Read by QxMD icon Read

Discontinuation of treatment amyloidosis

Cinzia Marchica, Faisal Zawawi, Dania Basodan, Rosie Scuccimarri, Sam J Daniel
BACKGROUND: Muckle-Wells syndrome (MWS) is a rare auto-inflammatory disease characterized by the presence of recurrent urticaria, deafness and amyloidosis. Progressive sensorineural hearing loss (SNHL) is reported to occur in up to 85% of patients occurring in the second and third decades and as early as the first decade in patients with a more severe phenotype, thus potentially having a significant impact on a child's development. IL-1 inhibitors, such as Anakinra, have been described to improve systemic inflammation, and stabilize or improve hearing status as well...
January 30, 2018: Journal of Otolaryngology—Head & Neck Surgery
James C Melville, Kelsey D Menegotto, Timothy C Woernley, Blake D Maida, Ibrahim Alava
Macroglossia is classified as true macroglossia, which exhibits abnormal histology with clinical findings, and relative macroglossia, in which normal histology does not correlate with pathologic enlargement. This report describes an atypical case of morbidity with massive macroglossia secondary to myxedema; the macroglossia enlarged over a 3-month period before being presented to the Department of Oral and Maxillofacial Surgery, University of Texas Health Science Center at Houston (Houston, TX). Substantial enlargement of the tongue (16 cm long × 10 cm wide) was first attributed to angioedema, which was refractory to the discontinuation of lisinopril and a C1 esterase inhibitor...
January 2018: Journal of Oral and Maxillofacial Surgery
Johanna Denman, Kaveh Manavi, Mark Cook
We present a case of an HIV-positive man with systemic immunoglobulin light chain (AL) amyloid with cardiac involvement. At relapse, he was treated with lenalidomide and dexamethasone having previously developed autonomic neuropathy with bortezomib-based chemotherapy. The patient achieved a serological complete response with symptomatic improvement. After 11 cycles, lenalidomide was discontinued due to extensive ischaemia of the gastrointestinal tract. The patient remains symptomatically stable with normal levels of serum-free light chains 11 months after the treatment was discontinued...
September 2017: International Journal of STD & AIDS
Yasunobu Sekiguchi, Haruko Takizawa, Tadaaki Inano, Yasutaka Fukuda, Mutsumi Wakabayashi, Keiji Sugimoto, Shigeki Tomita, Hiroshi Izumi, Noriko Nakamura, Tomohiro Sawada, Yasunori Ohta, Norio Komatsu, Masaaki Noguchi
Three patients under hemodialysis (HD) with relapsed/refractory multiple myeloma (MM) were administered panobinostat/bortezomib/dexamethasone (FVD). Case 1: The patient was a 66-year-old male with BJP-κ. FVD was effective, but HD could not be discontinued. He developed Grade 3 adverse events (AEs), including nausea, dehydration, and fatigue, following the common terminology criteria for adverse events v4.0. FVD was discontinued after the third course, while HD was continued. Case 2: The patient was a 65-year-old female with IgG-λ + BJP-λ...
October 2017: International Journal of Hematology
Erhan Tatar, Adam Uslu, Cenk Simsek, Ahmet Aykas, Giray Bozkaya, Cetin Imamoglu
Fever of unknown origin is a rare condition after solid organ transplant and is generally associated with atypical infections (eg, tuberculosis, fungal infections) and/or lymphoproliferative disorders. Here, we present a kidney transplant patient with a late diagnosis of E148Q mutation-positive familial Mediterranean fever as the cause of fever of unknown origin. A 22-year-old female patient with a previous history of 4 years of hemodialysis and unknown primary renal disease received a deceased-donor kidney transplant at our center 5 years previously...
February 2017: Experimental and Clinical Transplantation
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%...
December 1, 2016: JAMA Neurology
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"