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Eltrombopag

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https://www.readbyqxmd.com/read/28667351/efficacy-and-safety-of-eltrombopag-in-persistent-and-newly-diagnosed-itp-in-clinical-practice
#1
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
#2
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...
June 27, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28643468/eltrombopag-use-in-a-patient-with-wiskott-aldrich-syndrome
#3
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
#4
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
#5
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
#6
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...
June 1, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28566882/platelets-in-liver-disease-cancer-and-regeneration
#7
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
https://www.readbyqxmd.com/read/28537785/a-practical-guide-to-the-use-of-eltrombopag-in-children-with-chronic-immune-thrombocytopenia
#8
John D Grainger, Sharon Thind
INTRODUCTION: Pediatric immune thrombocytopenia (ITP) may be associated with significant burden on children and their parents/caregivers. Thrombopoietin (TPO) receptor agonists (RAs) have been used to treat adult patients with chronic ITP (cITP) for nearly a decade and following pediatric studies Eltrombopag has been recently approved for pediatric cITP in the United States and Europe. TPO-RA s may help reduce the risk of bleeding and the need for conventional ITP therapies. REVIEW: In this review, the clinical data demonstrating the efficacy and safety of TPO-RAs in pediatric ITP are evaluated, key recommendations regarding safe administration of eltrombopag are provided, and potential future directions in management of pediatric ITP are discussed...
March 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28473904/from-chronic-immune-thrombocytopenia-to-severe-aplastic-anemia-recent-insights-into-the-evolution-of-eltrombopag
#9
REVIEW
Harinder Gill, Raymond S M Wong, Yok-Lam Kwong
Thrombopoietin (TPO) is the most potent cytokine stimulating thrombopoiesis. Therapy with exogenous TPO is limited by the formation of antibodies cross-reacting with endogenous TPO. Mimetics of TPO are compounds with no antigenic similarity to TPO. Eltrombopag is an orally-active nonpeptide small molecule that binds to the transmembrane portion of the TPO receptor MPL. Initial trials of eltrombopag have centered on immune thrombocytopenia (ITP), which is due to both increased destruction and decreased production of platelets...
May 2017: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/28469947/successful-long-term-use-of-eltrombopag-in-a-patient-with-refractory-severe-thrombocytopenia-associated-with-chronic-lymphocytic-leukemia-that-allowed-oral-anticoagulant-treatment-for-severe-cardiomyopathy
#10
Juárez Salcedo Luis Miguel, Gil-Fernández Juan José
Autoimmune cytopenias (AICs) are frequently associated with chronic lymphocytic leukemia (CLL). The most common of these AICs is autoimmune hemolytic anemia (AIHA); the second most is immune thrombocytopenia (ITP). Here, we report on a patient with CLL-associated ITP, with thrombocytopenia refractory to corticosteroids and intravenous immunoglobulins, in which continuous oral treatment with Eltrombopag allowed initiation and maintenance of an oral anticoagulation treatment with Acenocoumarol that was indicated because of a severe arrhythmogenic cardiomyopathy...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/28423296/eltrombopag-added-to-standard-immunosuppression-for-aplastic-anemia
#11
Danielle M Townsley, Phillip Scheinberg, Thomas Winkler, Ronan Desmond, Bogdan Dumitriu, Olga Rios, Barbara Weinstein, Janet Valdez, Jennifer Lotter, Xingmin Feng, Marie Desierto, Harshraj Leuva, Margaret Bevans, Colin Wu, Andre Larochelle, Katherine R Calvo, Cynthia E Dunbar, Neal S Young
BACKGROUND: Acquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia...
April 20, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28295201/thrombopoetin-receptor-agonist-therapy-in-thrombocytopenia-itp-and-beyond
#12
Alice Taylor, John Paul Westwood, Faidra Laskou, Siobhan McGuckin, Marie Scully
Eltrombopag is well established in treatment of severe immune thrombocytopenia (ITP) and is increasingly commonplace in second-line management. A role is also suggested for both bridging therapy for surgery, as well as treating thrombocytopenia due to non-immune aetiologies. We present the largest single-centre experience with eltrombopag, with our cohort of 62 patients. Patients with severe ITP (n = 34) had 91·2% response, which was sustained over a median of 18·5 months. In 41·4% of ITP cases (n = 14), complete response (CR- platelet count >100 × 10(9) /l) was achieved and in 2 cases, therapy was stopped and CR maintained...
May 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28295192/state-of-the-art-how-i-manage-immune-thrombocytopenia
#13
REVIEW
Nichola Cooper
The management of patients with immune thrombocytopenia (ITP) is rapidly evolving. Over the last 15 years, a number of novel treatments have improved practice, with many steroid-sparing agents and a reduction in the progression to splenectomy. Although this has improved clinical care, many therapeutic challenges remain. There is no diagnostic test, no biomarkers to direct treatment and few comparative studies to help management decisions. Development of up to date guidelines is difficult with little high-grade evidence...
April 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28293407/thrombocytopenia-in-patients-with-chronic-hepatitis-c-virus-infection
#14
REVIEW
Sumit Dahal, Smrity Upadhyay, Rashmi Banjade, Prajwal Dhakal, Nabin Khanal, Vijaya Raj Bhatt
Thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection is a major problem. The pathophysiology is multifactorial, with auto-immunogenicity, direct bone marrow suppression, hypersplenism, decreased production of thrombopoietin and therapeutic adverse effect all contributing to thrombocytopenia in different measures. The greatest challenge in the care of chronic HCV patients with thrombocytopenia is the difficulty in initiating or maintaining IFN containing anti-viral therapy. Although at present, it is possible to avoid this challenge with the use of the sole Direct Antiviral Agents (DAAs) as the primary treatment modality, thrombocytopenia remains of particular interest, especially in cases of advanced liver disease...
2017: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/28271416/bleeding-tendency-and-platelet-function-during-treatment-with-romiplostim-in-children-with-severe-immune-thrombocytopenic-purpura
#15
Elena V Suntsova, Irina M Demina, Anastasia A Ignatova, Nikolay M Ershov, Natalia M Trubina, Juliya Dobrynina, Irina V Serkova, Zhanna S Supik, Ekaterina V Orekhova, Lili A Hachatryan, Natalia N Kotskaya, Aleksey V Pshonkin, Aleksey A Maschan, Galina A Novichkova, Mikhail A Panteleev
It has been suggested that platelet function in chronic immune thrombocytopenic purpura (ITP) may be abnormal. Thrombopoietin mimetics used for treatment can affect it, but the data remain limited. We investigated platelet function of 20 children diagnosed with severe ITP (aged 1-16 years, 12 females and eight males). Platelet functional activity in whole blood was characterized by flow cytometry before and after stimulation with SFLLRN plus collagen-related peptide. Levels of CD42b, PAC1, and CD62P, but not CD61 or annexin V, were significantly increased (P < 0...
June 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28162984/eltrombopag-versus-placebo-for-low-risk-myelodysplastic-syndromes-with-thrombocytopenia-eqol-mds-phase-1-results-of-a-single-blind-randomised-controlled-phase-2-superiority-trial
#16
Esther N Oliva, Caterina Alati, Valeria Santini, Antonella Poloni, Alfredo Molteni, Pasquale Niscola, Flavia Salvi, Grazia Sanpaolo, Enrico Balleari, Ulrich Germing, Pierre Fenaux, Aspasia Stamatoullas, Giuseppe A Palumbo, Prassede Salutari, Stefana Impera, Paolo Avanzini, Agostino Cortelezzi, Anna Marina Liberati, Paola Carluccio, Francesco Buccisano, Maria Teresa Voso, Stefano Mancini, Austin Kulasekararaj, Fortunato Morabito, Monica Bocchia, Patrizia Cufari, Maria Antonietta Aloe Spiriti, Irene Santacaterina, Maria Grazia D'Errigo, Irene Bova, Gina Zini, Roberto Latagliata
BACKGROUND: In myelodysplastic syndromes, thrombocytopenia is associated with mortality, but treatments in this setting are scarce. We tested whether eltrombopag, a thrombopoietin receptor agonist, might be effective in improving thrombocytopenia in lower-risk myelodysplastic syndromes and severe thrombocytopenia. METHODS: EQoL-MDS was a single-blind, randomised, controlled, phase 2 superiority trial of adult patients with low-risk or International Prognostic Scoring System intermediate-1-risk myelodysplastic syndromes and severe thrombocytopenia...
March 2017: Lancet Haematology
https://www.readbyqxmd.com/read/28151387/bidirectional-inefficacy-or-intolerability-of-thrombopoietin-receptor-agonists-new-data-and-a-concise-review
#17
Fabian Depré, Nasra Aboud, Beate Mayer, Abdulgabar Salama
BACKGROUND: There is evidence that the thrombopoietin-receptor agonists romiplostim and eltrombopag may have different therapeutic values and adverse reaction profiles. Here we present new data and provide a review of all studies dealing with switching between these two drugs. MATERIALS AND METHODS: A total of 89 patients (38 males and 51 females, aged between 14-87 years) were treated with eltrombopag and/or romiplostim between 2007 and 2016 at our institution...
January 25, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28142109/cytokine-changes-in-response-to-tpo-receptor-agonist-treatment-in-primary-immune-thrombocytopenia
#18
Ming-Ming Qu, Xue-Na Liu, Xin-Guang Liu, Qi Feng, Yang Liu, Xu Zhang, Shuang Liu, Lei Zhang, Guo-Sheng Li, Yuan-Yuan Zhu, Ming-Yun Lv, Jun Peng, Ming Hou
Thrombopoietin receptor agonists (TPO-RAs) have been clinically used in primary immune thrombocytopenia (ITP) with favorable outcomes, while their effect on cytokine regulation in ITP remains unknown. In the present study, plasma and mRNA expression levels of interleukin (IL)-2, interferon gamma (IFN-γ), IL-4, IL-17A, and transforming growth factor-β1 (TGF-β1) were determined by ELISA and real-time quantitative PCR in 26 corticosteroid-resistant/relapsed ITP patients receiving eltrombopag or rhTPO therapy and 15 healthy controls (HCs)...
April 2017: Cytokine
https://www.readbyqxmd.com/read/28088971/-eltrombopag-for-refractory-thrombocytopenia-in-patients-with-allogeneic-hematopoietic-stem-cell-transplantation
#19
Y R Ma, X J Huang, X D Mo, W Han, C H Yan, Y Chen, Y Ji, Y Y Chen, Y Wang, X H Zhang, K Y Liu, L P Xu
Objective: To evaluate the efficacy and safety of eltrombopag in post-HSCT thrombocytopenia. Methods: A total of 10 patients who underwent post-HSCT thrombocytopenia at Peking University center, who had been treated with eltrombopag, were retrospectively evaluated. Results: Of the 10 cases, 5 males and 5 females with a median of 34 years old (range, 17-54 years), 5 patients were acute myeloid leukemia, 3 with acute lymphoid leukemia and 2 with severe aplastic anemia. Nine patients had undergone haplo-identical donor transplantation, and one patient was a matched related recipient...
December 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/28056326/-a-case-report-of-combined-treatment-with-rituximab-and-eltrombopag-in-refractory-immune-thrombocytopenia
#20
(no author information available yet)
No abstract text is available yet for this article.
January 1, 2017: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
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