine-as-an-anti-inflammatory-and-cardioprotective-agent
#4
REVIEW
Armen Yuri Gasparyan, Lilit Ayvazyan, Marlen Yessirkepov, George D Kitas
INTRODUCTION: Colchicine has been successfully used for the treatment of neutrophilic disorders such as familial Mediterranean fever (FMF), Behçet disease (BD) and gout. There is a growing interest in its cardiovascular effects. AREAS COVERED: A MEDLINE/PubMed search for English articles published from January 1972 to June 2015 was completed using the following terms: therapy, pharmacokinetics, efficiency, side effects, toxicity, heart, colchicine, inflammation, FMF, amyloidosis, BD, gout, cardiovascular disorders, pericarditis, arrhythmias, inflammation, neutrophils, platelets...
2015: Expert Opinion on Drug Metabolism & Toxicology
https://www.readbyqxmd.com/read/26120866/therapeutic-blockade-of-interleukin-6-by-tocilizumab-in-the-management-of-aa-amyloidosis-and-chronic-inflammatory-disorders-a-case-series-and-review-of-the-literature
#5
REVIEW
Thirusha Lane, Julian D Gillmore, Ashutosh D Wechalekar, Philip N Hawkins, Helen J Lachmann
OBJECTIVES: AA amyloidosis is the most serious potential complication of chronic inflammatory disorders. The main aim of treatment is to suppress inflammation thereby inhibiting serum amyloid A protein (SAA), which is the precursor of AA amyloid fibrils, to prevent or halt amyloid deposition. Interleukin (IL)-6 blockade is frequently effective in inflammatory conditions, however, there are few published data on its use in AA amyloidosis or the systemic autoinflammatory diseases (SAIDs) or chronic inflammatory conditions...
November 2015: Clinical and Experimental Rheumatology
https://www.readbyqxmd.com/read/26017328/safety-and-efficacy-of-long-term-diflunisal-administration-in-hereditary-transthyretin-attr-amyloidosis
#6
RANDOMIZED CONTROLLED TRIAL
Yoshiki Sekijima, Kana Tojo, Hiroshi Morita, Jun Koyama, Shu-ichi Ikeda
BACKGROUND: A recent 2-year randomized controlled trial indicated that the transthyretin (TTR) tetramer stabilizer, diflunisal, inhibits polyneuropathy progression and preserves quality of life in hereditary ATTR amyloidosis. However, its long-term outcomes are unknown. Here, we report tolerance and efficacy of long-term diflunisal administration in hereditary ATTR amyloidosis. METHODS: Diflunisal was administered orally at 500 mg/day to 40 Japanese hereditary ATTR amyloidosis patents who were not candidates for liver transplantation...
2015: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/25974382/a-phase-ii-trial-of-lenalidomide-dexamethasone-and-cyclophosphamide-for-newly-diagnosed-patients-with-systemic-immunoglobulin-light-chain-amyloidosis
#7
MULTICENTER STUDY
Maria T Cibeira, Albert Oriol, Juan J Lahuerta, Maria-Victoria Mateos, Javier de la Rubia, Miguel T Hernández, Miquel Granell, Carlos Fernández de Larrea, Jesús F San Miguel, Joan Bladé
Immunomodulatory drugs have been shown to be of benefit in relapsed/refractory immunoglobulin light-chain (AL) amyloidosis. We designed a prospective, multicentre phase II trial of lenalidomide, dexamethasone and cyclophosphamide for newly diagnosed patients with AL amyloidosis not eligible for autologous stem-cell transplantation. Twenty-eight patients were included in the study. Cardiac involvement was present in 23 patients; 14 of them had cardiac stage III. The overall haematological response rate was 46%, including complete and very good partial responses in 25% and 18% of patients respectively...
September 2015: British Journal of Haematology
https://www.readbyqxmd.com/read/25872787/tafamidis-in-transthyretin-amyloid-cardiomyopathy-effects-on-transthyretin-stabilization-and-clinical-outcomes
#8
MULTICENTER STUDY
Mathew S Maurer, Donna R Grogan, Daniel P Judge, Rajiv Mundayat, Jeff Packman, Ilise Lombardo, Arshed A Quyyumi, Janske Aarts, Rodney H Falk
BACKGROUND: Transthyretin (TTR) amyloidosis is a progressive systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the heart and systemically as amyloid. METHODS AND RESULTS: This phase 2 open-label trial evaluated the stabilization of TTR tetramers using 20 mg of tafamidis daily at week 6 (primary end point), month 6, and month 12, as well as safety of tafamidis treatment and efficacy with respect to progression of TTR amyloid cardiomyopathy...
May 2015: Circulation. Heart Failure
https://www.readbyqxmd.com/read/25455933/successful-treatment-of-renal-light-chain-al-amyloidosis-with-bortezomib-and-dexamethasone-vd
#9
B Huang, J Li, X Xu, D Zheng, Z Zhou, J Liu
OBJECTIVE: To assess the efficacy and tolerability of bortezomib with dexamethasone for patients with renal light chain (AL) amyloidosis. METHODS: Twelve newly diagnosed patients with renal AL amyloidosis were treated with a combination of bortezomib (1.3mg/m(2)/d iv, d1, 4, 8, 11) and dexamethasone (20mg/d iv drip, d1-4). RESULTS: Median follow-up time was 22.5 months (range, 2.1-53.6). Ten patients were evaluable. Five out of 10 (50%) patients achieved complete hematologic responses (CHR), and totally 8/10 (80%) achieved hematologic responses (HR)...
February 2015: Pathologie-biologie
https://www.readbyqxmd.com/read/25022953/tafamidis-a-review-of-its-use-in-familial-amyloid-polyneuropathy
#10
REVIEW
Lesley J Scott
Oral tafamidis (Vyndaqel(®)) is indicated in the EU for the treatment of transthyretin (TTR) amyloidosis in adult patients with early stage symptomatic polyneuropathy to delay peripheral neurologic impairment and, in Argentina, Japan and Mexico, for delaying the peripheral neurological impairment of TTR familial amyloid polyneuropathy (TTR-FAP). It is the first disease-modifying pharmacotherapy to be approved for use in adult patients with early-stage TTR-FAP. The drug acts to kinetically stabilize the variant TTR tetramer and thereby prevent tetramer dissociation, the rate-limiting step in TTR misfolding and amyloidogenesis...
August 2014: Drugs
https://www.readbyqxmd.com/read/23461404/-heart-transplantation-and-the-subsequent-treatment-of-al-amyloidosis
#11
Z Adam, J Krejčí, M Krejčí, P Němec, L Spinarová, V Zampachová, Z Cermáková, T Pika, L Pour, Z Kořístek, M Tomíška, P Szturz, Z Král, J Mayer
UNLABELLED: Severe damage to the heart caused by AL amyloid deposits is a contraindication of high-dose chemotherapy with autologous haematopoietic stem cell transplantation. Severe heart damage caused by AL amyloid results in frequent life-threatening complications, even during the course of the classical chemotherapy treatment and it often makes keeping to the treatment schedule impossible. Scheduling heart transplantation before the treatment of AL amyloidosis will significantly improve the patients overall condition and enable them to undergo the intensive AL amyloidosis treatment with the hope that a long-term complete remission may be achieved...
February 2013: Vnitr̆ní Lékar̆ství
https://www.readbyqxmd.com/read/22747647/diflunisal-for-attr-cardiac-amyloidosis
#12
Adam Castaño, Stephen Helmke, Julissa Alvarez, Susan Delisle, Mathew S Maurer
Transthyretin (TTR) cardiac amyloidosis is an important, often under-recognized and potentially modifiable cause of heart failure with a preserved ejection fraction. The only proven treatment is liver or combined heart/liver transplantation, which, although effective, is not suitable for the vast majority of older adults with this condition. Diflunisal, a nonsteroidal anti-inflammatory drug, can stabilize the TTR tetramer in vitro and may prevent misfolding monomers and dimers from forming amyloid deposits in the heart...
November 2012: Congestive Heart Failure
https://www.readbyqxmd.com/read/22584381/green-tea-halts-progression-of-cardiac-transthyretin-amyloidosis-an-observational-report
#13
Arnt V Kristen, Stephanie Lehrke, Sebastian Buss, Derliz Mereles, Henning Steen, Philipp Ehlermann, Stefan Hardt, Evangelos Giannitsis, Rupert Schreiner, Uwe Haberkorn, Philipp A Schnabel, Reinhold P Linke, Christoph Röcken, Erich E Wanker, Thomas J Dengler, Klaus Altland, Hugo A Katus
BACKGROUND: Treatment options in patients with amyloidotic transthyretin (ATTR) cardiomyopathy are limited. Epigallocatechin-3-gallate (EGCG), the most abundant catechin in green tea (GT), inhibits fibril formation from several amyloidogenic proteins in vitro. Thus, it might also halt progression of TTR amyloidosis. This is a single-center observational report on the effects of GT consumption in patients with ATTR cardiomopathy. METHODS: 19 patients with ATTR cardiomyopathy were evaluated by standard blood tests, echocardiography, and cardiac MRI (n = 9) before and after consumption of GT and/or green tea extracts (GTE) for 12 months...
October 2012: Clinical Research in Cardiology: Official Journal of the German Cardiac Society
https://www.readbyqxmd.com/read/22573235/light-chain-deposition-disease-affecting-the-gastrointestinal-tract-in-the-setting-of-post-living-donor-kidney-transplantation
#14
Victor H Jimenez-Zepeda, Rajkumar Vajpeyi, Rohan John, Suzanne Trudel
Light chain deposition disease (LCDD) is an uncommon, clonal plasma cell proliferative disorder, in which monoclonal immunoglobulin light chains deposit in various tissues, resulting in organ dysfunction. Gastrointestinal (GI) involvement has been described in both primary and secondary amyloidosis, but has rarely been reported in LCDD, and only as an incidental finding. We report a case of LCDD in living related kidney transplant recipient presenting with severe GI dysmotility, weight loss and progressive allograft dysfunction...
July 2012: International Journal of Hematology
https://www.readbyqxmd.com/read/22551192/doxycycline-plus-tauroursodeoxycholic-acid-for-transthyretin-amyloidosis-a-phase-ii-study
#15
Laura Obici, Andrea Cortese, Alessandro Lozza, J Lucchetti, Marco Gobbi, Giovanni Palladini, Stefano Perlini, Maria J Saraiva, Giampaolo Merlini
We designed a phase II, open-label study to evaluate the efficacy, tolerability, safety, and pharmacokinetics of orally doxycycline (100 mg BID) and tauroursodeoxycholic acid (TUDCA) (250 mg three times/day) administered continuously for 12 months. Primary endpoint is response rate defined as nonprogression of the neuropathy and of the cardiomyopathy. Since July 2010, we enrolled 20 patients. Seventeen patients have hereditary ATTR, two patients have senile systemic amyloidosis, and one is a domino recipient...
June 2012: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/22260742/tocilizumab-improves-cardiac-disease-in-a-hemodialysis-patient-with-aa-amyloidosis-secondary-to-rheumatoid-arthritis
#16
Yoshinari Hattori, Yoshifumi Ubara, Keiichi Sumida, Rikako Hiramatsu, Eiko Hasegawa, Masayuki Yamanouchi, Noriko Hayami, Tatsuya Suwabe, Junichi Hoshino, Naoki Sawa, Kenichi Oohashi, Kenmei Takaichi
A 58-year-old Japanese woman on hemodialysis (HD) was admitted for intractable rheumatoid arthritis. Even after HD was started due to end-stage renal failure in 2004, her arthropathy worsened. A soluble tumor necrosis factor receptor inhibitor (etanercept at 25 mg twice weekly), tacrolimus (2 mg daily), and prednisolone (10 mg daily) had been administered since 2005, but high disease activity had persisted. She was admitted to our hospital in July 2007. C-reactive protein (CRP) was 6.8 mg/dL, and the DAS-CRP score was calculated to be 8...
March 2012: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/21770831/colchicine-sensitive-nephrotic-syndrome-due-to-aa-amyloidosis
#17
Michael J Hausmann, Esther Maor, Leonid Kachko
A 28-year-old women is presented who was evaluated for a new-onset postpartum nephrotic syndrome with normal renal function. Histological diagnosis was AA amyloidosis but no underlying disease has been diagnosed despite extensive workup. Complete remission was achieved with colchicine. Upon discontinuation of colchicines, the patient's nephrotic syndrome flared up but completely responded to reinstitution of colchicine therapy. Remission of this patient's nephrotic syndrome is thus not attributable to resolution of any "idiopathic" primary disease...
September 2011: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/21506142/use-of-plasma-exchange-in-patients-with-heparin-induced-thrombocytopenia-a-report-of-two-cases-and-a-review-of-the-literature
#18
REVIEW
Elizabeth A Jaben, A Sergio Torloni, Rajiv K Pruthi, Jeffrey L Winters
Heparin-induced thrombocytopenia (HIT), which is characterized by thrombocytopenia and potentially serious thromboses, may develop in patients exposed to heparin anticoagulation. HIT is caused by antibodies to the heparin/platelet factor 4 (PF4) complex. Management of HIT involves discontinuation of heparin and anticoagulation with a nonheparin alternative such as a direct thrombin inhibitor (DTI). This poses a challenge in the management of patients who need to undergo cardiopulmonary bypass surgery (CPB), because CPB requires anticoagulation with heparin and standardized protocols for use of DTIs are not widely available...
2011: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/21239830/dealing-with-neuropathy-in-plasma-cell-dyscrasias
#19
REVIEW
Pieter Sonneveld, Joost L M Jongen
Peripheral neuropathy (PN) is a frequent complication of plasma-cell dyscrasias such as monoclonal gammopathy of undetermined significance, multiple myeloma, Waldenström's disease, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, Castleman's disease, and light-chain amyloidosis. PN can be associated with the underlying disease or it can related to the treatment. The novel immunomodulatory drugs thalidomide and lenalidomide and the proteasome inhibitor bortezomib have changed the standard treatment of multiple myeloma...
2010: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/20872958/discordance-between-serum-cardiac-biomarker-and-immunoglobulin-free-light-chain-response-in-patients-with-immunoglobulin-light-chain-amyloidosis-treated-with-immune-modulatory-drugs
#20
COMPARATIVE STUDY
Angela Dispenzieri, David Dingli, Shaji K Kumar, S Vincent Rajkumar, Martha Q Lacy, Suzanne Hayman, Frances Buadi, Stephen Zeldenrust, Nelson Leung, Kristen Detweiler-Short, John A Lust, Stephen J Russell, Robert A Kyle, Morie A Gertz
We evaluated the capability of soluble cardiac biomarkers to predict tolerability and outcomes of IMiD-containing treatments among 106 patients treated on clinical trials. Baseline elevations in troponin T (TnT) and N-terminal brain naturietic protein (NT-proBNP) predicted for an inability to tolerate IMiD-based regimens. The best predictors for early attrition during cycle 1 were TnT ≥ 0.07 μg/L and NT-proBNP ≥ 11,939 ng/L. NT-proBNP-response underperformed TnT-response as a predictor for overall survival (OS), but both predicted for early protocol attrition...
October 2010: American Journal of Hematology
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