All Stories

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