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Fabian Depré, Nasra Aboud, Frauke Ringel, Abdulgabar Salama
BACKGROUND: Eltrombopag and romiplostim are thrombopoietin receptor agonists (TPOs) that have been increasingly used for the treatment of immune thrombocytopenia (ITP). Based on our experience, the incidence of abortive treatment with these drugs and the occurrence of adverse reactions that lead to therapy break-off despite response are higher than has been previously suggested. METHODS: During the last 8 years, a total of 65 patients were treated with eltrombopag and/or romiplostim at our institute...
September 2016: Transfusion Medicine and Hemotherapy
Q A Hill, L C Harrison, A D Padmakumar, R G Owen, K R Prasad, G F Lucas, P Tachtatzis
OBJECTIVE AND IMPORTANCE: Transplantation-mediated alloimmune thrombocytopenia (TMAT) occurs when leukocytes transferred in a donor organ from a patient with immune thrombocytopenia (ITP), mount a response against recipient platelets. We present the first fatal case of TMAT following liver transplantation and review its aetiology and treatment. CLINICAL PRESENTATION: The liver donor had ITP and died from an intracranial haemorrhage. The recipient platelet count fell to 2 × 10(9)/l on post-operative day 2...
October 21, 2016: Hematology (Amsterdam, Netherlands)
Reiko Watanabe, Takayuki Tabayashi, Tatsuki Tomikawa, Morihiko Sagawa, Tomoe Anan-Nemoto, Yuta Kimura, Yasuyuki Takahashi, Michihide Tokuhira, Satoshi Otaki, Hidenori Oi, Makoto Sawano, Satoshi Sugiyama, Masahiro Kizaki
Thrombopoietin receptor (TPO-R) agonists have been shown to be effective in refractory chronic immune thrombocytopenia (ITP); however, their efficacy in patients under critical care is not known. We report the case of a female patient with a newly diagnosed ITP who experienced severe bleeding from an external wound. The patient was administered the standard treatments for ITP, which are high-dose intravenous immunoglobulin (IVIg) and corticosteroids. However, following failure of these treatments, we administered romiplostim on day 6 after the onset of ITP...
October 5, 2016: International Journal of Hematology
Jyothis Purushothaman, Kochuthresia J Puthumana, Aswath Kumar, Susheela J Innah, Sareena Gilvaz
Immune thrombocytopenic purpura is a common acquired autoimmune disorder defined by a low platelet count secondary to accelerated platelet destruction or impaired thrombopoesis by anti-platelet antibodies. Thrombopoietin (TPO)-mimetic drugs such as eltrombopag and romiplostim have been used successfully in many nonpregnant individuals with immune thrombocytopenia (ITP) but studies based on its effects in pregnancy are limited. A 27-year-old multigravida who is a known case of ITP with bad obstetric history was referred to the Department of Obstetrics and Gynecology at 26 weeks of gestation with complaints of mucosal bleeding and recurrent abortions...
July 2016: Asian Journal of Transfusion Science
Ariela L Marshall, Roberta Scarpone, Melanie De Greef, Robert Bird, David J Kuter
No abstract text is available yet for this article.
September 1, 2016: Haematologica
Andrew N McCall, Johann P Ingimarsson, Griffin T Morrisson, Christopher C Hook, Amy E Krambeck
BACKGROUND: Percutaneous nephrolithotomy (PCNL) for large stone burden can be problematic in patients with significant risk of bleeding complications, specifically thrombocytopenia. This report demonstrates effective correction of two patients' thrombocytopenia, subsequently leading to removal of large stone burden through PCNL. CASE PRESENTATION: We present two Middle Eastern patients who presented with medical histories significant for thrombocytopenia, secondary to splenomegaly and hepatic vein thrombosis, and large volume nephrolithiasis...
2016: J Endourol Case Rep
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
Michael Steurer, Philippe Quittet, Helen A Papadaki, Dominik Selleslag, Jean-François Viallard, Georgia Kaiafa, Ann Janssens, Tomas Kozak, Hans Wadenvik, Marieke Schoonen, Laura Belton, Georg Kreuzbauer
OBJECTIVE: Romiplostim has maintained long-term platelet counts in patients with immune thrombocytopenia (ITP) for up to 5 years in clinical studies. This prospective observational study aimed to describe romiplostim utilisation and outcomes in European clinical practice. METHODS: Adults with primary ITP who received romiplostim in routine care were eligible. RESULTS: Three-hundred and forty patients were eligible for analysis, of whom 299 (88%) completed the two-year observation period...
August 25, 2016: European Journal of Haematology
Jose Perdomo
Romiplostim is a thrombopoietin receptor agonist (TPO-RA) used for the treatment of adult primary immune thrombocytopenia (ITP). ITP is an autoimmune condition characterized by low platelet counts due to increased destruction and reduced platelet production. First-line interventions include corticosteroids, anti-D, and intravenous immunoglobulins, while second-line therapies comprise splenectomy, rituximab, cyclosporine A, and TPO-RAs. The recognition that compromised platelet production is a critical part of the pathogenesis of ITP prompted the development of therapeutic strategies based on the stimulation of the TPO receptor...
2016: ImmunoTargets and Therapy
Christian A Di Buduo, Manuela Currao, Alessandro Pecci, David L Kaplan, Carlo L Balduini, Alessandra Balduini
Eltrombopag is a small non-peptide thrombopoietin mimetic that has been approved for increasing platelet count not only in immune thrombocytopenia and HCV-related thrombocytopenia, but also in aplastic anemia. Moreover, this drug is under investigation for increasing platelet count in myelodysplastic syndromes. Despite current clinical practice, the mechanisms governing Eltrombopag's impact on human hematopoiesis are largely unknown in part due to the impossibility of using traditional in vivo models. To investigate Eltrombopag's impact on megakaryocyte functions, we employed our established in vitro model for studying hematopoietic stem cell differentiation combined with our latest 3D silk-based bone marrow tissue model...
August 11, 2016: Haematologica
Shosaku Nomura
Immune thrombocytopenia (ITP) is an acquired hemorrhagic condition characterized by the accelerated clearance of platelets caused by antiplatelet autoantibodies. A platelet count in peripheral blood <100 × 10(9)/L is the most important criterion for the diagnosis of ITP. However, the platelet count is not the sole diagnostic criterion, and the diagnosis of ITP is dependent on additional findings. ITP can be classified into three types, namely, acute, subchronic, and persistent, based on disease duration...
2016: Clinical Medicine Insights. Blood Disorders
Shima Fayaz, Pezhman Fard-Esfahani, Majid Golkar, Mojgan Allahyari, Sedigheh Sadeghi
BACKGROUND: Romiplostim is a peptibody analogue of thrombopoietin (TPO) which regulates platelet production. This molecule consists of two main parts: Peptide sequences which like wild type TPO, mimics stimulation of TPO receptor and IgG1Fc, (Peptide + Antibody = Peptibody). This drug is used in treatment of chronic Immune Thrombocytopenic Purpura (ITP). METHODS: In this project E. coli bacteria were transformed by a construct harboring peptibody fusion gene...
2016: Daru: Journal of Faculty of Pharmacy, Tehran University of Medical Sciences
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
Barbara Kohn, Gürkan Bal, Aleksandra Chirek, Sina Rehbein, Abdulgabar Salama
BACKGROUND: Immune thrombocytopenia (ITP) in dogs is analogous to that in humans. Romiplostim, a novel thrombopoietin receptor (TPO-R) agonist, is currently used for the treatment of refractory ITP in humans, but not in dogs. Here, we describe the response to romiplostim in five dogs with refractory ITP. Five dogs with severe and refractory ITP (three primary and two secondary) received romiplostim subcutaneously. Four dogs were administered 3-5 μg/kg and one dog received 10-13 μg/kg body weight once weekly...
2016: BMC Veterinary Research
Xin-Guang Liu, Shuang Liu, Qi Feng, Xue-Na Liu, Guo-Sheng Li, Zi Sheng, Peng Chen, Yang Liu, Yu Wei, Xiao-Yuan Dong, Ping Qin, Chengjiang Gao, Chunhong Ma, Lei Zhang, Ming Hou, Jun Peng
Elevated expression of the activating Fcγ receptor (FcγR) I and FcγRIIa together with decreased expression of the inhibitory FcγRIIb are involved in the pathogenesis of primary immune thrombocytopenia (ITP). Thrombopoietin receptor agonists (TPO-RAs) have been used clinically for the management of ITP; however, little is known about the effect of TPO-RAs on FcγR modulation in ITP. In this prospective study, we measured the alteration in monocyte FcγR expression from 21 corticosteroid-resistant/relapsed patients with chronic ITP receiving eltrombopag therapy...
August 11, 2016: Blood
Cindy Neunert, Jenny Despotovic, Kristina Haley, Michele P Lambert, Kerri Nottage, Kristin Shimano, Carolyn Bennett, Robert Klaassen, Kimo Stine, Alexis Thompson, Yves Pastore, Travis Brown, Peter W Forbes, Rachael F Grace
BACKGROUND: Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option for children who require an increased platelet count. PROCEDURE: We performed a multicenter retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use. RESULTS: Seventy-nine children had a total of 87 treatments (28 eltrombopag, 43 romiplostim, and eight trialed on both)...
August 2016: Pediatric Blood & Cancer
Ann Janssens, Francesco Rodeghiero, David Anderson, Beng H Chong, Zoltán Boda, Ingrid Pabinger, Libor Červinek, Deirdra R Terrell, Xuena Wang, Janet Franklin
The effects of romiplostim on bone marrow morphology were evaluated in adults with immune thrombocytopenia (ITP). Patients with platelet counts <50 × 10(9)/L, ≥1 prior ITP therapies, and no collagen at baseline received weekly subcutaneous romiplostim starting at 1 μg/kg, adjusted to maintain platelet counts between 50 and 200 × 10(9)/L. Biopsies were scheduled after 1, 2, or 3 years of romiplostim (cohorts 1, 2, and 3, respectively). Irrespective of scheduled time, biopsies were performed earlier if patients discontinued or failed to achieve/maintain a response to romiplostim...
June 2016: Annals of Hematology
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