All Stories

  1. Which pediatric ITP patients will go on to lupus?
  2. Novel therapeutics and future directions for refractory immune thrombocytopenia
  3. The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective
  4. Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study
  5. Comorbidities and complications in adults with pyruvate kinase deficiency
  6. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
  7. Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream
  8. Inhibitor Development with Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia a: Final Results of the Nuprotect Study
  9. Second‐line treatments in children with immune thrombocytopenia: Effect on platelet count and patient‐centered outcomes
  10. Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia
  11. Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study
  12. Recombinant activated factor VII in approved indications: Update on safety
  13. Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States
  14. Focusing in on use of pharmacokinetic profiles in routine hemophilia care
  15. Performing and interpreting individual pharmacokinetic profiles in patients with Hemophilia A or B: Rationale and general considerations
  16. Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study
  17. Safety and pharmacokinetics of the oral iron chelator SP-420 in β-thalassemia
  18. Risk of post-procedural bleeding in children on intravenous fish oil
  19. Immunogenicity, efficacy and safety of Nuwiq® (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study
  20. Perioperative management of haemophilia B: A critical appraisal of the evidence and current practices
  21. Recombinant porcine factor VIII for high‐risk surgery in paediatric congenital haemophilia A with high‐titre inhibitor
  22. Ringed sideroblasts in β‐thalassemia
  23. Skin testing, graded challenge, and desensitization to von Willebrand factor (VWF) products in type III von Willebrand disease (VWD)
  24. Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era
  25. Safety and efficacy of recombinant factor VIIa by pediatric age cohort: reassessment of compassionate use and trial data supporting US label
  26. Refractory Autoimmune Disease: An overview of when first-line therapy is not enough
  27. Unrelated Donor Marrow (BMT) or Cord Blood Transplantation (UCBT) for Thalassemia Major after Reduced Intensity Conditioning (URTH Trial Extension)
  28. A Budget Impact Model of Hemophilia Bypassing Agent Prophylaxis Relative to Recombinant Factor VIIa On-Demand
  29. Center-Based Quality Initiative Targets Youth Preparedness for Medical Independence:HEMO-Milestones Toolin a Comprehensive Hemophilia Clinic Setting
  30. Erythrocyte pyruvate kinase deficiency: 2015 status report
  31. Platelet function tests, independent of platelet count, are associated with bleeding severity in ITP
  32. Sports Participation in Children and Adolescents with Immune Thrombocytopenia (ITP)
  33. Liver MRI is more precise than liver biopsy for assessing total body iron balance: a comparison of MRI relaxometry with simulated liver biopsy results
  34. Safety update on the use of recombinant activated factor VII in approved indications
  35. Author's response: ‘Transition considerations for extended half‐life factor products’
  36. Dysregulated arginine metabolism and cardiopulmonary dysfunction in patients with thalassaemia
  37. Transition considerations for extended half‐life factor products
  38. Guidelines for the Standard Monitoring of Patients With Thalassemia
  39. Skin Testing, Graded Challenge and Desensitization to Von Willebrand Factor (vWF) Products in Type III Von Willebrand Disease (VWD)
  40. Pediatric Heparin-Induced Thrombocytopenia: Prevalence, Thrombotic Risk, and Application of the 4Ts Scoring System
  41. CSF 5-Methyltetrahydrofolate Serial Monitoring to Guide Treatment of Congenital Folate Malabsorption Due to Proton-Coupled Folate Transporter (PCFT) Deficiency
  42. Correlation between dispensed and prescribed doses of factor products for bleeding disorders: can a small, centre‐based pharmacy hit the mark?
  43. Treatment and outcomes of immune cytopenias following solid organ transplant in children
  44. Impact of Acute Bleeding on Daily Activities of Patients with Congenital Hemophilia with Inhibitors and Their Caregivers and Families: Observations from the Dosing Observational Study in Hemophilia (DOSE)
  45. The use of erythropoietin-stimulating agents versus supportive care in newborns with hereditary spherocytosis: a single centre's experience
  46. MRI guided iron assessment and oral chelator use improve iron status in thalassemia major patients
  47. Relationship among chelator adherence, change in chelators, and quality of life in Thalassemia
  48. R2 and R2* are equally effective in evaluating chronic response to iron chelation
  49. Human T-cell lymphotropic virus type 1 infection among U.S. Thalassemia patients
  50. Rituximab for treatment of inhibitors in haemophilia A
  51. Safety of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors: overall rFVIIa exposure and intervals following high (>240 μg kg−1) rFVIIa doses across clinical...
  52. Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major
  53. Coagulopathy and Vascular Malformations
  54. Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME)
  55. Pregnancy outcomes in women with thalassemia in North America and the United Kingdom
  56. Human T Cell Lymphotropic Virus Type 1 Infection Among U.S. Thalassemia Patients
  57. Induced Pluripotent Stem Cells with a Mitochondrial DNA Deletion
  58. Novel dominant β-thalassemia: Hb Boston-Kuwait [Codon 139/140(+T)]
  59. Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension
  60. Assessment of individual dose utilization vs. physician prescribing recommendations for recombinant activated factor VII (rFVIIa) in paediatric and adult patients with congenital haemophilia and alloantibody inhibitors (CHwI): the Dosing Observational ...
  61. Dosing, efficacy, and safety of recombinant factor VIIa (rFVIIa) in pediatric versus adult patients: The experience of the Hemostasis and Thrombosis Research Society (HTRS) Registry (2004-2008)
  62. To Treat or Not To Treat—From Guidelines to Individualized Patient Management
  63. Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone
  64. Increased leucocyte apoptosis in transfused β-thalassaemia patients
  65. 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
  66. Bleeding risks are higher in children versus adults given prophylactic platelet transfusions for treatment-induced hypoproliferative thrombocytopenia
  67. Inadequate Dietary Intake in Patients with Thalassemia
  68. Pre-transplantation iron chelation in patients with MDS or acute leukemia and iron overload undergoing myeloablative allo-SCT
  69. A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload
  70. Does iron overload really matter in stem cell transplantation?
  71. Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort
  72. Tracking the impact of the National Institutes of Health Clinical and Translational Science Awards on child health research: developing and evaluating a measurement strategy
  73. Beliefs about chelation among thalassemia patients
  74. Trends in anti-D immune globulin for childhood immune thrombocytopenia: Usage, response rates, and adverse effects
  75. Recognition and Management of Immune Thrombocytopenic Purpura and Autoimmune Hemolytic Anemia in the Emergency Department
  76. Applicability of 2009 international consensus terminology and criteria for immune thrombocytopenia to a clinical pediatric population
  77. Thirty-day readmission rates following hospitalization for pediatric sickle cell crisis at freestanding children's hospitals: Risk factors and hospital variation
  78. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia
  79. Iron Overload in Patients with Acute Leukemia or MDS Undergoing Myeloablative Stem Cell Transplantation
  80. Transition from pediatric to adult care for sickle cell disease: Results of a survey of pediatric providers
  81. Risks Factors And Mortality Associated With Doppler-Defined-Pulmonary Hypertension In Thalassemia Major: A Report From The Thalassemia Clinical Research Network Longitudinal Cohort Study
  82. Iron chelation adherence to deferoxamine and deferasirox in thalassemia
  83. Renal dysfunction in patients with thalassaemia
  84. Red cell alloimmunization in a diverse population of transfused patients with thalassaemia
  85. Exposure and safety of higher doses of recombinant factor VIIa ≥250 μg kg−1 in individuals with congenital haemophilia complicated by alloantibody inhibitors: the Haemophilia and Thrombosis Research Society Registry experience (2004-2008)
  86. Hormonal Contraception and Thrombotic Risk: A Multidisciplinary Approach
  87. The frequency and management of asparaginase-related thrombosis in paediatric and adult patients with acute lymphoblastic leukaemia treated on Dana-Farber Cancer Institute consortium protocols
  88. A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload
  89. Quality of life in thalassemia: A comparison of SF‐36 results from the thalassemia longitudinal cohort to reported literature and the US norms
  90. Relationship between Chronic Transfusion Therapy and Body Composition in Subjects with Thalassemia
  91. Symptoms of depression and anxiety in patients with thalassemia: Prevalence and correlates in the thalassemia longitudinal cohort
  92. Oral chelation: Should it be used with young children?
  93. Education and employment status of children and adults with thalassemia in North America
  94. Preface to Cooley's Anemia: Ninth Symposium
  95. Primary Care Clinicians’ Knowledge and Confidence About Newborn Screening for Sickle Cell Disease: Randomized Assessment of Educational Strategies
  96. Observational Cohort Study of Pediatric Inpatients With Central Venous Catheters at “Intermediate Risk” of Thrombosis and Eligible for Anticoagulant Prophylaxis
  97. PSY45 IMPACT OF ACUTE BLEEDING AND ADMINISTRATION OF BYPASSING AGENTS (BPA) ON DAILY ACTIVITIES OF PATIENTS WITH CONGENITAL HEMOPHILIA WITH INHIBITORS (CHWI) AND THEIR CAREGIVERS AND FAMILIES: OBSERVATIONS FROM THE DOSING OBSERVATIONAL STUDY IN HEMOPHI...
  98. PSY46 EFFECT OF ACUTE BLEEDING EPISODES ON QUALITY OF LIFE (QOL) IN PATIENTS WITH CONGENITAL HEMOPHILIA WITH INHIBITORS (CHWI) AND THEIR FAMILIES: OBSERVATIONS OF BLEED VS. NON-BLEED DAY QOL FROM THE DOSING OBSERVATIONAL STUDY IN HEMOPHILIA (DOSE) AND ...
  99. Erratum: “Corticosteroids for acute chest syndrome in children with sickle cell disease: Variation in use and association with length of stay and readmission” by Sobota et al.,Am J Hematol2010 DOI number 21565
  100. Genetic studies in pediatric ITP: outlook, feasibility, and requirements
  101. Future research in ITP: an ICIS consensus
  102. Infantile Myofibroma or Lymphatic Malformation
  103. Pain as an emergent issue in thalassemia
  104. Reversible severe combined immunodeficiency phenotype secondary to a mutation of the proton-coupled folate transporter
  105. Thiamine-Responsive Megaloblastic Anemia: Identification of Novel Compound Heterozygotes and Mutation Update
  106. Hemoglobin H-constant spring in North America: An alpha thalassemia with frequent complications
  107. Systematic molecular genetic analysis of congenital sideroblastic anemia: Evidence for genetic heterogeneity and identification of novel mutations
  108. Remission from transfusion-dependence in a patient with congenital dyserythropoietic anemia (CDA) and increased intensity of iron chelation
  109. Compliance with immunizations in splenectomized individuals with hereditary spherocytosis
  110. Prospective longitudinal study of coagulation profiles in children with hypoplastic left heart syndrome from stage I through Fontan completion
  111. One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab
  112. Response to mercaptopurine for refractory autoimmune cytopenias in children
  113. Corticosteroids for acute chest syndrome in children with sickle cell disease: Variation in use and association with length of stay and readmission
  114. Compliance with Immunizations in Splenectomized Individuals: A Study of the Splenectomized Hereditary Spherocytosis Population
  115. Female monozygotic twins discordant for hemophilia A due to nonrandom X-chromosome inactivation
  116. Practical implications of liver and heart iron load assessment by T2*-MRI in children and adults with transfusion-dependent anemias
  117. The Incidence and Implications of Anti-Heparin-Platelet Factor 4 Antibody Formation in a Pediatric Cardiac Surgical Population
  118. Relative response of patients with myelodysplastic syndromes and other transfusion‐dependent anaemias to deferasirox (ICL670): a 1‐yr prospective study
  119. Treatment with Rituximab in Benign and Malignant Hematologic Disorders in Children
  120. Clinical Effects and Safety of Rituximab for Treatment of Refractory Pediatric Autoimmune Diseases
  121. Successful Use of Bivalirudin for Cardiac Transplantation in a Child With Heparin-induced Thrombocytopenia
  122. Urinary hepcidin in congenital chronic anemias
  123. Targeted ITP strategies: Do they elucidate the biology of ITP and related disorders?
  124. Relationship of Transfusion and Iron-Related Complications to Cost of Care in Thalassemia.
  125. Hemoglobin Jamaica Plain — A Sickling Hemoglobin with Reduced Oxygen Affinity
  126. Myocardial iron measurements by MRI: getting to the heart of the matter
  127. Male infertility and thiamine-dependent erythroid hypoplasia in mice lacking thiamine transporter Slc19a2
  128. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial
  129. Extracorporeal membrane oxygenation as a bridge to cardiac transplantation in a patient with cardiomyopathy and hemophilia A
  130. Thiamine-responsive megaloblastic anemia (TRMA) syndrome: consequences of defective high-affinity thiamine transport
  131. Time course of early induction of intracellular adhesion molecule-1 messenger RNA during reperfusion, following cardiopulmonary bypass with hypothermic circulatory arrest in lambs
  132. Chronic Disseminated Intravascular Coagulation and Childhood-Onset Skin Necrosis Resulting From Homozygosity for a Protein C Gla Domain Mutation, Arg15Trp
  133. Starting out right: Kozak sequences and clots
  134. Risk factors for thromboembolism in teens: when should I test?
  135. Coagulation abnormalities in patients with single-ventricle physiology precede the Fontan procedure
  136. Myocardial immediate early gene activation after cardiopulmonary bypass with cardiac ischemia-reperfusion
  137. Coagulation Abnormalities in Pediatric and Adult Patients After Sclerotherapy or Embolization of Vascular Anomalies
  138. Inflammatory Bowel Disease Associated With Immune Thrombocytopenic Purpura in Children
  139. Characterization of a Murine High-Affinity Thiamine Transporter, Slc19a2
  140. A novel mutation in the SLC19A2 gene in a Tunisian family with thiamine-responsive megaloblastic anaemia, diabetes and deafness syndrome
  141. Thiamine-Responsive Megaloblastic Anemia Syndrome: A Disorder of High-Affinity Thiamine Transport
  142. 3714 SOLITARY COLONIC ULCER ASSOCIATED WITH COAGULATION FACTOR DEFICIENCY.
  143. Direct measurement of LDL-C in children: performance of two surfactant-based methods in a general pediatric population
  144. Homeoproteins CDP and SATB1 Interact: Potential for Tissue-Specific Regulation
  145. Defective high-affinity thiamine transporter leads to cell death in thiamine-responsive megaloblastic anemia syndrome fibroblasts
  146. Cux/CDP Homeoprotein Is a Component of NF-μNR and Represses the Immunoglobulin Heavy Chain Intronic Enhancer by Antagonizing the Bright Transcription Activator
  147. TIME COURSE OF EARLY ICAM-1 mRNA INDUCTION IN LUNG AND VENTRICLE FOLLOWING CARDIOPULMONARY BYPASS IN LAMBS
  148. HEMOPHILIA
  149. UPDATE ON GENETIC RISK FACTORS FOR THROMBOSIS AND ATHEROSCLEROTIC VASCULAR DISEASE
  150. Prevalence of factor V Leiden in a population of patients with congenital heart disease
  151. PREFACE
  152. Refined mapping of the gene for thiamine-responsive megaloblastic anemia syndrome and evidence for genetic homogeneity
  153. Combined schedule of 7-valent pneumococcal conjugate vaccine followed by 23-valent pneumococcal vaccine in children and young adults with sickle cell disease
  154. Hair Defects and Pup Loss in Mice with Targeted Deletion of the First Cut Repeat Domain of theCux/CDPHomeoprotein Gene
  155. Localization of the Gene for Thiamine-Responsive Megaloblastic Anemia Syndrome, on the Long Arm of Chromosome 1, by Homozygosity Mapping
  156. CASP, a novel, highly conserved alternative-splicing product of the CDP/cut/cux gene, lacks cut-repeat and homeo DNA-binding domains, and interacts with full-length CDP in vitro
  157. Combined Schedule of Pneumococcal Conjugate Vaccine Followed by Pneumococcal Polysaccharide Vaccine in Patients With Sickle Cell Disease. • 779
  158. Blockade of selectin-mediated leukocyte adhesion improves postischemic function in lamb hearts
  159. Hypoxia enhances inflammatory regulation of E-selectin through a cAMP-dependent pathway
  160. ARMS test for diagnosis of factor V Leiden mutation, a common cause of inherited thrombotic tendency
  161. P-selectin expression in myocardium of children undergoing cardiopulmonary bypass
  162. Severe haemophilia A in a female resulting from two de novo factor VIII mutations
  163. Repressor Activity of CCAAT Displacement Protein in HL-60 Myeloid Leukemia Cells
  164. Inherited dyslipidemias in childhood
  165. Volume 187, Number 2 (1992), in the article "Sequence-Specific Binding of HMG-I(Y) to the Proximal Promoter of the gp91-phox Gene," by David G. Skalnik and Ellis J. Neufeld, pages 563-569
  166. Sequence-specific binding of HMG-I(Y) to the proximal promoter of the gp91-phox gene
  167. Human CCAAT displacement protein is homologous to the Drosophila homeoprotein, cut
  168. Neufeld Replies
  169. Phosphoinositide turnover provides a link in stimulus—response coupling
  170. Production of phosphoinositide-derived messengers
  171. A mutant fibroblast cell line defective in glycoprotein synthesis due to a deficiency of glucosamine phosphate acetyltransferase