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Severe aplastic anemia

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https://www.readbyqxmd.com/read/29434791/efficacy-and-safety-of-combined-immunosuppressive-therapy-plus-umbilical-cord-blood-infusion-in-severe-aplastic-anemia-patients-a-cohort-study
#1
Xiu Luo, Huina Lu, Bing Xiu, Hao Wu, Bing Li, Ping Li, Yuhua Chen, Lili Zhou, Wenjun Zhang, Yan Dong, Aibin Liang, Yi Ding
The present study aimed to evaluate the efficacy and safety of combined immunosuppressive therapy (IST) plus umbilical cord blood infusion (UCBI) in severe aplastic anemia (SAA) patients. A total of 68 patients with SAA were enrolled in the current prospective cohort study and divided into the IST (n=35; positive control) and IST+UCBI (n=33; experimental) groups according to the treatment conditions. Patients in the IST group were treated with rabbit antithymocyte globulin (r-ATG) at a dose of 2.5 mg/kg through intravenous infusion once a day for five days...
February 2018: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29432302/allogeneic-matched-related-donor-bone-marrow-transplantation-for-pediatric-patients-with-severe-aplastic-anemia-using-low-dose-cyclophosphamide-atg-plus-fludarabine
#2
Chayamon Takpradit, Susan E Prockop, Nancy A Kernan, Andromachi Scaradavou, Kevin Curran, Julianne Ruggiero, Nicole Zakak, Richard J O'Reilly, Farid Boulad
BACKGROUND: The combination of cyclophosphamide (CY) and antithymocyte globulin (ATG) has been used as a standard conditioning regimen for matched related donor transplantation in patients with severe aplastic anemia. PROCEDURE: To decrease the regimen-related toxicity while maintaining appropriate engraftment and survival rates, fludarabine (FLU) was added to the regimen. Four pediatric patients received matched related donor bone marrow transplantation with CY (50 mg/kg×2) (instead of the 50 mg/kg×4 standard dosing), equine ATG (30 mg/kg×3), with the addition of FLU (30 mg/m×4)...
February 9, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29419434/deep-sequencing-and-flow-cytometric-characterization-of-expanded-effector-memory-cd8-cd57-t-cells-frequently-reveals-t-cell-receptor-v%C3%AE-oligoclonality-and-cdr3-homology-in-acquired-aplastic-anemia
#3
Valentina Giudice, Xingmin Feng, Zenghua Lin, Wei Hu, Fanmao Zhang, Wangmin Qiao, Maria Del Pilar Fernandez Ibanez, Olga Rios, Neal S Young
Oligoclonal expansion of CD8+CD28- lymphocytes has been considered indirect evidence for a pathogenic immune response in acquired aplastic anemia. A subset of CD8+CD28- cells with CD57 expression termed effector memory cells is expanded in several immune mediated diseases and may have a role in immune surveillance. We hypothesized that effector memory CD8+CD28-CD57+ cells may drive aberrant oligoclonal expansion in aplastic anemia. We found CD8+CD57+ cells frequently expanded in the blood of aplastic anemia patients, with oligoclonal characteristics by flow cytometric Vβ usage analysis: skewing in 1 to 5 Vβ families and frequencies of immunodominant clones ranging from 1...
February 1, 2018: Haematologica
https://www.readbyqxmd.com/read/29410500/optimal-donor-for-severe-aplastic-anemia-patient-requiring-allogeneic-hematopoietic-stem-cell-transplantation-a-large-sample-study-from-china
#4
Yunjing Zeng, Sanbin Wang, Jishi Wang, Li Liu, Yi Su, Zhixiang Lu, Xuemei Zhang, Yanqi Zhang, Jiang Fan Zhong, Lihui Peng, Qiang Liu, Yinghao Lu, Lei Gao, Xi Zhang
HLA-haploidentical hematopoietic stem cell transplantation (HSCT) may be an option for severe aplastic anemia (SAA) patients. However, to date, no large-sample studies have been performed to determine which types of SAA patients are suitable for HLA-haploidentical HSCT. We retrospectively studied 189 consecutive patients with SAA who underwent HLA-identical or HLA-haploidentical HSCT at seven transplant centers in China. Propensity score matching (PSM) was applied in this study to reduce the influence of potential confounders...
February 6, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29410341/evaluation-of-a-machine-learning-based-prognostic-model-for-unrelated-hematopoietic-cell-transplantation-donor-selection
#5
Ljubomir Buturovic, Jason Shelton, Stephen R Spellman, Tao Wang, Lyssa Friedman, David Loftus, Lyndal Hesterberg, Todd Woodring, Katharina Fleischhauer, Katharine C Hsu, Michael R Verneris, Mike Haagenson, Stephanie J Lee
Survival of patients undergoing hematopoietic cell transplantation (HCT) from unrelated donors for acute leukemia exhibits considerable variation, even after stringent genetic matching. In order to improve the donor selection process, we attempted to create an algorithm to quantify the likelihood of survival to five years after unrelated donor HCT for acute leukemia, based on the clinical characteristics of the donor selected. All standard clinical variables were included in the model, which also included average leukocyte telomere length (ATL) of the donor based on its association with recipient survival in severe aplastic anemia, and links to multiple malignancies...
February 1, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29397846/-differential-expression-profiles-of-micrornas-between-de-novo-and-complete-response-severe-aplastic-anemia
#6
Ying-Qi Shao, Hong-Yu Dong, Mei-Li Ge, Feng Zhang, Jun Shi, Xing-Xin Li, Jin-Bo Huang, Zhen-Dong Huang, Jing Zhang, Neng Nie, Yi-Zhou Zheng
OBJECTIVE: To detect the expression of miRNA in de novo and complete response SAA patients and predict the targets of the miRNAs. METHODS: The expression profiles of miRNA from bone marrow mononuclear cells of the SAA patients with de novo and CR were detected by miRNA microarray. RESULTS: Totally 35 up-regulated and 37 down-regulated miRNA were identified in CR SAA patients in comparison with de novo SAA patients. Furthermore, by predicting the targets of the differentlly expressed miRNA, it was found that some targets associated with T cell receptor signaling pathway and cell adhesion molecules...
February 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/29390381/mesalazine-as-a-cause-of-fetal-anemia-and-hydrops-fetalis-a-case-report
#7
Sverker Ek, Staffan Rosenborg
RATIONALE: Mesalazine and its prodrug sulfasalazine are both used for inflammatory bowel disease. Sulfasalazine has been associated with hematological side-effects such as aplastic and hemolytic anemia in patients, but also in fetuses after intrauterine exposure. To our knowledge, we describe the first case of a fetus with severe anemia, and subsequent hydrops, where this drug was found at concentrations in the fetus corresponding to those in the mother and most likely responsible for the fetal condition...
December 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29377260/nationwide-survey-on-the-use-of-horse-antithymocyte-globulins-atgam-in-patients-with-acquired-aplastic-anemia-a-report-on-behalf-of-the-french-reference-center-for-aplastic-anemia
#8
Régis Peffault de Latour, Reza Tabrizi, Ambroise Marcais, Thierry Leblanc, Thierry Lamy, Mohamad Mohty, Suzanne Tavitian, Charlotte Jubert, Marlène Pasquet, Claire Galambrun, Stéphanie Nguyen, Jean Yves Cahn, Thorsten Braun, Eric Deconinck, Jacques Olivier Bay, Flore Sicre de Fontbrune, Fiorenza Barraco, Gérard Socié
Antithymocyte globulins (ATG) plus cyclosporine (CSA) is the gold standard immunosuppressive treatment (IST) for patients with aplastic anemia. A prospective randomized trial showed in 2011 that hATG was superior to rabbit ATG for first-line treatment of severe AA. The French Health Agency (ANSM) permitted a patient-named Authorization for Temporary Use (ATU) program of hATG (ATGAM, Pfizer) in patients with AA in 2011 since commercial access to hATG is not approved. We took advantage of this program to analyze the outcomes of 465 patients who received ATGAM plus CSA as first line treatment (n=379; 81...
January 27, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29374141/ipsc-modeling-of-severe-aplastic-anemia-reveals-impaired-differentiation-and-telomere-shortening-in-blood-progenitors
#9
Dario Melguizo-Sanchis, Yaobo Xu, Dheraj Taheem, Min Yu, Katarzyna Tilgner, Tomas Barta, Katja Gassner, George Anyfantis, Tengfei Wan, Ramu Elango, Sameer Alharthi, Ashraf A El-Harouni, Stefan Przyborski, Soheir Adam, Gabriele Saretzki, Sujith Samarasinghe, Lyle Armstrong, Majlinda Lako
Aplastic Anemia (AA) is a bone marrow failure (BMF) disorder, resulting in bone marrow hypocellularity and peripheral pancytopenia. Severe aplastic anemia (SAA) is a subset of AA defined by a more severe phenotype. Although the immunological nature of SAA pathogenesis is widely accepted, there is an increasing recognition of the role of dysfunctional hematopoietic stem cells in the disease phenotype. While pediatric SAA can be attributable to genetic causes, evidence is evolving on previously unrecognized genetic etiologies in a proportion of adults with SAA...
January 26, 2018: Cell Death & Disease
https://www.readbyqxmd.com/read/29373264/disseminated-fusariosis-emerged-from-prolonged-local-genital-infection-after-cord-blood-transplantation
#10
Kohei Okada, Tomoyuki Endo, Daigo Hashimoto, Tomoyuki Saga, Takahide Ara, Reiki Ogasawara, Atsushi Yasumoto, Makoto Ibata, Mutsumi Takahata, Akio Shigematsu, Takeshi Kondo, Yasunori Muraosa, Toshifumi Nomura, Hiromi Kanno-Okada, Satoshi Hashino, Shinya Tanaka, Katsuhiko Kamei, Takanori Teshima
Disseminated fusariosis (DF) is a rare life threatening fungal infection in immunocompromised hosts. We herein report a case of a fatal DF mimicking varicella zoster virus (VZV) infection that was emerged from a localized genital infection during cord blood transplantation (CBT) in a patient with severe aplastic anemia (SAA). The patient developed an ulcer following small painful vesicles mimics herpes simplex virus infection (HSV) on the glans penis before CBT, but a Fusarium species was identified. Despite administration of voriconazole, liposomal amphotericin B and granulocyte transfusion, the lesion was extended to extensive skin looked like VZV infection and the patients died after CBT...
January 16, 2018: Journal of Infection and Chemotherapy: Official Journal of the Japan Society of Chemotherapy
https://www.readbyqxmd.com/read/29357782/aplastic-anemia-secondary-to-propylthiouracil-a-rare-and-life-threatening-adverse-effect
#11
Hira Shaikh, Amir Kamran, Soorih Shaikh, Prerna Mewawalla
Background Propylthiouracil has been in use for more than half a century for the treatment of hyperthyroidism. While it is largely known to cause agranulocytosis, its association with aplastic anemia is rarely heard of. Our case will be the third in literature to suggest aplastic anemia as a manifestation of propylthiouracil, which unfortunately culminated in the death of the patient. Case A 67-year-old female, with recently diagnosed metastatic adenocarcinoma of the lung, developed hyperthyroidism after being started on Nivolumab and Iplimumab...
January 1, 2018: Journal of Oncology Pharmacy Practice
https://www.readbyqxmd.com/read/29333401/management-of-immune-thrombocytopenia-korean-experts-recommendation-in-2017
#12
REVIEW
Jun Ho Jang, Ji Yoon Kim, Yeung-Chul Mun, Soo-Mee Bang, Yeon Jung Lim, Dong-Yeop Shin, Young Bae Choi, Ho-Young Yhim, Jong Wook Lee, Hoon Kook
Management options for patients with immune thrombocytopenia (ITP) have evolved substantially over the past decades. The American Society of Hematology published a treatment guideline for clinicians referring to the management of ITP in 2011. This evidence-based practice guideline for ITP enables the appropriate treatment of a larger proportion of patients and the maintenance of normal platelet counts. Korean authority operates a unified mandatory national health insurance system. Even though we have a uniform standard guideline enforced by insurance reimbursement, there are several unsolved issues in real practice in ITP treatment...
December 2017: Blood Research
https://www.readbyqxmd.com/read/29330562/influence-of-genetic-polymorphisms-of-il23r-stat3-il12b-and-stat4-on-the-risk-of-aplastic-anemia-and-the-effect-of-immunosuppressive-therapy
#13
Li Zhao, Huanling Zhu, Bing Han, Lixin Wang, Yuming Sun, Xiaojun Lu, Chunyan Huang, Bin Tan, Chunxia Chen, Li Qin
Studies have suggested that IL-23/STAT3 and IL-12/STAT4 signaling pathways associate with aplastic anemia (AA) occurrence. Polymorphisms in pathway-related genes may contribute to AA risk. In the current study, we investigated the association between polymorphisms in genes of IL23R, STAT3, IL12B, and STAT4 and occurrence, severity, and immunosuppressive outcome of AA in the Han population in southwest China. In the current 164 AA cases and 211 controls study, we found T allele and TT genotype of rs7574865 were more frequent in the cases than that in the controls...
January 12, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/29316578/acute-hepatitis-as-a-prequel-to-very-severe-aplastic-anemia
#14
Christina Weiler-Normann, Johannes Hartl, Sören Weidemann, Ute-Marie von Pein, Walter Fiedler, Christoph Schramm, Florian Brinkert, Nicolaus Kröger, Maximilian Christopeit
Severe aplastic anemia is a rare and potentially life-threatening disease of the bone marrow often requiring allogeneic hematopoietic stem cell transplantation. Pathogenesis of the disease can vary and often remains enigmatic. Occasionally, severe aplastic anemia is associated with prior severe acute hepatitis. Differential diagnosis of acute non-viral hepatitis challenges the physician as pathogenesis remains unclear.We here present a case of a young patient presenting with acute hepatitis followed by severe aplastic anemia successfully treated with allogeneic hematopoietic stem cell transplantation...
January 2018: Zeitschrift Für Gastroenterologie
https://www.readbyqxmd.com/read/29306655/comparative-study-of-porcine-anti-human-lymphocyte-immunoglobulin-and-rabbit-anti-human-thymocyte-immunoglobulin-as-a-first-line-treatment-of-acquired-severe-aplastic-anemia
#15
Miao Chen, Chao Liu, Xinhui Qiao, Daobin Zhou, Junling Zhuang, Bing Han
Porcine anti-human lymphocyte immunoglobulin (pALG) and rabbit anti-human thymocyte immunoglobulin (rATG) are the only two ATGs for severe aplastic anemia (SAA) treatment in China. 148 treatment-naïve SAA patients who received ATG combined with cyclosporine A (CsA) therapy were analysed retrospectively. The patients were divided into a pALG group (n = 114) and a rATG group (n = 34). After three months, the pALG and rATG groups had an overall response (OR) of 65.8% and 44.1%, respectively (P = 0...
January 2, 2018: Leukemia Research
https://www.readbyqxmd.com/read/29301129/amegakaryocytic-thrombocytopenia-and-subsequent-aplastic-anemia-associated-with-apparent-epstein-barr-virus-infection
#16
Ilana Levy, Ruth Laor, Nizar Jiries, Jacob Bejar, Aaron Polliack, Tamar Tadmor
Acquired amegakaryocytic thrombocytopenia (AAT), a rare entity characterized by severe thrombocytopenia and the absence of megakaryocytes in the bone marrow, may mimic or precede the diagnosis of aplastic anemia (AA). Here, we describe a patient who presented with apparent Epstein-Barr virus (EBV)-associated immune thrombocytopenia resistant to several lines of therapies, which was in fact a form of AAT with some features of AA. He eventually responded to therapy with eltrombopag, cyclosporine A (CSA), and antithymocyte globulin (ATG) and recovered completely...
January 5, 2018: Acta Haematologica
https://www.readbyqxmd.com/read/29297952/first-line-choice-for-severe-aplastic-anemia-in-children-transplantation-from-a-haploidentical-donor-vs-immunosuppressive-therapy
#17
Yifei Cheng, Zhengli Xu, Yuanyuan Zhang, Jun Wu, Fengrong Wang, Xiaodong Mo, Yuhong Chen, Wei Han, Jinsong Jia, Yu Wang, Xiaohui Zhang, Xiaojun Huang, Leping Zhang, Lanping Xu
We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = ...
December 18, 2017: Clinical Transplantation
https://www.readbyqxmd.com/read/29281994/successful-treatment-of-cerebral-aspergillosis-case-report-of-a-patient-with-t-cell-large-granular-lymphocytic-leukemia-t-lgl
#18
Amin T Turki, Jassin Rashidi-Alavijeh, Jan Dürig, Guido Gerken, Peter-Michael Rath, Oliver Witzke
BACKGROUND: Invasive aspergillosis involving patients with neutropenia or severe immunosuppression, such as patients with hematologic malignancies is associated with high mortality. Patients with T-cell large granular lymphocytic leukemia (T-LGL) on the other hand are considered to be less vulnerable for severe opportunistic fungal infection as their course of disease is chronic and marked by less violent cytopenia then in e.g. Aplastic Anemia. Only neutropenia is regarded as independent risk factor for severe opportunistic infection in T-LGL patients...
December 28, 2017: BMC Infectious Diseases
https://www.readbyqxmd.com/read/29244176/parvovirus-b19-is-associated-with-a-significant-decrease-in-hemoglobin-level-among-children-5-years-of-age-with-anemia-in-northwestern-tanzania
#19
Yustina A Tizeba, Mariam M Mirambo, Neema Kayange, Tumaini Mhada, Emmanuela E Ambrose, Luke R Smart, Stephen E Mshana
Parvovirus B19 (B19) can cause transient aplastic crisis and lead to acute severe anemia. This study investigated the relationship between B19 and anemia among children <5 years old in the city of Mwanza, Tanzania. An enzyme immunoassay was used to detect B19 IgM- and IgG-specific antibodies among children with various categories of anemia according to the World Health Organization (WHO) guidelines. A total of 265 children with median age of 28.5 months (interquartile range 18-39.5) were investigated. Eighty-six children (32...
December 13, 2017: Journal of Tropical Pediatrics
https://www.readbyqxmd.com/read/29243948/shanghai-health-study-2001-2009-what-was-learned-about-benzene-health-effects
#20
Sherilyn A Gross, Dennis J Paustenbach
The Shanghai Health Study (SHS) was a large epidemiology study conducted as a joint effort between the University of Colorado and Fudan University in Shanghai, China. The study was funded by members of the American Petroleum Institute between 2001 and 2009 and was designed to evaluate the human health effects associated with benzene exposure. Two arms of the SHS included: an occupational-based molecular epidemiology study and several hospital-based case control studies. Consistent with historical literature, following sufficient exposure to relatively high airborne concentrations and years of exposure, the SHS concluded that exposure to benzene resulted in an increased risk of various blood and bone marrow abnormalities such as benzene poisoning, aplastic anemia (AA), myelodysplastic syndrome (MDS), and acute myeloid leukemia (AML)...
December 15, 2017: Critical Reviews in Toxicology
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