keyword
MENU ▼
Read by QxMD icon Read
search

Severe aplastic anemia

keyword
https://www.readbyqxmd.com/read/29932285/upfront-eltrombopag-monotherapy-induces-stable-hematologic-remission-in-pediatric-patients-with-nonsevere-idiopathic-aplastic-anemia
#1
Wenliang Geng, Susan Kearney, Stephen Nelson
Aplastic anemia (AA) is characterized by multilineage cytopenias and bone marrow hypocellularity. Severe AA can be treated with immunosuppressive therapy (IST) and/or allogeneic hematopoietic stem cell transplantation. The thrombopoietin agonist eltrombopag has been shown to induce hematopoietic recovery and transfusion independence in adults with refractory and relapsed AA. Recently, upfront eltrombopag therapy in patients with AA in combination with IST has shown efficacy. Data for its use without concurrent IST in pediatric patients with AA remain sparse...
June 22, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29922180/increased-reactive-oxygen-species-and-cell-cycle-defects-contribute-to-anemia-in-the-rasa3-mutant-mouse-model-s-cat
#2
Emily S Hartman, Elena C Brindley, Julien Papoin, Steven L Ciciotte, Yue Zhao, Luanne L Peters, Lionel Blanc
RASA3 is a Ras GTPase activating protein that plays a critical role in blood formation. The autosomal recessive mouse model scat (severe combined anemia and thrombocytopenia) carries a missense mutation in Rasa3 . Homozygotes present with a phenotype characteristic of bone marrow failure that is accompanied by alternating episodes of crisis and remission. The mechanism leading to impaired erythropoiesis and peripheral cell destruction as evidenced by membrane fragmentation in scat is unclear, although we previously reported that the mislocalization of RASA3 to the cytosol of reticulocytes and mature red cells plays a role in the disease...
2018: Frontiers in Physiology
https://www.readbyqxmd.com/read/29881557/idiopathic-severe-aplastic-anemia-with-a-delayed-response-to-immunosuppressive-therapy-a-case-report
#3
Nujood Alzahrani, Nshwa Ashor, Traji Fathi, Dania Bukhari, Galila Zaher
Bone marrow transplantation is the definitive treatment of severe aplastic anemia; however, with the absence of this option, combined immunosuppressive therapy with antithymocyte globulin (ATG) and cyclosporine A is used as a first-line therapy. This case report highlights the possible delay in response to ATG protocol in treating aplastic anemia.
June 2018: Clinical Case Reports
https://www.readbyqxmd.com/read/29879518/chromosomal-aberrations-and-survival-after-unrelated-donor-hematopoietic-stem-cell-transplant-in-patients-with-fanconi-anemia
#4
Youjin Wang, Weiyin Zhou, Blanche P Alter, Tao Wang, Stephen R Spellman, Michael Haagenson, Meredith Yeager, Stephanie J Lee, Stephen J Chanock, Sharon A Savage, Shahinaz M Gadalla
Studies of chromosomal aberrations in blood or bone marrow of patients with Fanconi anemia (FA) have focused on their associations with leukemic transformation. The role of such abnormalities on outcomes after hematopoietic cell transplantation (HCT) is unclear. We used genome-wide single nucleotide polymorphism (SNP) arrays to identify chromosomal aberrations in pre-HCT blood samples from 73 patients with FA who received unrelated donor HCT for severe aplastic anemia between 1991-2007. Outcome data and blood samples were available through the Center for International Blood and Marrow Transplant Research...
June 4, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29870142/increased-apoptosis-and-peripheral-blood-mononuclear-cell-suppression-of-bone-marrow-mesenchymal-stem-cells-in-severe-aplastic-anemia
#5
Yu-Hua Chao, Chiao-Wen Lin, Hui-Hsien Pan, Shun-Fa Yang, Te-Fu Weng, Ching-Tien Peng, Kang-Hsi Wu
BACKGROUND: Although immune-mediated pathogenesis is considered an important aspect of severe aplastic anemia (SAA), its underlying mechanisms remain unclear. Mesenchymal stem cells (MSCs) are essential to the formation of specialized microenvironments in the bone marrow (BM), and MSC insufficiency can trigger the development of SAA. METHODS: To find MSC alterations in the SAA BM, we compared BM MSCs from five children with SAA and five controls. Peripheral blood mononuclear cells (PBMCs) were cocultured with MSCs to evaluate the supportive effects of MSCs on hematopoiesis...
June 5, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29869462/effects-and-predictive-factors-of-immunosuppressive-therapy-combined-with-umbilical-cord-blood-infusion-in-patients-with-severe-aplastic-anemia
#6
Xia Zhang, Zhangzhi Li, Wei Geng, Bin Song, Chucheng Wan
PURPOSE: To investigate the efficacy and safety of umbilical cord blood (UCB) infusion (UCBI) plus immunosuppressive therapy (IST) treatment in comparison to IST treatment, as well as predictive factors for clinical responses, in severe aplastic anemia (SAA) patients. MATERIALS AND METHODS: Totally, 93 patients with SAA were enrolled in this cohort study. In the IST group, rabbit antithymocyte globulin (r-ATG) combined with cyclosporine A (CsA) was administered, while in the IST+UBCI group, r-ATG, CsA, and UCB were used...
July 2018: Yonsei Medical Journal
https://www.readbyqxmd.com/read/29862282/epstein-barr-virus-infection-affects-function-of-cytotoxic-t-lymphocytes-in-patients-with-severe-aplastic-anemia
#7
Tian Zhang, Chunyan Liu, Hui Liu, Lijuan Li, Ting Wang, Rong Fu
Severe aplastic anemia (SAA) is characterized by pancytopenia and failure of hematopoietic function and is caused by excessive functioning of cytotoxic T lymphocytes (CTLs). EBNA-1, a nucleoprotein of the Epstein Barr virus (EBV), can influence the proliferation and function of lymphocytes. We therefore tested the number of EBV copies in the CD8+ T cells of 27 patients with SAA and 10 healthy control subjects and observed the influences of EBNA-1 upon the CD8+ T cells of patients with SAA. The results showed that more EBV copies were found in the CD8+ T cells of patients with untreated SAA than in patients with SAA in remission or in the healthy control subjects...
2018: BioMed Research International
https://www.readbyqxmd.com/read/29773597/hematopoietic-stem-cell-loss-and-hematopoietic-failure-in-severe-aplastic-anemia-is-driven-by-macrophages-and-aberrant-podoplanin-expression
#8
Amanda McCabe, Julianne N P Smith, Angelica Costello, Jackson Maloney, Divya Katikaneni, Katherine C MacNamara
Severe aplastic anemia results from profound hematopoietic stem cell loss. T cells and interferon gamma have long been associated with severe aplastic anemia, yet the underlying mechanisms driving hematopoietic stem cell loss remain unknown. Using a mouse model of severe aplastic anemia, we demonstrate that interferon gamma-dependent hematopoietic stem cell loss required macrophages. Interferon gamma was necessary for bone marrow macrophage persistence, despite loss of other myeloid cells and hematopoietic stem cells...
May 17, 2018: Haematologica
https://www.readbyqxmd.com/read/29772350/unmanipulated-haploidentical-hematopoietic-stem-cell-transplantation-achieved-outcomes-comparable-with-matched-unrelated-donor-transplantation-in-young-acquired-severe-aplastic-anemia
#9
Yue Lu, Rui-Juan Sun, Yan-Li Zhao, Min Xiong, Xing-Yu Cao, Jing-Pin Zhang, Zhi-Jie Wei, Jia-Rui Zhou, De-Yan Liu, Dao-Pei Lu
Salvage haploidentical HSCT is considered in patients with severe aplastic anemia (SAA) if matched unrelated donor (MUD) is unavailable. However, studies on haplo- and MUD transplantation in SAA are yet lacking. The present study retrospectively analyzed the outcomes of 89 young SAA patients who underwent unmanipulated alternative hematopoietic stem cell transplantation (HSCT) between September 2012 and September 2016 at our single center. 41 patients received haploidentical donors, and 48 patients received MUD for HSCT...
May 14, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29760162/unrelated-cord-blood-transplantation-in-patients-with-acquired-refractory-aplastic-anemia-a-nationwide-phase-ii-study
#10
Regis Peffault de Latour, Sylvie Chevret, Charlotte Jubert, Anne Sirvent, Claire Galambrun, Annalisa Ruggeri, Virginie Gandemer, Jérôme Cornillon, Fanny Rialland, Jean-Hugues H Dalle, Edouard Forcade, Benedicte Bruno, Catherine Paillard, Pierre S Rorlich, Alexandra Salmon, Sabine Fürst, Flore Sicre de Fontbrune, Marie Therese Rubio, Jacques-Olivier Bay, Mohamad Mohty, Jerome Larghero, Eliane Gluckman, Gerard Socié
Outcomes remain poor for patients with refractory severe aplastic anemia (SAA). Alternative donor transplantation may be considered, but results from previous studies have not been encouraging. We conducted a prospective nationwide phase II study to assess the efficacy and safety of unrelated cord blood transplantation (CBT) in patients with refractory SAA (APCORD protocol, NCT 01343953). To demonstrate a significant difference in one-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients...
May 14, 2018: Blood
https://www.readbyqxmd.com/read/29749240/why-is-it-necessary-to-examine-retina-when-the-patient-suffers-from-aplastic-anemia
#11
D Tomcikova, A Gerinec, B Busanyova, M Gresikova, S Biskup, K Hortnagel
PURPOSES: To inform about a case of Revesz syndrome (RS) with initial ophthalmological symptomatology of severe proliferative vitreoretinopathy of the left eye (LE). After the aplastic anemia had developed, RS was established. The exudative retinopathy was successfully treated with photocoagulation on the right eye (RE). BACKGROUND: RS is characterized by fatal bone marrow failure, exudative retinopathy, neuroradiographic abnormalities, neurodevelopmental delay and skin abnormalities...
2018: Bratislavské Lekárske Listy
https://www.readbyqxmd.com/read/29743400/-epstein-barr-virus-associated-hemophagocytic-lymphohistiocytosis-subsequent-to-rabbit-antithymocyte-globulin-administration-and-successfully-treated-with-rituximab-in-a-patient-with-aplastic-anemia
#12
Hiroaki Nagata, Takahiro Fujino, Ayako Muramatsu, Yuka Kawaji, Kodai Kuriyama, Muneo Oshiro, Yoshiko Hirakawa, Toshiki Iwai, Hitoji Uchiyama
Rabbit antithymocyte globulin (ATG) is an effective immunosuppressive therapy for patients with aplastic anemia (AA). However, Epstein-Barr virus-associated lymphoproliferative disorder (EBV-LPD) is a rare but serious complication of the therapy. An 81-year-old man was diagnosed with severe AA on the occasion of melena. Because cyclosporine monotherapy did not improve his condition, rabbit ATG was additionally administered. Thirty-one days after the administration of rabbit ATG, the patient presented with fever and general malaise...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/29739053/fungal-infection-caused-by-geotrichum-capitatum-in-a-severe-aplastic-anemia-patient-a-case-report-and-review-of-the-literature
#13
Wulin Shan, Chunyang Dai, Jingsong Kan, Ming Li, Meiling Yin
BACKGROUND: Invasive fungal infections often occur in immunocompromised patients. Here, we report an infection case caused by Geotrichum capitatum in a severe aplastic anemia patient. METHODS: Identification of the pathogenic bacteria was done by sequencing and mass spectrometric analysis. RESULTS: The fungal infection was isolated from blood cultures. The pathogenic bacteria were identified as Geotrichum capitatum. The infection was primarily cured by voriconazole and caspofungin monotherapy...
May 1, 2018: Clinical Laboratory
https://www.readbyqxmd.com/read/29724291/comparison-of-two-dosages-of-rabbit-antithymocyte-globulin-r-atg-in-treating-children-with-severe-aplastic-anemia
#14
Shao-Fen Lin, Su Liu, Hong-Man Xue, Jun-Bin Huang, Jian Wang, Qi-Hui Chen, Bi-Hong Zhang, Chun Chen
In this study, efficacy and safety of two different dosages of rabbit antithymocyte globulin (r-ATG) combined with cyclosporine (CsA) in treating children with severe aplastic anemia (SAA) were compared. The clinical data of 122 SAA children treated by r-ATG/CsA between Jan 2005 and Jan 2017 at Sun Yat-sen Memorial Hospital of Sun Yat-sen University were retrospectively analyzed. The r-ATG dose of 55 cases was 2.5mg/(kg·d, group 1), and in the other 67 cases it was 3.5 mg/(kg·d, group 2). r-ATG was continuously administered for 5 days...
May 1, 2018: Die Pharmazie
https://www.readbyqxmd.com/read/29722478/successful-reduced-intensity-conditioning-hematopoietic-stem-cell-transplantation-for-paroxysmal-nocturnal-hemoglobinuria-with-aplastic-anemia-in-two-children
#15
Jeffrey R Andolina, Ariel L Reinish, Razia Akhtar, Suzie Noronha, Jessica C Shand, Angela Girvin, David N Korones, Lauren B Bruckner, Craig A Mullen, Kevin J Curran, Farid Boulad
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare cause of bone marrow failure in children. We report two children who presented with pancytopenia, and were diagnosed with PNH with severe aplastic anemia. Both children underwent upfront, successful hematopoietic stem cell transplantation with reduced-intensity conditioning. One patient had a syngeneic donor, and one patient had a 10/10 matched unrelated donor. Neither patient developed graft versus host disease, infections, or recurrent PNH. Reduced-intensity conditioning hematopoietic stem cell transplantation is a reasonable therapy for PNH with marrow failure in children...
May 3, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29713553/acquired-amegakaryocytic-thrombocytopenia-and-pure-red-cell-aplasia-in-thymoma
#16
Sumit Dahal, Eliza Sharma, Suyash Dahal, Binav Shrestha, Bikash Bhattarai
Association of thymoma with myasthenia gravis, pure red cell aplasia, and aplastic anemia is well documented. However, thymoma complicated by acquired amegakaryocytic thrombocytopenia (AAMT) is rarely reported. Here, we present a case of a 60-year-old male with past medical history of recurrent invasive thymoma who presented with cough and blood in sputum. He was found to have severe normocytic normochromic anemia and thrombocytopenia that did not improve with intravenous steroids or multiple transfusions of red cells and platelets...
2018: Case Reports in Hematology
https://www.readbyqxmd.com/read/29708883/graves-disease-following-allogenic-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-case-report-and-literature-review
#17
Ahu Paketçi, Korcan Demir, Özlem Tüfekçi, Sezer Acar, Ayhan Abacı, Şebnem Yılmaz, Ece Böber
BACKGROUND: Similar autoimmune processes (defective T-cell function) take place during the pathogenesis of aplastic anemia (AA) and Graves' disease (GD). Antithyroid drugs used for the management of GD may induce AA and GD may occur following treatment of severe aplastic anemia (SAA). CASE PRESENTATION: Clinical and laboratory investigations were performed for an 11-year-and-2-month-old girl who was referred for bilateral exophthalmus and abnormal thyroid function tests...
April 25, 2018: Journal of Pediatric Endocrinology & Metabolism: JPEM
https://www.readbyqxmd.com/read/29684566/eltrombopag-for-the-treatment-of-refractory-pure-rbc-aplasia-after-major-abo-incompatible-hematopoietic-stem-cell-transplantation
#18
Alessandro Busca, Chiara Dellacasa, Luisa Giaccone, Sara Manetta, Lucia Biale, Laura Godio, Semra Aydin, Moreno Festuccia, Lucia Brunello, Benedetto Bruno
Pure RBC aplasia (PRCS) is a well-recognized complication after allogeneic hematopoietic stem cell transplantation (HSCT). Many therapeutic options are available to treat this condition, including erythropoietin, rituximab, bortezomib, plasma exchange, immunoadsorption, donor lymphocyte infusion, mesenchymal stem cells, antithymocyte globulin, and high-dose steroids; however, treatment outcomes are often variable and can sometimes lead to disappointing results. In this brief article we report our experience with 2 patients with PRCA after major ABO-incompatible HSCT who were resistant to multiple therapeutic interventions and who eventually benefited from treatment with eltrombopag, a thrombopoietin mimetic approved by the US Food and Drug Administration for the treatment of patients with immune thrombocytopenic purpura or severe aplastic anemia refractory to immunosuppressive agents or not eligible for HSCT...
April 21, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29677078/therapeutic-outcomes-of-haploidentical-allogeneic-hematopoietic-stem-cell-transplantation-in-patients-with-severe-aplastic-anemia-a-multicenter-study
#19
Yonghua Li, Fengqi Duan, Haowen Xiao, Xiaoxiong Wu, Shunqing Wang, Duorong Xu, Qifa Liu, Zhiping Fan, Danian Nie, Yongrong Lai, Bingyi Wu, Dongjun Lin, Xin Du, Jianyu Weng, Xin Du, Zujun Jiang, Yan Pang, Lin Ouyang, Zenghui Liu, Leqin Zhang, Na Han, Lixuan Chen, Yang Xiao
BACKGROUND: Haploidentical donor allogeneic hematopoietic stem cell transplantation (HID-HSCT) is an alternative curative treatment for patients with severe aplastic anemia (SAA) who do not have suitable matched related donors (MRD). The aim of this study was to compare the therapeutic outcomes of HID-HSCT with those of MRD-HSCT for SAA. METHODS: A total of 235 SAA patients who underwent HID-HSCT (116) or MRD-HSCT (119) at 11 transplantation centers from January 2007 to January 2016 were included...
April 19, 2018: Transplantation
https://www.readbyqxmd.com/read/29674506/diagnostic-and-prognostic-roles-of-circulating-exosomal-micrornas-in-acquired-aplastic-anemia-and-myelodysplastic-syndromes
#20
Valentina Giudice, Lauren G Banaszak, Fernanda Gutierrez-Rodrigues, Sachiko Kajigaya, Reema Panjwani, Maria Del Pilar Fernandez Ibanez, Olga Rios, Christopher K Bleck, Erin S Stempinski, Diego Quinones Raffo, Danielle M Townsley, Neal S Young
Exosomal microRNAs modulate cancer cell metabolism and the immune response. Specific exosomal microRNAs have been reported to be reliable biomarkers of several solid and hematologic malignancies. We examined the possible diagnostic and prognostic values of exosomal microRNAs in two human bone marrow failure diseases, aplastic anemia and myelodysplastic syndromes. After screening of 372 microRNAs in a discovery set (n=42) of plasma exosome samples, we constructed a custom microRNA PCR plate, including 42 microRNAs, for validation in a larger cohort (n=99), and we identified 25 differentially expressed exosomal microRNAs uniquely or frequently present in aplastic anemia and/or myelodysplastic syndromes...
April 19, 2018: Haematologica
keyword
keyword
90246
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"