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Autologous stem cell transplant amyloidosis

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https://www.readbyqxmd.com/read/28181278/hematology-patient-reported-symptom-screen-to-assess-quality-of-life-for-al-amyloidosis
#1
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 2009-2014, 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...
February 9, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28145535/amyloid-cardiomyopathy
#2
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-over-the-years-2000-2014-cracking-the-glass-ceiling-of-early-death
#3
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, we evaluated the trends in presentation, management and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000-2014. As compared to the two 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 to earlier periods...
January 26, 2017: Blood
https://www.readbyqxmd.com/read/28124791/recovery-from-heart-failure-in-a-patient-with-cardiac-amyloidosis-treated-with-autologous-stem-cell-transplantation
#4
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
#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...
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
#6
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
#7
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...
November 9, 2016: 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
#8
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
#9
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
#10
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
#11
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
#12
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
#13
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
#14
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
https://www.readbyqxmd.com/read/27384856/spontaneous-splenic-rupture-in-a-patient-with-light-chain-deposition-disease-undergoing-autologous-peripheral-blood-stem-cell-transplantation
#15
REVIEW
Shojiro Haji, Junichi Kiyasu, Yoshimichi Tachikawa, Jiro Toyonaga, Motohiko Ikeda, Mariko Tsuda, Yasuhiro Tsukamoto, Mitsuo Kozuru, Yuji Yufu
Light-chain deposition disease (LCDD) is a rare plasma cell neoplasm that secretes an abnormal immunoglobulin light chain, which is deposited in tissues, leading to organ dysfunction. Spontaneous splenic rupture is a rare and life-threatening complication of treatment with granulocyte colony-stimulating factor (G-CSF). Herein, we describe spontaneous splenic rupture after the administration of lenograstim to a patient with LCDD undergoing autologous stem cell transplantation (ASCT). The patient was successfully treated by transcatheter embolization of the splenic artery, and long-term stringent complete remission was attained...
June 2016: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/27358783/long-term-outcomes-of-cardiac-transplant-for-immunoglobulin-light-chain-amyloidosis-the-mayo-clinic-experience
#16
Martha Grogan, Morie Gertz, Arleigh McCurdy, Lindsey Roeker, Robert Kyle, Sudhir Kushwaha, Richard Daly, Joseph Dearani, Richard Rodeheffer, Robert Frantz, Martha Lacy, Suzanne Hayman, Christopher McGregor, Brooks Edwards, Angela Dispenzieri
AIM: To determine the outcome of orthotopic heart transplantation (OHT) in immunoglobulin light chain (AL) amyloidosis. METHODS: The medical records of patients with AL who underwent orthotopic heart transplantation at the Mayo Clinic in Rochester Minnesota from 1992 to 2011 were reviewed. Patients met at least one of the following at: New York Heart Association class IV heart failure, ventricular thickness > 15 mm, ejection fraction < 40%. Selection guidelines for heart transplant included age < 60 years, absence of multiple myeloma and significant extra-cardiac organ involvement...
June 24, 2016: World Journal of Transplantation
https://www.readbyqxmd.com/read/27341539/induction-therapy-pre-autologous-stem-cell-transplantation-in-immunoglobulin-light-chain-amyloidosis-a-retrospective-evaluation
#17
Yi L Hwa, Shaji K Kumar, Morie A Gertz, Martha Q Lacy, Francis K Buadi, Taxiarchis V Kourelis, Wilson I Gonsalves, S Vincent Rajkumar, Ronald S Go, Nelson Leung, Prashant Kapoor, David Dingli, Robert A Kyle, Stephen Russell, John A Lust, Suzanne R Hayman, Yi Lin, Steven Zeldenrust, Angela Dispenzieri
There is no consensus on whether patients with immunoglobulin light chain amyloidosis (AL) should receive induction therapy prior to an autologous stem cell transplant (ASCT). This study investigated the relationships between baseline bone marrow plasmacytosis (BMPC), cardiac staging, and pre-transplant induction in AL patients. All patients who received ASCT for AL within 12 months of diagnosis were included. Patient characteristics and outcomes were abstracted. Univariate and multivariate modeling was performed...
October 2016: American Journal of Hematology
https://www.readbyqxmd.com/read/27296954/the-effect-of-bone-marrow-plasma-cell-burden-on-survival-in-patients-with-light-chain-amyloidosis-undergoing-high-dose-melphalan-and-autologous-stem-cell-transplantation
#18
Christopher Dittus, Nsabimana Uwumugambi, Fangui Sun, J Mark Sloan, Vaishali Sanchorawala
The prognosis in light chain (AL) amyloidosis has been linked to several variables, which are primarily related to end-organ damage. Recently, bone marrow plasma cell (BMPC) burden >10% has also been described as an adverse prognostic factor. We reviewed data pertaining to 546 patients with AL amyloidosis who underwent high-dose melphalan (HDM) and stem cell transplantation (SCT) to determine if BMPC > 10% was a negative prognostic factor. Of these patients, 445 had a BMPC burden ≤ 10% and 101 had a BMPC burden > 10%...
September 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27257181/prognostic-impact-of-cytogenetic-aberrations-in-al-amyloidosis-patients-after-high-dose-melphalan-a-long-term-follow-up-study
#19
Tilmann Bochtler, Ute Hegenbart, Christina Kunz, Axel Benner, Christoph Kimmich, Anja Seckinger, Dirk Hose, Hartmut Goldschmidt, Martin Granzow, Peter Dreger, Anthony D Ho, Anna Jauch, Stefan O Schönland
Cytogenetic aberrations detected by interphase fluorescence in situ hybridization (iFISH) of plasma cells are routinely evaluated as prognostic markers in multiple myeloma. This long-term follow-up study aimed to assess the prognosis of systemic light chain amyloidosis (AL) patients treated with high-dose melphalan (HDM) chemotherapy and autologous stem cell transplantation, depending on iFISH results. Therefore, we analyzed a consecutive cohort of 123 AL patients recruited from 2003 to 2014. HDM was safe, with only 1 of 123 patients dying as a result of treatment-related mortality, and effective, with a complete remission (CR) rate of 34%...
July 28, 2016: Blood
https://www.readbyqxmd.com/read/27236160/%C3%A2-liver-transplantation-followed-by-autologous-stem-cell-transplantation-for-acute-liver-failure-caused-by-al-amyloidosis-case-report-and-review-of-the-literature
#20
Mayada Elnegouly, Katja Specht, Heinz Zoller, Edouard Matevossian, Florian Bassermann, Andreas Umgelter
 Hepatic involvement in AL amyloidosis may present as acute liver failure. Historically, liver transplantation in these cases has achieved poor outcomes due to progress of amyloidosis and non-hepatic organ damage. In the era of bortezomib treatment, the prognosis of AL amyloidosis has been markedly improved and may also result in better post-transplant outcomes. We present a case of isolated acute liver failure caused by AL amyloidosis, bridged to transplantation with bortezomib and treated with sequential orthotopic liver transplantation (OLT) and autologous stem cell transplantation...
July 2016: Annals of Hepatology
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