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Pediatric ITP

Yue Li, Sixi Liu, Huihui Wang, Huirong Mai, Xiuli Yuan, Changgang Li, Xiaowen Chen, Feiqiu Wen
BACKGROUND: γ-Glutamyl hydrolase (GGH) regulates intracellular folates and antifolates such as methotrexate (MTX) for proper nucleotide biosynthesis and antifolate-induced cytotoxicity, respectively. In addition to genetic polymorphism and karyotypic abnormalities, methylation of CpG island 1 (CpG1) in the promoter region is found to modulate GGH activity by reducing GGH mRNA expression in acute lymphoblastic leukemia (ALL) cells. We aim to investigate methylation status of two CpG islands (CpG1 and CpG2) in the GGH promoter region in pediatric patients with ALL and acute myelogenous leukemia (AML)...
2017: PloS One
Sylwia Jasinski, Mark E Weinblatt, Chana L Glasser
BACKGROUND: Autoimmune cytopenias are characterized by immune-mediated destruction of hematopoietic cell lines with immune thrombocytopenia (ITP) affecting platelets and Evans syndrome (ES) affecting platelets and red blood cells. For patients with persistent disease, limited options for effective and well-tolerated therapies exist. OBJECTIVES: Our aim is to describe our institution's experience with sirolimus as therapy for pediatric patients with persistent ITP and ES...
March 6, 2017: Journal of Pediatric Hematology/oncology
Farida Hussein El-Rashedi, Mahmoud Ahmed El-Hawy, Mohamed Ahmed Helwa, Sameh Said Abd-Allah
OBJECTIVE/BACKGROUND: To assess the percentage of CD4(+), CD8(+), and natural killer cells (CD16(+), CD56(+)) in children with immune thrombocytopenic purpura (ITP) at presentation and study their impact on disease chronicity. METHODS: This case-control study was conducted at the Pediatric Hematology and Oncology Unit, Menoufia University Hospital (tertiary care center in Egypt). The study was held on 30 children presenting with ITP; they were followed-up and classified into two groups: 15 children with acute ITP; and 15 children with chronic ITP...
March 2017: Hematology/oncology and Stem Cell Therapy
S Kaba, G Keskindemirci, C Aydogmus, R Siraneci, F Erol Cipe
Intravenous immunoglobulin (IVIG) is commonly used in primary and secondary immunodeficiency diseases as well as autoimmune conditions as immunomodulatator treatment. Immediate adverse events which are generally mild and occur during infusion are seen in 6 hours. Reported immediate adverse events are in a wide range from 1%-40% in pediatric patients. 115 patients who received IVIG (except newborns) were included into this crosssectional study. IVIG was given to patients for primary immunodeficiencies (n=8), ITP (n=65), Kawasaki disease (n=11), secondary immunosupression (n=28), and passive immunization (n=3)...
January 2017: European Annals of Allergy and Clinical Immunology
Sarah Leonard, Lindsay M Anderson, Jude Jonassaint, Charles Jonassaint, Nirmish Shah
Iron chelation therapy can prevent iron overload for pediatric patients with sickle cell disease and β-thalassemia major; however, adherence is suboptimal. Therefore, we developed an intensive training program (ITP), to improve medication management and disease knowledge. The objectives were to determine feasibility of the ITP and its preliminary impact on adherence, disease knowledge, and health outcomes. Pediatric patients were recruited to participate in the ITP over a 90-day period and were followed for 6 months...
April 2017: Journal of Pediatric Hematology/oncology
Uri Hamiel, Iris Kventsel, Ilan Youngster
Immune thrombocytopenia (ITP) is an isolated autoimmune condition, often preceded by a viral infection. Vaccines, mainly the measles-mumps-rubella vaccine, have also been associated with an increased risk of developing the disease. Although some case reports of ITP after influenza immunization in adults have been published, epidemiologic studies examining the role of the influenza vaccine as a trigger of ITP have not conclusively proven causality. We report a child with 3 occurrences of ITP, each within 1 week of receiving the influenza trivalent inactivated vaccine...
December 2016: Pediatrics
Li-Juan Tang, Xiao-Heng Zhou, Qiong Bin, Xiao-Hua Huang, Jiang-Ming Luo
OBJECTIVE: To investigate the frequency distribution of human platelet antigen allele HPA-2 and HPA-15 among the pediatric patients with acute or chronic idiopathic thrombocytopenic purpura (ITP) in Chinese Guangxi area, and to explore the potential correlation of ITP with HPA-2 and HPA-15 gene polymorphisms. METHODS: The clinical and laboratorial data of 46 children diagnosed as acute ITP and 46 children diagnosed as chronic ITP between January 2007 and December 2014 were collected...
October 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
Michelle L Schoettler, Dionne Graham, Wen Tao, Margaret Stack, Elaine Shu, Lauren Kerr, Ellis J Neufeld, Rachael F Grace
An observational approach is recommended in newly diagnosed children with immune thrombocytopenia (ITP) at low risk of bleeding; however, there is no standard definition of risk. A standardized clinical assessment and management plan (SCAMP(®) ), a modifiable practice guideline, was implemented and revised (SCAMP-1 and SCAMP-2) and applied to 71 newly diagnosed patients with ITP. The Buchanan and Adix bleeding score guided treatment and was modified by stratifying by low- and high-risk grade 3 bleeding in SCAMP-2...
May 2017: Pediatric Blood & Cancer
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...
February 2017: 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) for pediatric patients with chronic immune thrombocytopenia (ITP). METHODS: Clinical and laboratory data for 46 Japanese patients aged <16 years who were diagnosed as having chronic ITP in 14 hospitals during 2001-2011, and were treated with CEP for >12 months, were analyzed. RESULTS: Median daily CEP dose was 1 mg/kg (range, 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...
January 1, 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
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