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Pediatric ITP

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https://www.readbyqxmd.com/read/29659042/physician-decision-making-in-selection-of-second-line-treatments-in-immune-thrombocytopenia-in-children
#1
Rachael F Grace, Jenny M Despotovic, Carolyn M Bennett, James B Bussel, Michelle Neier, Cindy Neunert, Shelley E Crary, Yves D Pastore, Robert J Klaassen, Jennifer A Rothman, Kerry Hege, Vicky R Breakey, Melissa J Rose, Kristin A Shimano, George R Buchanan, Amy Geddis, Kristina M Haley, Adonis Lorenzana, Alexis Thompson, Michael Jeng, Ellis J Neufeld, Travis Brown, Peter W Forbes, Michele P Lambert
Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second-line treatments for ITP which examined treatment decisions...
April 16, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29589486/advances-in-chemical-pharmacotherapy-for-the-treatment-of-pediatric-immune-thrombocytopenia
#2
Thomas Kühne
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder of heterogeneous pathophysiological mechanisms. Treatment endpoints include elevation of platelets and reduction of bleeding risk, elevation of quality of life, reduction of concomitant therapies and prevention from bleeding. Persistent and chronic ITP is more common in adults but occurs in children. Standard therapies include corticosteroids and immunoglobulins, both associated with side effects. There are new treatments, such as thrombopoietin-receptor agonists and promising investigational drugs...
March 28, 2018: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/29516627/a-comparative-prospective-observational-study-of-children-and-adults-with-immune-thrombocytopenia-2-year-follow-up
#3
Alexandra Schifferli, Andreas Holbro, Meera Chitlur, Michael Coslovsky, Paul Imbach, Hugo Donato, Mohsen Elalfy, Elena Graciela, John Grainger, Susanne Holzhauer, Cecilia Riccheri, Francesco Rodeghiero, Marco Ruggeri, Hannah Tamary, Tatjana Uglova, Runhui Wu, Thomas Kühne
Comparative clinical studies of children and adults with immune thrombocytopenia (ITP) are poorly covered in the literature. However, the accepted classification of ITP -childhood ITP and adult ITP-results in considerable differences in treatment protocols and practice guidelines. The analysis of the Pediatric and Adult Registry on Chronic ITP (PARC-ITP) of patients at first presentation demonstrated fewer differences in clinical and laboratory findings at initial diagnosis between children and adults than expected...
March 8, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29487060/eltrombopag-for-use-in-children-with-immune-thrombocytopenia
#4
REVIEW
Taylor Olmsted Kim, Jenny Despotovic, Michele P Lambert
Eltrombopag is currently the only US Food and Drug Administration-approved thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown favorable efficacy and adverse effect profiles in children. Two multicenter, double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body]) demonstrated efficacy in raising platelet counts, reducing bleeding, and reducing the need for concomitant ITP therapies with relatively few adverse effects...
February 27, 2018: Blood Advances
https://www.readbyqxmd.com/read/29471711/enhancing-autonomy-in-biobank-decisions-too-much-of-a-good-thing
#5
Phoebe B Mitchell, Sonja I Ziniel, Sarah K Savage, Kurt D Christensen, Elissa R Weitzman, Robert C Green, Noelle L Huntington, Debra J Mathews, Ingrid A Holm
The opportunity to receive individual research results (IRRs) in accordance with personal preferences may incentivize biobank participation and maximize perceived benefit. This trial investigated the relationship between parents' preferences and intent to participate (ITP) in biobank research utilizing their child's genetic information. We randomized parents of pediatric patients to four hypothetical biobanks, one of which employed a preference-setting model for return of results regarding their child. ITP was highest among those desiring all types of IRRs (93...
January 1, 2018: Journal of Empirical Research on Human Research Ethics: JERHRE
https://www.readbyqxmd.com/read/29467954/efficacy-and-safety-of-thrombopoietin-receptor-agonists-in-children-with-chronic-immune-thrombocytopenia-a-meta-analysis
#6
Jian-Chun Guo, Yi Zheng, Hai-Tao Chen, Haixia Zhou, Xian-Hui Huang, Li-Ping Zhong, Huai-Bin Zhou, Yu Huang, Dan-Li Xie, Yong-Liang Lou
Background and Aim: Thrombopoietin receptor agonists (TPO-RAs) have been shown to be safe and effective for adults with chronic immune thrombocytopenia (ITP). The aim of this meta-analysis is to assess the efficacy and safety of thrombopoietin receptor agonists for children with chronic ITP. Materials and Methods: Clinical randomized controlled trials (RCTs) evaluating the efficacy and safety of TPO-RAs in pediatric ITP patients published up to June 2017 were retrieved from PubMed, Cochrane Library, and Embase databases...
January 23, 2018: Oncotarget
https://www.readbyqxmd.com/read/29407001/subacute-sclerosing-panencephalitis-and-immune-thrombocytopenia-more-than-a-coincidence
#7
I Oncel, S Saltik, B Anlar
Subacute sclerosing panencephalitis (SSPE) is a progressive disease caused by persistent measles virus (MV). It has an incidence of 0.4-2.0/million in Turkey. Immune thrombocytopenia (ITP) is a bleeding disorder whose estimated incidence is 4.2/100.000 person/years in the pediatric age group. We observed three cases with ITP in our cohort of 315 pediatric SSPE cases, an incidence higher than coincidentally expected in the general population. We hypothesize an association between SSPE and ITP. Our three cases had measles 1-2 years before the onset of ITP and 8-10 years before first symptoms of SSPE...
February 2018: Medical Hypotheses
https://www.readbyqxmd.com/read/29334536/nationwide-trend-analysis-of-pediatric-inpatients-with-immune-thrombocytopenia-in-the-united-states
#8
Yusuke Okubo, Atsuhiko Handa
BACKGROUND: Several studies have reported the epidemiology of immune thrombocytopenia (ITP) among children in the United States and other countries. However, recent trends in ITP among hospitalized children and hospital course remain unknown at a national level in the United States. METHOD: Hospital discharge records of patients with ITP aged 19 years and younger were obtained for the years 2003, 2006, 2009, and 2012 using the Kids' Inpatient Database. Data were weighted to estimate the annual hospitalization rates in the United States with trend analyses...
April 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29330464/eltrombopag-versus-romiplostim-in-treatment-of-children-with-persistent-or-chronic-immune-thrombocytopenia-a-systematic-review-incorporating-an-indirect-comparison-meta-analysis
#9
Jiaxing Zhang, Yi Liang, Yuan Ai, Xiaosi Li, Juan Xie, Youping Li, Wenyi Zheng, Rui He
In absence of direct comparison, we conducted an indirect-comparison meta-analysis to evaluate the efficacy and safety of thrombopoietin-receptor agonists(TPO-RAs) in treatment of pediatric persistent or chronic immune thrombocytopenia(ITP). PubMed, Embase, Cochrane Library, Clinical Trials.gov, China National Knowledge Infrastructure, and Chinese Biomedical Literature Database were searched from their earliest records to May 2017. Randomized controlled trials comparing the TPO-RAs with placebo in pediatric ITP were included...
January 12, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29313460/thrombopoietin-and-its-receptor-expression-in-pediatric-patients-with-chronic-immune-thrombocytopenia
#10
Shanshan Li, Jingbo Shao, Min Xia, Na Zhang, Jingwei Yang, Hong Li, Hui Jiang
OBJECTIVES: Chronic immune thrombocytopenia (cITP) is common in children. However, the pathogenesis has not been fully elucidated. This study aimed to determine whether thrombopoietin (TPO) and its receptor c-mannosylation of the TPO receptor (c-Mpl) have an impact on childhood cITP. METHODS: Sixty-four patients with newly diagnosed ITP (nITP), 64 patients with persistent ITP, 80 patients with cITP, and 64 healthy children (control) were enrolled in this study. Plasma TPO was measured with an ELISA, and c-Mpl was determined by flow cytometry...
January 9, 2018: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/29296878/management-of-newly-diagnosed-immune-thrombocytopenia-can-we-change-outcomes
#11
REVIEW
Cindy E Neunert
Immune thrombocytopenia resulting from antibody-mediated platelet destruction combined with impaired platelet production is a common cause of thrombocytopenia. The decision to treat newly diagnosed patients is based on several factors including ceasing hemorrhagic manifestations, increasing the platelet count, prevention of bleeding, and inducing remission. Current standard first-line therapy is a course of corticosteroids. Although this treatment paradigm increases the platelet count in the majority of patients, a high percentage relapse after discontinuation of corticosteroid therapy...
November 14, 2017: Blood Advances
https://www.readbyqxmd.com/read/29222284/management-of-newly-diagnosed-immune-thrombocytopenia-can-we-change-outcomes
#12
REVIEW
Cindy E Neunert
Immune thrombocytopenia resulting from antibody-mediated platelet destruction combined with impaired platelet production is a common cause of thrombocytopenia. The decision to treat newly diagnosed patients is based on several factors including ceasing hemorrhagic manifestations, increasing the platelet count, prevention of bleeding, and inducing remission. Current standard first-line therapy is a course of corticosteroids. Although this treatment paradigm increases the platelet count in the majority of patients, a high percentage relapse after discontinuation of corticosteroid therapy...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29173312/treatment-of-children-with-persistent-and-chronic-idiopathic-thrombocytopenic-purpura-4-infusions-of-rituximab-and-three-4-day-cycles-of-dexamethasone
#13
Joseph H Oved, Christina S Y Lee, James B Bussel
OBJECTIVES: To assess initial and long-term outcome of children with persistent/chronic idiopathic thrombocytopenic purpura (ITP) treated with 4 infusions of rituximab and three 4-day cycles of dexamethasone (4R+3Dex) including cohorts with most benefit and/or treatment associated toxicity. STUDY DESIGN: All pediatric patients with ITP at Weill-Cornell who received 4R+3Dex were included in this retrospective study. Duration was median time from first rituximab infusion to treatment failure...
December 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/29068869/dural-venous-sinus-thrombosis-and-pulmonary-embolism-following-immunoglobulin-treatment-in-pediatric-patient-with-immune-thrombocytopenic-purpura
#14
Kyung Mi Park, Eu Jeen Yang, Young Tak Lim
Intravenous immunoglobulin (IVIG) is a widely used agent as the first choice of treatment of immune thrombocytopenic purpura (ITP). IVIG has several side effects, but it is a relatively safe treatment. Life-threatening thrombosis has been reported in adults and rarely in children. We report a case of a 14-year-old boy with dural venous sinus thrombosis and pulmonary embolism after treatment with IVIG for ITP. The patient was treated with low-molecular-weight heparin followed by warfarin and the symptoms were recovered...
November 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29067848/distinct-clinical-correlates-of-immune-thrombocytopenic-purpura-at-diagnosis-of-childhood-onset-and-adult-sle
#15
Gladys Cherres Xavier Esteves, Natali Weniger Spelling Gormezano, Oriany L Pereira, David Kern, Clovis Almeida Silva, Rosa Maria Rodrigues Pereira, Katia Tomie Kozu, Eloisa Bonfá, Nadia Emi Aikawa
OBJECTIVES: To compare clinical and laboratorial features between childhood-onset systemic lupus erythematosus (cSLE) and adult SLE (aSLE) at concomitant diagnosis of immune thrombocytopenic purpura (ITP). METHODS: This study evaluated 56 cSLE and 73 aSLE patients regularly followed at Pediatric and Rheumatology Divisions of the same University hospital with ITP (platelets count <100,000/mm3 in the absence of other causes) at lupus onset. RESULTS: Median current age was 11...
October 25, 2017: Modern Rheumatology
https://www.readbyqxmd.com/read/29020887/the-potential-association-of-tumor-necrosis-factor-%C3%AE-eta-252-g-a-cytokine-gene-polymorphism-with-immune-thrombocytopenic-purpura-among-egyptian-children
#16
Dalia Saber Morgan, Rasha Abdel-RaoufAbdel-Aziz Afifi, Shereen Mohamed El-Hoseiny, Dalia Gamil Amin, Sara Yahia Gaber Ibrahim
OBJECTIVES: The main objective of this study was to study tumor necrosis factor beta (TNFB) + 252G/A gene polymorphism, known to be related to autoimmunity, in immune thrombocytopenia (ITP) patients. We also aimed to investigate the association between TNFB + 252G/A polymorphism and susceptibility to develop persistent/chronic ITP. METHODS: One hundred pediatric ITP patients, as well as 50 age- and sex-matched healthy Egyptian subjects, were included. Genotyping of TNF-β gene (G252A) was done using the PCR-RFLP method...
October 12, 2017: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/28974109/clinical-significance-of-t-cell-immunoglobulin-mucin-3-expression-on-peripheral-blood-mononuclear-cells-in-pediatric-acute-immune-thrombocytopenia
#17
Asmaa M Zahran, Mervat A M Youssef, Khalid I Elsayh, Mustafa M Embaby, Ahmad I M Ibrahim
T-cell immunoglobulin mucin 3 (TIM-3) is a transmembrane protein that plays an important role in several autoimmune diseases. The relationship between TIM-3 and excessive immune responses in immune thrombocytopenia (ITP) is still unknown. In this study, we evaluated the relationship between the expression of TIM-3 on peripheral blood mononuclear cells in patients with ITP and the disease severity. The frequency of lymphocyte and monocyte subsets and their TIM-3 expression were evaluated in patients with acute ITP (n = 45) and in healthy control (n = 20) using flow cytometry...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/28971174/investigation-of-celiac-disease-followed-by-immune-thrombocytopenic-purpura-diagnosis-in-patients-and-comparison-with-literature
#18
Hakan Sarbay, Halil Kocamaz, Mehmet Akin, Bayram Ozhan
OBJECTIVE: Celiac disease (CD) and Immune thrombocytopenic purpura (ITP) may occur together as a result of similar autoimmune mechanisms. The aim of this study was to assess the frequency of CD in a group of ITP patients and in the literature. METHODS: A total of 29 patients in Pamukkale University Faculty of Medicine Hospital Pediatric Hematology and Oncology Department with ITP were included in the study. Test was performed for the antibodies related to CD. Positive result for celiac antibodies was confirmed with biopsy...
2017: Northern Clinics of Istanbul
https://www.readbyqxmd.com/read/28921855/utility-of-the-immature-platelet-fraction-in-pediatric-immune-thrombocytopenia-differentiating-from-bone-marrow-failure-and-predicting-bleeding-risk
#19
Alicia McDonnell, Karen L Bride, Derick Lim, Michele Paessler, Char M Witmer, Michele P Lambert
BACKGROUND: Differentiating childhood immune thrombocytopenia (ITP) from other cause of thrombocytopenia remains a diagnosis of exclusion. Additionally factors that predict bleeding risk for those patients with ITP are currently not well understood. Previous small studies have suggested that immature platelet fraction (IPF) may differentiate ITP from other causes of thrombocytopenia and in combination with other factors may predict bleeding risk. METHODS: We performed a retrospective chart review of thrombocytopenic patients with an IPF measured between November 1, 2013 and July 1, 2015...
February 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28845713/thrombopoietin-receptor-agonists-for-children-with-immune-thrombocytopenia-a-systematic-review
#20
REVIEW
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
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