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Aplastic anemia

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https://www.readbyqxmd.com/read/29224329/-allogeneic-hematopoietic-stem-cell-transplantation-for-the-treatment-of-severe-aplastic-anemia-complicated-with-hepatocellular-carcinoma-a-case-report-and-literature-review
#1
S W Yang, Z M Zhu, X L Yuan, L Jiang, R J Ma, Y Zhang
No abstract text is available yet for this article.
November 14, 2017: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29222240/old-and-new-tools-in-the-clinical-diagnosis-of-inherited-bone-marrow-failure-syndromes
#2
REVIEW
Allison H West, Jane E Churpek
Patients with inherited bone marrow failure syndromes (IBMFSs) classically present with specific patterns of cytopenias along with congenital anomalies and/or other physical features that are often recognizable early in life. However, increasing application of genomic sequencing and clinical awareness of subtle disease presentations have led to the recognition of IBMFS in pediatric and adult populations more frequently than previously realized, such as those with early onset myelodysplastic syndrome (MDS). Given the well-defined differences in clinical management needs and outcomes for aplastic anemia, acute myeloid leukemia, and MDS in patients with an IBMFS vs those occurring sporadically, as well as nonhematologic comorbidities in patients with IBMFSs, it is critical for hematologists to understand how to approach screening for the currently known IBMFSs...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29222238/clinical-implications-of-somatic-mutations-in-aplastic-anemia-and-myelodysplastic-syndrome-in-genomic-age
#3
REVIEW
Jaroslaw P Maciejewski, Suresh K Balasubramanian
Recent technological advances in genomics have led to the discovery of new somatic mutations and have brought deeper insights into clonal diversity. This discovery has changed not only the understanding of disease mechanisms but also the diagnostics and clinical management of bone marrow failure. The clinical applications of genomics include enhancement of current prognostic schemas, prediction of sensitivity or refractoriness to treatments, and conceptualization and selective application of targeted therapies...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29217821/il-11-promotes-the-treatment-efficacy-of-hematopoietic-stem-cell-transplant-therapy-in-aplastic-anemia-model-mice-through-a-nf-%C3%AE%C2%BAb-microrna-204-thrombopoietin-regulatory-axis
#4
Yan Wang, Zhi-Yun Niu, Yu-Jie Guo, Li-Hua Wang, Feng-Ru Lin, Jing-Yu Zhang
Hematopoietic stem cell (HSC) transplantation could be of therapeutic value for aplastic anemia (AA) patients, and immunosuppressants may facilitate the efficiency of the procedure. As anti-inflammatory cytokine interleukin-11 (IL-11) has a thrombopoietic effect, its use in cases of chronic bone marrow failure, such as AA, has been proposed to induce HSC function. However, the putative mechanisms that may support this process remain poorly defined. We found that decreased miR-204-5p levels were coincident with increased proliferation in mouse HSCs following exposure to IL-11 in vitro...
December 8, 2017: Experimental & Molecular Medicine
https://www.readbyqxmd.com/read/29203412/comparable-outcomes-after-hla-matched-sibling-and-alternative-donor-hematopoietic-cell-transplantation-for-children-with-fanconi-anemia-and-severe-aplastic-anemia
#5
Christen L Ebens, Todd E DeFor, Rebecca Tryon, John E Wagner, Margaret L MacMillan
Fanconi anemia (FA) associated severe aplastic anemia (SAA) requires allogeneic hematopoietic cell transplantation (HCT) for cure. With the evolution of conditioning regimens over time, alternative donor HCT (AD-HCT) outcomes have dramatically improved. We compared outcomes of HLA-matched sibling donor HCT (MSD-HCT, n=17) to AD-HCT (n=57) for FAassociated SAA at a single institution from 2001-2016. Overall survival at five years was 94 vs. 86%, neutrophil engraftment 100 vs. 95%, platelet recovery 100 vs. 89%, grade II-IV acute GVHD 6 vs...
December 1, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29184906/parvovirus-b19-in-the-context-of-hematopoietic-stem-cell-transplantation-evaluating-cell-donors-and-recipients
#6
Bianca E Gama, Vanessa E Emmel, Michelle Oliveira-Silva, Luciana M Gutiyama, Leonardo Arcuri, Marta Colares, Rita de Cássia Tavares, Luis F Bouzas, Eliana Abdelhay, Rocio Hassan
Background: Parvovirus B19 (B19V) is a common human pathogen, member of the family Parvoviridae. Typically, B19V has been found to infect erythroid progenitors and cause hematological disorders, such as anemia and aplastic crisis. However, the persistence of genomic deoxyribonucleic acid (DNA) has been demonstrated in tonsils, liver, skin, brain, synovial, and testicular tissues as well as bone marrow, for both symptomatic and asymptomatic subjects. Although the molecular and cellular mechanisms of persistence remain undefined, it raises questions about potential virus transmissibility and its effects in the context of allogeneic hematopoietic stem cell transplantation (allo-HSCT) recipients...
November 2017: Transplantation Direct
https://www.readbyqxmd.com/read/29181263/cytogenetic-study-is-not-essential-in-patients-with-aplastic-anemia
#7
Atreyee Dutta, Rajib De, Tuphan K Dolai, Pradip K Mitra, Ajanta Halder
Depending on contemporary treatment approach of aggressive immunosuppression, Aplastic Anemia (AA) is caused by immunological destruction of otherwise normal hematopoietic stem cells. The aim was to summarize the cytogenetic abnormalities in AA patients and the frequency of Fanconi Anemia (FA) in morphologically normal AA patients in eastern India. Ethical clearances were obtained from both institutions involved in this study. Out of 72800 patients attending the outpatient department, 520 pancytopenia patients were screened for AA after Bone marrow (BM) aspiration and biopsy...
2017: American Journal of Blood Research
https://www.readbyqxmd.com/read/29173808/epigenetic-and-microenvironmental-alterations-in-bone-marrow-associated-with-ros-in-experimental-aplastic-anemia
#8
Ritam Chatterjee, Sujata Law
Aplastic anemia or bone marrow failure often develops as an effect of chemotherapeutic drug application for the treatment of various pathophysiological conditions including cancer. The long-term bone marrow injury affects the basic hematopoietic population including hematopoietic stem/progenitor cells (HSPCs). The present study aimed in unearthing the underlying mechanisms of chemotherapeutics mediated bone marrow aplasia with special focus on altered redox status and associated effects on hematopoietic microenvironment and epigenetic status of hematopoietic cells...
November 22, 2017: European Journal of Cell Biology
https://www.readbyqxmd.com/read/29170252/nationwide-survey-on-the-use-of-eltrombopag-in-patients-with-severe-aplastic-anemia-a-report-on-behalf-of-the-french-reference-center-for-aplastic-anemia
#9
Etienne Lengline, Bernard Drenou, Pierre Peterlin, Olivier Tournilhac, Julie Abraham, Ana Berceanu, Brigitte Dupriez, Gaelle Guillerm, Emmanuel Raffoux, Flore Sicre de Fontbrune, Lionel Ades, Marie Balsat, Driss Chaoui, Paul Coppo, Selim Corm, Thierry Leblanc, Natacha Maillard, Louis Terriou, Gerard Socié, Regis Peffault de la Tour
Few therapeutic options are available for aplastic anemia patients ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag recently showed tri-lineage responses in refractory patients. However, real-life use of this drug remains unknown. This retrospective study (2012-2016) was conducted by the French Reference Center for Aplastic anemia on patients with relapse/refractory aplastic anemia, and patients ineligible for anti-thymocyte globulin (naive) or transplantation, who received eltrombopag for at least two months...
November 23, 2017: Haematologica
https://www.readbyqxmd.com/read/29167806/aplastic-anemia-is-related-to-alterations-in-t-cell-receptor-signaling
#10
REVIEW
Yankai Xiao, Suwen Zhao, Bo Li
Aplastic anemia (AA) is a disease characterized by bone marrow hematopoietic dysfunction and peripheral blood pancytopenia, which is thought to be mediated by an abnormal T cell-induced immune response. T cell receptor (TCR) signaling is pivotal for T cell development and function. An aberrant TCR signaling leads to an unbalanced immune system that can result in a range of immune-related disorders, including autoimmune diseases, chronic infections, and tumors. In this article, we briefly review the T cell immune pathophysiology of AA, the physiology of normal TCR signaling and its regulatory factors, and clinical and laboratory findings of TCR signaling molecules and their regulatory factors in AA...
2017: Stem Cell Investigation
https://www.readbyqxmd.com/read/29164009/next-generation-gene-sequencing-differentiates-hypoplastic-myelodysplastic-syndrome-from-aplastic-anemia
#11
Jeffrey L Lew, Joshua L Fenderson, Mark G Carmichael
Hypoplastic Myelodysplastic Syndrome (h-MDS) comprises 15% of all MDS and has traditionally been difficult to distinguish from aplastic anemia (AA) by current testing. Accurate differentiation is important because treatment and prognosis differ. Since the publication of the 2008 World Health Organization classification of MDS, next-generation DNA sequencing has discovered novel mutations strongly associated with AA and MDS. Recent research supports the utility of identifying these mutations in the diagnosis and management of MDS; however, use of next-generation sequencing is not yet recommended in guidelines and the study is not routinely performed...
November 2017: Hawai'i Journal of Medicine & Public Health: a Journal of Asia Pacific Medicine & Public Health
https://www.readbyqxmd.com/read/29146883/a-landscape-of-germline-mutations-in-a-cohort-of-inherited-bone-marrow-failure-patients
#12
Olivier Bluteau, Marie Sebert, Thierry Leblanc, Régis Peffault de Latour, Samuel Quentin, Elodie Lainey, Lucie Hernandez, Jean-Hugues Dalle, Flore Sicre de Fontbrune, Etienne Lengline, Raphael Itzykson, Emmanuelle Clappier, Nicolas Boissel, Naddia Vasquez, Mélanie Da Costa, Julien Masliah-Planchon, Wendy Cuccuini, Anna Raimbault, Louis De Jaegere, Lionel Adès, Pierre Fenaux, Sébastien Maury, Claudine Schmitt, Marc Muller, Carine Domenech, Nicolas Blin, Bénédicte Bruno, Isabelle Pellier, Mathilde Hunault, Stéphane Blanche, Arnaud Petit, Guy Leverger, Gérard Michel, Yves Bertrand, André Baruchel, Gérard Socié, Jean Soulier
Bone marrow failure (BMF) in children and young adults is often suspected to be inherited, but in many cases diagnosis remains uncertain. We studied a cohort of 179 patients (from 173 families) with BMF of suspected inherited origin but unresolved diagnosis after medical evaluation and Fanconi anemia exclusion. All patients had cytopenias, and 12.0% presented ≥5% bone marrow blast cells. Median age at genetic evaluation was 11 years; 20.7% of patients were aged ≤2 years and 36.9% were ≥18 years. We analyzed genomic DNA from skin fibroblasts using whole-exome sequencing, and were able to assign a causal or likely causal germline mutation in 86 patients (48...
November 16, 2017: Blood
https://www.readbyqxmd.com/read/29143887/diagnosis-and-treatment-of-aplastic-anemia
#13
REVIEW
Scott A Peslak, Timothy Olson, Daria V Babushok
Acquired aplastic anemia (AA) is a rare, life-threatening bone marrow failure (BMF) disorder that affects patients of all ages and is caused by lymphocyte destruction of early hematopoietic cells. Diagnosis of AA requires a comprehensive approach with prompt evaluation for inherited and secondary causes of bone marrow aplasia, while providing aggressive supportive care. The choice of frontline therapy is determined by a number of factors including AA severity, age of the patient, donor availability, and access to optimal therapies...
November 16, 2017: Current Treatment Options in Oncology
https://www.readbyqxmd.com/read/29140934/response-of-symptomatic-persistent-chronic-disseminated-candidiasis-to-corticosteroid-therapy-in-immunosuppressed-pediatric-patients-case-study-and-review-of-the-literature
#14
Vered Shkalim-Zemer, Itzhak Levi, Salvador Fischer, Hannah Tamary, Joanne Yakobovich, Gali Avrahami, Gil Gilad, Sara Elitzur, Isaac Yaniv, Ronit Elhasid, Michal Manistersky, Itamar Shalit
BACKGROUND: Chronic disseminated candidiasis (CDC) is a severe invasive fungal infection principally observed during neutrophil recovery in patients with acute leukemia treated with intensive chemotherapy. Its pathophysiology remains unclear. We describe the management of six children with symptomatic CDC who did not respond to antifungal therapy. METHODS: The databases of the hematology-oncology departments of two tertiary pediatric medical centers were searched for all patients diagnosed with CDC from 2003 to 2015 who responded to corticosteroids after failing antifungal therapy...
November 14, 2017: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/29137385/improved-outcome-of-haploidentical-transplantation-in-severe-aplastic-anemia-using-reduced-intensity-fludarabine-based-conditioning
#15
Wu Yamei, Luo Rongmu, Cao Yongbin, Si Yingjian, Li Xiaohong, Zhang Xiaomei, Yan Pei, Du Zhenlan, Wang Haitao, Wang Jing, Wang Bojing, Wu Xiaoxiong, Da Wanming
Significant improvements in hematopoietic stem cell transplantation (HSCT) with haploidentical family donors (HFD) have confirmed its therapeutic role in severe aplastic anemia (SAA) and led to the evolution of treatment algorithms. However, the optimal conditioning regimen for HFD-HSCT remains undefined, especially the dosage of cyclophosphamide (Cy). A total of 77 patients with SAA from two research centers, who received HFD-HSCT with reduced-intensity fludarabine + cyclophosphamide + thymoglobulin ± busulfan conditioning regimen plus third-party cells infusion were included in this study, of which 67 pairs had 4-5 loci mismatched...
October 13, 2017: Oncotarget
https://www.readbyqxmd.com/read/29131151/combined-intensive-immunosuppression-and-eculizumab-for-aplastic-anemia-in-the-context-of-hemolytic-paroxysmal-nocturnal-hemoglobinuria-a-retrospective-analysis
#16
S Pagliuca, A M Risitano, F S De Fontbrune, M Robin, A P Iori, S Marotta, D Michonneau, A Villate, D Desmier, G Socié, R P De Latour
No abstract text is available yet for this article.
November 13, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29128072/paroxysmal-nocturnal-hemoglobinuria-assessment-by-flow-cytometric-analysis
#17
REVIEW
Mike Keeney, Andrea Illingworth, D Robert Sutherland
Paroxysmal nocturnal hemoglobinuria (PNH) is an uncommon but frequently debilitating disease that, if untreated, may lead to death in up to 35% of patients within 5 years. Assessment of PNH clone size by flow cytometric analysis has increased in importance with the availability of therapeutic treatments, which prevent the hemolysis of red blood cells and, hence, the myriad symptoms that accompany the disease. This article addresses flow cytometric methodologies and highlights areas of importance in implementing testing, not only for classic PNH but also for other related bone marrow failure disorders, such as aplastic anemia and low-grade myelodysplastic syndrome...
December 2017: Clinics in Laboratory Medicine
https://www.readbyqxmd.com/read/29127946/seasonal-trends-of-diagnosis-of-childhood-malignant-diseases-and-viral-prevalence-in-south-korea
#18
Kyu Seok Shim, Min Hyung Kim, Choong Nam Shim, Minkyu Han, In Seok Lim, Soo Ahn Chae, Sin Weon Yun, Na Mi Lee, Dae Yong Yi, Hyery Kim
BACKGROUND: Several studies have reported a seasonal trend in the diagnosis of childhood cancer suggesting seasonal factors such as infection. The present study aimed to analyze the diagnosis pattern of childhood malignant diseases using public health data, and to compare this pattern with seasonal viral infection trends. METHOD: Using the open data source of the Health Insurance Review and Assessment Service, we extracted data regarding all patients under 21 years of age and who had any cancer, aplastic anemia or myelodysplastic syndrome between September 2009 and December 2013...
November 8, 2017: Cancer Epidemiology
https://www.readbyqxmd.com/read/29125443/neurosurgical-management-in-children-with-bleeding-diathesis-auditing-neurological-outcome
#19
Zaitun Zakaria, Chandrasekaran Kaliaperumal, Darach Crimmins, John Caird
OBJECTIVE The aim of this study was to assess the outcome of neurosurgical treatment in children with bleeding diathesis and also to evaluate the current management plan applied in the authors' service. METHODS The authors retrospectively analyzed all cases in which neurosurgical procedures were performed in pediatric patients presenting with intracranial hematoma due to an underlying bleeding tendency over a 5-year period at their institution. They evaluated the patients' neurological symptoms from the initial referral, hematological abnormalities, surgical treatment, neurological outcome, and scores on the Pediatric Glasgow Outcome Scale-Extended (GOS-E Peds) obtained 1 year after the last operation...
November 10, 2017: Journal of Neurosurgery. Pediatrics
https://www.readbyqxmd.com/read/29119669/sinusitis-caused-by-exserohilum-rostratum-after-cord-blood-transplantation-for-myelodysplastic-syndrome-a-case-report-and-literature-review
#20
Sumiko Kohashi, Takaaki Toyama, Norisato Hashimoto, Masatoshi Sakurai, Jun Kato, Taku Kikuchi, Yuya Koda, Kayoko Sugita, Naoki Hasegawa, Kyoko Yarita, Katsuhiko Kamei, Shinichiro Okamoto, Takehiko Mori
Invasive fungal disease is a serious infectious complication of allogeneic hematopoietic stem cell transplantation (HSCT). Exserohilum rostratum is a species causing phaeohyphomycosis, which rarely causes invasive disease in humans. We treated a case of sinusitis caused by E. rostratum after cord blood transplantation (CBT). A 60-year-old man with myelodysplastic syndrome, who had a medical history of an operation to correct deviation of the nasal septum, developed sinusitis caused by E. rostratum under prolonged profound neutropenia after a second CBT because of the graft rejection of the first transplantation...
November 8, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
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