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pediatrics, thrombocytopenia

Ulkem Kocoglu Barlas, Hasan Serdar Kıhtır, Nilufer Goknar, Melike Ersoy, Nihal Akcay, Esra Sevketoglu
BACKGROUND: Hemolytic uremic syndrome (HUS) is a clinical syndrome characterized by hemolytic anemia, thrombocytopenia, and acute kidney injury. Atypical hemolytic uremic syndrome (aHUS) is a devastating disease with significant mortality and high risk of progression to end-stage kidney disease. It is mostly caused by dysregulation of the alternative complement pathway. Cobalamin C (Cbl C) defect is a genetic disorder of cobalamin metabolism and is a rare cause of HUS. CASE-DIAGNOSIS/TREATMENT: We present a 6-month-old male infant who was admitted to the pediatric intensive care unit (PICU) due to restlessness, severe hypertension, anemia, respiratory distress, and acute kidney injury...
March 20, 2018: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
Revathi Rajagopal, Marianne Phillips, Nicholas G Gottardo
The initial signs of hepatic sinusoidal obstruction syndrome (HSOS) can be challenging to recognize in children, especially outside the hematopoietic stem cell transplantation setting. To assist clinicians to promptly identify HSOS, the European Society for Blood and Marrow Transplantation has proposed pediatric HSOS diagnostic criteria which emphasize unexplained consumptive and transfusion-refractory thrombocytopenia. To highlight the importance of these "bellwether" early signs of HSOS and the efficacy of pre-emptive treatment with defibrotide, we describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment...
March 14, 2018: Pediatric Blood & Cancer
Mary B Wire, Xiaobin Li, Jianping Zhang, William Sallas, Vassilios Aslanis, Taoufik Ouatas
Our objective was to support initial eltrombopag doses and dose titration based on modeling and simulation of plasma exposure and platelet count response in pediatric patients aged 1-17 years with previously treated chronic immune thrombocytopenia enrolled in 2 clinical studies. Data from 168 pediatric patients were used to develop a life-span population pharmacokinetic and pharmacodynamic model including 3 pharmacokinetic and 4 pharmacodynamic compartments enabling simulation of platelet counts for various starting doses and dose titration schedules...
March 14, 2018: Clinical Pharmacology and Therapeutics
E Palmerini, R L Jones, E Setola, P Picci, E Marchesi, R Luksch, G Grignani, M Cesari, A Longhi, M E Abate, A Paioli, Z Szucs, L D'ambrosio, K Scotlandi, F Fagioli, S Asaftei, S Ferrari
BACKGROUND: Data on temozolomide (TEM) and irinotecan (IRI) activity in recurrent Ewing sarcoma (EWS), especially in adult patients, are limited. METHODS: Patients receiving TEM 100 mg/m2 /day oral, and IRI 40 mg/m2 /day intravenous, days 1-5, every 21 days, were included in this multi-institutional retrospective study. Disease control rate (DCR) [overall response rate (ORR) [complete response (CR) + partial response (PR)] + stable disease (SD)], 6-months progression-free survival (6-mos PFS) and 1-year overall survival (OS) were assessed...
March 13, 2018: Acta Oncologica
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
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
Emily Y Jen, Chia-Wen Ko, Jee Eun Lee, Pedro L Del Valle, Antonina Aydanian, Charles Jewell, Kelly J Norsworthy, Donna Przepiorka, Lei Nie, Jiang Liu, Christopher M Sheth, Marjorie Shapiro, Ann T Farrell, Richard Pazdur
On September 1, 2017, FDA granted approval for gemtuzumab ozogamicin (GO) (Mylotarg; Pfizer, Inc) in combination with daunorubicin and cytarabine (DA) and as a monotherapy for the treatment of adult patients with newly-diagnosed CD33-positive acute myeloid leukemia (AML). GO is a CD33-targeted antibody-drug conjugate joined to calicheamicin. Approval of GO combination treatment was based on a randomized trial of 271 patients with newly-diagnosed AML treated with DA with or without 3 mg/m2 fractionated GO, which resulted in an event-free survival (EFS) of 13...
February 23, 2018: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
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
Katherine L Zaleski, James A DiNardo, Viviane G Nasr
Bivalirudin (Angiomax; The Medicines Company, Parsippany, NJ), a direct thrombin inhibitor, has found increasing utilization as a heparin alternative in the pediatric population, most commonly for the treatment of thrombosis secondary to heparin-induced thrombocytopenia. Due to the relative rarity of heparin-induced thrombocytopenia as well as the lack of Food and Drug Administration-approved indications in this age group, much of what is known regarding the pharmacokinetics and pharmacodynamics of bivalirudin in this population has been extrapolated from adult data...
February 14, 2018: Anesthesia and Analgesia
Suzanne S Puentes, Michele Dunstan
Escherichia coli is a bacterium that is an important part of the intestinal tract; however, it has the potential to become pathogenic. Shiga toxin-producing E coli (STEC) is a leading cause of E coli infections and has led to outbreaks in North America. Transmission is through ingestion of contaminated food sources, and via infected humans and animals. Young children infected with STEC are at high risk for developing hemolytic uremic syndrome (HUS). HUS is a clinical syndrome characterized by macroangiopathic hemolytic anemia, thrombocytopenia, and acute renal failure...
March 2018: Critical Care Nursing Clinics of North America
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
Cristina Oana Mărginean, Lorena Elena Meliţ, Minodora Dobreanu, Maria Oana Mărginean
RATIONALE: Hypertriglyceridemia is defined as a level of triglycerides above 150 mg/dL. The complex causes and classification of hypertriglyceridemia lead to difficulties in the diagnosis and management of this condition. PATIENT CONCERNS: We present the case of a 15 years and 6 months old female teenager, admitted in our clinic for the following complaints: severe abdominal pain predominantly in the lateral left quadrant, nausea, vomiting, and the lack of stools for 2 days...
December 2017: Medicine (Baltimore)
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...
January 12, 2018: Journal of Pediatric Hematology/oncology
Sidharth Totadri, Amita Trehan, Deepak Bansal, Richa Jain
Context: Survival rates exceed 90% in Wilms' tumor (WT). Actinomycin-D (ACT-D) which is indispensable in the management of WT is associated with the development of sinusoidal obstruction syndrome (SOS), a potentially fatal complication. Aims: The aim is to study the presentation, management, and outcome of SOS complicating ACT-D administration in WT. Settings and Design: Retrospective file review conducted in a Pediatric Hematology-Oncology unit...
October 2017: Indian Journal of Medical and Paediatric Oncology
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, 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
Caroline Besnard, Eva Levy, Nathalie Aladjidi, Marie-Claude Stolzenberg, Aude Magerus-Chatinet, Olivier Alibeu, Patrick Nitschke, Stéphane Blanche, Olivier Hermine, Eric Jeziorski, Judith Landman-Parker, Guy Leverger, Nizar Mahlaoui, Gérard Michel, Isabelle Pellier, Felipe Suarez, Isabelle Thuret, Geneviève de Saint-Basile, Capucine Picard, Alain Fischer, Bénédicte Neven, Frédéric Rieux-Laucat, Pierre Quartier
Evans syndrome (ES) is defined by the combination of autoimmune hemolytic anemia and immune thrombocytopenia. Clinical presentation includes manifestations of immune dysregulation, found in primary immune deficiencies, autoimmune lymphoproliferative syndrome with FAS (ALPS-FAS), Cytotoxic T Lymphocyte Antigen-4 (CTLA-4) and Lipopolysaccharide-Responsive vesicle trafficking Beige-like and Anchor protein (LRBA) defects. We report the clinical history and genetic results of 18 children with ES after excluding ALPS-FAS...
January 10, 2018: Clinical Immunology: the Official Journal of the Clinical Immunology Society
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)
Roya Sherkat, Mohammad Reza Sabri, Bahar Dehghan, Hamid Bigdelian, Nahid Reisi, Nooshin Afsharmoghadam, Hamid Rahimi, Narges Rahmanian, Cristoph Klein
RATIONALE: Primary cardiac lymphoma (PLC) is an extremely uncommon malignancy. PCL is more common in secondary immunodeficient patients. In this report, we describe a unique case of PLC who had been diagnosed as a STK4 deficient patient. This case is the first Primary immunodeficiency (PID) patient developing PCL in the world. PATIENT CONCERNS: An eleven-year-old girl, a known case of PID, was referred to the pediatric cardiology department because of chest pain and dyspnea...
December 2017: Medicine (Baltimore)
Y S Sun, I T Sun, H K Wang, A H Yang, C Y Tsai, C J Huang, D F Huang, C C Lai
Objective The objective of this paper is to identify the risk of complications of real-time ultrasound-guided renal biopsy in adult and pediatric patients with systemic lupus erythematosus (SLE). Materials and methods This retrospective study examined outcomes of 296 renal biopsy procedures in 275 SLE patients. Imaging-confirmed symptomatic hematoma was regarded as a major complication when intervention (blood transfusion, angiographic embolization, or surgery) was required or as a minor complication otherwise...
January 1, 2018: Lupus
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
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