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Paediatric Haematology

S-M Wang, W-X Zeng, W-S Wu, L-L Sun, D Yan
WHAT IS KNOWN AND OBJECTIVE: Thymidylate synthase (TYMS) is an important target for methotrexate (MTX). Genetic variations in the TYMS gene contribute to the differences in treatment responses to MTX. The aim of this study was to investigate the distribution of a microRNA (miRNA) binding site polymorphism (rs2790 A > G) in the 3'-untranslated region (3'-UTR) of TYMS and its association with MTX concentration and haematological toxicity in Chinese paediatric patients with acute lymphoblastic leukaemia (ALL)...
March 3, 2018: Journal of Clinical Pharmacy and Therapeutics
Melina Heinemann, Andreas Ranft, Thorsten Langer, Herbert Jürgens, Justus Kreyer, Volker Vieth, Michael Schäfers, Matthias Weckesser, Thorsten Simon, Wolf Hassenpflug, Selim Corbacioglu, Stefan Bielack, Regina Mayer-Steinacker, Thomas Kühne, Henk van den Berg, Hans Gelderblom, Sebastian Bauer, Lars Stegger, Uta Dirksen
BACKGROUND: The Cooperative Ewing Sarcoma Study and the Late Effects Surveillance System of the Society for Paediatric Oncology and Haematology recommend a structured follow-up imaging protocol (FUIP) for patients with Ewing sarcoma (EwS) with decreasing frequency of imaging over the first 5 years. The present study aims to assess the effectiveness of the FUIP for EwS patients regarding survival after relapse. PATIENTS AND METHODS: A retrospective multicenter analysis on 160 eligible patients with EwS recurrence was performed...
February 26, 2018: Pediatric Blood & Cancer
Ari Zimran, Michael Wajnrajch, Betina Hernandez, Gregory M Pastores
Taliglucerase alfa is an enzyme replacement therapy (ERT) approved for treatment of adult and paediatric patients with Type 1 Gaucher disease (GD) in several countries and the first plant cell-expressed recombinant therapeutic protein approved by the US Food and Drug Administration for humans. Here, we review the findings across six key taliglucerase alfa clinical studies. A total of 33 treatment-naïve adult patients were randomized to taliglucerase alfa 30 U/kg or 60 U/kg in a 9-month, multicentre, randomized, double-blind, parallel-group, dose-comparison pivotal study, after which eligible patients continued into two consecutive extension studies; 17 treatment-naïve adult patients completed 5 total years of treatment with taliglucerase alfa...
February 23, 2018: Orphanet Journal of Rare Diseases
J Guitard, F Isnard, M-D Tabone, M Antignac, E Brissot, Y Senghor, A Petit, G Leverger, C Hennequin
OBJECTIVES: Definitive diagnosis of invasive candidiasis (IC) may be difficult to achieve in patients with haematological malignancy (PHM). We aimed to evaluate the performance of BDG for the diagnosis and the follow-up of IC in PHM. PATIENTS AND METHODS: We retrospectively reviewed the serological data of BDG assay in adult and paediatric PHM, who developed candidemia or chronic disseminated candidiasis (CDC) through a 4-year period. Sensitivity and kinetics of BDG were determined for both clinical forms...
February 9, 2018: Journal of Infection
G T Chua, J S Rosa Duque, P C Y Chong, P P W Lee, Y L Lau, M H K Ho
DRESS (drug reaction with eosinophilia and systemic symptoms) is a rare but potentially life-threatening disorder characterized by fever, skin eruption, haematological abnormalities and multi-organ dysfunction after drug exposure. The pathophysiology is thought to be related to interactions between culprit drugs, viral reactivation and T-lymphocytes activation. We report 4 paediatric patients with DRESS who were treated at our centre over the past 12 years. Most cases improved after corticosteroids. Other immunosuppressive medications were attempted in refractory cases with varied outcomes...
November 30, 2017: European Annals of Allergy and Clinical Immunology
Holly Lindsay, Saleh Bhar, Challice Bonifant, Sarah Sartain, Sarah B Whittle, Youngna Lee-Kim, Mona D Shah
Paediatric haematology, oncology and bone marrow transplant (BMT) patients frequently require transfusion of blood products. Our institution required a new transfusion consent be obtained every admission. The objectives of this project were to: revise inpatient blood products consent form to be valid for 1 year, decrease provider time spent consenting from 15 to <5 min per admission, and improve provider frustration with the consent process. Over 6 months, we determined the average number of hospitalisations requiring transfusions in a random sampling of haematology/oncology/BMT inpatients...
2018: BMJ Open Quality
A Juan Ribelles, P Berlanga, G Schreier, M Nitzlnader, B Brunmair, V Castel, S Essiaf, A Cañete, R Ladenstein
BACKGROUND: Under the ExPO-r-NeT project (European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment), we aimed to identify paediatric oncology tumour boards in Europe to investigate the kind of technologies and logistics that are in place in different countries and to explore current differences between regions. METHODS: A 20-question survey regarding several features of tumor boards was designed. Data collected included infrastructure, organization, and clinical decision-making information from the centres...
January 8, 2018: Clinical & Translational Oncology
Ana Forjaz de Lacerda, Barbara Gomes
BACKGROUND: Children and adolescents dying from complex chronic conditions require paediatric palliative care. One aim of palliative care is to enable a home death if desired and well supported. However, there is little data to inform care, particularly from countries without paediatric palliative care, which constitute the majority worldwide. METHODS: This is an epidemiological study analysing death certificate data of decedents aged between 0 and 17 years in Portugal, a developed Western European country without recognised provision of paediatric palliative care, from 1987 to 2011...
December 22, 2017: BMC Pediatrics
Trausti Oskarsson, Stefan Söderhäll, Johan Arvidson, Erik Forestier, Thomas Leth Frandsen, Marit Hellebostad, Päivi Lähteenmäki, Ólafur G Jónsson, Ida Hed Myrberg, Mats Heyman
BACKGROUND: Treatment of relapsed childhood acute lymphoblastic leukemia (ALL) is particularly challenging due to the high treatment intensity needed to induce and sustain a second remission. To improve results, it is important to understand how treatment-related toxicity impacts survival. PROCEDURE: In this retrospective population-based study, we described the causes of death and estimated the risk for treatment-related mortality in patients with first relapse of childhood ALL in the Nordic Society of Paediatric Haematology and Oncology ALL-92 and ALL-2000 trials...
April 2018: Pediatric Blood & Cancer
Chiara Mainardi, Martin Ebinger, Sigrid Enkel, Tobias Feuchtinger, Heiko-Manuel Teltschik, Matthias Eyrich, Michael Schumm, Armin Rabsteyn, Patrick Schlegel, Christian Seitz, Carl-Phillip Schwarze, Ingo Müller, Johann Greil, Peter Bader, Paul-Gerhardt Schlegel, David Martin, Ursula Holzer, Michaela Döring, Rupert Handgretinger, Peter Lang
Poor graft function (PGF) is a severe complication of haematopoietic stem cell transplantation (HSCT) and administration of donor stem cell boosts (SCBs) represents a therapeutic option. We report 50 paediatric patients with PGF who received 61 boosts with CD34+ selected peripheral blood stem cells (PBSC) after transplantation from matched unrelated (n = 25) or mismatched related (n = 25) donors. Within 8 weeks, a significant increase in median neutrophil counts (0·6 vs. 1·516 × 109 /l, P < 0·05) and a decrease in red blood cell and platelet transfusion requirement (median frequencies 1 and 7 vs...
January 2018: British Journal of Haematology
Maria José Santiago-Lozano, Marta Lucía Barquín-Conde, Lucía Fuentes-Moreno, Roberto Manuel León-Vela, Lucas Madrid-Vázquez, Amelia Sánchez-Galindo, Jesús López-Herce Cid
INTRODUCTION: The aim of this study was to analyse the incidence, treatment and evolution of infections in children treated with ECMO. METHODS: A retrospective study based on a prospective database was performed. Children under the age of 18 years treated with ECMO from September 2006 to November 2015 were included. The patients' clinical characteristics were collected, together with ECMO technique, cultures and treatment of infection. RESULTS: One hundred patients with a median age of 11 months were analysed...
December 1, 2017: Enfermedades Infecciosas y Microbiología Clínica
B-M Schmidt, M Tameris, H Geldenhuys, A Luabeya, E Bunyasi, T Hawkridge, J B McClain, H Mahomed, T J Scriba, H McShane, M Hatherill
OBJECTIVE: Paediatric laboratory reference intervals used in Africa and Asia may be derived from historical intervals of predominantly Caucasian infants in Europe or North America. These intervals may therefore not be compatible with the range of normality for developing country populations. We aimed to compare haematology and biochemistry parameters in healthy South African infants with local laboratory reference intervals. METHODS: We compared the baseline haematology and biochemistry results of 634 (316 male and 318 female) HIV-unexposed infants, aged 3-6 months, living in a rural area of the Western Cape Province, South Africa, against laboratory reference intervals supplied by the South African National Health Laboratory Services (NHLS)...
January 2018: Tropical Medicine & International Health: TM & IH
J Pw Chung, T Th Lao, T C Li
INTRODUCTION: Individuals can be exposed to gonadotoxic agents in the course of treatment for cancers and other medical conditions. Fertility preservation refers to strategies that aim to preserve fertility by protecting it against the damage inflicted by gonadotoxic treatment. Many young patients are prescribed gonadotoxic treatment without prior counselling. This study aimed to study the awareness of, attitude to, and knowledge about fertility preservation among clinicians in Hong Kong...
December 2017: Hong Kong Medical Journal, Xianggang Yi Xue za Zhi
Jenny N Poynter, Michaela Richardson, Michelle Roesler, Mark Krailo, James F Amatruda, A Lindsay Frazier
BACKGROUND: Studies of family history of cancer in paediatric germ cell tumours (GCTs) are few, and none has had sufficient sample size to specifically evaluate family history of GCT. METHODS: We utilised family history data from a paediatric GCT study to calculate standardised incidence ratios (SIR) for GCT and other cancers using age- and sex-specific incidence rates from the SEER Program. RESULTS: This analysis included 7998 relatives of paediatric GCT probands...
October 24, 2017: British Journal of Cancer
Augustine O Ebonyi, Stephen Oguche, Martha O Ochoga, Oche O Agbaji, Joseph A Anejo-Okopi, Isaac O Abah, Prosper I Okonkwo, John A Idoko
BACKGROUND: Prior to commencing antiretroviral therapy (ART), haematological abnormalities are a common occurrence in individuals diagnosed with human immunodeficiency virus (HIV). In the course of receiving ART, these abnormalities usually improve. We determined the prevalence of haematological abnormalities in children diagnosed with HIV-1 and the changes in haematological parameters that occur after 6 and 12 months of being on ART. METHODS: A cross-sectional study of HIV-1 infected children aged 2 months to 15 years, between July 2005 and March 2013, at the paediatric HIV clinic of the Jos University Teaching Hospital, Jos...
October 1, 2017: Journal of Virus Eradication
Francisco Bautista, Lucas Moreno, Lynley Marshall, Andrew D J Pearson, Birgit Geoerger, Xavier Paoletti
BACKGROUND: Dose-escalation trials aim to identify the maximum tolerated dose and, importantly, the recommended phase II dose (RP2D) and rely on the occurrence of dose-limiting toxicities (DLTs) during the first treatment cycle. Molecularly targeted agents (MTAs) often follow continuous and prolonged administrations, displaying a distinct toxicity profile compared to conventional chemotherapeutics, and classical DLT criteria might not be appropriate to evaluate MTAs' toxicity. We investigated this issue in children...
November 2017: European Journal of Cancer
Francesca Perrino, Serena Licchelli, Giulia Serra, Giorgia Piccini, Cristina Caciolo, Patrizio Pasqualetti, Flavia Cirillo, Chiara Leoni, Maria Cristina Digilio, Giuseppe Zampino, Marco Tartaglia, Paolo Alfieri, Stefano Vicari
INTRODUCTION: Noonan syndrome (NS) is an autosomal dominant disorder characterized by short stature, skeletal and haematological/lymphatic defects, distinctive facies, cryptorchidism, and a wide spectrum of congenital heart defects. Recurrent features also include variable cognitive deficits and behavioural problems. Recent research has been focused on the assessment of prevalence, age of onset and characterization of psychiatric features in this disorder. Herein, we evaluated the prevalence of attention deficit and hyperactivity disorder (ADHD), anxiety and depressive symptoms and syndromes in a cohort of individuals with clinical and molecular diagnosis of NS...
January 2018: European Journal of Paediatric Neurology: EJPN
Jennifer Ann Geel, Brent T Stevenson, Rebecca B Jennings, Lisa E Krook, Sinead J Winnan, Bracha T Katz, T J Fox, Lukhanyo Nyati
BACKGROUND: The reported incidence of childhood cancer in upper-middle-income South Africa (SA) is much lower than in high-income countries, partly due to under-diagnosis and under-reporting. Documented survival rates are disturbingly low, prompting an analysis of potential factors that may be responsible. OBJECTIVES: To determine final-year medical students' level of knowledge of early warning signs of childhood cancer and whether a correlation existed between test scores and participants' age, gender and previous exposure to a person with cancer...
June 30, 2017: South African Medical Journal, Suid-Afrikaanse Tydskrif Vir Geneeskunde
Morten Tulstrup, Thomas L Frandsen, Jonas Abrahamsson, Bendik Lund, Kim Vettenranta, Olafur Gisli Jonsson, Hanne Vibeke Hansen Marquart, Birgitte Klug Albertsen, Mats Heyman, Kjeld Schmiegelow
OBJECTIVES: This randomized controlled trial tested the hypothesis that children with non-high risk acute lymphoblastic leukemia could benefit from individualized 6-mercaptopurine increments during consolidation therapy (NCT00816049). Primary and secondary end points were end of consolidation minimal residual disease (MRD) positivity and event-free survival. METHODS: 392 patients were randomized to experimental and 396 to standard therapy. Patients allocated to standard therapy received oral 6-mercaptopurine (25 mg/m(2) /day) from days 30 to 85, while the experimental arm received stepwise increments of additional 25 mg/m(2) /day beginning on days 50 and/or 71 unless dose-limiting myelosuppression had occurred...
October 6, 2017: European Journal of Haematology
Vijay Surampalli, Srinath Ramaswamy, Deepanjali Surendran, Chanaveerappa Bammigatti, Rathinam Palamalai Swaminathan
Primary Intestinal Lymphangiectasia (PIL) is a rare disease of unknown aetiology which presents in the paediatric age group with anasarca, diarrhoea, hypoproteinaemia, lymphoedema and chylous effusions. Tuberculosis, filariasis, chest trauma, malignancies and haematological disorders usually contribute to most cases of secondary lymphangiectasia and chylous effusions. We hereby describe a case of PIL presenting with chylous effusions which remained undiagnosed for eight years.
August 2017: Journal of Clinical and Diagnostic Research: JCDR
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