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https://www.readbyqxmd.com/read/29172661/-novelties-in-the-treatment-of-pediatric-immune-thrombocytopenia-2017
#1
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/29143887/diagnosis-and-treatment-of-aplastic-anemia
#2
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/29042367/safety-and-efficacy-of-long-term-treatment-of-chronic-persistent-itp-with-eltrombopag-final-results-of-the-extend-study
#3
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/28877042/hematopoietic-cell-transplantation-for-aplastic-anemia
#4
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/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
#5
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/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
#6
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/28034281/is-there-any-role-for-splenectomy-in-adulthood-onset-chronic-immune-thrombocytopenia-in-the-era-of-tpo-receptors-agonists-a-critical-overview
#7
Vibor Milunovic, Inga Mandac Rogulj, Slobodanka Kolonic Ostojic
Immune thrombocytopenia (ITP) in adulthood is characterized by chronic relapsing course. Despite the efficacious first line treatment (corticosteroid, intravenous immunoglobulin), majority of patients will enter the chronic phase warranting another treatment approach. Until recently, splenectomy performed in ITP chronic phase represented the standard of care with long-term remissions in more than 70% of patients, but however has never been tested in clinical trials. However, with the advances of our understanding of ITP pathophysiology and the shifting focus on megakaryocyte impairment, novel drugs were introduced in treatment paradigm, mainly trombopoietin receptor agonists (TPO-RAs); romiplostim and eltrombopag...
December 29, 2016: Cardiovascular & Hematological Disorders Drug Targets
https://www.readbyqxmd.com/read/28006767/a-2-year-longitudinal-prospective-study-of-the-effects-of-eltrombopag-on-bone-marrow-in-patients-with-chronic-immune-thrombocytopenia
#8
MULTICENTER STUDY
Russell K Brynes, Raymond S M Wong, Maung M Thein, Kalpana K Bakshi, Paul Burgess, Dickens Theodore, Attilio Orazi
BACKGROUND: The long-term effects of eltrombopag on bone marrow (BM) reticulin and/or collagen deposition in previously treated adults with chronic immune thrombocytopenia (ITP) were assessed. METHODS: Three BM biopsies were collected at baseline and after 1 and 2 years of eltrombopag treatment. Specimens were centrally processed, stained for reticulin and collagen, independently reviewed by 2 hematopathologists, and rated according to the European Consensus 0-3 scale of marrow fibrosis (MF)...
2017: Acta Haematologica
https://www.readbyqxmd.com/read/28005311/eltrombopag-a-thrombopoietin-mimetic-crosses-the-blood-brain-barrier-and-impairs-iron-dependent-hippocampal-neuron-dendrite-development
#9
T W Bastian, K A Duck, G C Michalopoulos, M J Chen, Z-J Liu, J R Connor, L M Lanier, M C Sola-Visner, M K Georgieff
Essentials Potential neurodevelopmental side effects of thrombopoietin mimetics need to be considered. The effects of eltrombopag (ELT) on neuronal iron status and dendrite development were assessed. ELT crosses the blood-brain barrier and causes iron deficiency in developing neurons. ELT blunts dendrite maturation, indicating a need for more safety studies before neonatal use. SUMMARY: Background Thrombocytopenia is common in sick neonates. Thrombopoietin mimetics (e.g. eltrombopag [ELT]) might provide an alternative therapy for selected neonates with severe and prolonged thrombocytopenia, and for infants and young children with different varieties of thrombocytopenia...
March 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/27928110/stable-liver-function-during-long-term-administration-of-eltrombopag-a-thrombopoietin-receptor-agonist-in-patients-with-chronic-liver-disease
#10
Tomohiro Kurokawa, Soichiro Murata, Nobuhiro Ohkohchi
Liver cirrhosis is the end stage of chronic liver disease, but no definitive pharmacological treatment is currently available. It has been reported that thrombopoietin (TPO) promotes liver regeneration and improves liver cirrhosis by increasing platelet count. We have shown the direct effect of platelet transfusion on the improvement of liver function in patients with chronic liver disease. However, platelet transfusion often causes adverse events, such as platelet transfusion refractoriness and pruritus. Therefore, we conducted an exploratory clinical trial and administered eltrombopag, an orally bioavailable, small-molecule, non-peptide TPO receptor agonist that has been approved for the treatment of chronic idiopathic thrombocytopenic purpura...
December 2016: Tohoku Journal of Experimental Medicine
https://www.readbyqxmd.com/read/27913466/nontransplant-therapy-for-bone-marrow-failure
#11
REVIEW
Danielle M Townsley, Thomas Winkler
Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27565278/cost-effectiveness-of-eltrombopag-versus-romiplostim-for-the-treatment-of-chronic-immune-thrombocytopenia-in-england-and-wales
#12
COMPARATIVE STUDY
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
https://www.readbyqxmd.com/read/27354722/characteristics-outcome-and-response-to-therapy-of-multirefractory-chronic-immune-thrombocytopenia
#13
COMPARATIVE STUDY
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
https://www.readbyqxmd.com/read/27312163/immunomodulation-and-immune-thrombocytopenia-some-unmet-needs-questions-and-outlook
#14
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
https://www.readbyqxmd.com/read/27312161/thrombopoietin-receptor-agonists-a-new-immune-modulatory-strategy-in-immune-thrombocytopenia
#15
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
https://www.readbyqxmd.com/read/27307776/long-term-safety-and-efficacy-of-romiplostim-for-treatment-of-immune-thrombocytopenia
#16
REVIEW
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
https://www.readbyqxmd.com/read/27046441/eltrombopag-after-allogeneic-haematopoietic-cell-transplantation-in-a-case-of-poor-graft-function-and-systematic-review-of-the-literature
#17
REVIEW
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
https://www.readbyqxmd.com/read/26364898/eltrombopag-for-treatment-of-thrombocytopenia-associated-disorders
#18
REVIEW
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
https://www.readbyqxmd.com/read/26322297/use-of-thrombopoietin-receptor-agonists-in-childhood-immune-thrombocytopenia
#19
REVIEW
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
https://www.readbyqxmd.com/read/26258668/efficacy-and-safety-of-eltrombopag-in-treatment-refractory-primary-immune-thrombocytopenia-a-retrospective-study
#20
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
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