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Pediatric ITP

Thomas Kühne
Evidence-based medicine is growing in immune thrombocytopenia (ITP), but solid clinical data are still lacking in many areas. A majority of children has self-limited ITP, but chronic symptomatic ITP exists also in pediatrics. Management includes a watch-and-wait strategy for children with newly diagnosed ITP and no or mild bleeding, and immunoglobulins and corticosteroids, if more bleeding and mucous membrane involvement is present. Treatment endpoints differ in clinical research and in clinical practice. The requirement of platelet enhancing drugs needs to be better defined in guidelines...
October 4, 2016: Hämostaseologie
Xin Yao, Chengrong Li, Jun Yang, Guobing Wang, Changgang Li, Yu Xia
OBJECTIVE: This study aims to investigate the role of T follicular helper (TFH) cells in the immunopathogenesis of pediatric immune thrombocytopenia (ITP), as well as differences in TFH expansion and its regulation between newly diagnosed ITP (nITP) and chronic pediatric ITP (cITP). METHODS: Eighty-five children with ITP and 20 age-matched healthy controls were enrolled into this study. TFH cell frequencies and TFH cell-associated regulatory factors before and after treatment were analyzed by flow cytometry, RT-PCR and ELISA...
October 2016: Blood Cells, Molecules & Diseases
Arun Panigrahi, Amy Clark, John Myers, Ashok Raj
BACKGROUND: Successful treatment of both pediatric autoimmune hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP), specifically those that are refractory to first-line therapies, remains unsatisfactory in terms of long-term remission and medication side effects. Here, we propose a novel combination therapy of mycophenolate mofetil (MMF), an adjunct immunosuppressive, and short-term corticosteroids for the treatment of persistent or chronic autoimmune cytopenias in children...
September 12, 2016: Pediatric Blood & Cancer
Taro Yamazaki, Atsushi Shibuya, Saori Ishii, Nobuyuki Miura, Akira Ohtake, Nozomu Sasaki, Ryuichiro Araki, Yatio Ota, Mitsuhiro Fujiwara, Yuji Miyajima, Kimiaki Uetake, Keigo Hamahata, Koji Kato, Kiyoshi Kawakami, Hidemi Toyoda, Naohiko Moriguchi, Masahiko Okada, Masanori Nishi, Yoshiyasu Ogata, Tomohito Takimoto, Shouichi Ohga, Shigeru Ohta, Shin Amemiya
BACKGROUND: A nationwide, multicenter and observational study was retrospectively conducted to evaluate the clinical utility of cepharanthin (CEP) therapy for pediatric patients with chronic immune thrombocytopenia (ITP). METHODS: Clinical and laboratory data of 46 Japanese patients less than 16 years of age were analyzed, who were diagnosed as having chronic ITP in 14 hospitals during the 2001-2011 decade, and were treated with CEP for more than 12 months. RESULTS: The median daily dose of CEP was 1 mg/kg (0...
September 6, 2016: Pediatrics International: Official Journal of the Japan Pediatric Society
Heba M Gouda, Nermine M Kamel, Safa S Meshaal
BACKGROUND: DNA methylation is an epigenetic process that refers to chromatin-based mechanisms in the regulation of gene expression without DNA alternation. It is mediated by DNA methyltransferases (DNMTs). The DNA methyltransferase 3B (DNMT3B) gene contains a C-to-T single nucleotide polymorphism (SNP; rs2424913) in the Promotor region, 149 base pairs from the transcription start site, which is reported to significantly increase the Promotor activity. OBJECTIVE: To investigate the prevalance of rs2424913 single nucleotide polymorphism located in the DNMT3B gene Promotor...
November 2016: Laboratory Medicine
Joanne Yacobovich, Sabreen Abu-Ahmed, Orna Steinberg-Shemer, Tracie Goldberg, Miriam Cohen, Hannah Tamary
The purpose of this study was to assess the efficacy and side effect profile of the repeated use of anti-D for the treatment of pediatric immune thrombocytopenia (ITP) in a large pediatric hematology center. We performed a retrospective analysis of patient records for children (aged 4 months-18 years) treated for ITP at Schneider Children's Medical Center of Israel from 1995-2015. Demographic and clinical data, reported adverse events, and therapy response were extracted from written and electronic files for all patients having received anti-D...
April 2016: Seminars in Hematology
J Sipurzynski, B Fahrner, R Kerbl, R Crazzolara, N Jones, G Ebetsberger, B Jauk, V Strenger, B Wohlmuther, W Schwinger, H Lackner, C Urban, W Holter, M Minkov, L Kager, M Benesch, M G Seidel
Chronic immune thrombocytopenia (cITP) is often associated with an underlying predisposition towards autoimmunity, recognition of which is relevant to guide treatment. International recommendations on diagnostic steps and therapeutic measures of cITP in childhood exist. However, due to the low prevalence (1-2/100,000) and a variation of availability of immunological and hematological tests and treatments across pediatric units, we postulated that these guidelines are not uniformly adhered to and that immune dysregulation syndromes remained undiscovered...
April 2016: Seminars in Hematology
Barina Aqil, Jyotinder N Punia, Choladda V Curry, Andrea N Marcogliese, M Tarek Elghetany
BACKGROUND: Micromegakaryocytes (microMKs) are considered the most reliable dysplastic feature for myelodysplastic syndrome (MDS), particularly refractory cytopenia of childhood (RCC); there is no minimal threshold for the diagnosis of RCC. Since most RCC patients present with thrombocytopenia, the presence of microMKs should raise concern for MDS/RCC. This study attempted to investigate the prevalence of microMKs and associated marrow fibrosis in patients with thrombocytopenia unrelated to MDS/RCC and the need for establishing a threshold for microMKs for the diagnosis of MDS/RCC...
August 2016: Leukemia Research
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
Aniruddha Dayama, Jasmita Dass, Manoranjan Mahapatra, Renu Saxena
INTRODUCTION: Antiphospholipid antibodies (APLAs) have been variably reported in 14% to 75% of patients with immune thrombocytopenia (ITP). There is lack of Indian data on incidence of APLA in ITP. OBJECTIVE: We studied the incidence of APLA in patients with pediatric and adult Indian ITP. MATERIALS AND METHODS: We prospectively studied 100 patients including acute (n = 37), persistent (n = 13), and chronic (n = 50) ITP. Male to female ratio was 1...
April 10, 2016: Clinical and Applied Thrombosis/hemostasis
Galila M Mokhtar, Nagham M S El-Beblawy, Amira A Adly, Nancy S Elbarbary, Tarek M Kamal, Esraa M Hasan
To evaluate the association between development, progression, and response to therapy among patients with immune thrombocytopenia (ITP) and different cytokine gene polymorphisms known to be related to autoimmunity [tumor necrosis factor (TNF)-alpha, interleukin (IL)-10, IL-6, IL-17, IL-1Ra]. A total of 50 pediatric patients with ITP (20 newly diagnosed, 30 chronic) and 50 healthy controls were investigated via PCR-restriction fragment length polymorphism analysis for cytokine gene polymorphism. Compared with controls, all patients showed a higher frequency of IL-6-174 CC [P = 0...
April 2016: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
Heng Zhang, Li Wang, Meijie Quan, Jie Huang, Peng Wu, Qin Lu, Yongjun Fang
BACKGROUND: The concept of health-related quality of life (HRQoL) was brought up decades ago and has been well utilized in many different areas. Regarding immune thrombocytopenia (ITP) management, much work has been done to emphasize the necessity of taking HRQoL into consideration. However, data on HRQoL of children with chronic ITP remain rare. METHODS: This is a cross-sectional study. Children with chronic ITP aged from 2 to 18 and their parents were recruited...
2016: Health and Quality of Life Outcomes
Michael D Tarantino, Mark Danese, Robert J Klaassen, Jennifer Duryea, Melissa Eisen, James Bussel
To examine utilization and outcomes in pediatric immune thrombocytopenia (ITP) hospitalizations, we used ICD-9 code 287.31 to identify hospitalizations in patients with ITP in the 2009 HCUP KID, an all-payer sample of pediatric hospitalizations from US community hospitals. Diagnosis and procedure codes were used to estimate rates of ITP-related procedures, comorbidity prevalence, costs, length of stay (LOS), and mortality. In 2009, there were an estimated 4499 hospitalizations in children aged 6 months-17 years with ITP; 43% in children aged 1-5 years; and 47% with emergency department encounters...
July 2016: Platelets
Char M Witmer, Michele P Lambert, Sarah H O'Brien, Cindy Neunert
BACKGROUND: Recent pediatric immune thrombocytopenia (ITP) guidelines have significantly altered and are encouraging an observational approach for patients without significant bleeding regardless of their platelet count. PROCEDURE: This retrospective multicenter cohort study utilized the Pediatric Health Information Systems (PHIS) administrative database. Subjects were 6 months to 18 years of age, admitted to a PHIS hospital between January 1, 2008 and September 30, 2014, with a primary diagnosis code for ITP...
July 2016: Pediatric Blood & Cancer
Xiao-Li Wang, Ai-Min Li
Treatment of refractory immune thrombocytopenia (ITP) continues to challenge medical practitioners because of the lack of well tolerated and effective drugs. Eltrombopag is a second-generation thrombopoietin approved to treat adult chronic/refractory ITP in 2008. The use of eltrombopag to treat pediatric refractory ITP remains in the clinical trial stage, and no reports on its use in China are yet available. In this article, we describe a pediatric case of refractory ITP that was successfully treated with eltrombopag...
December 8, 2015: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
Paola Giordano, Simona Cascioli, Giuseppe Lassandro, Valentina Marcellini, Fabio Cardinale, Federica Valente, Franco Locatelli, Rita Carsetti
BACKGROUND: Immune thrombocytopenic purpura (ITP) is characterized by reduced platelet count secondary to immune-mediated destruction, this results in an increased bleeding risk. Autoantibodies binding to platelets tag them for premature destruction in the spleen. For this reason, splenectomy is often performed as treatment of chronic forms of disease that are resistant to pharmacological therapy. METHODS: We studied 30 patients with ITP and compared them with age-matched controls...
February 2016: Pediatric Research
Nathalie Aladjidi, Helder Fernandes, Thierry Leblanc, Amélie Vareliette, Frédéric Rieux-Laucat, Yves Bertrand, Hervé Chambost, Marlène Pasquet, Françoise Mazingue, Corinne Guitton, Isabelle Pellier, Françoise Roqueplan-Bellmann, Corinne Armari-Alla, Caroline Thomas, Aude Marie-Cardine, Odile Lejars, Fanny Fouyssac, Sophie Bayart, Patrick Lutz, Christophe Piguet, Eric Jeziorski, Pierre Rohrlich, Philippe Lemoine, Damien Bodet, Catherine Paillard, Gérard Couillault, Frédéric Millot, Alain Fischer, Yves Pérel, Guy Leverger
Evans syndrome (ES) is a rare autoimmune disorder whose long-term outcome is not well known. In France, a collaborative pediatric network set up via the National Rare Disease Plan now provides comprehensive clinical data in children with this disease. Patients aged less than 18 years at the initial presentation of autoimmune cytopenia have been prospectively included into a national observational cohort since 2004. The definition of ES was restricted to the simultaneous or sequential association of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP)...
2015: Frontiers in Pediatrics
Ming'en Lyu, Yang Li, Feng Xue, Xiaofan Liu, Wenjie Liu, Tiantian Sun, Cuicui Lyu, Rongfeng Fu, Lei Zhang, Renchi Yang
OBJECTIVE: To explore the clinical value of immature platelet fraction (IPF), absolute immature platelet fraction (A- IPF) and thrombelastograph (TEG) on assessment of bleeding risk of immune thrombocytopenia (ITP). METHODS: two hundred and seventy- one patients with ITP were assessed based on ITP-BAT bleeding grading system. IPF, A-IPF were determined in 271 patients ,TEG in 125 patients. The correlations between bleeding grades and IPF, A-IPF, variables of TEG in subgroups were analyzed by statistical method...
September 2015: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
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
Brian LaMoreaux, Fatima Barbar-Smiley, Stacy Ardoin, Hareth Madhoun
INTRODUCTION: Romiplostim is a thrombopeitin-receptor agonist approved for raising platelet counts in patients with immune thrombocytopenia (ITP). Several hematologic adverse effects have been reported including acute myeloid leukemia, myelofibrosis, and thrombosis. METHODS: We report two cases, one pediatric and one adult patient, who had antiphospholipid antibodies and received romiplostim for ITP. Additionally, we conducted medline, Food and Drug Administration (FDA) Adverse Events reports website, and manufacturer's adverse events database...
February 2016: Seminars in Arthritis and Rheumatism
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