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Autologous stem cell transplant amyloidosis

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https://www.readbyqxmd.com/read/28512563/acquired-von-willebrand-syndrome-associated-to-secondary-igm-mgus-emerging-after-autologous-stem-cell-transplantation-for-al-amyloidosis
#1
Hina Qamar, Adrienne Lee, Karen Valentine, Leslie Skeith, Victor H Jimenez-Zepeda
Acquired von Willebrand syndrome (AVWS) is a rare hemorrhagic disorder that occurs in patients with no prior personal or family history of bleeding. Here, we describe a case of AVWS occurring after autologous stem cell transplantation (ASCT). Interestingly, AVWS developed after bortezomib-based induction and conditioning regimens. Recent evidence suggests that the proximity of the bortezomib therapy to the collection of stem cells with consequent depletion of regulatory T cells after the conditioning regimen could explain some of the unusual autoimmune complications reported in patients receiving bortezomib prior to ASCT...
2017: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/28457657/stem-cell-transplantation-for-immunoglobulin-light-chain-amyloidosis
#2
REVIEW
Taimur Sher, Morie A Gertz
Systemic chemotherapy aimed at eradicating transformed plasma cells is the mainstay of treatment for immunoglobulin light chain amyloidosis (AL). Autologous stem cell transplantation (SCT) is a highly effective treatment for AL and can lead to long term survival in excess of 10 years in patients who achieve complete remission. Since AL is a unique disease characterized by multiple organ dysfunction, SCT poses unique challenges in this disease. Morbidity and mortality of SCT has remarkably improved over time primarily due to careful selection of patients and evolution of predictive and prognostic models based on serum immunoglobulin light chains and cardiac biomarkers...
March 16, 2017: Current Problems in Cancer
https://www.readbyqxmd.com/read/28434927/impact-of-autologous-stem-cell-transplantation-on-the-incidence-and-outcome-of-oligoclonal-bands-in-patients-with-light-chain-amyloidosis
#3
Luis Gerardo Rodríguez-Lobato, Carlos Fernández de Larrea, M Teresa Cibeira, Natalia Tovar, Juan I Aróstegui, Laura Rosiñol, Tania Díaz, Ester Lozano, Montserrat Elena, Jordi Yagüe, Joan Bladé
The emergence of oligoclonal bands (OB) in patients with multiple myeloma achieving a complete remission (CR) after autologous stem cell transplantation (ASCT) and the use of novel agents is a well-recognized event. The presence of OB is associated with favorable outcome. However, the emergence of OB in light-chain (AL) amyloidosis has never been investigated. The aim of the study was to determine the incidence, natural history, and prognostic impact of OB in 50 patients with AL amyloidosis who achieved at least a partial response either after upfront ASCT (20 patients [40%]) or after conventional treatment in patients ineligible for transplantation (30 patients [60%])...
April 18, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28434321/renal-outcomes-of-autologous-stem-cell-transplantation-among-patients-with-light-chain-amyloidosis-a-single-centre-spanish-experience
#4
Sebastián Ernesto Azorín, María Teresa Cibeira, Manel Solé, Carlos Fernández de Larrea, Laura Rosiñol, Montse Rovira, Josep Maria Campistol, Joan Bladé
No abstract text is available yet for this article.
March 2017: Amyloid: the International Journal of Experimental and Clinical Investigation
https://www.readbyqxmd.com/read/28394369/revisiting-conditioning-dose-in-newly-diagnosed-light-chain-amyloidosis-undergoing-frontline-autologous-stem-cell-transplant-impact-on-response-and-survival
#5
N Tandon, E Muchtar, S Sidana, A Dispenzieri, M Q Lacy, D Dingli, F K Buadi, S R Hayman, R Chakraborty, W J Hogan, W Gonsalves, R Warsame, T V Kourelis, N Leung, P Kapoor, S K Kumar, M A Gertz
Autologous stem cell transplantation (ASCT) is an important treatment modality in light chain (AL) amyloidosis. Use of reduced-dose melphalan conditioning is common, given the associated organ and functional decline. The impact of full-intensity melphalan conditioning (n=314) was compared to reduced-dose conditioning (n=143). Patients in the full-intensity group were younger, with better performance status, fewer involved organs, lower tumor burden and lower Mayo stage. Full-dose conditioning was associated with higher rate of very good partial response or better (79% vs 62%; P<0...
April 10, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28290973/total-artificial-heart-implantation-as-a-bridge-to-heart-transplantation-in-an-active-duty-service-member-with-amyloid-cardiomyopathy
#6
Michael S Scully, Dylan E Wessman, James M McKee, Gregory M Francisco, Keshav R Nayak, Jon A Kobashigawa
INTRODUCTION: Cardiac involvement by light-chain (AL) amyloid occurs in up to 50% of patients with primary AL amyloidosis. The prognosis of amyloid heart disease is poor with 1-year survival rates of 35 to 40%. Historically, heart transplantation was considered controversial for patients with AL amyloid cardiomyopathy (CM) given the systemic nature of the disease and poor survival. We present a case report of an active duty service member diagnosed with advanced cardiac amyloid who underwent total artificial heart transplant as a bridge to heart transplant and eventual autologous stem cell transplant...
March 2017: Military Medicine
https://www.readbyqxmd.com/read/28181278/hematology-patient-reported-symptom-screen-to-assess-quality-of-life-for-al-amyloidosis
#7
Rahma Warsame, Shaji K Kumar, Morie A Gertz, Martha Q Lacy, Francis K Buadi, Suzanne R Hayman, Nelson Leung, David Dingli, John A Lust, Yi Lin, Stephen Russell, Prashant Kapoor, Ronald S Go, Taxiarchis Kourelis, Wilson Gonsalves, Steven R Zeldenrust, Robert A Kyle, S Vincent Rajkumar, Tyler Zemla, Jeffrey Sloan, Angela Dispenzieri
Patients with light chain amyloidosis (AL) often have delayed diagnosis and present with significant symptomatology; this may result in decreased quality of life (QOL). We prospectively employ a "Hematology Patient Reported Symptom Screen" (HPRSS), which is three questions about fatigue, pain, and QOL, scored 0-10. The aim of this study is to better understand QOL and determine if HPRSS parameters predict for clinical outcomes. From 2009 to 2014, 302 newly diagnosed AL patients were included. Baseline median scores [interquartile range] for fatigue, pain, and QOL were 6 [3,7], 2 [0,5], 5 [3,8], respectively...
May 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28145535/amyloid-cardiomyopathy
#8
Lucie Karafiatova, Tomas Pika
Amyloidosis is a heterogeneous group of diseases characterized by the deposition of amyloid. It is caused by extracellular deposition of insoluble fibrils with beta-pleated sheet configuration. The protein misfolding abnormalities result in amyloid fibrils and may manifest as primary, secondary, or familial amyloidosis. Amyloid deposition can occur in multiple organs (eg, heart, liver, kidney, skin, eyes, lungs, nervous system) resulting in a variety of clinical manifestations. Cardiac involvement can occur as part of a systemic disease or as a localized phenomenon...
February 1, 2017: Biomedical Papers of the Medical Faculty of the University Palacký, Olomouc, Czechoslovakia
https://www.readbyqxmd.com/read/28126928/improved-outcomes-for-newly-diagnosed-al-amyloidosis-between-2000-and-2014-cracking-the-glass-ceiling-of-early-death
#9
Eli Muchtar, Morie A Gertz, Shaji K Kumar, Martha Q Lacy, David Dingli, Francis K Buadi, Martha Grogan, Suzanne R Hayman, Prashant Kapoor, Nelson Leung, Amie Fonder, Miriam Hobbs, Yi Lisa Hwa, Wilson Gonsalves, Rahma Warsame, Taxiarchis V Kourelis, Stephen Russell, John A Lust, Yi Lin, Ronald S Go, Steven Zeldenrust, Robert A Kyle, S Vincent Rajkumar, Angela Dispenzieri
In light of major advances in immunoglobulin light chain (AL) amyloidosis, we evaluated the trends in presentation, management, and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000 to 2014. As compared with the 2 intervals 2000-2004 and 2005-2009, patients diagnosed in 2010-2014 were less likely to have >2 involved organs. Utilization of autologous stem cell transplant (ASCT) was similar across all periods, about one-third of patients, but there was an increase in the use of pre-ASCT bortezomib induction and of unattenuated melphalan conditioning in 2010-2014 compared with earlier periods...
April 13, 2017: Blood
https://www.readbyqxmd.com/read/28124791/recovery-from-heart-failure-in-a-patient-with-cardiac-amyloidosis-treated-with-autologous-stem-cell-transplantation
#10
Monika Gawor, Łukasz Mazurkiewicz, Blanka Milanowska, Jacek Grzybowski
No abstract text is available yet for this article.
2017: Kardiologia Polska
https://www.readbyqxmd.com/read/28002971/new-and-developing-therapies-for-al-amyloidosis
#11
REVIEW
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...
February 2017: Expert Opinion on Pharmacotherapy
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
#12
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/27862330/predictors-of-inferior-clinical-outcome-in-patients-with-standard-risk-multiple-myeloma
#13
Talha Badar, Samer Srour, Qaiser Bashir, Nina Shah, Gheath Al-Atrash, Chitra Hosing, Uday Popat, Yago Nieto, Robert Z Orlowski, Richard Champlin, Muzaffar H Qazilbash
INTRODUCTION: Outcome of patients with standard-risk (SR) multiple myeloma (MM) has improved; however, subsets of patients do worse than expected. We sought to identify the factors associated with inferior outcome. METHODS: We evaluated 51 patients with SR MM that received upfront autologous hematopoietic stem cell transplantation (auto-HCT) after induction and had a progression-free survival (PFS) of ≤18 months. RESULTS: The median age of patients was 61 yr...
March 2017: European Journal of Haematology
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
#14
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
https://www.readbyqxmd.com/read/27580556/aa-negative-and-kappa-positive-amyloidosis-in-a-patient-with-rheumatoid-arthritis
#15
Toshiharu Ueno, Keiichi Sumida, Junichi Hoshino, Tatsuya Suwabe, Koki Mise, Ryo Hazue, Noriko Hayami, Rikako Hiramatsu, Masahiro Kawada, Aya Imafuku, Eiko Hasegawa, Naoki Sawa, Kenmei Takaichi, Keiichi Kinowaki, Kenichi Ohashi, Takeshi Fujii, Aya Nishida, Yoshifumi Ubara
A 57-year-old Japanese woman with a 5-year history of rheumatoid arthritis (RA) was admitted to our hospital for an evaluation of nephrotic range proteinuria (4.8 g/day). A renal biopsy led to the diagnosis of amyloidosis according to strong positivity for Congo red staining and the detection of microfibrillar structures on electron microscopy that were negative for AA and positive for kappa light chain. Combination therapy with high-dose melphalan and autologous stem cell transplantation was performed according to the regimen for AL amyloidosis...
2016: Internal Medicine
https://www.readbyqxmd.com/read/27536687/the-prevalence-and-management-of-systemic-amyloidosis-in-western-countries
#16
REVIEW
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
https://www.readbyqxmd.com/read/27536686/the-clinical-presentation-and-management-of-systemic-light-chain-amyloidosis-in-china
#17
REVIEW
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
https://www.readbyqxmd.com/read/27501122/outcomes-of-patients-with-renal-monoclonal-immunoglobulin-deposition-disease
#18
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...
November 2016: American Journal of Hematology
https://www.readbyqxmd.com/read/27497715/newer-therapies-for-amyloid-cardiomyopathy
#19
REVIEW
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)...
October 2016: Current Heart Failure Reports
https://www.readbyqxmd.com/read/27479823/immunoparesis-status-in-immunoglobulin-light-chain-amyloidosis-at-diagnosis-affects-response-and-survival-by-regimen-type
#20
Eli Muchtar, Angela Dispenzieri, Shaji K Kumar, David Dingli, Martha Q Lacy, Francis K Buadi, Suzanne R Hayman, Prashant Kapoor, Nelson Leung, Rajshekhar Chakraborty, Stephen Russell, John A Lust, Yi Lin, Ronald S Go, Steven Zeldenrust, Robert A Kyle, S Vincent Rajkumar, Morie A Gertz
Clinical tools to guide in the appropriate treatment selection in immunoglobulin light chain (AL) amyloidosis are not well developed. We evaluated the response and outcome for various regimens at first-line treatment (n=681) and first progression (n=240) stratified by the immunoparesis status at diagnosis. Immunoparesis was assessed by the average relative difference of the uninvolved immunoglobulins, classifying patients into a negative average relative difference (i.e. significant immunoparesis) or a positive average relative difference (no/modest immunoparesis)...
September 2016: Haematologica
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