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AL amyloidosis treatment

Iuliana Vaxman, Morie Gertz
The term amyloidosis refers to a group of disorders in which protein fibrils accumulate in certain organs, disrupt their tissue architecture, and impair the function of the effected organ. The clinical manifestations and prognosis vary widely depending on the specific type of the affected protein. Immunoglobulin light-chain (AL) amyloidosis is the most common form of systemic amyloidosis, characterized by deposition of a misfolded monoclonal light-chain that is secreted from a plasma cell clone. Demonstrating amyloid deposits in a tissue biopsy stained with Congo red is mandatory for the diagnosis...
January 16, 2019: Acta Haematologica
Avery A Rizio, Michelle K White, Kristen L McCausland, Tiffany P Quock, Spencer D Guthrie, Miyo Yokota, Martha S Bayliss
Background: Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment...
November 2018: American Health & Drug Benefits
Vishal K Gupta, Dina Brauneis, Anthony C Shelton, Karen Quillen, Shayna Sarosiek, J Mark Sloan, Vaishali Sanchorawala
In light-chain (AL) amyloidosis, the depth of hematologic response to treatment is associated with improved survival and organ responses. We conducted a clinical trial utilizing bortezomib in induction and in conditioning with melphalan prior to stem cell transplantation (SCT) for AL amyloidosis. The results of this clinical trial with a median follow-up of 36 months have been reported previously. Here we report the long-term results of this clinical trial with a median follow-up of 77 months. The objectives of this follow-up report are to describe survival, durability of hematologic and organ responses, and relapse rates...
January 10, 2019: Biology of Blood and Marrow Transplantation
V Gödecke, J J Schmidt, J H Bräsen, C Koenecke, H Haller
BACKGROUND: Kidney involvement is a common complication in patients with plasma cell diseases. OBJECTIVE: This article outlines the spectrum of renal involvement in plasma cell dyscrasia and describes diagnostic and therapeutic measures to guide clinical management. MATERIAL AND METHODS: Evaluation and discussion of the current literature as well as existing guidelines and recommendations of professional societies. RESULTS: The clinical manifestations of renal involvement in plasma cell disorders are heterogeneous and range from acute cast nephropathy in multiple myeloma to rare forms of glomerulonephritis...
January 11, 2019: Der Internist
Tomáš Jelínek, Jana Mihályová, Roman Hájek
CD38 antigen is highly and uniformly expressed on plasma cells and thus represents an ideal target for the treatment of multiple myeloma (MM) with anti-CD38 monoclonal antibodies (mAbs). Daratumumab is the most advanced anti-CD38 mAb in the clinical development with approval in several indications, nevertheless isatuximab that targets completely different epitope of CD38 molecule is also very promising drug. Anti-CD38 possess pleiotropic mechanism of action that have been described also in other mAbs, but quite specific, novel and very important seems to be the immunomodulatory effect provided by depletion of several CD38+ immunosuppressive immune cell populations...
2018: Vnitr̆ní Lékar̆ství
Maria Gavriatopoulou, Despoina Fotiou, Ioannis Ntanasis-Stathopoulos, Efstathios Kastritis, Evangelos Terpos, Meletios Athanasios Dimopoulos
Plasma cell dyscrasias are a rare heterogeneous group of hematological disorders which are more prevalent in the older part of the population. The introduction of novel agents, improved understanding of disease biology and better supportive management have improved outcomes considerably and in the era of the aging population the question of how to best manage older patients with plasma cell dyscrasias has never been more relevant. Data on how to treat these patients comes mostly from subgroup analysis as they are underrepresented in clinical trials...
December 18, 2018: Aging
Andree Hermes Koop, Omar Y Mousa, Ming-Hsi Wang
Gastrointestinal (GI) amyloidosis is rare and has varying clinical and endoscopic presentations. In this case series, we present three patients with primary systemic amyloid-light chain (AL) amyloidosis with GI involvement and complications of GI bleeding. We also provide a brief review of the literature, including clinical presentation, endoscopic findings, pathology, and management of GI amyloidosis. The endoscopic findings of GI amyloidosis can vary, including friable mucosa with erosions, ulcers, and submucosal hematomas or mucosal thickening with polypoid protrusions...
October 2018: Clujul Medical (1957)
Wencui Chen, Guisheng Ren, Ke Zuo, Xianghua Huang
RATIONALE: Immunoglobulin light chain amyloidosis (AL amyloidosis) is characterized by the deposition of abnormal amyloid protein produced by a pathological plasma cell clone in various organs and soft tissues. Hematopoietic stem cell transplantation (HSCT) is an effective way to treat AL amyloidosis. Psoriasis is a common autoimmune disease (AID) and HSCT is a potential treatment for severe AIDs. We report a rare case of AL amyloidosis coincidence with psoriasis obtained continuous complete remission of the 2 diseases by autologous hematopoietic stem cell transplantation (ASCT)...
December 2018: Medicine (Baltimore)
Sebastian Küchlin, Johannes Duffner, Sophia Scheubeck, Katja Schoeller, Lars Maruschke, Maximilian Seidl, Monika Engelhardt, Gerd Walz, Eric Peter Prager, Johannes Moritz Waldschmidt
Despite substantial improvements following the introduction of novel agents and antibodies, amyloid light-chain (AL)-amyloidosis still carries a grim prognosis. Here, we report on the case of a severely frail 86-year-old patient suffering from monoclonal gammopathy of renal significance (MGRS)-associated AL-amyloidosis with a diuretic-refractory nephrotic syndrome. In this patient, treatment with bortezomib-dexamethasone effectively induced a serological response, but was unfortunately poorly tolerated and failed to promote renal recovery fast enough to prevent secondary complications...
December 13, 2018: Hemodialysis International
Panagiota Kyriakou, Dimitrios Mouselimis, Anastasios Tsarouchas, Angelos Rigopoulos, Constantinos Bakogiannis, Michel Noutsias, Vasileios Vassilikos
BACKGROUND: Cardiac Amyloidosis (CA) pertains to the cardiac involvement of a group of diseases, in which misfolded proteins deposit in tissues and cause progressive organ damage. The vast majority of CA cases are caused by light chain amyloidosis (AL) and transthyretin amyloidosis (ATTR). The increased awareness of these diseases has led to an increment of newly diagnosed cases each year. METHODS: We performed multiple searches on MEDLINE, EMBASE and the Cochrane Database of Systematic Reviews...
December 4, 2018: BMC Cardiovascular Disorders
Justin R Arnall, Saad Z Usmani, Hawawu Adamu, Joseph Mishkin, Manisha Bhutani
Systemic light-chain (AL) amyloidosis is a rare hematologic disorder where proteins infiltrate tissues leading to organ failure and death. Cardiac involvement, present in ∼70% of patients, determines stage and prognosis of the disease, with advanced involvement having a median survival of six months. The treatment of light-chain amyloidosis is directed at recovering organ function with therapeutic strategies following those of multiple myeloma with plasma cell-directed therapies. The use of single agent daratumumab has been reported in light-chain amyloidosis achieving rapid and deep responses...
November 26, 2018: Journal of Oncology Pharmacy Practice
Kar Wah Fuah, Christopher Thiam Seong Lim
BACKGROUND: Amyloidosis is a disorder caused by extracellular tissue deposition of insoluble fibrils which may result in a wide spectrum of symptoms depending upon their types, sites and amount of deposition. Amyloidosis can be divided into either systemic or localized disease. CASE PRESENTATION: We present a case of a middle-aged gentleman who presented with persistent nephrotic syndrome with worsening renal function. Repeated renal biopsies showed the presence of renal-limited AL amyloidosis...
November 6, 2018: BMC Nephrology
Divyesh P Engineer, Vivek B Kute, Himanshu V Patel, Pankaj R Shah
The kidney is the most common organ involved in systemic amyloidosis. We aimed to study etiology and clinicopathological profile of renal amyloidosis. This was a retrospective study of 40 consecutive adult patients with biopsy-proven renal amyloidosis evaluated over a period of two years. Emphasis was given to describing the clinical presentation, renal function, proteinuria, type of amyloidosis, and its etiology. Mean age of the study cohort was 44 ± 15 years (with a male-to-female ratio of 3:1). Amyloid A (AA) amyloidosis was the most common type of amyloidosis observed in 72...
September 2018: Saudi Journal of Kidney Diseases and Transplantation
Tiffany P Quock, Eunice Chang, Jennifer S Munday, Anita D'Souza, Sohum Gokhale, Tingjian Yan
AIMS: Examine mortality and healthcare costs in Medicare beneficiaries with newly diagnosed immunoglobulin light chain (AL) amyloidosis. PATIENTS & METHODS: Cases were identified in 2012-2015 Medicare 5% data with ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and ≥1 AL-specific treatment. Cases were matched 3:1 with disease-free controls. Descriptive statistics were reported. RESULTS:  A total of 249 (33.3%) cases were matched to 747 (66...
November 2018: Journal of Comparative Effectiveness Research
Anthony L Nguyen, Muhammad Kamal, Ravi Raghavan, Gayathri Nagaraj
A 52 year-old male presented with neck pain after undergoing thyroidectomy for a goiter three weeks prior which was complicated by a neck hematoma requiring evacuation. Computed tomography (CT) scan showed a neck hematoma requiring evacuation and he received desmopressin with cessation of bleeding. Coagulation studies were normal. He returned eighteen months later with severe oral mucosal bleeding after a dental procedure and required transfusions with red blood cells, platelets, and fresh frozen plasma (FFP) in addition to desmopressin, Humate-P, aminocaproic acid, and surgical packing...
September 5, 2018: Hematology Reports
Juan Gonzalez, Ahsan Wahab, Kavitha Kesari
Dysphagia is an uncommon presentation of systemic immunoglobulin light-chain (AL) amyloidosis with multiple myeloma (MM). Gastrointestinal (GI) involvement usually manifests with altered motility, malabsorption or bleeding. Furthermore, patients identified with GI amyloidosis, without previous diagnosis of a plasma cell disorder, are extremely rare. We report an elderly woman who presented with acute on chronic cardiac dysfunction, sick sinus syndrome and acute renal failure. While admitted, she developed intermittent dysphagia to both solids and liquids...
October 21, 2018: BMJ Case Reports
Romana Ryšavá
AL amyloidosis (light chain; previously also called primary amyloidosis) is a systemic disease characterized by an amyloid deposition process affecting many organs, and which still has unsatisfactory survival of patients. The monoclonal light chains kappa (κ) or lambda (λ) or their fragments form the fibrils that deposit and accumulate in different tissues. Renal involvement is very frequent in AL amyloidosis and can lead to the development of nephrotic syndrome followed by renal failure in some cases. AL amyloidosis ultimately leads to destruction of tissues and progressive disease...
October 8, 2018: Nephrology, Dialysis, Transplantation
Mirela Andrei, Jen Chin Wang
OBJECTIVE/BACKGROUND: Cutaneous immunoglobulin (Ig) amyloid light-chain (AL) amyloidosis associated with overt multiple myeloma (MM) is rare and optimal treatment is not well defined. The recently developed highly efficacious MM therapy has brought on a new set of challenges to this field for consideration. The goal of this paper is to describe the characteristics of cutaneous manifestations of systemic AL amyloidosis associated with MM according to age, sex, race, Ig type, plasma cell percentage, and cytogenetic and fluorescent in situ hybridization studies along with their outcomes...
September 21, 2018: Hematology/oncology and Stem Cell Therapy
Tomoya Iida, Daisuke Hirayama, Gota Sudo, Kei Mitsuhashi, Hisayoshi Igarashi, Kentaro Yamashita, Hiroo Yamano, Hiroshi Nakase
A 59-year-old woman presented to our hospital with a 6-month history of nausea, weight loss, and abdominal distension. Physical examination revealed abdominal distension without tenderness, and edema, numbness, and multiple peripheral neuropathy in the limbs. Blood test results showed anemia, hypoproteinemia, and hypoalbuminemia. Immunoelectrophoresis detected kappa-type Bence-Jones protein in both the serum and urine. Bone marrow examination did not reveal an increase of plasma cells. Computed tomography showed intestinal distension and retention of intestinal contents...
September 19, 2018: Clinical Journal of Gastroenterology
Talha Badar, Anita D'Souza, Parameswaran Hari
Immunoglobulin (Ig) light chain (AL) amyloidosis is a clonal plasma cell disorder characterized by misfolded Ig light chain deposition in vital organs of the body, resulting in proteotoxicity and organ dysfunction. Owing to its diverse clinical presentations and a tendency to mimic common medical conditions, AL amyloidosis is often diagnosed late and results in dismal outcomes. Early referral to a specialized center with expertise in management of AL amyloidosis is always recommended. The availability of sensitive biomarkers and novel therapies is reforming our approach to how we manage AL amyloidosis...
2018: F1000Research
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