19,537 results match your criteria Aplastic Anemia


CD8GITR T cells may negatively regulate T cell overactivation in aplastic anemia.

Immunol Invest 2020 May 28:1-10. Epub 2020 May 28.

Institute of Hematology, School of Medicine, Jinan University , Guangzhou, China.

Aplastic anemia (AA) is a T cell immune-mediated autoimmune disease. Overactivated CD8 T cells play a leading role in the pathogenesis of AA, which may be due to disbalance in costimulatory and coinhibitory signals in T cells. In this study, we firstly investigated the expression of OX40, 4-1BB, GITR, ICOS, CTLA-4, LAG-3, and TIM-3 on CD8 T cells from untreated patients with AA and healthy individuals (HIs) by flow cytometry. Read More

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http://dx.doi.org/10.1080/08820139.2020.1770785DOI Listing

Transient aplastic crisis triggered by parvovirus B19 in a family with hereditary spherocytosis.

IDCases 2020 11;21:e00802. Epub 2020 May 11.

Pathogenesis and Control of Chronic Infections, University of Montpellier, INSERM, EFS, Montpellier University Medical Centre, 34090, Montpellier, France.

Acute parvovirus B19 infection may lead to erythroblastopenia crisis in patients with underlying red blood cells disorders. We report herein an uncommon concomitant transient aplastic crisis in a mother and her daughter, both affected by hereditary spherocytosis. The diagnosis was confirmed by the detection of a very high parvovirus B19 DNA load in both the mother's and daughter's sera, associated with the presence of parvovirus B19 specific immunoglobulin-M antibodies. Read More

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http://dx.doi.org/10.1016/j.idcr.2020.e00802DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7242858PMC

Treating a Patient with Your Hands Tied: Acute Chest Syndrome in a Jehovah's Witness.

Cureus 2020 Apr 21;12(4):e7769. Epub 2020 Apr 21.

Pulmonary Critical Care, University of Connecticut Health Center, Farmington, USA.

Acute chest syndrome (ACS), a vaso-occlusive crisis in patients with sickle cell anemia, is a life-threatening condition and a leading cause of death in these patients. It is treated with analgesics, antibiotics, intravenous fluid, supplemental oxygen (or ventilatory support in severe cases) with simple or exchange transfusion, being the mainstay of therapy. We report a young Jehovah's Witness (JW) patient with sickle cell anemia who presented with ACS. Read More

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http://dx.doi.org/10.7759/cureus.7769DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7243639PMC

COVID-19-related acute necrotizing encephalopathy with brain stem involvement in a patient with aplastic anemia.

Neurol Neuroimmunol Neuroinflamm 2020 09 26;7(5). Epub 2020 May 26.

From the Department of Imaging (L.D., A.G., D. Mallon, F.T., B.J.), Imperial College Healthcare NHS Trust; Department of Neurosciences (J.V., I.H.J., A.E.), Imperial College Healthcare NHS Trust; Northwest London Pathology (D. Muir); Department of Hematology (A.L.), Imperial College Healthcare NHS Trust; Centre for Neuroinflammation and Neurodegeneration (R.N.), Faculty of Medicine, Imperial College London; and Department of Visual Neuroscience (R.N.), UCL Institute of Ophthalmology, London, United Kingdom.

Objective: To describe a novel case of coronavirus disease 2019 (COVID-19)-associated acute necrotizing encephalopathy (ANE) in a patient with aplastic anemia where there was early brain stem-predominant involvement.

Methods: Evaluation of cause, clinical symptoms, and treatment response.

Results: A 59-year-old woman with a background of transfusion-dependent aplastic anemia presented with seizures and reduced level of consciousness 10 days after the onset of subjective fever, cough, and headache. Read More

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http://dx.doi.org/10.1212/NXI.0000000000000789DOI Listing
September 2020

FLAER-negative CD15+ neutrophils can be used for the simplified screening of suspected PNH cases.

Int J Lab Hematol 2020 May 25. Epub 2020 May 25.

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.

Background: The flow cytometry analysis of GPI-linked proteins on red blood cells and leukocytes is crucial for paroxysmal nocturnal hemoglobinuria (PNH) diagnostics. However, the commonly used multicolor panels cannot be implemented in low-resourced hematology laboratories. In order to develop a simple prediagnostic test for PNH screening, we analyzed the diagnostic accuracy of the two-color (FLAER/CD15) detection of GPI-deficient neutrophils. Read More

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http://dx.doi.org/10.1111/ijlh.13239DOI Listing

Pharmacokinetic and pharmacodynamic effects of ravulizumab and eculizumab on complement component 5 in adults with paroxysmal nocturnal haemoglobinuria: results of two phase 3 randomised, multicentre studies.

Br J Haematol 2020 May 24. Epub 2020 May 24.

Clinical Haematology at Peter MacCallum Cancer Centre, The Royal Melbourne Hospital and University of Melbourne, Melbourne, Australia.

Ravulizumab, a novel long-acting complement component 5 (C5) inhibitor administered every 8 weeks (q8w), was non-inferior to eculizumab for all efficacy outcomes in two randomised, open-label, phase 3 trials in C5 inhibitor-naïve (Study 301) and eculizumab-experienced (Study 302) adult patients with paroxysmal nocturnal haemoglobinuria (PNH). This pre-specified analysis characterised ravulizumab pharmacokinetics (PK), pharmacodynamics (PD; free C5 levels), and PD differences between medications (Study 301, n = 246; Study 302, n = 195). Ravulizumab PK parameters were determined using non-compartmental analysis. Read More

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http://dx.doi.org/10.1111/bjh.16711DOI Listing

Conditioning regimen for allogeneic bone marrow transplantation in children with acquired bone marrow failure: fludarabine/melphalan vs. fludarabine/cyclophosphamide.

Bone Marrow Transplant 2020 May 23. Epub 2020 May 23.

Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.

Fludarabine/cyclophosphamide-based conditioning regimens are standard in bone marrow transplantation (BMT) for acquired bone marrow failure in children, however, graft failure may occur. Using the data from a nationwide transplantation registry, we compared the outcomes of children aged <16 years with acquired aplastic anemia and refractory cytopenia of childhood who underwent allogeneic BMT with either fludarabine/melphalan (n = 71) or fludarabine/cyclophosphamide (n = 296) between 2000 and 2016. The fludarabine/melphalan regimen provided excellent outcomes, with 3-year overall survival and failure-free survival rates of 98% and 97%, respectively. Read More

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http://dx.doi.org/10.1038/s41409-020-0948-8DOI Listing

Second Allogeneic Hematopoietic Stem Cell Transplantation After Donor Replacement in Children With Severe Aplastic Anemia.

Transplant Proc 2020 May 19. Epub 2020 May 19.

Department of Hematology, The Affiliated Cancer Hospital of Zhengzhou University/Henan Cancer Hospital, Zhengzhou, China. Electronic address:

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective measure for the treatment of severe aplastic anemia (SAA). While infection, graft failure, and graft-vs-host disease (GVHD) are the main causes of allo-HSCT failure, a second HSCT is needed to eliminate the dependence of blood transfusion and maintain disease-free survival. We applied low-dose total body irradiation (TBI) + fludarabine (FLU) + cyclophosphamide (CTX) + antilymphocyte globulin (ALG) + busulfan (BU) as a conditioning regimen of second HSCT after a transplantation with an HLA-mismatched donor. Read More

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http://dx.doi.org/10.1016/j.transproceed.2020.01.138DOI Listing

Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome.

Haematologica 2020 May 21. Epub 2020 May 21.

National Heart, Lung, and Blood Institute.

Myelodysplastic syndromes (MDS) are a group of clonal myeloid disorders characterized by cytopenia and a propensity to develop acute myeloid leukemia (AML). The management of lower-risk (LR) MDS with persistent cytopenias remains suboptimal. Eltrombopag (EPAG), a thrombopoietin receptor agonist, can improve platelet counts in LR-MDS and tri-lineage hematopoiesis in aplastic anemia (AA). Read More

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http://dx.doi.org/10.3324/haematol.2020.249995DOI Listing

A frequent nonsense mutation in exon 1 across certain HLA-A and -B alleles in leukocytes of patients with acquired aplastic anemia.

Haematologica 2020 May 21. Epub 2020 May 21.

Department of Hematology, Kanazawa University, Kanazawa, Japan;

Leukocytes that lack HLA allelic expression are frequently detected in patients with acquired aplastic anemia (AA) who respond to immunosuppressive therapy (IST), although the exact mechanisms underlying the HLA loss and HLA allele repertoire likely to acquire loss-of-function mutations are unknown. We identified a common nonsense mutation at position 19 (c.19C>T, p. Read More

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http://dx.doi.org/10.3324/haematol.2020.247809DOI Listing

Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria.

Blood 2020 May 19. Epub 2020 May 19.

University of Pennsylvania, Philadelphia, Pennsylvania, United States.

Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are pathogenically related non-malignant bone marrow failure disorders linked to T-cell mediated autoimmunity and associated with an increased risk of secondary myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Approximately 15-20% of AA patients and 2-6% of PNH patients go on to develop secondary MDS/AML by ten years of follow-up. Factors determining an individual patient's risk of malignant transformation remain poorly defined. Read More

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http://dx.doi.org/10.1182/blood.2019000940DOI Listing

Hematopoietic Stem Cell Transplant for Shwachman Diamond Syndrome.

Biol Blood Marrow Transplant 2020 May 16. Epub 2020 May 16.

Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI. Electronic address:

We report the outcome of hematopoietic cell transplantation for 52 patients with Shwachman-Diamond syndrome (SDS) transplanted between 2000 and 2017. The median age at transplant was 11 years with a median follow-up of 60 months. Transplant indication was bone marrow failure (BMF; cytopenia or aplastic anemia) in 39 patients and myelodysplasia or acute myeloid leukemia in 13 patients. Read More

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http://dx.doi.org/10.1016/j.bbmt.2020.04.029DOI Listing

Aplastic Crisis in a Woman with Autoimmune Hemolytic Anemia.

Indian J Hematol Blood Transfus 2020 Apr 25;36(2):422-423. Epub 2019 Oct 25.

2Department of Pathology, Apollo Hospitals, Hyderabad, 500034 India.

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http://dx.doi.org/10.1007/s12288-019-01222-6DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7229053PMC

Inhibition of 15-PGDH Protects Mice from Immune-mediated Bone Marrow Failure.

Biol Blood Marrow Transplant 2020 May 15. Epub 2020 May 15.

Department of Medicine, Case Western Reserve University, Cleveland, OH, 44106, USA. Electronic address:

Aplastic anemia (AA) is a human immune mediated bone-marrow failure syndrome that is treated by stem cell transplantation for patients who have a matched related donor or immunosuppressive therapy (IST) for those who do not. Responses to IST are variable, with patients still at risk for prolonged neutropenia, transfusion-dependence, immune suppression, and severe opportunistic infections. Therefore, additional therapies to accelerate hematologic recovery in patients receiving front line IST are needed. Read More

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http://dx.doi.org/10.1016/j.bbmt.2020.04.010DOI Listing

New Insights of Human Parvovirus B19 in Modulating Erythroid Progenitor Cell Differentiation.

Viral Immunol 2020 May 15. Epub 2020 May 15.

Pediatrics Department, Children Digital and Health Data Research Center, Zhongnan Hospital of Wuhan University, Wuhan, China.

Human parvovirus B19 (B19), a human pathogen of the erythroparvovirus genus, is responsible for a variety of diseases. B19 cause less symptoms in healthy individuals, also cause acute and chronic anemia in immunodeficiency patients. Transient aplastic crisis and pure red cell aplasia are two kinds of anemic hemogram, respectively, in acute and chronic B19 infection phase, especially occurring in patients with a shortened red cell survival or with immunodeficiency. Read More

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http://dx.doi.org/10.1089/vim.2020.0013DOI Listing

BK virus encephalitis and end-stage renal disease in a child with hematopoietic stem cell transplantation.

Pediatr Transplant 2020 May 15:e13739. Epub 2020 May 15.

Division of Pediatric Nephrology, Department of Pediatrics, University of Florida, Gainesville, FL, USA.

BK virus encephalitis after HSCT is uncommon. Several reports of native kidney BKVN in patients with HSCT, hematologic malignancies, human immunodeficiency virus infection, and non-renal solid organ transplantation have been described. However, an uncommon combination of BK encephalitis and ESRD of native kidneys secondary to BK virus in a child with HSCT has not been described. Read More

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http://dx.doi.org/10.1111/petr.13739DOI Listing

Morphogen signaling by Wnt/β-catenin pathway and microenvironmental alteration in the bone marrow of agricultural pesticide exposure-induced experimental aplastic anemia.

J Biochem Mol Toxicol 2020 May 14:e22523. Epub 2020 May 14.

Stem Cell Research and Application Unit, Department of Biochemistry and Medical Biotechnology, Calcutta School of Tropical Medicine, Kolkata, West Bengal, India.

The etiologic link between pesticide toxicity and aplastic anemia in agricultural and agro-industrial setting has been frequently reported in epidemiological studies conducted worldwide. Chronic pesticide toxicity causes long-term bone marrow injury and perturbs the normal hematopoietic physiology, including survival of hematopoietic progenitor cells and bone marrow's blood cell forming ability. The purpose of this study is to understand the mechanism of pesticide toxicity-mediated bone marrow aplasia by studying Wnt/β-catenin signaling pathway and microenvironmental stromal components. Read More

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http://dx.doi.org/10.1002/jbt.22523DOI Listing

COVID-19 transmission and blood transfusion: A case report.

J Infect Public Health 2020 May 13. Epub 2020 May 13.

Department of Internal Medicine, Kyungpook National University Hospital, School of Medicine, Kyungpook National University, Daegu, Republic of Korea.

The recent outbreak of the novel coronavirus disease 2019 (COVID-19) has been labelled as a pandemic by the World Health Organization. Although person-to-person transmission of the etiologic agent, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has been confirmed, it is not known whether COVID-19 may be transmitted by blood transfusion. Notwithstanding the urgent requirement of blood, it is critical to know whether the SARS-CoV-2 virus can be transmitted by blood transfusion because many individuals may be asymptomatic carriers and may donate blood. Read More

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http://dx.doi.org/10.1016/j.jiph.2020.05.001DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7218366PMC

[Eltrombopag combined with cyclosporine alone for treatment of very severe aplastic anemia: a case report].

Authors:
H Cheng X Wang K L Xu

Zhonghua Xue Ye Xue Za Zhi 2019 Jul;40(7):612

Department of Hematology, The Affiliated Hospital of Xuzhou Medical University, Key Laboratory of Bone Marrow Stem Cell, Xuzhou 221000, China.

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http://dx.doi.org/10.3760/cma.j.issn.0253-2727.2019.07.017DOI Listing

Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases.

Blood Adv 2020 May;4(9):2084-2094

Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University, New York, NY.

We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Read More

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http://dx.doi.org/10.1182/bloodadvances.2019000839DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7218429PMC

Opportunistic bacterial, viral and fungal infections of the lung.

Medicine (Abingdon) 2020 May 8. Epub 2020 May 8.

is a Consultant in respiratory medicine at the Royal Brompton & Harefield NHS Foundation Trust, London, UK. His specialist interest is in respiratory infections and bronchiectasis in immunocompromised hosts, particularly immunodeficiency secondary to cancer and biological therapy. Competing interests: none declared.

Opportunistic infections are a major cause of morbidity and mortality in severely immunocompromised patients, such as those given chemotherapy or biological therapies, and those with haematological malignancy, aplastic anaemia or HIV infection, or recipients of solid organ or stem cell transplants. The type and degree of immune defect dictates the profile of potential opportunistic pathogens; T-cell-mediated defects increase the risk of viral (cytomegalovirus, respiratory viruses) and infections, whereas neutrophil defects are associated with bacterial pneumonia and invasive aspergillosis. However, patients often have combinations of immune defects, and a wide range of other opportunistic infections can cause pneumonia. Read More

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http://dx.doi.org/10.1016/j.mpmed.2020.03.006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7206443PMC

Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia.

Biol Blood Marrow Transplant 2020 May 8. Epub 2020 May 8.

Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Division of Hematology-Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.

With the COVID-19 pandemic and the ensuing barriers to the collection and transport of donor cells, it is often necessary to collect and cryopreserve grafts before initiation of transplantation conditioning. The effect on transplantation outcomes in nonmalignant disease is unknown. This analysis examined the effect of cryopreservation of related and unrelated donor grafts for transplantation for severe aplastic anemia in the United States during 2013 to 2019. Read More

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http://dx.doi.org/10.1016/j.bbmt.2020.04.027DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7206419PMC

An Unusual Case of Morphea in the Setting of Aplastic Anemia.

Cureus 2020 Apr 6;12(4):e7562. Epub 2020 Apr 6.

Department of Dermatology, University of Nevada Las Vegas School of Medicine, Las Vegas, USA.

Cutaneous sclerosis occurs in association with a variety of systemic diseases, including hematologic malignancy, plasma cell dyscrasias, solid organ tumors, and other systemic autoimmune conditions. Herein, we present a unique case of morphea/lichen sclerosus overlap arising in association with aplastic anemia. To expand upon this rare case, we also review the literature surrounding paraneoplastic sclerosing skin disorders. Read More

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http://dx.doi.org/10.7759/cureus.7562DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7202578PMC

Immunosuppression and growth factors for severe aplastic anemia: new data for old questions.

Haematologica 2020 May;105(5):1170-1171

Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA.

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http://dx.doi.org/10.3324/haematol.2020.246512DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7193508PMC

Proteomic profiling and bioinformatics analysis identify key regulators during the process from fanconi anemia to acute myeloid leukemia.

Am J Transl Res 2020 15;12(4):1415-1427. Epub 2020 Apr 15.

Department of Hematology & Oncology, Children's Hospital of Soochow University Suzhou 215025, Jiangsu, China.

Fanconi anemia (FA) is a congenital aplastic anemia, characterized as congenital bone marrow failure, developmental malformation, and the malignant tendency, which may develop into acute myeloid leukemia (AML). However, few studies have been conducted on the progression from FA to AML. In this study, we used proteomic profiling, together with bioinformatics analyses, to explore the molecular mechanisms by which FA progresses to AML. Read More

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7191167PMC

Comparative study on hemoglobin A1c, glycated albumin and glycosylated serum protein in aplastic anemia patients with Type 2 diabetes mellitus.

Biosci Rep 2020 May;40(5)

Laboratory Medicine Centre, Zhongshan Hospital Affiliated to Sun Yat-sen University, Zhongshan 528403, Guangdong, China.

Objective: To differentiate the value of hemoglobin A1c (HbA1c), glycated albumin (GA) and glycosylated serum protein (GSP) in monitoring blood glucose of patients with aplastic anemia.

Methods: 42 patients with aplastic anemia (AA) and 30 patients with AA and Type 2 diabetes mellitus (T2DM) were enrolled in the study, in comparison with 114 healthy subjects and 88 subjects with T2DM. HbA1c, GA, GSP, fasting plasma glucose (FPG), hemoglobin (Hb) and albumin (ALB) were measured, and group comparison and correlation analysis were carried out. Read More

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http://dx.doi.org/10.1042/BSR20192300DOI Listing

Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT).

Bone Marrow Transplant 2020 Apr 28. Epub 2020 Apr 28.

King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.

Aplastic anemia (AA) is a serious hematological disorder, which is solely cured by hematopoietic stem cell transplantation (HSCT). Haploidentical HSCT is an emerging modality with encouraging outcomes in several blood conditions. The present study aims to comprehensively assess the feasibility and safety of haploidentical HSCT in patients with severe and very severe AA. Read More

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http://dx.doi.org/10.1038/s41409-020-0897-2DOI Listing

Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide.

Blood Adv 2020 Apr;4(8):1770-1779

Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.

Severe aplastic anemia (SAA) is a stem cell disorder often treated with bone marrow transplantation (BMT) to reconstitute hematopoiesis. Outcomes of related HLA-haploidentical (haplo) donors after reduced-intensity conditioning with intensive graft-versus-host disease (GVHD) prophylaxis including posttransplantation cyclophosphamide are presented here from 37 SAA, 20 relapsed/refractory (R/R), and 17 treatment-naïve (TN) SAA patients. Median follow-up is 32 months (90% confidence interval [CI], 29-44). Read More

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http://dx.doi.org/10.1182/bloodadvances.2020001729DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7189283PMC

Up-front Treatment With Romiplostim in Children With Acquired Bone Marrow Failure: A Single Institutional Pediatric Case Series.

J Pediatr Hematol Oncol 2020 Apr 24. Epub 2020 Apr 24.

Stead Family Department of Pediatrics.

Background: Thrombopoietin receptor agonists are emerging as a therapeutic option for patients with aplastic anemia (AA) and myelodysplastic syndrome (MDS). We report our experience of treating children with AA/MDS with romiplostim, thrombopoietin receptor agonist.

Observations: Three children (AA, 2; MDS, 1) received romiplostim treatment at a median dose of 10 μg/kg/week (starting dose: 5 μg/kg/wk; 2. Read More

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http://dx.doi.org/10.1097/MPH.0000000000001810DOI Listing

Identification of potential miRNA-mRNA interaction network in bone marrow T cells of acquired aplastic anemia.

Hematology 2020 Dec;25(1):168-175

Department of Pediatrics, Tongji Hospital, Tongji University School of Medicine, Shanghai, People's Republic of China.

MicroRNAs(miRNAs) can regulate T cell differentiation and plasticity by targeting their corresponding message RNAs (mRNAs), which play important roles in many autoimmune diseases. But the effect of miRNAs and their targeted mRNAs in acquired AA is not fully understood. The Gene Expression Omnibus data-sets of bone marrow T cells in acquired AA patients were performed to integrated analysis. Read More

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http://dx.doi.org/10.1080/16078454.2020.1757332DOI Listing
December 2020

Disappearance of monosomy 7 in a patient with aplastic anemia after eltrombopag treatment.

Hematology 2020 Dec;25(1):165-167

Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.

We present the case of a patient with aplastic anemia (AA) who was treated with eltrombopag. To the best of our knowledge, this is the first report of the disappearance of monosomy 7 after eltrombopag treatment. The patient was a 77-year-old woman with intraoral hematoma and purpura who was diagnosed with very severe AA with a normal karyotype. Read More

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http://dx.doi.org/10.1080/16078454.2020.1757331DOI Listing
December 2020

Eltrombopag for patients with moderate aplastic anemia or uni-lineage cytopenias.

Blood Adv 2020 Apr;4(8):1700-1710

Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.

There is no standard or widely effective treatment of patients with moderate aplastic anemia (MAA) or hypo-productive uni-lineage cytopenias (UC). Eltrombopag (EPAG), a small molecule thrombopoietin mimetic, has previously been shown to result in durable multi-lineage hematologic responses with low toxicity in patients with refractory severe aplastic anemia (SAA). Its safety and efficacy in MAA are unknown. Read More

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http://dx.doi.org/10.1182/bloodadvances.2020001657DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7189303PMC

Pulmonary acute graft-versus-host disease and infections after allogeneic hematopoietic stem cell transplantation in pediatric recipients: A comparative study on CT.

Transpl Infect Dis 2020 Apr 23:e13285. Epub 2020 Apr 23.

Department of Radiology, Beijing Friendship Hospital, Capital Medical University, Beijing, China.

Objective: To compare the chest CT patterns of acute graft-versus-host disease (aGVHD) and infections within 100 days after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in pediatric recipients to help hematologist make definitive diagnosis as early as possible.

Methods: A total of 143 pediatric recipients from January 2015 to June 2019 who were diagnosed pulmonary aGVHD or infections within 100 days after allo-HSCT were enrolled in this study. Two observers evaluated the extent and distribution (unilateral, bilateral) of the CT patterns. Read More

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http://dx.doi.org/10.1111/tid.13285DOI Listing

Regulation And Effect Of Telomerase And Telomeric Length In Stem Cells.

Curr Stem Cell Res Ther 2020 Apr 21. Epub 2020 Apr 21.

Address: Hacettepe University, Faculty of Medicine, Department of Medical Biochemistry, Sihhiye-Ankara. Turkey.

Telomeres are the protective end caps of eukaryotic chromosomes and they decide the proliferative lifespan of somatic cells, as the guardians of the cell replication. Telomere length in leucocytes reflects telomere length in other somatic cells. Leucocyte telomere length can be a biomarker of human ageing. Read More

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http://dx.doi.org/10.2174/1574888X15666200422104423DOI Listing

[Efficacy of Oral Cyclophosphamide for Treatment of Refractory T-cell Large Granular Lymphocytic Leukemia Accompanied with Pure Erythrocyte Aplastic Anemia].

Zhongguo Shi Yan Xue Ye Xue Za Zhi 2020 Apr;28(2):418-423

Department of Hematology, Foshan Hospitol Affiliated to Sun Yat-sen University, Foshan 536000, Guangdong Province, China.

Objective: To explore the clinical effects of oral small dose of cyclophosphamide (CTX) in the treatment of T-cell large granular lymphocytic leukemia (T-LGLL) accompanied with pure red cell aplasia (PRCA).

Methods: The clinical features, characteristics of laboratory examinations and the process of oral small dose of CTX treatment after the ineffective treatment of cyclosporine A combining with prednisone in 1 case of T-LGLL with PRCA were reported and discussed with related references.

Results: The elderly female patient had indolent process, mainly presenting with anemia and significant low hyperplasia of bone marrow erythrocyte cells. Read More

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http://dx.doi.org/10.19746/j.cnki.issn.1009-2137.2020.02.010DOI Listing

[Prognostic factors of cyclosporine A combined with androgen in the treatment of transfusion dependent non-severe aplastic anemia].

Zhonghua Xue Ye Xue Za Zhi 2020 Mar;41(3):234-238

State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.

To analyze the prognostic factors of transfusion-dependent non-severe aplastic anemia (TD-NSAA) patients treated with cyclosporine A (CsA) and androgen. Clinical data of 77 consecutive TD-NSAA patients treated with CsA and androgen were retrospectively analyzed between 2010 and 2013. We obtained clinical manifestations and baseline parameters of routine blood test from responders, and compared those with non-responders. Read More

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http://dx.doi.org/10.3760/cma.j.issn.0253-2727.2020.03.009DOI Listing

Deficit of circulating CD19 CD24 CD38 regulatory B cells in severe aplastic anaemia.

Br J Haematol 2020 Apr 20. Epub 2020 Apr 20.

Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA.

Immune aplastic anaemia (AA) is caused by cytotoxic T lymphocytes (CTLs) that destroy haematopoietic stem and progenitor cells. Enhanced type 1 T helper (Th1) responses and reduced regulatory T cells (Tregs) are involved in the immune pathophysiology. CD24 CD38 regulatory B cells (Bregs) suppress CTLs and Th1 responses, and induce Tregs via interleukin 10 (IL-10). Read More

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http://dx.doi.org/10.1111/bjh.16651DOI Listing

Delayed Presentation of Thymoma-Related Aplastic Anemia: An Unusual Presentation of a Rare Complication.

J Hematol 2019 Dec 25;8(4):160-164. Epub 2019 Dec 25.

Mount Sinai Comprehensive Cancer Center, Miami Beach, FL, USA.

A 68-year-old Caucasian man presented with gross hematuria and oral mucosal bleeding. The patient was known to have an anterior mediastinal mass, highly suspicious for thymoma, which was incidentally identified on imaging, 8 years prior. The patient then declined treatment and was lost to follow-up. Read More

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http://dx.doi.org/10.14740/jh557DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7155810PMC
December 2019

Large Cell Change in a Small Liver: A Histologic Clue to Short Telomere Syndromes?

Hepatology 2020 Apr 15. Epub 2020 Apr 15.

Department of Pathology and Laboratory Medicine, The University of North Carolina School of Medicine, Chapel Hill, NC, 27514, United States.

Telomeres are DNA-protein complexes at the ends of chromosomes essential for maintaining genetic stability. Defects in genes coding telomere maintenance proteins rarely occur, resulting in accelerated telomere shortening. Termed short telomere syndromes (STSs), these conditions have diverse clinical manifestations, including premature hair greying, bone marrow failure, pulmonary fibrosis, early-onset malignancy, and cirrhosis. Read More

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http://dx.doi.org/10.1002/hep.31272DOI Listing
April 2020
11.055 Impact Factor

Treg sensitivity to FasL and relative IL-2 deprivation drive Idiopathic Aplastic Anemia immune dysfunction.

Blood 2020 04 15. Epub 2020 Apr 15.

Department of Haematology, School of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences and Medicine, King’s College London, London, United Kingdom

Idiopathic aplastic anemia (AA) has two key characteristics: an autoimmune response against hematopoietic stem/progenitor cells and regulatory T cells (Tregs) deficiency. We have previously demonstrated reduction in a specific subpopulation of Treg in AA, which predict response to immunosuppression. The aims of the present study were to define mechanisms of Treg subpopulation imbalance and identify potential for therapeutic intervention. Read More

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http://dx.doi.org/10.1182/blood.2019001347DOI Listing
April 2020
10.452 Impact Factor

Epstein-Barr Virus-Induced Post-Transplant Lymphoproliferative Disorder of the Central Nervous System Successfully Treated with Chemo-immunotherapy.

Viruses 2020 Apr 8;12(4). Epub 2020 Apr 8.

Department of Hematology and Rheumatology, Kindai University Faculty of Medicine, Osaka 589-8511, Japan.

Aplastic anemia is a rare blood disease characterized by the destruction of the hematopoietic stem cells (HSC) in the bone marrow that, in the majority of cases, is caused by an autoimmune reaction. Patients with aplastic anemia are treated with immunosuppressive drugs and some of them, especially younger individuals with a donor available, can be successfully treated with hematopoietic stem cell transplantation (HSCT). We report here a rare case of post-transplant lymphoproliferative disorder (PTLD) associated with Epstein-Barr virus (EBV) reactivation in a 30-year-old female patient who underwent allogeneic HSCT for severe aplastic anemia. Read More

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http://dx.doi.org/10.3390/v12040416DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7232501PMC

Cancer and myeloid clonal evolution in the short telomere syndromes.

Curr Opin Genet Dev 2020 Feb 7;60:112-118. Epub 2020 Apr 7.

Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States; Telomere Center, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States; Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States. Electronic address:

The short telomere syndromes are considered the most common premature aging disorders. Although studies in genetically modified cells and animal models have suggested telomere dysfunction may promote genome instability, only a minority of humans with inherited loss-of-function mutations in telomerase and related genes develop cancer. Solid tumors are overall rare, and the vast majority of cancers are bone marrow-derived with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) comprising three-quarter of cases. Read More

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http://dx.doi.org/10.1016/j.gde.2020.02.019DOI Listing
February 2020

Acquired Aplastic Anemia as a Clonal Disorder of Hematopoietic Stem Cells.

Stem Cell Rev Rep 2020 Jun;16(3):472-481

Department of Hematology and Bone Marrow Transplantation, Poznań University of Medical Sciences, Poznań, Poland.

Aplastic anemia is rare disorder presenting with bone marrow failure syndrome due to autoimmune destruction of early hematopoietic stem cells (HSCs) and stem cell progenitors. Recent advances in newer genomic sequencing and other molecular techniques have contributed to a better understanding of the pathogenesis of aplastic anemia with respect to the inflammaging, somatic mutations, cytogenetic abnormalities and defective telomerase functions of HSCs. These have been summarized in this review and may be helpful in differentiating aplastic anemia from hypocellular myelodysplastic syndrome. Read More

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http://dx.doi.org/10.1007/s12015-020-09971-yDOI Listing

Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: A retrospective analysis on behalf of EBMT group.

Am J Hematol 2020 Apr 8. Epub 2020 Apr 8.

UOC Ematologia, Istituto Giannina Gaslini, Genoa, Italy.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is curative for bone marrow failure in patients with Fanconi anemia (FA), but the presence of a malignant transformation is associated with a poor prognosis and the management of these patients is still challenging. We analyzed outcome of 74 FA patients with a diagnosis of myelodysplastic syndrome (n = 35), acute leukemia (n = 35) or with cytogenetic abnormalities (n = 4), who underwent allo-HSCT from 1999 to 2016 in EBMT network. Type of diagnosis, pre-HSCT cytoreductive therapies and related toxicities, disease status pre-HSCT, donor type, and conditioning regimen were considered as main variables potentially influencing outcome. Read More

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http://dx.doi.org/10.1002/ajh.25810DOI Listing

Occupational exposure to cytostatic fumes during hyperthermic intraperitoneal chemotherapy.

Occup Med (Lond) 2020 Apr 8. Epub 2020 Apr 8.

Department of Occupational Diseases and Environmental Health, Nofer Institute of Occupational Medicine, Lodz, Poland.

Hyperthermic intraperitoneal chemotherapy (HIPEC)-heated, intra-abdominal chemotherapy-has become the treatment of choice for treating peritoneal metastases from ovarian, stomach or colorectal cancers. HIPEC has several advantages and disadvantages. The major benefit is minimal systemic toxicity for the patient, but there is a risk of occupational exposure for operating room staff. Read More

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http://dx.doi.org/10.1093/occmed/kqaa029DOI Listing

Risk factors associated with poor response to immunosuppressive therapy in acquired aplastic anemia: A meta-analysis of retrospective studies.

Exp Ther Med 2020 Apr 19;19(4):3104-3112. Epub 2020 Feb 19.

Department of Orthopaedics, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 201999, P.R. China.

Acquired aplastic anemia (AA) is a rare hematological disease characterized by bone marrow hypocellularity and varying degrees of pancytopenia. Immunosuppressive therapy (IST) is currently one of the first-line treatments for AA; however, unresponsiveness remains a major concern. Although previous studies have suggested several common risk factors for unresponsiveness, there are currently no widely accepted predictors. Read More

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http://dx.doi.org/10.3892/etm.2020.8536DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7086236PMC
April 2020
0.941 Impact Factor