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eltrombopag long term

Rachel Allen, Peter Bryden, Kelly M Grotzinger, Ceilidh Stapelkamp, Bethan Woods
OBJECTIVES: To evaluate the cost-effectiveness of eltrombopag compared with romiplostim to be used in the treatment of chronic immune thrombocytopenia in patients in England and Wales who are splenectomized or ineligible for splenectomy and are refractory to other treatments. METHODS: A Markov cohort model in which patients were administered a sequence of treatments was used to predict long-term outcomes associated with each treatment. The model was informed by data from the eltrombopag clinical trial program and the available literature...
July 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Matthieu Mahévas, Mathieu Gerfaud-Valentin, Guillaume Moulis, Louis Terriou, Sylvain Audia, Sophie Guenin, Guillaume Le Guenno, Gilles Salles, Olivier Lambotte, Nicolas Limal, Jean-François Viallard, Stephane Cheze, Cecile Tomowiak, Bruno Royer, Antoine Neel, Odile Debouverie, Arnaud Hot, Isabelle Durieu, Antoinette Perlat, Manuel Cliquennois, Clémence Deteix, Marc Michel, Bertrand Godeau
Refractory immune thrombocytopenia (ITP) was previously defined as lack of a minimum response to splenectomy and the requirement for long-term treatment to reduce the risk of significant bleeding events. In this multicenter study, we included 37 patients with multirefractory ITP, defined as no response to splenectomy, rituximab, romiplostim, and eltrombopag. As compared with a historical cohort of 183 ITP patients, matched on the calendar year of ITP diagnosis with a 5:1 ratio, patients with multirefractory ITP were more likely to have secondary ITP (odds ratio [OR], 4...
September 22, 2016: Blood
Bertrand Godeau
During the last two decades, new therapeutic strategies have been developed, particularly anti-CD20 agents and thrombopoietin-receptor (TPO-r) mimetics, for immune thrombocytopenia (ITP). However, although the new efficient drugs have deeply modified the therapeutic strategy and the disease prognosis, there are still unmet needs and challenges. Concerning rituximab, reassuring data concerning its safety have recently been reported. The main limitation of the treatment is its modest long-term efficacy, with frequent disease relapse...
April 2016: Seminars in Hematology
Alexandra Schifferli, Thomas Kühne
In 2008, new drugs that mimic the effects of thrombopoietin became available for the treatment of primary immune thrombocytopenia, eg, romiplostim and eltrombopag. These drugs activate the thrombopoietin receptor, stimulate the production of megakaryocytes, and increase the production of platelets. Important clinical observation has been gained, such as unexpected long-term remission after stopping thrombopoietin receptor agonists. The pathophysiology of this unforeseen cure is currently the subject of discussion and is investigated in clinical trials and laboratory research projects...
April 2016: Seminars in Hematology
Prakash Vishnu, David M Aboulafia
Inhibition of platelet production and mediated by antiplatelet antibodies is a well-known mechanism causing low platelet counts in immune thrombocytopenia (ITP). Use of thrombopoietin receptor agonists increases platelet counts and decreases the risk of bleeding in patients with ITP. Two such thrombopoietin receptor agonists, romiplostim and eltrombopag, are approved by the US Food and Drug Administration to treat thrombocytopenia in adults, and most recently, children with persistent or chronic ITP. This review focuses on the efficacy data and safety analysis of the pooled data from the clinical trials evaluating romiplostim for treatment of adults with ITP...
2016: Journal of Blood Medicine
J Dyba, A Tinmouth, C Bredeson, J Matthews, D S Allan
BACKGROUND: Late graft failure after allogeneic haematopoietic cell transplantation (HCT) can result from the failed engraftment of long-term engrafting cells. The use of thrombopoietin (TPO) receptor agonists (TRA) has been extensively studied and remains an important component of experimental ex vivo stem cell expansion protocols, but its use in allogeneic transplantation is still evolving. METHODS: We describe the use of eltrombopag, a TRA, to stimulate the rescue of late graft failure in a patient following allogeneic HCT, and we performed a systematic review of published studies describing the use of TRAs following allogeneic transplantation...
June 2016: Transfusion Medicine
Pietro Merli, Luisa Strocchio, Luciana Vinti, Giuseppe Palumbo, Franco Locatelli
INTRODUCTION: Eltrombopag is an orally bioavailable, non-peptide thrombopoietin receptor agonist capable of stimulating platelet production through the differentiation of CD34+ hematopoietic progenitor cells into committed CD41+ megakaryocyte precursors and proliferation of megakaryocyte progenitor cells. AREAS COVERED: This drug has been tested in several clinical trials in adult patients with chronic immune thrombocytopenia (ITP), demonstrating the ability of the drug to reduce the burden of thrombocytopenia and its associated side effects...
2015: Expert Opinion on Pharmacotherapy
Angelica Maria Garzon, William Beau Mitchell
Most children with immune thrombocytopenia (ITP) will have spontaneous remission regardless of therapy, while about 20% will go on to have chronic ITP. In those children with chronic ITP who need treatment, standard therapies for acute ITP may have adverse effects that complicate their long-term use. Thus, alternative treatment options are needed for children with chronic ITP. Thrombopoietin receptor agonists (TPO-RA) have been shown to be safe and efficacious in adults with ITP, and represent a new treatment option for children with chronic ITP...
2015: Frontiers in Pediatrics
Ali Eser, Tayfur Toptas, Osman Kara, Aslihan Sezgin, Figen Noyan-Atalay, Guven Yilmaz, Toluy Ozgumus, Funda Pepedil-Tanrikulu, Isik Kaygusuz-Atagunduz, Tulin Firatli-Tuglular
Eltrombopag was used in patients with chronic primary immune thrombocytopenia (ITP) who did not tolerate or were refractory to two or more previous treatments. The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment. Data were extracted from medical chart records retrospectively. Platelet count of at least 50 000/μl at any time point during the treatment was defined as the 'response'. Median duration of eltrombopag treatment was 29 weeks (11-74). The number of patients who had a platelet count of at least 50 000/μl at any time point was 26 (83...
January 2016: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
Füsun Özdemirkıran, Bahriye Payzın, H Demet Kiper, Sibel Kabukçu, Gülsüm Akgün Çağlıyan, Selda Kahraman, Ömür Gökmen Sevindik, Cengiz Ceylan, Gürhan Kadıköylü, Fahri Şahin, Ali Keskin, Öykü Arslan, Mehmet Ali Özcan, Gülnur Kabukçu, Gülsüm Görgün, Zahit Bolaman, Filiz Büyükkeçeci, Oktay Bilgir, İnci Alacacıoğlu, Filiz Vural, Murat Tombuloğlu, Zafer Gökgöz, Güray Saydam
OBJECTIVE: Immune thrombocytopenia (ITP) is an immune-mediated disease characterized by transient or persistent decrease of the platelet count to less than 100x109/L. Although it is included in a benign disease group, bleeding complications may be mortal. With a better understanding of the pathophysiology of the disease, thrombopoietin receptor agonists, which came into use in recent years, seem to be an effective option in the treatment of resistant cases. This study aimed to retrospectively assess the efficacy, long-term safety, and tolerability of eltrombopag in Turkish patients with chronic ITP in the Aegean region of Turkey...
December 2015: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
Hideki Hattori, Maki Kuwayama, Hiroyuki Takamori, Nobuko Nishiura, Takahiro Karasuno
We report a 77-year-old Japanese man with idiopathic thrombocytopenic purpura (ITP) which developed into chronic myelogenous leukemia (CML) during treatment with eltrombopag, a thrombopoetin (TPO) receptor agonist, because the disease was refractory to prednisolone. Eltrombopag can induce a good reaction in terms of the platelet count. However, CML in the chronic phase developed in about 19 months in our present case. Dasatinib was administered because he had diabetes. However, a blastic crisis immediately occurred...
December 2014: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Antonio M Risitano
Acquired idiopathic AA is the most typical form of immune-mediated bone marrow failure; the standard treatment of AA for patients who lack a transplant option is immunosuppressive treatment (IST). The standard IST regimen is horse anti-thymocyte globuline (h-ATG) combined with cyclosporine A (CsA), which results in a long term survival around 65-70%. In the past two decades several efforts have been made to improve these results, however results were quite disappointing. Indeed, the addition of a third immunosuppressive agent (micophenolate and sirolimus) on the h-ATG/CsA platform has not resulted in any benefit...
January 25, 2015: Current Drug Targets
Valeria Santini, Pierre Fenaux
Myelodysplastic syndromes (MDS) are clinically, genetically, and molecularly heterogeneous neoplastic diseases characterized by ineffective hematopoiesis leading to peripheral cytopenias. The severity of cytopenias influences oucome and is considered in prognostic scoring systems; thrombocytopenia, although not the most frequently observed at disease onset, is estimated to affect 40%-80% of MDS patients. As thrombocytopenia in MDS is determined by premature marrow destruction and programmed cell death, the use of thrombomimetic agents has been proposed in order to stimulate megakaryocyte differentation and proliferation...
January 2015: Seminars in Hematology
Seung Hwan Shin, Sung Eun Lee, Jong Wook Lee
Recent advances in the treatment of aplastic anemia (AA) made most of patients to expect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT) from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatment option for younger patients with severe or very severe AA, whereas immunosuppressive treatment (IST) is an alternative option for others. Horse anti-thymocyte globuline (ATG) with cyclosporin A (CsA) had been a standard IST regimen with acceptable response rate. Recently, horse ATG had been not available and replaced with rabbit ATG in most countries...
November 2014: Korean Journal of Internal Medicine
Efthymia Vlachaki, Nikolaos Sousos, Vasilios Perifanis, Georgia Kaiafa, Ilias Onoufriadis, Apostolos Hatzitolios, Panagiota Boura
BACKGROUND/AIM: Thrombopoietin receptor agonists (romiplostim and eltrombopag) have recently been licensed for the treatment of thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) with an insufficient response to corticosteroids, immunoglobulins or splenectomy. In the present case series, we present 4 nonresponding patients with chronic ITP who achieved maintenance of complete response (CR) for a period of at least 6 months on eltrombopag treatment administered in a modified regimen of 25 mg for 2, 3 or 5 days a week...
2015: Acta Haematologica
Kavitha Ramaswamy, Loan Hsieh, Emily Leven, Michaela V Thompson, Diane Nugent, James B Bussel
OBJECTIVES: To determine the safety, tolerability, or efficacy of 2 licensed thrombopoietic agents in children with persistent and chronic immune thrombocytopenia (ITP). STUDY DESIGN: Retrospective analysis approved by the institutional review board of children with ITP not on-study who received thrombopoietin (TPO) therapy; 21 received romiplostim (11 at Children's Hospital of Orange County, 10 at Weill Cornell Medical Center) and 12 received eltrombopag (all at Weill Cornell Medical Center)...
September 2014: Journal of Pediatrics
Sammy Saab, Vignan Manne, Sherona Bau, Justin A Reynolds, Ruby Allen, Leonard Goldstein, Francisco Durazo, Mohammed El-Kabany, Steven Han, Ronald W Busuttil
BACKGROUND: There has been increasing interest in using protease inhibitors with pegylated interferon and ribavirin to treat recurrent hepatitis C (HCV) disease in liver transplant recipients. METHODS: We retrospectively evaluated the safety and efficacy in liver transplant recipients treated for recurrent hepatitis C genotype 1 with the combination of peginterferon, ribavirin and boceprevir. RESULTS: Twenty liver transplant recipients were treated for recurrent hepatitis C...
January 2015: Liver International: Official Journal of the International Association for the Study of the Liver
Anil Kumar Tripathi, Ayush Shukla, Sanjay Mishra, Yogendra Singh Yadav, Deependra Kumar Yadav
Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterised by isolated thrombocytopenia (peripheral blood platelet count <100 × 10(9)/L) in the absence of other causes or disorders that may be associated with thrombocytopenia. The upfront treatment in newly diagnosed ITP patients is steroids; however, about one-third patients do not respond, and require other treatment, including IVIg, anti-D, or splenectomy. Previous studies have shown decreased platelet production in some ITP patients, aside from the evidence of enhanced platelet destruction...
April 2014: International Journal of Hematology
Tomás José González-López, Blanca Sánchez-González, Cristina Pascual, Maryam Arefi, Erik de Cabo, Arancha Alonso, Mónica Martín-Salces, Reyes Jiménez-Bárcenas, María Calbacho, Pilar Galan, Abelardo Barez, José Ramón González-Porras
Eltrombopag is effective and safe in chronic immune thrombocytopenia (ITP) patients not responding to previous therapy. However, when eltrombopag is discontinued, platelet counts usually return to baseline within 2 weeks. Here, we describe the clinical characteristics of the, to the best of our knowledge, largest case series of patients with ITP, who presented sustained responses after discontinuing eltrombopag (n = 12). The median time from diagnosis to eltrombopag initiation was 24 months (range, 1-480)...
2015: Platelets
Ronan Desmond, Danielle M Townsley, Bogdan Dumitriu, Matthew J Olnes, Phillip Scheinberg, Margaret Bevans, Ankur R Parikh, Kinneret Broder, Katherine R Calvo, Colin O Wu, Neal S Young, Cynthia E Dunbar
About a quarter of patients with severe aplastic anemia remain pancytopenic despite immunosuppressive therapy. We have previously demonstrated that eltrombopag has efficacy in this setting with 44% (11/25) of patients having clinically significant hematologic responses. We now report safety and efficacy data on a further 18 patients and long-term follow-up on the entire cohort of 43 patients. The overall response rate was 17 of 43 patients (40%) at 3 to 4 months, including tri- and bilineage responses. The majority of patients who remained on eltrombopag in an extension study (14/17) continued to show improvement, and 7 eventually had significant increases in neutrophil, red cell, and platelet lineages...
March 20, 2014: Blood
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