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https://www.readbyqxmd.com/read/27923195/aplastic-anemia-and-mds-international-foundation-aamdsif-bone-marrow-failure-disease-scientific-symposium-2016
#1
Amer M Zeidan, Minoo Battiwalla, Deborah Berlyne, Thomas Winkler
Patients with acquired and inherited bone marrow failure syndromes (BMFS) have ineffective hematopoiesis due to impairments of the hematopoietic stem cell compartment. Common manifestations of BMFS include varying degrees of peripheral blood cytopenias and, sometimes, progression to acute myelogenous leukemia. Research efforts have been made all over the world to improve understanding of the pathogenesis of these diseases and their clinical implications. The Aplastic Anemia and MDS International Foundation (AAMDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMFS...
November 24, 2016: Leukemia Research
https://www.readbyqxmd.com/read/27923178/aplastic-anemia-and-risk-of-deep-vein-thrombosis-and-pulmonary-embolism-a-nationwide-cohort-study
#2
Chun-Liang Lin, Cheng-Li Lin, Shu-Ling Tzeng, Wei-Sheng Chung
OBJECTIVES: Deep vein thrombosis (DVT) and pulmonary embolism (PE) constitute venous thromboembolism (VTE), which is not fully known in aplastic anemia (AA). Therefore, we investigated the incidence and risk of VTE in AA patients. METHODS: We conducted a nationwide cohort study to investigate the risk of DVT and PE in patients with AA. We identified patients with newly diagnosed AA as the AA cohort between 2000 and 2010 from the National Health Insurance Research Database (NHIRD)...
November 21, 2016: Thrombosis Research
https://www.readbyqxmd.com/read/27917629/identification-of-hla-class-i-misreads-dropouts-using-serological-typing-in-comparison-with-dna-based-typing
#3
Hamid Nawaz Tipu, Muhammad Mukarram Bashir, Muhammad Noman
Serology and DNA techniques are employed for Human Leukocyte Antigen (HLA) typing in different transplant centers. Results may not always correlate well and may need retyping with different technique. All the patients (with aplastic anemia, thalassemia, and immunodeficiency) and their donors, requiring HLA typing for bone marrow transplant were enrolled in the study. Serological HLA typing was done by complement-dependent lymphocytotoxicity while DNA-based typing was done with sequence specific primers (SSP)...
October 2016: Iranian Journal of Allergy, Asthma, and Immunology
https://www.readbyqxmd.com/read/27916046/-a-comparative-study-of-unrelated-donor-and-matched-sibling-donor-allogeneic-hematopoietic-stem-cell-transplantation-in-children-and-adolescents-with-acquired-severe-aplastic-anemia
#4
J Zhou, Y W Fu, L J Liang, Q Wang, L J Han, Y L Zu, Yanli Zhang, X H Zhu, F K Yu, B J Fang, X D Wei, Y P Song
Objective: To evaluate the efficacy of unrelated donor allogeneic hematopoietic stem cell transplantation(URD allo-HSCT) for children and adolescents with severe aplastic anemia (SAA). Methods: Clinical data of 34 SAA children and adolescents undergoing allo-HSCT were retrospectively analyzed from October 2001 to October 2015. According to the source of donor, the patients were divided into matched sibling donor allo-HSCT group (MSD group) and unrelated donor group (URD group). The clinical outcome of SAA children and adolescents receiving URD allo-HSCT was assessed, and patients in MSD allo-HSCT group were enrolled as control at the same period...
December 1, 2016: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
https://www.readbyqxmd.com/read/27913534/myelodysplastic-and-myeloproliferative-disorders-of-childhood
#5
Henrik Hasle
Myelodysplastic syndrome (MDS) and myeloproliferative disorders are rare in children; they are divided into low-grade MDS (refractory cytopenia of childhood [RCC]), advanced MDS (refractory anemia with excess blasts in transformation), and juvenile myelomonocytic leukemia (JMML), each with different characteristics and management strategies. Underlying genetic predisposition is recognized in an increasing number of patients. Germ line GATA2 mutation is found in 70% of adolescents with MDS and monosomy 7. It is challenging to distinguish RCC from aplastic anemia, inherited bone marrow failure, and reactive conditions...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27913467/transplantation-for-bone-marrow-failure-current-issues
#6
Régis Peffault de Latour
The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27913466/nontransplant-therapy-for-bone-marrow-failure
#7
Danielle M Townsley, Thomas Winkler
Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27913465/aplastic-anemia-and-clonal-evolution-germ-line-and-somatic-genetics
#8
Akiko Shimamura
Clonal progression to myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) remains a dreaded complication for a subset of patients with bone marrow failure (BMF). Recognizing risk factors for the development of MDS or AML would inform individualized treatment decisions and identify patients who may benefit from early or upfront hematopoietic stem cell transplantation. Now that next-generation DNA sequencing is available in the clinical laboratory, research has focused on the implications of germ line and somatic mutations for diagnosing and monitoring patients with BMF...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27904106/fatal-hemorrhagic-gastrointestinal-angioectasia-after-bone-marrow-transplantation-for-dyskeratosis-congenita
#9
Jin Imai, Takayoshi Suzuki, Marie Yoshikawa, Makiko Dekiden, Hirohiko Nakae, Fumio Nakahara, Shingo Tsuda, Hajime Mizukami, Jun Koike, Muneki Igarashi, Hiromasa Yabe, Tetsuya Mine
Dyskeratosis congenita (DC) is a rare inherited disease in which the telomere complex cannot be maintained. Shortened telomeres can cause a number of clinical conditions. We herein report a case of unrelated bone marrow transplantation due to aplastic anemia associated with DC. The patient died of uncontrollable refractory intestinal bleeding. Three cases of DC with life-threatening hemorrhaging after transplantation have been reported; however, the bleeding origin could not be determined. Our case is the only patient in which a gastrointestinal bleeding point, jejunal multiple angioectasia, was determined...
2016: Internal Medicine
https://www.readbyqxmd.com/read/27903525/glycosylphosphatidylinositol-specific-t-cells-ifn%C3%AE-producing-t-cells-and-pathogenesis-of-idiopathic-aplastic-anemia
#10
Lucia Gargiulo, Yoshitaka Zaimoku, Barbara Scappini, Hiroyuki Maruyama, Rie Ohumi, Lucio Luzzatto, Shinji Nakao, Rosario Notaro
No abstract text is available yet for this article.
November 30, 2016: Blood
https://www.readbyqxmd.com/read/27885510/left-main-crossover-stenting-in-a-patient-with-severe-thrombocytopenia-due-to-aplastic-anemia
#11
Marie Nishikawa, Jun Shiraishi, Muneo Ohshiro, Masaki Yashige, Masayuki Hyogo, Takahisa Sawada
A 76-year-old man with aplastic anemia presented with recurrent acute myocardial infarction (AMI) with heart failure. After the initial appearance of AMI approximately 2 months earlier, he had received conservative treatment/transfusion alone because of severe thrombocytopenia and anemia (platelet 11 × 10(3)/μL, hemoglobin 6.4 g/dL). Refractory heart failure persisted despite repeated conservative treatment/transfusion for the second AMI, and therefore, we performed transradial coronary angiography and left main crossover stenting with a bare metal stent...
November 24, 2016: Cardiovascular Intervention and Therapeutics
https://www.readbyqxmd.com/read/27885340/acute-kidney-injury-in-hematopoietic-stem-cell-transplantation-a-review
#12
REVIEW
Vinod Krishnappa, Mohit Gupta, Gurusidda Manu, Shivani Kwatra, Osei-Tutu Owusu, Rupesh Raina
Hematopoietic stem cell transplantation (HSCT) is a highly effective treatment strategy for lymphoproliferative disorders and bone marrow failure states including aplastic anemia and thalassemia. However, its use has been limited by the increased treatment related complications, including acute kidney injury (AKI) with an incidence ranging from 20% to 73%. AKI after HSCT has been associated with an increased risk of mortality. The incidence of AKI reported in recipients of myeloablative allogeneic transplant is considerably higher in comparison to other subclasses mainly due to use of cyclosporine and development of graft-versus-host disease (GVHD) in allogeneic groups...
2016: International Journal of Nephrology
https://www.readbyqxmd.com/read/27843799/etiological-causes-of-pancytopenia-a-report-of-137-cases
#13
Osman Yokuş, Habip Gedik
OBJECTIVE: The aim of this study is to evaluate the etiological causes of pancytopenia in patients who were admitted to the hematology ward due to pancytopenia. MATERIALS AND METHODS: All patients who were admitted to the hematology ward of Ministry of Health Istanbul Training and Research Hospital due to pancytopenia between 2013 and 2015 were retrospectively evaluated regarding etiological causes. RESULTS: A total of 137 patients was evaluated...
October 2016: Avicenna Journal of Medicine
https://www.readbyqxmd.com/read/27837250/development-of-a-disease-specific-quality-of-life-questionnaire-for-patients-with-aplastic-anemia-and-or-paroxysmal-nocturnal-hemoglobinuria-qlq-aa-pnh-report-on-phases-i-and-ii
#14
Martha Groth, Susanne Singer, Cathrin Niedeggen, Andrea Petermann-Meyer, Alexander Röth, Hubert Schrezenmeier, Britta Höchsmann, Tim H Brümmendorf, Jens Panse
Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated ultra-rare diseases. Quality of life (QoL) evaluation tools used in studies for AA and PNH are unspecific and designed for cancer patients (e.g., the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, EORTC QLQ-C30). Given the complexity of AA and PNH, variation in symptoms and treatments, younger age of many patients, and the fact that AA and PNH are not classified as malignant diseases, it is likely that cancer-specific questionnaires are inappropriate...
November 11, 2016: Annals of Hematology
https://www.readbyqxmd.com/read/27816648/mixed-t-cell-chimerism-after-allogeneic-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-using-an-alemtuzumab-containing-regimen-is-shaped-by-persistence-of-recipient-cd8-t-cells
#15
Francesco Grimaldi, Victoria Potter, Pilar Perez-Abellan, John P Veluchamy, Muhammad Atif, Rosemary Grain, Monica Sen, Steven Best, Nicholas Lea, Carmel Rice, Antonio Pagliuca, Ghulam J Mufti, Judith C W Marsh, Linda D Barber
Prevention of graft-versus-host disease (GVHD) is paramount for allogeneic hematopoietic stem cell transplantation (HSCT) to treat nonmalignant diseases. We previously reported that allogeneic HSCT for severe aplastic anemia (SAA) using the fludarabine, cyclophosphamide, and alemtuzumab (Campath-1H) (FCC) regimen is associated with a very low risk of GVHD and excellent clinical outcomes. We now report a single-center study of 45 patients with longer follow-up and investigation of lymphocyte recovery. Overall survival (OS) was 93%, and event-free survival (EFS) was 90...
November 2, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27815446/blood-stem-cell-activity-is-arrested-by-th1-mediated-injury-preventing-engraftment-following-nonmyeloablative-conditioning
#16
Antonia M S Müller, Mareike Florek, Holbrook E K Kohrt, Natascha J Küpper, Alexander Filatenkov, Jessica A Linderman, Husein Hadeiba, Robert S Negrin, Judith A Shizuru
T cells are widely used to promote engraftment of hematopoietic stem cells (HSCs) during an allogeneic hematopoietic cell transplantation. Their role in overcoming barriers to HSC engraftment is thought to be particularly critical when patients receive reduced doses of preparative chemotherapy and/or radiation compared with standard transplantations. In this study, we sought to delineate the effects CD4(+) cells on engraftment and blood formation in a model that simulates clinical hematopoietic cell transplantation by transplanting MHC-matched, minor histocompatibility-mismatched grafts composed of purified HSCs, HSCs plus bulk T cells, or HSCs plus T cell subsets into mice conditioned with low-dose irradiation...
November 15, 2016: Journal of Immunology: Official Journal of the American Association of Immunologists
https://www.readbyqxmd.com/read/27811186/camitta-bm-thomas-ed-nathan-dg-et-al-severe-aplastic-anemia-a-prospective-study-of-the-effect-of-early-marrow-transplantation-on-acute-mortality-blood-1976-48-1-63-70
#17
https://www.readbyqxmd.com/read/27808465/long-term-outcomes-of-95-children-with-moderate-aplastic-anemia-treated-with-horse-antithymocyte-globulin-and-cyclosporine
#18
Eri Nishikawa, Hiroshi Yagasaki, Asahito Hama, Hiromasa Yabe, Akira Ohara, Yoshiyuki Kosaka, Kazuko Kudo, Ryoji Kobayashi, Shouichi Ohga, Akira Morimoto, Ken-Ichiro Watanabe, Nao Yoshida, Hideki Muramatsu, Yoshiyuki Takahashi, Seiji Kojima
BACKGROUND: Currently, the standard management of moderate aplastic anemia (MAA) has not been well described, although the superiority of the combination of antithymocyte globulin (ATG) and cyclosporine (CyA) over CyA alone has been demonstrated in terms of hematological responses and failure-free survival (FFS). PROCEDURE: We adopted this therapeutic strategy and treated 95 children with MAA who were enrolled in two consecutive prospective studies between October 1992 and August 2009...
November 3, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27806787/-analysis-of-clinical-characteristics-and-prognosis-of-non-severe-aplastic-anemia-children-with-chromosomal-abnormalities
#19
S Zhu, W B An, Y Wan, Y Y Ren, R R Zhang, J L Zhang, C Liu, C C Sun, L X Chang, X J Chen, W Y Yang, Y Guo, Y M Chen, Y Zou, X F Zhu
Objective: To analyze the clinical characteristics and prognosis of non-severe aplastic anemia (NSAA) with chromosomal abnormalities in children. Method: A retrospective analysis of 304 cases with NSAA with successful karyotyping from 2001 to 2014 in the Institute of Hematology & Blood Disease Hospital was carried out. The treatment response, condition of blood transfusion were analyzed using χ(2) test, the cumulative survival was estimated by the Kaplan-Meier method. Result: Out of 304 patients, 28 patients had chromosomal abnormalities with trisomy 8 (7 cases, 25...
November 2, 2016: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/27806503/anemia-related-mortality-in-inner-mongolia-in-2008-2012
#20
Lifu Chen, Maolin Du, Hairong Zhang, Yuanzhi Xu, Qingxia Wang, Fengyun Zuo, Yun Li, Sun Juan
OBJECTIVE: This study aimed to assess the characteristics of anemia-related deaths and the relationship between socio-demographic status and mortality risk of anemia in Inner Mongolia from 2008 to 2012. METHODS: We obtained our data for 2008-2012 from the Death Registry System of Inner Mongolia. We calculated the mortality, percentage, and potential years of life lost (PYLL) of anemia in diseases of the blood and blood-forming organs and certain disorders involving the immune mechanism (DBDIM)...
July 12, 2016: Global Journal of Health Science
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