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Eltrombopag

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https://www.readbyqxmd.com/read/29467954/efficacy-and-safety-of-thrombopoietin-receptor-agonists-in-children-with-chronic-immune-thrombocytopenia-a-meta-analysis
#1
Jian-Chun Guo, Yi Zheng, Hai-Tao Chen, Haixia Zhou, Xian-Hui Huang, Li-Ping Zhong, Huai-Bin Zhou, Yu Huang, Dan-Li Xie, Yong-Liang Lou
Background and Aim: Thrombopoietin receptor agonists (TPO-RAs) have been shown to be safe and effective for adults with chronic immune thrombocytopenia (ITP). The aim of this meta-analysis is to assess the efficacy and safety of thrombopoietin receptor agonists for children with chronic ITP. Materials and Methods: Clinical randomized controlled trials (RCTs) evaluating the efficacy and safety of TPO-RAs in pediatric ITP patients published up to June 2017 were retrieved from PubMed, Cochrane Library, and Embase databases...
January 23, 2018: Oncotarget
https://www.readbyqxmd.com/read/29374141/ipsc-modeling-of-severe-aplastic-anemia-reveals-impaired-differentiation-and-telomere-shortening-in-blood-progenitors
#2
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/29330464/eltrombopag-versus-romiplostim-in-treatment-of-children-with-persistent-or-chronic-immune-thrombocytopenia-a-systematic-review-incorporating-an-indirect-comparison-meta-analysis
#3
Jiaxing Zhang, Yi Liang, Yuan Ai, Xiaosi Li, Juan Xie, Youping Li, Wenyi Zheng, Rui He
In absence of direct comparison, we conducted an indirect-comparison meta-analysis to evaluate the efficacy and safety of thrombopoietin-receptor agonists(TPO-RAs) in treatment of pediatric persistent or chronic immune thrombocytopenia(ITP). PubMed, Embase, Cochrane Library, Clinical Trials.gov, China National Knowledge Infrastructure, and Chinese Biomedical Literature Database were searched from their earliest records to May 2017. Randomized controlled trials comparing the TPO-RAs with placebo in pediatric ITP were included...
January 12, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29303047/eltrombopag-in-the-management-of-aplastic-anaemia-real-world-experience-in-a-non-trial-setting
#4
Yu-Yan Hwang, Harinder Gill, Thomas S Y Chan, Garret M K Leung, Carol Y M Cheung, Yok-Lam Kwong
OBJECTIVE: The thrombopoietin mimetic eltrombopag has been used in clinical trials for the frontline and salvage treatment of aplastic anaemia (AA). Eltrombopag was investigated in AA patients on a non-trial all-comer basis. METHODS: Consecutive newly diagnosed and relapsed/refractory AA patients were treated with eltrombopag. RESULTS: In a 4.5-year period, 20 consecutive AA patients (newly diagnosed, N = 10; relapsed/refractory, N = 10) at a median age of 47 (22-84) years were treated with eltrombopag...
January 5, 2018: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/29301129/amegakaryocytic-thrombocytopenia-and-subsequent-aplastic-anemia-associated-with-apparent-epstein-barr-virus-infection
#5
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/29296963/novel-tpo-receptor-agonist-ta-316-contributes-to-platelet-biogenesis-from-human-ips-cells
#6
Ayako Aihara, Tomo Koike, Natsuki Abe, Sou Nakamura, Akira Sawaguchi, Takanori Nakamura, Naoshi Sugimoto, Hiromitsu Nakauchi, Taito Nishino, Koji Eto
Signaling by thrombopoietin (TPO) in complex with its receptor, c-MPL, is critical for hematopoietic stem cell (HSC) homeostasis and platelet generation. Here we show that TA-316, a novel chemically synthesized c-MPL agonist (CMA), is useful for ex vivo platelet generation from human-induced pluripotent stem (iPS) cell-derived immortalized megakaryocyte progenitor cell lines (imMKCLs). Moreover, the generation is clinically applicable, because self-renewal expansion and platelet release is tightly controllable...
February 28, 2017: Blood Advances
https://www.readbyqxmd.com/read/29285951/seifem-2017-from-real-life-to-an-agreement-on-the-use-of-granulocyte-transfusions-and-colony-stimulating-factors-for-prophylaxis-and-treatment-of-infectious-complications-in-patients-with-hematologic-malignant-disorders
#7
Alessandro Busca, Simone Cesaro, Luciana Teofili, Mario Delia, Chiara Cattaneo, Marianna Criscuolo, Francesco Marchesi, Nicola Stefano Fracchiolla, Caterina Giovanna Valentini, Francesca Farina, Roberta Di Blasi, Lucia Prezioso, Angelica Spolzino, Anna Candoni, Maria Ilaria Del Principe, Luisa Verga, Annamaria Nosari, Franco Aversa, Livio Pagano
The rapid spread of severe infections mainly due to resistant pathogens, justifies the search for therapies aiming to restore immune functions severely compromised in patients with hematologic malignancies. Areas covered: The present review summarizes the current knowledge on the role of granulocyte transfusions and colony-stimulating factors as treatment strategy for hematologic patients with serious infectious complications. In addition, a survey among 21 hematologic centers, to evaluate the clinical practice for the use of G-CSF originator and biosimilars was performed...
January 3, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29241763/eltrombopag-reduces-clinically-relevant-thrombocytopenic-events-in-higher-risk-mds-and-aml
#8
Aditi Shastri, Amit K Verma
No abstract text is available yet for this article.
January 2018: Lancet Haematology
https://www.readbyqxmd.com/read/29241762/eltrombopag-for-advanced-myelodysplastic-syndromes-or-acute-myeloid-leukaemia-and-severe-thrombocytopenia-aspire-a-randomised-placebo-controlled-phase-2-trial
#9
Moshe Mittelman, Uwe Platzbecker, Boris Afanasyev, Sebastian Grosicki, Raymond S M Wong, Achilles Anagnostopoulos, Benjamin Brenner, Claudio Denzlinger, Giuseppe Rossi, Arnon Nagler, Regina Garcia-Delgado, Maria Socorro O Portella, Zewen Zhu, Dominik Selleslag
BACKGROUND: Thrombocytopenia is a life-threatening complication in patients with advanced myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML). In this study (ASPIRE), we aimed to assess eltrombopag, an oral thrombopoietin receptor agonist, for thrombocytopenia (grade 4) treatment in adult patients with advanced MDS or AML. METHODS: ASPIRE consisted of an open-label, double-blind phase for 8 weeks and a randomised, double-blind phase (parts 1 and 2, reported here) for 12 weeks, and an open-label extension (part 3)...
December 11, 2017: Lancet Haematology
https://www.readbyqxmd.com/read/29188582/a-low-birth-weight-infant-with-no-malformations-delivered-by-a-primary-immune-thrombocytopenia-patient-treated-with-eltrombopag
#10
Naruko Suzuki, Junji Hiraga, Yumi Hariyama, Yusuke Takagi, Haruhiko Ohashi, Yasuyuki Kishigami, Hidenori Oguchi, Yoshitoyo Kagami
Primary immune thrombocytopenia (ITP) is defined by a low platelet count secondary to antibody-mediated platelet destruction or reductions in platelet production. Although eltrombopag is a thrombopoietin receptor agonist that increases platelet production in refractory or relapsed ITP, the influence on pregnancy is limited. We present the case of a pregnant 25-year-old ITP patient referred to our hospital with a history of two induced abortions. After eradication of Helicobacter pylori and with oral prednisolone at 8 mg/day, platelet count remained below 10,000/µl...
November 29, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/29181179/efficacy-of-bendamustine-on-thrombocytopenia-and-hemolytic-anemia-secondary-to-cd5-positive-b-cell-lymphoma-with-massive-splenomegaly-in-a-patient-with-rheumatoid-arthritis
#11
Yuzuru Hosoda, Hiroshi Hagino, Norihiko Hino, Toru Motokura
Chemotherapy for lymphoma may be avoided in the presence of coincident cytopenia. In case of immune cytopenia secondary to lymphoma, treatment of cytopenia is the same for primary cases, however, chemotherapy for lymphoma may be effective at the cost of severe hematological toxicity. The present study reports a complex case of thrombocytopenia and direct antiglobulin test-negative hemolytic anemia, thus mimicking Evans syndrome, secondary to cluster of differentiation 5-positive B-cell lymphoma with massive splenomegaly, in a patient suffering from rheumatoid arthritis for two decades...
November 2017: Molecular and Clinical Oncology
https://www.readbyqxmd.com/read/29178132/thrombopoietin-receptor-agonists-for-prevention-and-treatment-of-chemotherapy-induced-thrombocytopenia-in-patients-with-solid-tumours
#12
REVIEW
Xia Zhang, Yunhai Chuai, Wei Nie, Aiming Wang, Guanghai Dai
BACKGROUND: Chemotherapy-induced thrombocytopenia (CIT) is defined as a peripheral platelet count less than 100×10 9 /L, with or without bleeding in cancer patients receiving myelosuppressive chemotherapy. CIT is a significant medical problem during chemotherapy, and it carries the risk of sub-optimal overall survival and bleeding. Alternative interventions to platelet transfusion are limited. Different stages of preclinical and clinical studies have examined the thrombopoietin receptor agonists (TPO-RAs) for CIT in patients with solid tumours...
November 27, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/29172661/-novelties-in-the-treatment-of-pediatric-immune-thrombocytopenia-2017
#13
Gábor Kovács, Csongor Kiss
Immune thrombocitopenia in children is a very variable disease. International recommendations give therapeutic possibilities without strong protocols. In 2011, a therapeutic algorithm was published based on Hungarian practice. Recently, new innovative drugs have been available even in Hungary, so there is a need for modification of the therapeutic protocols. In this summary we give an overview about the current up-to-date management. In infancy and in childhood, high-dose immunglobulin treatment is recommended henceforward...
December 2017: Orvosi Hetilap
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
#14
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/29143887/diagnosis-and-treatment-of-aplastic-anemia
#15
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/29101508/a-sticky-situation-myocardial-infarction-in-a-young-woman-with-immune-thrombocytopenia-on-eltrombopag-and-a-history-of-mediastinal-radiation
#16
Jennifer Teichman, Ahmed Taher, Abdulaziz Hashi, Akshay Bagai, Michelle Sholzberg
More recent immune thrombocytopenia (ITP) treatment strategies enhance platelet production with the use of thrombopoietin receptor agonists (TPO-RA) such as eltrombopag. Patients receiving TPO-RA agents may be at an increased risk of thromboembolism, however the pathophysiology and common underlying risk factors are not well understood. We present the case of a young asplenic woman on eltrombopag for chronic ITP with acute myocardial infarction involving the right coronary artery. Past medical history was significant for remote mediastinal radiation for lymphoma and splenectomy for ITP...
November 3, 2017: Journal of Thrombosis and Thrombolysis
https://www.readbyqxmd.com/read/29069007/successful-eltrombopag-treatment-of-severe-refractory-thrombocytopenia-in-chronic-myelomonocytic-leukemia-two-cases-reports-a-care-compliant-article
#17
Yayue Gao, Ming Gong, Chunxia Zhang, Xudong Kong, Yigai Ma
RATIONALE: Thrombocytopenia in chronic myelomonocytic leukemia (CMML) is usually attributed to impaired marrow production resulting from cytotoxic drug use or CMML itself ("CMML-induced thrombocytopenia"). In very rare cases, immune thrombocytopenia (ITP) can be a complication of CMML ("CMML-associated ITP"). However, treatment of severe thrombocytopenia in patients with CMML is still a challenge. PATIENT CONCERNS: Case 1 was a 61-year-old female patient admitted to our hospital because of skin petechiae and purpura for 6 days...
October 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29042367/safety-and-efficacy-of-long-term-treatment-of-chronic-persistent-itp-with-eltrombopag-final-results-of-the-extend-study
#18
Raymond S M Wong, Mansoor N Saleh, Abderrahim Khelif, Abdulgabar Salama, Maria Socorro O Portella, Paul Burgess, James B Bussel
In phase 2/3 trials, eltrombopag treatment of 6 months or less in patients with chronic/persistent immune thrombocytopenia (ITP) increased platelet counts and reduced bleeding. The open-label EXTEND study evaluated long-term safety and efficacy of eltrombopag in adults with ITP who had completed a previous eltrombopag study. For the 302 patients enrolled, median duration of eltrombopag treatment was 2.37 years (2 days-8.76 years). Median platelet counts increased to 50 × 10 9 /L or more by week 2 and were sustained throughout the treatment period...
December 7, 2017: Blood
https://www.readbyqxmd.com/read/29026567/proactive-use-of-eltrombopag-before-the-onset-of-clinical-bleeding-in-two-children-with-immune-thrombocytopenia-and-lifestyle-restrictions
#19
Shayla Bergmann
Children with immune thrombocytopenia (ITP) are often managed using a watch-and-wait approach to avoid conventional treatment that may be poorly tolerated. However, in some patients, this approach may lead to lifestyle restrictions due to risk of injury-related bleeding. Eltrombopag is a well-tolerated new option that may help these children.
October 2017: Clinical Case Reports
https://www.readbyqxmd.com/read/28978824/the-cutting-edge-of-aplastic-anemia-treatment
#20
Naoshi Obara
Aplastic anemia is a syndrome in which hematopoietic stem cells are decreased and bone marrow hypoplasia and pancytopenia are observed; it is considered as a T cell-mediated autoimmune disease. Recently, it has been reported that gene mutations suggestive of clonal hematopoiesis are detected in approximately one third of the patients with aplastic anemia. Among treatment approaches other than hematopoietic stem cell transplantation, immunosuppressive therapy with antithymocyte globulin (ATG) plus cyclosporin is a basic approach, although it has been shown that eltrombopag, a thrombopoietin receptor agonist, is effective and that the recovery of hematopoiesis in three blood lineage is observed in some patients...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
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