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AL amyloidosis treatment

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https://www.readbyqxmd.com/read/28103898/the-burden-of-amyloid-light-chain-amyloidosis-on-health-related-quality-of-life
#1
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
#2
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
#3
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 1980's. 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...
January 10, 2017: Human Pathology
https://www.readbyqxmd.com/read/28031465/aggregation-of-full-length-immunoglobulin-light-chains-from-al-amyloidosis-patients-is-remodeled-by-epigallocatechin-3-gallate
#4
Kathrin Andrich, Ute Hegenbart, Christoph Kimmich, Niraja Kedia, H Robert Bergen, Stefan Schönland, Erich E Wanker, Jan Bieschke
Intervention into amyloid deposition with anti-amyloid agents like the polyphenol Epigallocatechin-3-gallate (EGCG) is emerging as an experimental secondary treatment strategy in systemic light chain amyloidosis (AL). In both AL and Multiple Myeloma (MM), soluble immunoglobulin light chains (LC) are produced by clonal plasma cells, but only in AL they form amyloid deposits in vivo. We investigated the amyloid formation of patient-derived LC and their susceptibility to EGCG in vitro to probe commonalities and systematic differences in their assembly mechanisms...
December 28, 2016: Journal of Biological Chemistry
https://www.readbyqxmd.com/read/28002971/new-and-developing-therapies-for-al-amyloidosis
#5
Giulia Zumbo, Omid Sadeghi-Alavijeh, Philip N Hawkins, Marianna Fontana
Systemic light-chain (AL) amyloidosis is an infiltrative disorder associated with an underlying plasma cells dyscrasia, in which monoclonal immunoglobulin light chains accumulate in an abnormal misfolded form as amyloid fibrils in the extracellular space. Symptoms and prognosis are governed by which organs are affected, and cardiac involvement is the major determinant of survival. Diagnosis requires demonstration of amyloid deposition and confirmation of the fibril protein type. Areas covered: This review will focus on the available treatments for systemic AL amyloidosis and on new drug targets and therapeutic approaches...
December 29, 2016: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/27965280/longitudinal-systolic-strain-cardiac-function-improvement-and-survival-following-treatment-of-light-chain-al-cardiac-amyloidosis
#6
Francesco Salinaro, Hans K Meier-Ewert, Edward J Miller, Shivda Pandey, Vaishali Sanchorawala, John L Berk, David C Seldin, Frederick L Ruberg
AIMS: To determine whether echocardiographic longitudinal systolic strain (LS) parameters identify short-term improvement following chemotherapy for light-chain (AL) cardiac amyloidosis (CA). Among patients with CA, standard echocardiographic measures are commonly unchanged at 1 year following successful chemotherapy, despite observed reductions in cardiac biomarkers. METHODS AND RESULTS: We retrospectively identified 61 patients with AL-CA treated with high-dose melphalan or bortezomib-based regimens...
December 12, 2016: European Heart Journal Cardiovascular Imaging
https://www.readbyqxmd.com/read/27927236/chronic-infantile-neurological-cutaneous-and-articular-cinca-syndrome-a-review
#7
REVIEW
Martina Finetti, Alessia Omenetti, Silvia Federici, Roberta Caorsi, Marco Gattorno
INTRODUCTION: The Chronic Infantile Neurological Cutaneous and Articular (CINCA, or Neonatal-onset multisystem inflammatory disease NOMID) is a rare autoinflammatory disease identified in 1987 by Prieur et al., typically characterized by the triad of skin rash, arthropathy and central nervous system manifestations. It represents the most severe phenotype of the cryopyrin-associated periodic syndrome (CAPS). CLINICAL DESCRIPTION AND ETIOLOGY: The syndrome is due to autosomal dominant gain of function mutations in NLRP3, which encodes a key component of the innate immunity that regulates the activation and secretion of interleukin (IL)-1β...
December 7, 2016: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/27904139/interphase-fluorescence-in-situ-hybridization-in-untreated-al-amyloidosis-has-an-independent-prognostic-impact-by-abnormality-type-and-treatment-category
#8
E Muchtar, A Dispenzieri, S K Kumar, R P Ketterling, D Dingli, M Q Lacy, F K Buadi, S R Hayman, P Kapoor, N Leung, R Chakraborty, W Gonsalves, R Warsame, T V Kourelis, S Russell, J A Lust, Y Lin, R S Go, S Zeldenrust, R A Kyle, S V Rajkumar, M A Gertz
The significance of interphase fluorescence in situ hybridization (iFISH) by regimen type was assessed in 692 immunoglobulin light-chain (AL) amyloidosis patients with iFISH at diagnosis. First-line treatment was categorized as stem cell transplant and three non-transplant regimens. The most common abnormality was t(11;14) (49% of patients) followed by monosomy 13/del(13q) (36%) and trisomies (26%). A lower rate of very good partial response (VGPR) or better was observed in patients with t(11;14) treated with bortezomib-based (52% vs 77%; P=0...
December 16, 2016: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/27900617/new-and-evolving-concepts-regarding-the-prognosis-and-treatment-of-cardiac-amyloidosis
#9
REVIEW
Stefano Perlini, Roberta Mussinelli, Francesco Salinaro
Systemic amyloidoses are rare and proteiform diseases, caused by extracellular accumulation of insoluble misfolded fibrillar proteins. Prognosis is dictated by cardiac involvement, which is especially frequent in light chain (AL) and in transthyretin variants (ATTR, both mutated, (ATTRm), and wild-type, (ATTRwt)). Recently, ATTRwt has emerged as a potentially relevant cause of a heart failure with preserved ejection fraction (HFpEF). Cardiac amyloidosis is an archetypal example of restrictive cardiomyopathy, with signs and symptoms of global heart failure and diastolic dysfunction...
December 2016: Current Heart Failure Reports
https://www.readbyqxmd.com/read/27885144/treatment-of-multiple-myeloma-in-a-heart-transplant-recipient-focus-on-the-immunomodulatory-effects-of-lenalidomide
#10
Lola Xie, Bartosz Jozwik, Phillip Weeks, L Maximilian Buja, Robert Brown, Sriram Nathan, Keshava Rajagopal, Christina Paruthi, Biswajit Kar, Pranav Loyalka, Indranee Rajapreyar
Malignancy following solid organ transplant remains a significant threat to the survival of cardiac transplant recipients. Plasma cell dyscrasias including multiple myeloma have been encountered in this population, and medication treatments traditionally used to treat these disorders demonstrate immunomodulatory effects that may have implications on the transplanted allograft. Lenalidomide is an immunomodulatory agent that has been used to treat plasma cell disorders, including light-chain amyloidosis (AL) and multiple myeloma, and represents such a class of medications in which the risks and benefits in the solid organ transplant population remain to be fully elucidated...
November 24, 2016: Progress in Transplantation
https://www.readbyqxmd.com/read/27879147/pre-transplantation-novel-agent-induction-predicts-progression-free-survival-for-patients-with-immunoglobulin-light-chain-amyloidosis-undergoing-high-dose-melphalan-and-autologous-stem-cell-transplantation
#11
Andrew J Cowan, Zandra K Klippel, Philip A Stevenson, Teresa S Hyun, Sherilyn Tuazon, Pamela S Becker, Damian J Green, Leona A Holmberg, David G Coffey, Ajay K Gopal, Edward N Libby
INTRODUCTION: High-dose melphalan and autologous stem cell transplantation (HDM/SCT) is an effective treatment modality for immunoglobulin light-chain (AL) amyloidosis; however, its application remains restricted to patients with good performance status and limited organ involvement. In recent years, the paradigm for AL amyloidosis has changed with the introduction of novel agents such as immunomodulatory drugs (IMiDs) and proteasome inhibitors (PIs). We hypothesized that use of novel agent induction regimens has improved outcomes for patients with AL amyloidosis undergoing HDM/SCT at our center...
December 2016: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/27832515/bortezomib-dexamethasone-versus-high-dose-melphalan-for-japanese-patients-with-systemic-light-chain-al-amyloidosis-a-retrospective-single-center-study
#12
Nagaaki Katoh, Akihiro Ueno, Takuhiro Yoshida, Ko-Ichi Tazawa, Yasuhiro Shimojima, Takahisa Gono, Yoshiki Sekijima, Masayuki Matsuda, Shu-Ichi Ikeda
Bortezomib-dexamethasone (BD) and high-dose melphalan (HDM) are effective for systemic light-chain (AL) amyloidosis, but have not been compared in detail. We retrospectively investigated patients treated with BD or HDM at our center between September 2001 and June 2016. Among 234 patients, 20 were treated with BD and 30 received HDM. With the exception of age, transplant eligibility, and previous history of other chemotherapy, there were no significant differences in most background parameters between the two groups...
November 10, 2016: International Journal of Hematology
https://www.readbyqxmd.com/read/27825470/monoclonal-igm-related-al-amyloidosis
#13
REVIEW
Paolo Milani, Giampaolo Merlini
Monoclonal immunoglobulin M (IgM)-related light chain (AL) amyloidosis, which accounts for 5%-7% of all AL amyloidosis cases, is a distinct clinical entity that poses specific challenges to clinicians. Several studies reported that although there is a substantial overlap, the pattern of organ involvement is peculiar, with higher frequencies of lung, lymph nodes, and peripheral nervous system involvement. A recent collaborative study from three European referral centers, defined that cardiac involvement, advanced Mayo disease stage, neuropathic, and liver involvement were independent factors that had impact on survival in IgM-AL amyloidosis patients...
June 2016: Best Practice & Research. Clinical Haematology
https://www.readbyqxmd.com/read/27815860/phase-2-trial-of-daily-oral-epigallocatechin-gallate-in-patients-with-light-chain-amyloidosis
#14
Sohsuke Meshitsuka, Sumito Shingaki, Masatoshi Hotta, Miku Goto, Makoto Kobayashi, Yuuichi Ukawa, Yuko M Sagesaka, Yasuyo Wada, Masanori Nojima, Kenshi Suzuki
Previous studies have suggested that an increase in mitochondrial reactive oxygen species may cause organ damage in patients with light-chain (AL) amyloidosis; however, this damage can be decreased by antioxidant-agent treatment. Epigallocatechin gallate (EGCG), the major natural catechin in green tea, has potent antioxidant activity. Because EGCG has recently been reported to have a favorable toxicity profile for treating amyloidosis, we sought to examine the clinical efficacy and toxicity of EGCG in patients with AL amyloidosis...
November 4, 2016: International Journal of Hematology
https://www.readbyqxmd.com/read/27804150/progressive-refractory-light-chain-amyloidosis-and-multiple-myeloma-patients-are-responsive-to-the-addition-of-clarithromycin-to-imid-based-therapy
#15
Adir Shaulov, Chezi Ganzel, Noam Benyamini, Yossef Barshay, Neta Goldschmidt, David Lavie, Diana Libster, Alex Gural, Batia Avni, Moshe E Gatt
Multiple myeloma (MM) and primary systemic light chain amyloidosis (AL) are both chronic plasma cell dyscrasias with different clinical expression but limited treatment options for relapsed refractory disease. We report the effect of the addition of clarithromycin on 31 MM and 17 AL with relapsed or refractory disease who had an insufficient response or disease progression while on an IMiD based therapy. In this high risk population, hematological response was reported in 48% of MM patients and 94% of AL patients...
November 2, 2016: American Journal of Hematology
https://www.readbyqxmd.com/read/27780577/bendamustine-induced-nephrogenic-diabetes-insipidus-in%C3%A2-a%C3%A2-patient-with-al-amyloidosis
#16
Nsabimana A Uwumugambi, Vaishali Sanchorawala, Anthony C Shelton, Lauren Stern, Craig E Gordon
Nephrogenic diabetes insipidus is a condition characterized by polyuria with dilute urine due to the inability of the principal cells of the renal collecting ducts to respond to antidiuretic hormone and concentrate urine. Nephrogenic diabetes insipidus can be drug induced, and several chemotherapeutic agents have been reported to cause it. Bendamustine is a traditional chemotherapeutic agent being studied for treatment for relapsed systemic AL amyloidosis. We report a case of a 59-year-old man with AL amyloidosis who developed partial nephrogenic diabetes insipidus after receiving bendamustine for treatment of AL amyloidosis...
October 22, 2016: American Journal of Kidney Diseases: the Official Journal of the National Kidney Foundation
https://www.readbyqxmd.com/read/27764008/primary-laryngotracheal-amyloidosis-with-bilateral-vocal-cord-involvement-and-associated-bronchiectasis
#17
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
https://www.readbyqxmd.com/read/27729322/the-prognostic-value-of-multiparametric-flow-cytometry-in-al-amyloidosis-at-diagnosis-and-at-the-end-of-first-line-treatment
#18
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
https://www.readbyqxmd.com/read/27696268/immunoglobulin-light-chain-systemic-amyloidosis
#19
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
https://www.readbyqxmd.com/read/27694943/outcome-of-al-amyloidosis-after-high-dose-melphalan-and-autologous-stem-cell-transplantation-in-sweden-long-term-results-from-all-patients-treated-in-1994-2009
#20
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...
December 2016: Bone Marrow Transplantation
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