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Amyloidosis AL

Felicia D'Alitto, Amina Scherz, Cristina Margini, Hendrik Von Tengg-Kobligk, Matteo Montani, Thomas Pabst, Annalisa Berzigotti
Portal hypertension (PH) is a common clinical syndrome leading to severe complications. In the western world, about 90% of cases of PH are due to liver cirrhosis, and thanks to the availability of ultrasound elastography methods, this diagnosis is usually confirmed at bedside. We report a case of a patient presenting with PH and ascites initially suspected of suffering from liver cirrhosis. The finding of large hepatomegaly and a massive increase in liver stiffness prompted us to perform a liver biopsy. This revealed no fibrosis, but diffuse primary amyloidosis (AL amyloidosis)...
June 8, 2018: Curēus
Faye A Sharpley, Richa Manwani, Shameem Mahmood, Sajitha Sachchithanantham, Helen Lachmann, Julian Gilmore, Carol Whelan, Phillip Hawkins, Ashutosh Wechalekar
Pomalidomide is a next-generation immunomodulatory agent with activity in relapsed light chain (AL) amyloidosis, but real world outcomes are lacking. We report the experience of the UK National Amyloidosis Centre. All patients with AL amyloidosis treated with pomalidomide between 2009 and 2017 were included. Data was collected on treatment toxicity and clonal response. Survival was calculated by the Kaplan-Meier method and outcomes reported on an intent-to-treat (ITT) basis. A total of 29 patients treated with pomalidomide were identified...
August 10, 2018: British Journal of Haematology
Cindy Varga, Raymond L Comenzo
The application of high-dose melphalan and autologous stem cell transplant (SCT) to systemic AL amyloidosis (AL) has evolved over the past two decades and remains an important component of therapy for patients with AL. The era of novel agents created the opportunity to provide well -tolerated induction and post-SCT consolidation to AL patients eligible for SCT and the current availability of new monoclonal antibody therapies will likely provide additional opportunities to enhance the outcomes with SCT. In this review, we touch on the history of SCT for AL and examine the data on eligibility, mobilization, induction, risk-adapted melphalan dosing, engraftment, consolidation and maintenance, and long-term outcomes with SCT...
August 8, 2018: Bone Marrow Transplantation
Luis M Blancas-Mejia, Pinaki Misra, Christopher J Dick, Shawna A Cooper, Keely R Redhage, Michael R Bergman, Torri L Jordan, Khansaa Maar, Marina Ramirez-Alvarado
Light chain (AL) amyloidosis is a devastating, complex, and incurable protein misfolding disease. It is characterized by an abnormal proliferation of plasma cells (fully differentiated B cells) producing an excess of monoclonal immunoglobulin light chains that are secreted into circulation, where the light chains misfold, aggregate as amyloid fibrils in target organs, and cause organ dysfunction, organ failure, and death. In this article, we will review the factors that contribute to AL amyloidosis complexity, the findings by our laboratory from the last 16 years and the work from other laboratories on understanding the structural, kinetics, and thermodynamic contributions that drive immunoglobulin light chain-associated amyloidosis...
August 8, 2018: Chemical Communications: Chem Comm
Efstathios D Pagourelias, Oana Mirea, Georgios Vovas, Jürgen Duchenne, Blazej Michalski, Johan Van Cleemput, Jan Bogaert, Vasilios P Vassilikos, Jens-Uwe Voigt
Aims: We aimed at investigating the relationship between segmental morphology [wall thickness (WT) and WT location (LT: base-mid-apex)], loading conditions and underlying pathological substrate [histology (H), hypertrophy vs. infiltration] with segmental longitudinal (SLS) and circumferential (SCS) strain in a group of patients with hypertrophic cardiomyopathy (HCM) and cardiac amyloidosis (CA). Methods and results: We included 30 patients with biopsy-proven CA (65...
August 1, 2018: European Heart Journal Cardiovascular Imaging
Anja Seckinger, Jens Hillengass, Martina Emde, Susanne Beck, Christoph Kimmich, Tobias Dittrich, Michael Hundemer, Anna Jauch, Ute Hegenbart, Marc-Steffen Raab, Anthony D Ho, Stefan Schönland, Dirk Hose
Monoclonal antibodies against the cell surface antigen CD38, e.g., isatuximab, daratumumab, or Mor202, have entered the therapeutic armamentarium in multiple myeloma due to single agent overall response rates of 29 vs. 36 vs. 31%, effectivity in combination regimen, e.g., with lenalidomide or bortezomib plus dexamethasone, and tolerable side effects. Despite clinical use, many questions remain. In this manuscript, we address three of these: first, upfront CD38 target-expression in AL-amyloidosis, monoclonal gammopathy of unknown significance (MGUS), asymptomatic, symptomatic, and relapsed multiple myeloma...
2018: Frontiers in Immunology
Sara Bringhen, Alberto Milan, Claudio Ferri, Ralph Wäsch, Francesca Gay, Alessandra Larocca, Marco Salvini, Evangelos Terpos, Hartmut Goldschmidt, Michele Cavo, Maria Teresa Petrucci, Heinz Ludwig, Holger W Auner, Jo Caers, Martin Gramatzki, Mario Boccadoro, Hermann Einsele, Pieter Sonneveld, Monika Engelhardt
Cardiovascular disease in myeloma patients may derive from factors unrelated to the disease (age, diabetes, dyslipidemia, obesity, prior cardiovascular diseases), related to the disease (cardiac AL-amyloidosis, hyperviscosity, high-output failure, arteriovenous shunting, anemia, renal dysfunction) and linked to antimyeloma treatment (anthracyclines, corticosteroids, alkylating agents, immunomodulatory drugs, proteasome inhibitors). An accurate knowledge of cardiovascular events, effective dose reductions, prevention and management of early and late cardiovascular side effects of chemotherapeutic agents are essential in current clinical practice...
July 26, 2018: Haematologica
Antonio Vitarelli, Silvia Lai, Maria Teresa Petrucci, Carlo Gaudio, Lidia Capotosto, Enrico Mangieri, Serafino Ricci, Giuseppe Germanò, Simone De Sio, Giovanni Truscelli, Federico Vozella, Mario Sergio Pergolini, Marcello Giordano
BACKGROUND: Given that in patients with cardiac amyloidosis (CA), deposition of amyloid protein is not restricted to the left ventricular (LV) myocardium, it can be hypothesized that the diagnostic value of deformation mechanics would be enhanced by considering right ventricular (RV) strain measures. The aim of the present study was to examine the potential utility of left ventricular (LV) and right ventricular (RV) deformation and rotational parameters derived from three-dimensional speckle-tracking echocardiograph (3DSTE) to diagnose cardiac amyloidosis and differentiate this disease from other forms of myocardial hypertrophy...
July 23, 2018: International Journal of Cardiology
Naveed Malek, Dominic G O'Donovan, Hadi Manji
An elderly Caucasian man presented with a 10-month history of proximal myopathy and dysphagia. His serum creatine kinase (CK) was elevated at 877 U/L (normal 40-320) and electromyography confirmed a myopathic process. Blood and urine tests suggested myeloma; bone marrow examination showed 30% plasma cells and stained positive for amyloid. The muscle biopsy was initially reported as normal but in the light of the bone marrow report, the biopsy specimen was stained for amyloid, which was positive. We diagnosed systemic amyloidosis causing a myopathy and have started treatment for myeloma...
July 24, 2018: Practical Neurology
Morie A Gertz
DISEASE OVERVIEW: Immunoglobulin light chain amyloidosis is a clonal, nonproliferative plasma cell disorder in which fragments of immunoglobulin light or heavy chain are deposited in tissues. Clinical features depend on organs involved but can include restrictive cardiomyopathy, nephrotic syndrome, hepatic dysfunction, peripheral/autonomic neuropathy, and "atypical multiple myeloma." DIAGNOSIS: Tissue biopsy stained with Congo red demonstrating amyloid deposits with apple-green birefringence is required for diagnosis...
July 24, 2018: American Journal of Hematology
Maria Gavriatopoulou, Pellegrino Musto, Jo Caers, Giampaolo Merlini, Efstathios Kastritis, Niels van de Donk, Francesca Gay, Ute Hegenbart, Roman Hajek, Sonja Zweegman, Benedetto Bruno, Christian Straka, Meletios A Dimopoulos, Hermann Einsele, Mario Boccadoro, Pieter Sonneveld, Monika Engelhardt, Evangelos Terpos
The introduction of novel agents in the management of multiple myeloma and related plasma cell dyscrasias has changed our treatment approaches and subsequently the outcome of patients. Due to current advances, the European Myeloma Network updated the diagnostic and therapeutic recommendations for patients with Waldenström's macroglobulinemia (WM), AL-amyloidosis, monoclonal immunoglobulin deposition disease (MIDD), POEMS syndrome, and primary plasma cell leukemia. For patients with WM, the combination of rituximab with chemotherapy remains the treatment cornerstone, while the Bruton-tyrosine kinase inhibitor ibrutinib has been introduced and approved for relapsed/refractory disease...
July 23, 2018: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
M Hasib Sidiqi, Morie A Gertz
Autologous stem cell transplantation (ASCT) has been used as treatment for immunoglobulin light-chain (AL) amyloidosis for over two decades with improving outcomes; however, the majority of patients are not candidates for this therapy at diagnosis. Novel agents such as immunomodulatory drugs, proteasome inhibitors, and immunotherapy with monoclonal antibodies targeting CD38 have been adopted from the multiple myeloma spheres with encouraging results. Herein, we discuss the role of daratumumab, a monoclonal antibody to CD38, in the treatment of AL amyloidosis...
July 22, 2018: Leukemia & Lymphoma
Nicholas L Wright, Kathryn E Flynn, Ruta Brazauskas, Parameswaran Hari, Anita D'Souza
We conducted this retrospective study to assess patient-reported distress in light chain (AL) amyloidosis, using the Distress Thermometer (DT) survey at first evaluation at our center. Of 78 patients who completed the survey, 75 scored their distress (distress: <4 - low, 4-6 - moderate, >6 - high). Moderate and high distress were self-reported by 30% and 17% patients, respectively. More patients with distress lived alone and had lower haemoglobin than patients without. AL stage did not correlate with distress (Stage I/II median DT 4 compared to 3 in Stage III/IV, p = ...
July 21, 2018: Amyloid: the International Journal of Experimental and Clinical Investigation
Vina P Nguyen, Allison Rosenberg, Lisa M Mendelson, Raymond L Comenzo, Cindy Varga, Vaishali Sanchorawala
Serum free light chains (sFLC) are independent prognostic markers of disease in light chain (AL) amyloidosis, and are used in the haematologic response criteria for treatment. However, up to 20% of patients have low sFLCs at diagnosis, with a difference between involved and uninvolved free light chains (dFLC) of less than 50 mg/L, making responses to treatment difficult to evaluate. In order to characterize this distinct subgroup of patients, we retrospectively analyzed 123 AL amyloidosis patients with dFLC <50 mg/L who were diagnosed between 2002 and 2013...
July 21, 2018: Amyloid: the International Journal of Experimental and Clinical Investigation
Jennifer Kollmer, Markus Weiler, Jan Purrucker, Sabine Heiland, Stefan O Schönland, Ernst Hund, Christoph Kimmich, John M Hayes, Tim Hilgenfeld, Mirko Pham, Martin Bendszus, Ute Hegenbart
OBJECTIVE: To detect, localize, and quantify peripheral nerve lesions in amyloid light chain (AL) amyloidosis by magnetic resonance neurography (MRN) in correlation with clinical and electrophysiologic findings. METHODS: We prospectively examined 20 patients with AL-polyneuropathy (PNP) and 25 age- and sex-matched healthy volunteers. After detailed neurologic and electrophysiologic testing, the patient group was subdivided into mild and moderate PNP. MRN in a 3...
July 20, 2018: Neurology
Matthew Hoscheit, Afrin Kamal, Michael Cline
Systemic amyloidosis is a group of complex disorders characterized by the extracellular deposition of nonbranching fibrils in various tissues, ultimately leading to a variety of clinical presentations including isolated or multiorgan dysfunction. Amyloid involvement of the gastrointestinal tract is common depending on the subtype of this disease; light chain primary amyloidosis (AL) and secondary amyloidosis (AA) affect the gastrointestinal tract in unique ways due to differing pathophysiology. A case is reported of gastroparesis associated with AL amyloidosis diagnosed by esophagogastroduodenoscopy and study of gastric emptying, then subsequently confirmed by Congo red staining performed on endoscopic biopsies...
May 2018: Case Reports in Gastroenterology
Yong Joong Kim, Sejin Ha, Yong-Il Kim
BACKGROUND: Recent progress in amyloid positron emission tomography (PET) has enabled the targeted imaging of cardiac amyloidosis with accuracy. We performed a systematic review and meta-analysis on the diagnostic performance of cardiac amyloidosis using amyloid PET. METHODS: A systematic search was performed using key words: cardiac amyloidosis, amyloid, and PET. We estimated the pooled sensitivity, specificity, positive and negative likelihood ratio (LR), and diagnostic odds ratio (DOR)...
July 18, 2018: Journal of Nuclear Cardiology: Official Publication of the American Society of Nuclear Cardiology
K H G Rutten, R A P Raymakers, M C Minnema
Amyloid light chain (AL) amyloidosis and multiple myeloma (MM) are both clonal plasma cell disorders, and may be concurrently present in patients. However, symptomatic MM seldom develops in patients with AL amyloidosis, while the other way around is common. With this case report, we discuss the difficulties in the differential diagnosis between AL amyloidosis and MM, and extend on the possible mechanisms involved in the development of these overlapping disorders. In addition, we provide clinicians with tools that may help improve their management and monitoring of such patients...
July 2018: Netherlands Journal of Medicine
Aimaz Afrough, Rima M Saliba, Amir Hamdi, Medhavi Honhar, Ankur Varma, A Megan Cornelison, Gabriela Rondon, Simrit Parmar, Nina D Shah, Qaiser Bashir, Chitra Hosing, Uday Popat, Donna M Weber, Sheeba Thomas, Robert Z Orlowski, Richard E Champlin, Muzaffar H Qazilbash
BACKGROUND: With the availability of immunomodulatory agents (IMiD) and proteasome inhibitors (PI), most patients with immunoglobulin light chain amyloidosis (AL) receive induction therapy prior to autologous hematopoietic stem cell transplant (auto-HCT). In this study, we evaluated the type of induction therapy and its impact on the outcome of auto-HCT in AL. METHODS: We identified 128 patients with AL who underwent high-dose chemotherapy and auto- HCT at our institution between 1997 and 2013...
July 14, 2018: Biology of Blood and Marrow Transplantation
Jonah Rubin, D Eric Steidley, Martin Carlsson, Moh-Lim Ong, Mathew S Maurer
BACKGROUND: Transthyretin amyloidosis (ATTR) is often associated with cardiac involvement manifesting as conduction disease as well as a restrictive cardiomyopathy causing heart failure and death. Myocardial contraction fraction (MCF), the ratio of left ventricular stroke volume (SV) to myocardial volume (MV), is a volumetric measure of myocardial shortening that is superior to EF in predicting mortality in patients with primary amyloid light chain (AL) cardiac amyloidosis. We hypothesized that MCF would be an independent predictor of survival in ATTR...
July 12, 2018: Journal of Cardiac Failure
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