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Eltrombopag

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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
#1
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
#2
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
https://www.readbyqxmd.com/read/28965224/effect-of-thrombopoietin-receptor-agonists-on-leukocyte-and-haematopoietic-stem-and-progenitor-cells-in-the-peripheral-blood-of-patients-with-immune-thrombocytopenic-purpura
#3
Gürkan Bal, Depré Fabian, Dzamashvili Maia, Frauke Ringel, Abdulgabar Salama
The thrombopoietin receptor agonists (TPO-RAs), romiplostim and eltrombopag, stimulate megakaryopoiesis and thereby increase platelet counts. Both drugs are increasingly used in the treatment of immune thrombocytopenic purpura (ITP). To assess the effect of TPO-RAs on trilineage haematopoiesis, colony-forming cell (CFC) assays were performed on peripheral blood mononuclear cells of 8 healthy donors and 52 ITP patients. Additionally, we revaluated the regular and complete blood counts (CBCs) performed during romiplostim therapy in 45 patients and the CBCs performed in 9 patients during eltrombopag therapy...
September 30, 2017: Annals of Hematology
https://www.readbyqxmd.com/read/28962071/thrombopoietin-mimetics-for-patients-with-myelodysplastic-syndromes
#4
REVIEW
Helga Dodillet, Karl-Anton Kreuzer, Ina Monsef, Nicole Skoetz
BACKGROUND: Myelodysplastic syndrome (MDS) is one of the most frequent haematologic malignancies of the elderly population and characterised by progenitor cell dysplasia with ineffective haematopoiesis and a high rate of transformation to acute myeloid leukaemia (AML). Thrombocytopenia represents a common problem for patients with MDS. ranging from mild to serious bleeding events and death. To manage thrombocytopenia, the current standard treatment includes platelet transfusion, unfortunately leading to a range of side effects...
September 30, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28941711/persistent-elevation-of-plasma-thrombopoietin-levels-after-treatment-in-severe-aplastic-anemia
#5
Xin Zhao, Xingmin Feng, Zhijie Wu, Thomas Winkler, Ronan Desmond, Matthew Olnes, Bogdan Dumitriu, Danielle M Townsley, Cynthia E Dunbar, Neal S Young
Although hematopoietic growth factors are found at high levels in aplastic anemia (AA) patients, little is known about their dynamic change over time after treatment. We examined plasma concentrations of hematopoietic growth factors sequentially in 55 severe AA patients, including 45 treatment-naive patients who had received immunosuppressive therapy (IST) or IST and eltrombopag and 10 IST-refractory patients who had received eltrombopag only, focusing on thrombopoietin (TPO). TPO concentrations were much higher than normal in patients before treatment and then decreased in responders but not in nonresponders...
September 20, 2017: Experimental Hematology
https://www.readbyqxmd.com/read/28882075/comparative-treatment-related-adverse-event-cost-burden-in-immune-thrombocytopenic-purpura
#6
Prina Z Donga, Sara P Bilir, Gregg Little, Tim Babinchak, Julie Munakata
AIMS: Real-world evidence on the safety profile and costs associated with immune thrombocytopenic purpura (ITP) treatment in adults is lacking. This study quantifies and compares adverse event (AE) crude rates and costs associated with ITP treatments as found in claims data. MATERIALS AND METHODS: A retrospective claims-based analysis was conducted using IMS Pharmetrics Plus database. Included patients were ≥18 years old, with a diagnosis of ITP (2007-2012); an ITP-related claim for anti-D, intravenous immunoglobulin (IVIG), rituximab, romiplostim, or eltrombopag; and 1-year continuous enrollment (3-years for rituximab) during follow-up...
November 2017: Journal of Medical Economics
https://www.readbyqxmd.com/read/28877042/hematopoietic-cell-transplantation-for-aplastic-anemia
#7
Rajat Kumar, Carmem Bonfim, Biju George
PURPOSE OF REVIEW: Improvements in allogeneic hematopoietic cell transplantation (HCT) with better donor selection, conditioning regimens and graft vs. host disease prophylaxis make it reasonable to move HCT earlier in the algorithm for management of severe aplastic anemia (SAA). Recent progress in transplantation is reviewed whereas issues related to developing countries are also addressed. RECENT FINDINGS: Multiple research centers are reporting on clonality, mutations and telomere disorders in SAA, which may help to choose the most appropriate therapy upfront...
November 2017: Current Opinion in Hematology
https://www.readbyqxmd.com/read/28864871/eltrombopag-for-thrombocytopenia-in-patients-with-advanced-solid-tumors-receiving-gemcitabine-based-chemotherapy-a-randomized-placebo-controlled-phase-2-study
#8
Eric S Winer, Howard Safran, Boguslawa Karaszewska, Sebastian Bauer, Dilawar Khan, Steffen Doerfel, Paul Burgess, Stacey Kalambakas, Yasser Mostafa Kamel, Frederic Forget
In this phase 2 study, patients with solid tumors receiving gemcitabine monotherapy or gemcitabine plus cisplatin/carboplatin were randomized 2:1 to eltrombopag 100 mg (n = 52) or placebo (n = 23) for 5 days before and after chemotherapy was started. The primary endpoint was prechemotherapy (Day 1) platelet count across ≤6 cycles. Prechemotherapy platelet counts were numerically higher with eltrombopag than placebo. Frequencies of grades 3/4 thrombocytopenia were lower with eltrombopag in both the combination therapy (77 vs...
September 1, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28864815/eltrombopag-a-powerful-chelator-of-cellular-or-extracellular-iron-iii-alone-or-combined-with-a-second-chelator
#9
Evangelia Vlachodimitropoulou, Yu-Lin Chen, Maciej Garbowski, Pimpisid Koonyosying, Bethan Psaila, Martha Sola-Visner, Nichola Cooper, Robert Hider, John Porter
Eltrombopag (ELT) is a thrombopoietin receptor agonist, also reported to decrease labile iron in leukemia cells. Here we examine the previously undescribed iron(III)-coordinating and cellular iron-mobilizing properties of ELT. We find a high binding constant for iron(III) (log β2=35). Clinically achievable concentrations (1μM) progressively mobilised cellular iron from hepatocyte, cardiomyocyte and pancreatic cell lines, rapidly decreasing intracellular ROS and also restoring insulin secretion in pancreatic cells...
September 1, 2017: Blood
https://www.readbyqxmd.com/read/28845713/thrombopoietin-receptor-agonists-for-children-with-immune-thrombocytopenia-a-systematic-review
#10
Jiaxing Zhang, Yi Liang, Yuan Ai, Juan Xie, Youping Li, Wenyi Zheng
OBJECTIVE: We conducted a systematic review to assess the efficacy and safety of Thrombopoietin-receptor agonists (TPOras) for pediatric immune thrombocytopenia (ITP). METHODS: We searched PubMed, Embase and Cochrane Library from their earliest records to January 2017. Randomized controlled trials (RCTs) were included. Primary outcomes were durable response and clinically significant bleeding. Secondary outcomes were overall response, overall bleeding events, the use of rescue medication and adverse events (AEs)...
October 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28795988/eltrombopag-to-treat-thrombocytopenia-during-last-month-of-pregnancy-in-a-woman-with-myh9-related-disease-a-case-report
#11
Remi Favier, Celine De Carne, Elisabeth Elefant, Ruxanda Lapusneanu, Vasiliki Gkalea, Agnès Rigouzzo
MYH9-related disease (MYH9-RD) is an inherited rare autosomal dominant macrothrombocytopenia. Patients with MYH9-RD have giant platelets and leukocyte inclusion bodies caused by mutations in the MYH9 gene encoding the non-muscle myosin heavy chain II-A. Before identification of the causative gene, patients were diagnosed as Epstein or Fechtner or Sebastian syndromes or May-Hegglin anomaly. As with other inherited thrombocytopenias, the risk of increased bleeding during perioperative period or delivery is a major concern...
August 8, 2017: A & A Case Reports
https://www.readbyqxmd.com/read/28781864/repeated-successful-use-of-eltrombopag-in-chronic-primary-immune-thrombocytopenia-description-of-an-intriguing-case
#12
Cristina Santoro, Paola Volpicelli, Erminia Baldacci, Grazia Ferrara, Alice Di Rocco, Antonietta Ferretti, Marika Porrazzo, Maria Gabriella Mazzucconi
Thrombopoietin receptor agonists (TPO-RAs) are used as effective alternative treatments in ITP patients unresponsive to first-/second-line therapies. TPO-RAs can also be used to normalize platelet count to safely perform invasive procedures and chemotherapy, in case of malignancies. In few responsive patients, TPO-RAs can be suspended maintaining a sustained response.
August 2017: Clinical Case Reports
https://www.readbyqxmd.com/read/28759125/thrombopoietin-receptor-agonist-switch-in-adult-primary-immune-thrombocytopenia-itp-patients-a-retrospective-collaborative-survey-involving-four-spanish-centers
#13
Sunil Lakhwani, Maria Perera, Fernando Fernández-Fuerte, Mario A Rios de Paz, Melisa Torres, José María Raya, Miguel T Hernandez
OBJECTIVE: To describe the reasons for and result of thrombopoietin receptor agonists (TPO-RA) switching in adult ITP patients of four Spanish centres. METHODS: We retrospectively analized all patients who received sequential treatment with both TPO-RA between 2010 and 2015 recording clinical and biological parameters. RESULTS: 26 patients were included; 17 received first romiplostim and 9 received first eltrombopag. Reasons for switching were inefficacy (n=10), patient preference (n=8), side effects (n=5), and excessive platelet-count fluctuation (n=3)...
July 31, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28667351/efficacy-and-safety-of-eltrombopag-in-persistent-and-newly-diagnosed-itp-in-clinical-practice
#14
Tomás José González-López, Fernando Fernández-Fuertes, José Angel Hernández-Rivas, Blanca Sánchez-González, Violeta Martínez-Robles, María Teresa Alvarez-Román, Gloria Pérez-Rus, Cristina Pascual, Silvia Bernat, Esther Arrieta-Cerdán, Carlos Aguilar, Abelardo Bárez, María Jesús Peñarrubia, Pavel Olivera, Angeles Fernández-Rodríguez, Erik de Cabo, Luis Javier García-Frade, José Ramón González-Porras
Eltrombopag is safe and effective in primary chronic ITP. However, lack of clinical trials avoids a clear demonstration of its utility in newly diagnosed and persistent ITP. Our aim here is to report Spanish results for this type of patients. We retrospectively evaluated 220 adult primary ITP patients. According to standard definition, patients were allocated to newly diagnosed (n = 30), persistent (n = 30), and chronic (n = 160) ITP. Groups were homogenous regarding most relevant parameters. 180 (90%) of 220 patients achieved a platelet response (R) with 167 (75...
June 30, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28653480/eltrombopag-for-secondary-failure-of-platelet-recovery-post-allogeneic-hematopoietic-stem-cell-transplant-in-children
#15
Salah Ali, Adam Gassas, Melanie Kirby-Allen, Joerg Krueger, Muhammad Ali, Tal Schechter
Secondary failure of platelet engraftment occurs in 20% of patients undergoing allogeneic HSCT and is associated with poor outcome. Currently, there are no guidelines for treatment of late thrombocytopenia and platelet transfusion is the mainstay of treatment. Here, we describe the use of Eltrombopag to treat secondary failure of platelet recovery following HSCT in a child with severe aplastic anemia. Eltrombopag resulted in recovery of platelet count with no need for platelet transfusion support with no reported side effects...
September 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28643468/eltrombopag-use-in-a-patient-with-wiskott-aldrich-syndrome
#16
Maria Gabelli, Antonio Marzollo, Lucia Dora Notarangelo, Giuseppe Basso, Maria Caterina Putti
Wiskott-Aldrich syndrome (WAS) is an inherited X-linked disorder characterized by microthrombocytopenia, immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice. Eltrombopag, a thrombopoietin receptor agonist, may be useful to prevent bleeding while awaiting HSCT. We present a case of a male with WAS, profound thrombocytopenia, and bleeding diathesis successfully managed with eltrombopag before HSCT. Eltrombopag was given for 32 weeks obtaining a stable platelet count without any platelet transfusion...
June 23, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28627134/u-s-food-and-drug-administration-approval-summary-eltrombopag-for-the-treatment-of-pediatric-patients-with-chronic-immune-idiopathic-thrombocytopenia
#17
Lori A Ehrlich, Virginia E Kwitkowski, Gregory Reaman, Chia-Wen Ko, Lei Nie, Richard Pazdur, Ann T Farrell
The U.S. Food and Drug Administration (FDA) approved eltrombopag for pediatric patients with chronic immune (idiopathic) thrombocytopenia (ITP) ages ≥6 on June 11, 2015, and ages ≥1 on August 24, 2015. Approval was based on the FDA review of two randomized trials that included 159 pediatric patients with chronic ITP who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This manuscript describes the basis for approval of these applications. The FDA concluded that eltrombopag has shown efficacy and a favorable benefit to risk profile for pediatric patients with chronic ITP...
June 19, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28592755/acute-myeloid-leukemia-developing-secondary-immune-thrombocytopenia-after-umbilical-cord-blood-transplantation
#18
Rena Matsumoto, Kazuhiro Ito, Naoko Hosono, Yasufumi Matsuda, Katsunori Tai, Ippei Sakamaki, Goh Aoki, Hirohito Yamazaki, Shinji Nakao, Takahiro Yamauchi
A 64-year-old man was diagnosed with acute myeloid leukemia M2 (FLT3-ITD-positive). After induction chemotherapy and four courses of consolidation therapy, he underwent umbilical cord blood transplantation (CBT) in his first remission. He developed acute graft-versus-host disease (skin stage 2) after successful engraftment. On post-transplantation day 147, he was admitted to the hospital suffering from pneumonia. During the treatment, drastic thrombocytopenia was observed on day 251. Both platelet-associated immunoglobulin G and platelet antibody producing B cells were detected, and he was diagnosed with immune thrombocytopenia (ITP)...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28573819/use-of-eltrombopag-for-secondary-immune-thrombocytopenia-in-clinical-practice
#19
MULTICENTER STUDY
Tomás J González-López, María T Alvarez-Román, Cristina Pascual, Blanca Sánchez-González, Fernando Fernández-Fuentes, Gloria Pérez-Rus, José A Hernández-Rivas, Silvia Bernat, José M Bastida, María P Martínez-Badas, Violeta Martínez-Robles, Inmaculada Soto, Pavel Olivera, Estefanía Bolaños, Rafael Alonso, Laura Entrena, Marta Gómez-Nuñez, Arancha Alonso, María Yera Cobo, Isabel Caparrós, María Tenorio, Esther Arrieta-Cerdán, Elsa Lopez-Ansoar, Javier García-Frade, José R González-Porras
Eltrombopag is a second-line treatment in primary immune thrombocytopenia (ITP). However, its role in secondary ITP is unknown. We evaluated the efficacy and safety of eltrombopag in secondary ITP in daily clinical practice. Eighty-seven secondary ITP patients (46 with ITP secondary to autoimmune syndromes, 23 with ITP secondary to a neoplastic disease subtype: lymphoproliferative disorders [LPDs] and 18 with ITP secondary to viral infections) who had been treated with eltrombopag were retrospectively evaluated...
September 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28566882/platelets-in-liver-disease-cancer-and-regeneration
#20
REVIEW
Tomohiro Kurokawa, Nobuhiro Ohkohchi
Although viral hepatitis treatments have evolved over the years, the resultant liver cirrhosis still does not completely heal. Platelets contain proteins required for hemostasis, as well as many growth factors required for organ development, tissue regeneration and repair. Thrombocytopenia, which is frequently observed in patients with chronic liver disease (CLD) and cirrhosis, can manifest from decreased thrombopoietin production and accelerated platelet destruction caused by hypersplenism; however, the relationship between thrombocytopenia and hepatic pathogenesis, as well as the role of platelets in CLD, is poorly understood...
May 14, 2017: World Journal of Gastroenterology: WJG
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