Search Results - "Ellis J. Neufeld"
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Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: new data, new questions
Published in Blood (01-05-2006)“…For nearly 30 years, patients with transfusional iron overload have depended on nightly deferoxamine infusions for iron chelation. Despite dramatic gains in…”
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Novel therapeutics and future directions for refractory immune thrombocytopenia
Published in British journal of haematology (01-10-2023)“…Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder affecting approximately 1 in 20 000 people. While most patients with ITP are successfully…”
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3
Asymptomatic and Symptomatic SARS-CoV-2 Infections After BNT162b2 Vaccination in a Routinely Screened Workforce
Published in JAMA : the journal of the American Medical Association (22-06-2021)“…A 2-dose regimen of the BNT162b2 vaccine (Pfizer-BioNTech) against SARS-CoV-2 was authorized in Dec 2020 based on reported 94.8% efficacy. Although an…”
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Erythrocyte pyruvate kinase deficiency: 2015 status report
Published in American journal of hematology (01-09-2015)“…Over the last several decades, our understanding of the genetic variation, pathophysiology, and complications of the hemolytic anemia associated with red cell…”
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Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study
Published in Haematologica (Roma) (01-02-2019)Get full text
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Pediatric Heparin-Induced Thrombocytopenia: Prevalence, Thrombotic Risk, and Application of the 4Ts Scoring System
Published in The Journal of pediatrics (01-01-2015)“…Objective To characterize heparin-induced thrombocytopenia (HIT) at a single pediatric center including the prevalence and the accuracy of the 4Ts scoring…”
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Which pediatric ITP patients will go on to lupus?
Published in Blood (18-04-2024)Get full text
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Safety update on the use of recombinant activated factor VII in approved indications
Published in Blood reviews (01-06-2015)“…Abstract This updated safety review summarises the large body of safety data available on the use of recombinant activated factor VII (rFVIIa) in approved…”
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Systematic molecular genetic analysis of congenital sideroblastic anemia: Evidence for genetic heterogeneity and identification of novel mutations
Published in Pediatric blood & cancer (01-02-2010)“…Background Sideroblastic anemias are heterogeneous congenital and acquired bone marrow disorders characterized by pathologic iron deposits in mitochondria of…”
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Risk of Post-Procedural Bleeding in Children on Intravenous Fish Oil
Published in The American journal of surgery (01-10-2017)“…ABSTRACT Background Intestinal failure-associated liver disease (IFALD) can be treated with parenteral fish oil (FO) monotherapy, but practitioners have raised…”
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Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol
Published in BMJ open (27-08-2021)“…IntroductionImmune thrombocytopaenia (ITP) is an acquired disorder of low platelets and risk of bleeding. Although many children can be observed until…”
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Increasing observation rates in low‐risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP®)
Published in Pediatric blood & cancer (01-05-2017)“…An observational approach is recommended in newly diagnosed children with immune thrombocytopenia (ITP) at low risk of bleeding; however, there is no standard…”
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Prospective longitudinal study of coagulation profiles in children with hypoplastic left heart syndrome from stage I through Fontan completion
Published in The Journal of thoracic and cardiovascular surgery (01-04-2009)“…Objective The risk for thrombosis is increased after the Fontan operation. It is unknown whether children with univentricular heart disease have an intrinsic…”
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X-linked gray platelet syndrome due to a GATA1 Arg216Gln mutation
Published in Blood (15-04-2007)“…We identified a family with gray platelet syndrome (GPS) segregating as a sex-linked trait. Affected males had a mild bleeding disorder, thrombocytopenia, and…”
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15
Does iron overload really matter in stem cell transplantation?
Published in American journal of hematology (01-06-2012)“…A growing body of evidence suggests that iron overload is associated with inferior outcomes after myeloablative allogeneic hematopoietic stem cell…”
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Effect of Acute Bleeding on Daily Quality of Life Assessments in Patients with Congenital Hemophilia with Inhibitors and Their Families: Observations from the Dosing Observational Study in Hemophilia
Published in Value in health (01-09-2012)“…Abstract Introduction Quality-of-life (QOL) assessments in frequently bleeding patients with congenital hemophilia with inhibitors and their families are…”
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Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream
Published in Blood advances (23-12-2019)“…Hemophilia A (HA) is a bleeding disorder caused by mutations in the F8 gene encoding coagulation factor VIII (FVIII). Current treatments are based on regular…”
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Inadequate Dietary Intake in Patients with Thalassemia
Published in Journal of the Academy of Nutrition and Dietetics (01-07-2012)“…Abstract Background Patients with thalassemia have low circulating levels of many nutrients, but the contribution of dietary intake has not been assessed…”
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Thiamine-Responsive Megaloblastic Anemia: Identification of Novel Compound Heterozygotes and Mutation Update
Published in The Journal of pediatrics (01-12-2009)“…Objective To determine causative mutations and clinical status of 7 previously unreported kindreds with TRMA syndrome, (thiamine-responsive megaloblastic…”
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MRI guided iron assessment and oral chelator use improve iron status in thalassemia major patients
Published in American journal of hematology (01-07-2014)“…Oral iron chelators and magnetic resonance imaging (MRI) assessment of heart and liver iron burden have become widely available since the mid 2000s, allowing…”
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