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AL amyloidosis treatment

Charles W Lanks, Timothy L Van Natta, David W Hsia
Localized tracheobronchial amyloidosis is a rare disease that results from submucosal deposition of insoluble amyloid proteins in the large airways. Amyloidosis affecting the larynx and subglottic space typically results in unilateral, nodular vocal cord infiltration. It rarely can present with bilateral vocal cord involvement and can progress to lifethreatening respiratory failure due to upper airway obstruction. In these patients, typical treatment modalities such as CO2 laser ablation are often ineffectual...
October 2016: Journal of Bronchology & Interventional Pulmonology
Eli Muchtar, Dragan Jevremovic, Angela Dispenzieri, David Dingli, Francis K Buadi, Martha Q Lacy, Wilson Gonsalves, Suzanne R Hayman, Prashant Kapoor, Nelson Leung, Stephen Russell, John A Lust, Yi Lin, Ronald S Go, Rajshekhar Chakraborty, Steven Zeldenrust, Shaji K Kumar, Robert A Kyle, S Vincent Rajkumar, Morie A Gertz
Multiparametric flow cytometry (MFC) in AL amyloidosis has not been widely adopted and consequently there is little information on its clinical relevance. We studied 173 AL amyloidosis patients who underwent MFC immunophenotyping of bone marrow sample at diagnosis and 82 patients at the end of the first line of treatment (EOT). The number of monotypic plasma cells (PCs) and the polytypic PCs/bone marrow PCs (pPCs/BMPCs) ratio were analyzed. At diagnosis, ≥2.5% monotypic PCs was associated with a shorter progression-free survival (PFS) and overall survival (OS) compared to patients with <2...
October 11, 2016: Blood
Angela Dispenzieri, Giampaolo Merlini
Immunoglobulin light chain amyloidosis (AL) is a rare, complex disease caused by misfolded free light chains produced by a usually small, indolent plasma cell clone. Effective treatments exist that can alter the natural history, provided that they are started before irreversible organ damage has occurred. The cornerstones of the management of AL amyloidosis are early diagnosis, accurate typing, appropriate risk-adapted therapy, tight follow-up, and effective supportive treatment. The suppression of the amyloidogenic light chains using the cardiac biomarkers as guide to choose chemotherapy is still the mainstay of therapy...
2016: Cancer Treatment and Research
S Rosengren, U-H Mellqvist, H Nahi, K Forsberg, S Lenhoff, O Strömberg, L Ahlberg, O Linder, K Carlson
High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality (24% in a randomised study comparing HDM/ASCT with oral melphalan/dexamethasone). We report here on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. Seventy-two patients were treated at eight Swedish centres. Median follow-up was 67...
October 3, 2016: Bone Marrow Transplantation
Kanji Miyazaki, Kenshi Suzuki
Capillary electrophoresis/immunosubtraction (CE/IS) is a simple method for detecting and immunotyping serum or urine monoclonal proteins. To our knowledge, there are no previous reports of the use of CE/IS for characterizing patients with Immunoglobulin light chain (AL) amyloidosis, and there are no convincing data available to compare CE/IS with serum immunofixation electrophoresis (IFE) and free light chain (FLC) assay. The aim of this study was to evaluate the clinical utility of CE/IS in patients with AL amyloidosis as a diagnostic accuracy study...
September 28, 2016: Amyloid: the International Journal of Experimental and Clinical Investigation
T Jelinek, E Kryukova, Z Kufova, F Kryukov, R Hajek
Proteasome inhibitors are the backbone in the treatment of multiple myeloma with 3 of its representatives (bortezomib, carfilzomib, and ixazomib) having already been approved. There is a different situation altogether in the treatment of amyloid light chain (AL) amyloidosis where owing to the rarity of this entity neither of these drugs has currently gained approval. Amyloid light chain plasma cells are possibly more vulnerable to bortezomib than myeloma plasmocytes because of a slightly distinct mechanism of action, which is described in depth in this manuscript...
September 20, 2016: Hematological Oncology
Seok Jin Kim, Hyun-Tae Shin, Hae-Ock Lee, Nayoung K D Kim, Jae Won Yun, Jee Hyang Hwang, Kihyun Kim, Woong-Yang Park
Clinically applicable platforms revealing actionable genomic alterations may improve the treatment efficacy of myeloma patients. In this pilot study, we used a high depth targeted sequencing panel containing 83 anti-cancer drug target genes to sequence genomic DNAs extracted from bone marrow aspirates of 23 patients with myeloma and 12 patients with amyloid light-chain (AL) amyloidosis. Mutation analysis revealed NRAS as the most commonly mutated gene (30%, 7/23) in myeloma patients followed by KRAS (26%, 6/23) and BRAF (22%, 5/23)...
September 15, 2016: Oncotarget
Rodney H Falk, Kevin M Alexander, Ronglih Liao, Sharmila Dorbala
The amyloidoses are a group of protein-folding disorders in which ≥1 organ is infiltrated by proteinaceous deposits known as amyloid. The deposits are derived from 1 of several amyloidogenic precursor proteins, and the prognosis of the disease is determined both by the organ(s) involved and the type of amyloid. Amyloid involvement of the heart (cardiac amyloidosis) carries the worst prognosis of any involved organ, and light-chain (AL) amyloidosis is the most serious form of the disease. The last decade has seen considerable progress in understanding the amyloidoses...
September 20, 2016: Journal of the American College of Cardiology
Yoshitaka Kikukawa, Hiroyuki Hata, Mitsuharu Ueda, Taro Yamashita, Singo Nasu, Kazuhiko Ide, Shikiko Ueno, Yukio Ando, Hiroaki Mitsuya, Yutaka Okuno
A 70-year-old woman with Charcot-Marie-Tooth disease (CMT) suffered from nephrotic syndrome and a renal biopsy revealed non-AA amyloid depositions that contained immunoglobulin light chain λ. Her serum λ free LC was elevated to 80.8 mg/L and she was diagnosed with primary amyloid light-chain (AL) amyloidosis. She was subsequently treated with lenalidomide, cyclophosphamide, and dexamethasone (RCD). After 14 cycles of RCD, she achieved complete remission. Her serum albumin levels gradually normalized to 3...
2016: Internal Medicine
Xia Wu, Jun Feng, Xinxin Cao, Lu Zhang, Daobin Zhou, Jian Li
BACKGROUND: Primary immunoglobulin light chain amyloidosis (AL amyloidosis) is a plasma cell disorder which mainly affects heart, kidneys, liver, and peripheral nervous system. Cases of atypical AL amyloidosis presented as spontaneous vertebral compression fractures have been rarely reported, and data about the management and clinical outcomes of the patients are scarce. METHODS: Herein, we present 3 new cases of AL amyloidosis with spontaneous vertebral compression fracture and review 13 cases retrieved from the literature...
September 2016: Medicine (Baltimore)
Yutaka Tsukune, Yuriko Yahata, Makoto Sasaki, Makoto Hiki, Miyuki Tsutsui, Yasuharu Hamano, Seigo Itoh, Tetsuro Miyazaki, Tomotaka Dohi, Masaki Maruyama, Akihiko Gotoh, Norio Komatsu
Cardiac amyloid light-chain amyloidosis (AL amyloidosis) is a rare disease with a very poor prognosis, associated with plasma cell dyscrasias such as monoclonal gammopathy of undetermined significance and multiple myeloma. Though bortezomib-containing regimens have achieved high hematologic response rates, there are still few reports describing the outcomes of Japanese patients. Six patients with severe cardiac AL amyloidosis were treated with bortezomib-containing regimens. Involved free light chain (iFLC) decreased immediately in most of these cases...
August 2016: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
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 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)...
August 18, 2016: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
Mario Nuvolone, Giampaolo Merlini
Systemic amyloidosis is caused by misfolding and extracellular deposition of one of an ever-growing list of circulating proteins, resulting in vital organ dysfunction and eventually death. Despite different predisposing conditions, including plasma cell dyscrasias [immunoglobulin light chain (AL) amyloidosis], long-lasting inflammation [reactive (AA) amyloidosis] or mutations (hereditary amyloidoses), clinical manifestations are conspicuously overlapping and mimic more prevalent conditions, significantly complicating and often delaying the recognition of these rare, complex diseases...
August 18, 2016: Nephrology, Dialysis, Transplantation
Delphine Gobert, Romain Paule, Denise Ponard, Pierre Levy, Véronique Frémeaux-Bacchi, Laurence Bouillet, Isabelle Boccon-Gibod, Christian Drouet, Stéphane Gayet, David Launay, Ludovic Martin, Arsène Mekinian, Véronique Leblond, Olivier Fain
Acquired angioedema (AAE) due to C1-inhibitor (C1INH) deficiency is rare. Treatment options for acute attacks are variable and used off-label. Successful treatment of the associated lymphoma with rituximab seems to prevent acute attacks in subjects with AAE. The aim of this study was to describe AAE manifestations, its associated diseases, and patients' responses to treatments in a representative cohort.A retrospective nationwide study was conducted in France. The inclusion criteria were recurrent angioedema attacks and an acquired decrease in functional C1INH <50% of the reference value...
August 2016: Medicine (Baltimore)
Hans L A Nienhuis, Johan Bijzet, Bouke P C Hazenberg
BACKGROUND: Amyloidosis has been a mystery for centuries, but research of the last decennia has clarified many of the secrets of this group of diseases. A protein-based classification of amyloidosis helps to understand problems that were part of the obsolete clinical classification in primary, secondary, and familial amyloidosis. All types of amyloid are secondary to some underlying precursor-producing process: each type is caused by a misfolded soluble precursor protein that becomes deposited as insoluble amyloid fibrils...
April 2016: Kidney Diseases
Xiang-Hua Huang, Zhi-Hong Liu
BACKGROUND: Amyloidosis includes a group of diseases characterized by the extracellular deposition of various fibrillary proteins that can autoaggregate in a highly abnormal fibrillary conformation. The amyloid precursor protein of systemic light-chain (AL) amyloidosis is comprised of monoclonal light chains that are due to plasma cell dyscrasia. The clinical presentation of patients with AL amyloidosis varies from patient to patient. Current treatment strategies target the clone in order to decrease the production of the pathologic light chains...
April 2016: Kidney Diseases
Taxiarchis V Kourelis, Samih H Nasr, Angela Dispenzieri, Shaji K Kumar, Morie A Gertz, Fernando C Fervenza, Francis K Buadi, Martha Q Lacy, Stephen B Erickson, Fernando G Cosio, Prashant Kapoor, John A Lust, Suzanne R Hayman, Vincent Rajkumar, Steven R Zeldenrust, Stephen J Russell, David Dingli, Yi Lin, Wilson Gonsalves, Elizabeth C Lorenz, Ladan Zand, Robert A Kyle, Nelson Leung
Recent reports suggest that deep hematologic responses to chemotherapy are associated with improved renal outcomes in monoclonal immunoglobulin deposition disease (MIDD). Here we describe the long term outcomes and identify prognostic factors after first line treatment of the largest reported series of patients with MIDD. Between March 1992 and December 2014, 88 patients with MIDD were seen at Mayo Clinic, MN. Renal responses were defined using criteria used for light chain amyloidosis (AL) or those used by the IMWG...
August 8, 2016: American Journal of Hematology
Rajshekhar Chakraborty, Eli Muchtar, Morie A Gertz
The heart and the kidneys are the most commonly involved organs in systemic amyloidosis. Cardiac involvement is associated with an increased morbidity, treatment intolerance, and poorer overall survival. The most common types of amyloidosis that are associated with cardiac involvement include light chain (AL) amyloidosis and transthyretin (TTR) amyloidosis (both mutant and wild type). The traditional first-line treatment for AL amyloidosis includes alkylator-based chemotherapy or high-dose melphalan followed by autologous stem cell transplantation (ASCT)...
August 6, 2016: Current Heart Failure Reports
Mark Renz, Ronald Torres, Philip J Dolan, Stephen J Tam, Jose R Tapia, Lauri Li, Joshua R Salmans, Robin M Barbour, Paul J Shughrue, Tarlochan Nijjar, Dale Schenk, Gene G Kinney, Wagner Zago
Amyloid light chain (AL) amyloidosis is characterized by misfolded light chain (LC) (amyloid) deposition in various peripheral organs, leading to progressive dysfunction and death. There are no regulatory agency-approved treatments for AL amyloidosis, and none of the available standard of care approaches directly targets the LC protein that constitutes the amyloid. NEOD001, currently in late-stage clinical trials, is a conformation-specific, anti-LC antibody designed to specifically target misfolded LC aggregates and promote phagocytic clearance of AL amyloid deposits...
August 5, 2016: Amyloid: the International Journal of Experimental and Clinical Investigation
Diane Bodez, Arnault Galat, Aziz Guellich, Jean-François Deux, Jean Rosso, Fabien Le Bras, Benoît Funalot, Pascale Fanen, Nicole Benhaiem, Violaine Planté-Bordeneuve, Jean-Luc Dubois-Randé, Nicolas Lellouche, Soulef Guendouz, Valérie Molinier-Frenkel, Dania Mohty, Thibaud Damy
Cardiac amyloidosis must be suspected in all cases of hypertrophic cardiomyopathy with preserved left ventricular ejection fraction to allow specific management. Final diagnosis needs pathological evidence, but bone scintigraphy may be an alternative for TTR amyloidosis. Invasive samplings are limited by new tools. Amyloidosis typing is required to start specific therapies if possible. Main specific treatments that are available are chemotherapy for AL; transthyretin stabilizer or gene therapy, studied for TTR-related cardiac amyloidosis...
August 1, 2016: La Presse Médicale
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