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Severe aplastic anemia

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https://www.readbyqxmd.com/read/27933374/polymorphisms-of-the-tgf-%C3%AE-1-gene-and-the-risk-of-acquired-aplastic-anemia-in-a-chinese-population
#1
Xue-Hong Liang, Liucheng Rong, Guangsheng He, Hailong He, Shengyun Lin, Yan Yang, Yao Xue, Yongjun Fang
Acquired aplastic anemia (AA) is a hematological disease characterized by failure of bone marrow hematopoiesis resulting in pancytopenia. While immune-mediated destruction of hematopoietic stem/progenitor cells (HSPCs) plays a central role in the pathophysiology of acquired AA, the transforming growth factor-β1 (TGF-β1) is crucial in adjusting the immune system. The aim of our study was to investigate the role of TGF-β1 gene polymorphisms rs1800469 and rs2317130 in susceptibility to acquired AA. Via the approach of SNaPshot, we genotyped rs1800469 and rs2317130 in 101 patients with acquired AA and 165 controls...
December 9, 2016: Annals of Hematology
https://www.readbyqxmd.com/read/27923195/aplastic-anemia-and-mds-international-foundation-aamdsif-bone-marrow-failure-disease-scientific-symposium-2016
#2
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/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
#3
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/27913467/transplantation-for-bone-marrow-failure-current-issues
#4
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/27885510/left-main-crossover-stenting-in-a-patient-with-severe-thrombocytopenia-due-to-aplastic-anemia
#5
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/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
#6
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
#7
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/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
#8
https://www.readbyqxmd.com/read/27808465/long-term-outcomes-of-95-children-with-moderate-aplastic-anemia-treated-with-horse-antithymocyte-globulin-and-cyclosporine
#9
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
#10
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/27801332/-progress-in-the-treatment-of-severe-aplastic-anemia-with-porcine-anti-human-lymphocyte-globulin
#11
H Q Zhang, H Q Wang, Z H Shao
No abstract text is available yet for this article.
October 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/27763888/very-severe-aplastic-anemia-during-treatment-with-doxycycline
#12
Prabhsimranjot Singh, Ankur Sinha, Anisha Kamath, Sonali Malhotra
No abstract text is available yet for this article.
October 18, 2016: American Journal of Therapeutics
https://www.readbyqxmd.com/read/27746432/successful-treatment-of-ascites-using-a-denver-%C3%A2-peritoneovenous-shunt-in-a-patient-with-paroxysmal-nocturnal-hemoglobinuria-and-budd-chiari-syndrome
#13
Tomomi Kogiso, Etsuko Hashimoto, Taito Ito, Toshifumi Hara, Yuichi Ikarashi, Kazuhisa Kodama, Makiko Taniai, Nobuyuki Torii, Kentaro Yoshinaga, Satoru Morita, Yutaka Takahashi, Junji Tanaka, Shuji Sakai, Masakazu Yamamoto, Katsutoshi Tokushige
A 56-year-old man was diagnosed with aplastic anemia and paroxysmal nocturnal hemoglobinuria at 43 years of age and treatment with cyclosporin A was started. Liver cirrhosis, ascites, and thrombus in the hepatic veins were found at 56 years of age and Budd-Chiari syndrome (BCS) was diagnosed according to angiography findings. He was treated with diuretics and paracentesis was performed several times, but with limited efficacy. A Denver(®) peritoneovenous shunt (PVS) was inserted into the right jugular vein; his ascites and renal function improved immediately and his general condition has remained good for 12 months since starting the above treatment regimen...
2016: Internal Medicine
https://www.readbyqxmd.com/read/27725585/aplastic-anemia
#14
Kensuke Usuki
Treatments of aplastic anemia are comprised of supportive therapy and aplastic anemia-specific therapy aimed at restoring hematopoiesis. Supportive therapies include transfusion, G-CSF, and the administration of iron chelation agents, as well as dealing specifically with individual symptoms. Aplastic anemia-specific treatments given with the aim of achieving hematopoietic recovery include immunosuppressive therapy, allogeneic hematopoietic stem cell transplantation, and anabolic hormone therapy. Although transplantation provides complete recovery of hematopoiesis (cure), there is a risk of death due to transplant-related complications...
2016: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/27719726/-the-clinical-feature-and-outcomes-of-severe-aplastic-anemia-patients-suffered-from-bacteremia-following-antithymocyte-globulin
#15
X X Li, M Wang, Y Q Shao, N Nie, J Shi, Z D Huang, J Zhang, M L Ge, J B Huang, Y Z Zheng
Objective: To assess the clinical feature and outcomes of severe aplastic anemia (SAA) patients suffered from bacteremia following antithymocyte globulin (ATG). Methods: A total of 264 cases hospitalized in our hospital between Jan 2000 and July 2011 were enrolled into this study. We evaluated the associated pathogens of bacteremia, analyzed the risk factors by Logistic regression and estimated the overall survival (OS) by Kaplan-Meier method for the cohort of patients. Results: Bloodstream infections occurred in 49 patients, with a median age of 20 (4-62) years, including 38 cases with very SAA (VSAA) and 11 SAA patients...
September 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/27715384/allogeneic-hematopoietic-stem-cell-transplantation-for-nonmalignant-hematologic-disorders-using-chemotherapy-only-cytoreductive-regimens-and-t-cell-depleted-grafts-from-human-leukocyte-antigen-matched-or-mismatched-donors
#16
Alberto Mussetti, Nancy A Kernan, Susan E Prockop, Andromachi Scaradavou, Rachel Lehrman, Julianne M Ruggiero, Kevin Curran, Rachel Kobos, Richard O'Reilly, Farid Boulad
Nonmalignant hematologic disorders (NMHD) of childhood comprise a variety of disorders, including acquired severe aplastic anemia and inherited marrow failure syndromes. Patients with high-risk NMHD without matched related donors fare poorly with allogeneic hematopoietic alternative donor stem cell transplantation (allo-HSCT) and are at high risk for developing graft-versus-host disease following unmodified grafts. The authors retrospectively analyzed data on 18 patients affected by NMHD, lacking a human leukocyte antigen (HLA)-identical sibling donor, who underwent an alternative donor allo-HSCT at their institution between April 2005 and May 2013...
October 7, 2016: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/27709450/analysis-of-allogeneic-hematopoietic-stem-cell-transplantation-with-high-dose-cyclophosphamide-induced-immune-tolerance-for-severe-aplastic-anemia
#17
Zhi Guo, Hong-Yan Gao, Tian-Yan Zhang, Xiao-Dong Liu, Kai Yang, Jing-Xing Lou, Xue-Peng He, Yuan Zhang, Peng Chen, Hui-Ren Chen
The study was aimed to explore the efficacy and safety of allo-HSCT with high-dose cyclophosphamide-induced immune tolerance for SAA. In the present study, 20 cases (12 male, 8 female; average age = 17.8 years) received reduced-intensity conditioning allo-HSCT from August 2012 to August 2014 in the Beijing Military Region General Hospital. All were HLA mismatched and received CSA; 11 received ATG-intensive immune therapy. Donors underwent mobilization with cell colony-stimulating factor. The modified preconditioning regimen included reduced-strength fludarabine combined with Busulfex and cytarabine, cyclophosphamide...
October 5, 2016: International Journal of Hematology
https://www.readbyqxmd.com/read/27695669/environmental-determinants-of-aplastic-anemia-in-pakistan-a-case-control-study
#18
Mehwesh Taj, Tayyaba Shah, Syeda Kanwal Aslam, Sidra Zaheer, Faryal Nawab, Sumaira Shaheen, Kashif Shafique, Tahir Sultan Shamsi
AIM: Aplastic anemia (AA) affects the Asian population two to three fold more than people in other regions. Besides the host genetics and socioeconomic status, several other environmental exposures have been linked with an AA etiology. We aimed to examine the association of various environmental exposures with AA occurrence among Pakistani individuals. SUBJECTS AND METHODS: A case-control study was conducted in Karachi, Pakistan, where cases (diagnosed AA patients) were selected from the National Institute of Blood Disease and Bone Marrow Transplantation (NIBD), while for each case, a single control (who was free of AA and visited the outpatient department of the same hospital for the treatment of minor ailments) was selected matched by age and sex...
2016: Zeitschrift Für Gesundheitswissenschaften, Journal of Public Health
https://www.readbyqxmd.com/read/27695288/eltrombopag-for-the-treatment-of-aplastic-anemia-current-perspectives
#19
REVIEW
Su Han Lum, John D Grainger
Aplastic anemia (AA) is a potential life-threatening hematopoietic stem cell (HSC) disorder resulting in cytopenia. The mainstays of treatment for AA are definitive therapy to restore HSCs and supportive measures to ameliorate cytopenia-related complications. The standard definitive therapy is HSC transplantation for young and medically fit patients with suitable donors and immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine for the remaining patients. A significant proportion of patients are refractory to IST or relapse after IST...
2016: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/27668766/immunosuppressive-therapy-versus-alternative-donor-hematopoietic-stem-cell-transplantation-for-children-with-severe-aplastic-anemia-who-lack-an-hla-matched-familial-donor
#20
Y B Choi, E S Yi, J W Lee, K W Sung, H H Koo, K H Yoo
We compared the outcomes of immunosuppressive treatment (IST) with those of alternative donor hematopoietic stem cell transplantation (HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 42 patients with SAA who received frontline IST (N=19) or frontline HSCT with an alternative donor (N=23) between 1998 and 2012 were analyzed retrospectively. Six patients responded in the frontline IST group, whereas 11 underwent salvage HSCT after IST failure. Twenty-one of 23 patients who underwent frontline HSCT survived without treatment failure...
September 26, 2016: Bone Marrow Transplantation
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