Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. The Lancet Haematology. 2024; 11:e228-e239.
.2024; 144:5351.
. Understanding Sickle Cell Disease Pain: Perception from Patients, Parents and Healthcare Providers in Northwestern Tanzania. Blood.2024; 144:2495.
. Effects of Splenomegaly in Children with Sickle Cell Anemia Treated with Hydroxyurea: Secondary Analysis of the Sphere Trial. Blood.COVID-19 mRNA vaccination responses in individuals with sickle cell disease: an ASH RC Sickle Cell Research Network Study. Blood Advances. 2024; 8:4549-4553.
.2024; 144:175.
. Individualized, PK-Guided Dosing of Hydroxyurea for Young Children with Sickle Cell Anemia: Final Results from the Hydroxyurea Optimization through Precision Study (HOPS). Blood.2024; 144:541.
. Newborn Screening for Sickle Cell Disease in Sub-Saharan Africa: Initial Results of the ASH Consortium on Newborn Screening in Africa (CONSA) Program. Blood.Hydroxyurea dose optimisation for children with sickle cell anaemia in sub-Saharan Africa (REACH): extended follow-up of a multicentre, open-label, phase 1/2 trial. The Lancet Haematology. 2024; 11:e425-e435.
.2024; 144:1071.
. Inducible, Erythroid-Specific Knockout of Codanin-1 in Adult Mice Replicates Congenital Dyserythropoietic Anemia Type-Ia. Blood.2024; 144:1257.
. Anticoagulation Therapy for Venous Thromboembolism in the Pediatric Population: A Systematic Review and Meta-Analysis. Blood.De Novo Brain Vascular Malformations in Hereditary Hemorrhagic Telangiectasia. Pediatric Neurology. 2024; 155:120-125.
.Brain AVM compactness score in children with hereditary hemorrhagic telangiectasia. Child's Nervous System. 2024; 40:2101-2108.
.Current Practice: Rationale for Screening Children with Hereditary Hemorrhagic Telangiectasia for Brain Vascular Malformations. American Journal of Neuroradiology. 2024; 45:1177-1184.
.Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial. Blood Advances. 2024; 8:5790-5795.
.Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study. Pediatric Blood and Cancer. 2024; 71:e30779.
.Impact of inflammation and steroids on anti-coagulation in children supported on a ventricular assist device. Journal of Artificial Organs. 2024; 27:368-374.
.2024; 144:5512.
. Epik-P2: A Phase 2 Study of Alpelisib (ALP) in Pediatric and Adult Patients (pts) with PIK3CA -Related Overgrowth Spectrum (PROS). Blood.Hemoglobin concentration and body mass index are determinants of plasma von Willebrand factor and factor VIII levels. Thrombosis Research. 2024; 240:109061.
.Hepatocyte-independent PAR1-biased signaling controls liver pathology in experimental obesity. Journal of Thrombosis and Haemostasis. 2024; 22:3191-3198.
.Emicizumab use in infancy: A survey of United States Pediatric Hemophilia Treatment Center Medical Directors. Haemophilia. 2024; 30:848-851.
.2024; 3:1331485.
. Case report: Childhood erythrocytosis due to hypermanganesemia caused by homozygous SLC30A10 mutation.Successes and pitfalls in orphan drug development for sickle cell disease. Blood Advances. 2024; 8:2455-2465.
.2024; 144:5018.
. Bivalirudin Monitoring By Dilute Thrombin Time Is Cost-Efficient in Pediatric ECMO Patients. Blood.How we use angiopoietin-2 in the diagnosis and management of vascular anomalies. Pediatric Blood and Cancer. 2024; 71:e30921.
.Bivalirudin Monitoring in Pediatric Ventricular Assist Device and Extracorporeal Membrane Oxygenation: Analysis of Single-Center Retrospective Cohort Data 2018-2022. Pediatric Critical Care Medicine. 2024; 25:e328-e337.
.Prevalence and Predictors of Hereditary Hemorrhagic Telangiectasia and Capillary-Malformation Arteriovenous Malformation Syndrome Among Children with Neurovascular Malformations. The Journal of Pediatrics. 2024; 264:113761.
.The key roles of thrombin and fibrinogen in human infant and mice atopic dermatitis models. Allergy: European Journal of Allergy and Clinical Immunology. 2024; 79:239-242.
.2024; 144:617.
. Community Health Worker Engagement with Adolescents and Young Adults with Sickle Cell Disease: Findings from the Comets Study. Blood.2024; 144:2458.
. The Dominant Negative Acting VPS4AE228Q Mutation Causes Dyserythropoiesis in Human iPSC-Derived Erythroblast Cultures That Phenocopies Cimdag Syndrome-Associated Anemia. Blood.Medical Management of Nonmalignant Vascular Tumors of the Head and Neck: Part 1. Oral and Maxillofacial Surgery Clinics of North America. 2024; 36:103-113.
.Medical Management and Therapeutic Updates on Vascular Anomalies of the Head and Neck: Part 2. Oral and Maxillofacial Surgery Clinics of North America. 2024; 36:115-123.
.Capillary malformations. The Journal of Clinical Investigation. 2024; 134:e172842.
.Loss of TBC1D2B causes a progressive neurological disorder with gingival overgrowth. European Journal of Human Genetics. 2024; 32:558-566.
.Hereditary hemorrhagic telangiectasia - pediatric review. Current Opinion in Pediatrics. 2024; 36:592-598.
.2024; 144:1066.
. Community Health Worker and Mobile Health Programs to Help Young Adults with Sickle Cell Disease Transition to Using Adult Healthcare Services – the Comets Study; Results from Patient-Reported Outcomes at 6 Months. Blood.2024; 144:2633.
. Thrombolytic Therapy for Venous Thromboembolism in the Pediatric Population: A Systematic Review and Meta-Analysis. Blood.Unmasking the Impact of Oxygenator-Induced Hypocapnia on Blood Lactate in Veno-Arterial Extracorporeal Membrane Oxygenation. ASAIO Journal. 2024; 70:795-802.
.Factor XII promotes the thromboinflammatory response in a rat model of venoarterial extracorporeal membrane oxygenation. Journal of Thoracic and Cardiovascular Surgery. 2024; 168:e37-e53.
.Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. Blood Advances. 2024; 8:5529-5538.
.2024; 2:120-134.
. Longitudinal prospective study of Sturge–Weber syndrome urine angiogenic factors and neurological outcome.2024; 144:520.
. Building Capacity in Sub-Saharan Africa to Address Sickle Cell Disease: The Consortium on Newborn Screening in Africa (CONSA). Blood.Red cell abnormalities characterized by ektacytometry in children with cholestasis. Pediatric Research. 2024; 95:1035-1040.
.2024; 144:2242.
. Stroke Training, Research, and Education Toward Capacity with Hydroxyurea (STRETCH). Blood.Pancytopenia in the Emergency Department. The Journal of Pediatrics. 2024; 272:114111.
.Development of a multimodal geomarker pipeline to assess the impact of social, economic, and environmental factors on pediatric health outcomes. Journal of the American Medical Informatics Association : JAMIA. 2024; 31:1471-1478.
.Evaluation, analysis, and reporting of medication adherence for clinical trials of anticoagulants in children: guidance from the ISTH SSC Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis. Journal of Thrombosis and Haemostasis. 2024; 22:2081-2092.
.A Mixed-Methods Evaluation of a Project ECHO Program for the Evidence-Based Management of Sickle Cell Disease. International Journal of Environmental Research and Public Health. 2024; 21:530.
.Thrombotic risk associated with gender-affirming hormone therapy. Journal of Thrombosis and Haemostasis. 2024; 22:2129-2139.
.2024; 144:5360.
. Location, Location(s), Location(s+): Moving from Acute to Chronic Pain in SCD Pain in School-Age Children. Blood.Hydroxyurea reduces infections in children with sickle cell anemia in Uganda. Blood. 2024; 143:1425-1428.
.2024; 144:2312.
. Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub. Blood.2024; 144:799.
. Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease. Blood.2024; 144:1084.
. Loss of ATG5 in Humans Causes Syndromic Congenital Dyserythropoietic Anemia with Impaired Mitophagy in Late Stages of Terminal Erythropoiesis. Blood.Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia. Gene Therapy (Basingstoke). 2024; 31:273-284.
.Rationale, Development, and Validation of HdxSim, a Clinical Decision Support Tool for Model-Informed Precision Dosing of Hydroxyurea for Children with Sickle Cell Anemia. Clinical Pharmacology and Therapeutics. 2024; 116:670-677.
.2024; 144:542.
. Hydroxyurea Pharmacokinetics in Children with Sickle Cell Anemia: Comparison of Global Cohorts. Blood.Reducing transfusion utilization for children with sickle cell anemia in sub-Saharan Africa with hydroxyurea: Analysis from the phase I/II REACH trial. American Journal of Hematology. 2024; 99:625-632.
.2024; 144:5555.
. The Fuvid Study: Functional Characterization of Children with Pulmonary Embolism in a Prospective Multicenter Cohort Study. Blood.2024; 144:4032.
. Low Rate of Red Blood Cell Alloimmunization Among Transfused Children with Sickle Cell Anemia in Malawi without Pre-Transfusion Screening or Antigen Matching. Blood.Medical Therapeutics for the Treatment of Vascular Anomalies: Part 3. Oral and Maxillofacial Surgery Clinics of North America. 2024; 36:125-136.
.A pharmacokinetic-pharmacodynamic analysis of l-glutamine for the treatment of sickle cell disease: Implications for understanding the mechanism of action and evaluating response to therapy. British Journal of Haematology. 2024; 205:1147-1158.
.A Population Pharmacokinetic Analysis of L-Glutamine Exposure in Patients with Sickle Cell Disease: Evaluation of Dose and Food Effects. Clinical Pharmacokinetics. 2024; 63:357-365.
.Multicenter, phase 1 study of etavopivat (FT-4202) treatment for up to 12 weeks in patients with sickle cell disease. Blood Advances. 2024; 8:4459-4475.
.Seeing haemoglobin SC: Challenging the misperceptions. British Journal of Haematology. 2024; 205:404-405.
.Baseline characteristics of Ghanaian children and adults enrolled in PIVOT, a randomised clinical trial of hydroxyurea in HbSC disease in sub-Saharan Africa. British Journal of Haematology. 2024; 205:2470-2480.
.Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shift. The Analyst. 2024; 149:2561-2572.
.2024; 144:163.
. Heterogeneity of the Erythromyeloblastic Island (EMBI) Niche during Baseline and Stress Erythropoiesis. Blood.2024; 1.
. A health equity ECHO for clinicians of individuals with SCD.Attitudes, Beliefs, and Intention to Receive a COVID-19 Vaccine for Pediatric Patients With Sickle Cell Disease. Journal of Pediatric Hematology/Oncology. 2024; 46:e305-e312.
.Screening for haemoglobin disorders: One size may not fit all. British Journal of Haematology. 2024; 204:26-28.
.2024; 1:100001.
. Angiopoietin-2 is associated with sickle cell complications, including stroke risk, and decreases with hydroxyurea therapy.2024; 144:289.
. Pharmacokinetic (PK)-Guided Dosing of Hydroxyurea for Tanzanian Children with Sickle Cell Anemia. Blood.An Institutional Approach to Equity and Improvement in Child Health Outcomes. Pediatrics. 2024; 154:e2023064994.
.2024; 144:5602.
. RED Blood Cell Alloimmunization in Sickle Cell Anaemia Patients in Kilifi, Kenya. Blood.The bold promise of gene therapy for sickle cell disease. British Journal of Haematology. 2024; 204:381-382.
.2024; 144:5315.
. Growth Curves for Children Living with Sickle Cell Anemia in Kilifi County, Kenya, Do Not Follow Who Curves for Normal Children. Blood.