Susceptibility to BK polyomavirus-associated hemorrhagic cystitis in children undergoing allogeneic transplant. Haematologica. 2025; 110(2):527-531.
Bkpyv Replication Kinetics and Association with Hemorrhagic Cystitis in Pediatric HSCT Recipients. Transplantation and Cellular Therapy. 2025; 31(2):s515-s516.
Infections in patients with relapsed refractory multiple myeloma receiving idecabtagene vicleucel therapy: A real-world analysis from CIBMTR. Blood. 2025; 146(Supplement 1):716.
Comparisons Between Multiple Breath Washout and Standard-of-Care Spirometry in Children Undergoing Allogenic Hematopoietic Stem Cell Transplant: A Transpire Study. American Journal of Respiratory and Critical Care Medicine. 2025; 211(Supplement_1):a5125-a5125.
Selumetinib in pediatric patients with neurofibromatosis type 1 and plexiform neurofibroma: Propensity score analysis of SPRINT vs. natural history control arm. Neuro-Oncology Advances. 2025; 7(1):vdaf101.
Microbial metabolite drives ageing-related clonal haematopoiesis via ALPK1. Nature. 2025; 642(8066):201-211.
Smoking-Associated Carcinogen-Induced Inflammation Promotes Lung Carcinogenesis via IRAK4 Activation. Clinical Cancer Research. 2025; 31(4):746-755.
Daytime SHP2 inhibitor dosing, when immune cell numbers are elevated, shrinks neurofibromas. Life Science Alliance. 2025; 8(12).
Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report. Blood advances. 2025.
Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome - Impact of Age, Donor, and Infection: A Pidtc Report. Transplantation and Cellular Therapy. 2025; 31(2):s39-s41.
RAS Pathway Mutations and Therapeutics in Vascular Anomalies. Pediatric Blood and Cancer. 2025; 72(5):e31605.
Emapalumab for Maladaptive Interferon-γ-Mediated Inflammation in Drug Reaction With Eosinophilia and Systemic Symptoms Complicating First-Line Antituberculosis Therapy. Pediatric Blood and Cancer. 2025; 72(12):e32052.
Assessing Acute and Long-Term Toxicities of Proton Radiation Therapy in Pediatric Pelvic Tumors: Findings From the Pediatric Proton Consortium Registry. Pediatric Blood and Cancer. 2025; 72(10):e31811.
Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report. American Journal of Hematology. 2025; 100(10):1813-1827.
Alpha+ Thalassemia in Northwestern Tanzania: Molecular and Hematological Insights From Newborn Screening. Journal of Blood Medicine. 2025; 16:241-250.
Hydroxyurea to decrease stroke risk in children with sickle cell anemia: a systematic review and meta-analysis. Blood Global Hematology. 2025; 1(1).
When a Trait Becomes a Disease: A Rare Hematologic Overlap of Sickle Cell Trait and Hereditary Spherocytosis. American Journal of Hematology. 2025.
RAS G12 and Q61 codon mutations confer distinct disease characteristics in Acute Myeloid Leukemia and exhibit differential response to RAS(ON) inhibitor RMC-7977. Blood. 2025; 146(Supplement 1):3229-3229.
CONCORDANCE OF SOMATIC GENETIC TESTING IN BLOOD VS. BONE MARROW IN SHWACHMAN-DIAMOND SYNDROME. EJC Paediatric Oncology. 2025; 6:100332.
Liberalizing hospital proximity requirements for children/young adults with low-burden B-ALL receiving tisagenlecleucel. Blood advances. 2026; 10(1):106-110.
Liberalizing Requirements for Hospital Proximity Post-CD19 CAR for Children with Low Burden B-ALL at Infusion. Transplantation and Cellular Therapy. 2025; 31(2):s230-s231.
Treatment of focal anaplastic Wilms tumor: A report from the Children's Oncology Group AREN0321 and AREN03B2 studies. Cancer. 2025; 131(2):e35713.
Clinical Characteristics, Management, and Hematopoietic Cell Transplantation of Patients with TLR8 Gain-of-Function. Blood advances. 2025.
Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report. Transplantation and Cellular Therapy. 2026; 32(1):89.e1-89.e10.
Underlying Diagnosis and RBC Transfusions Increase HLA Antibodies in Children before Hematopoietic Stem Cell Transplantation. Transplantation and Cellular Therapy. 2025.
A 10-Year History of Graft Failure in Pediatric Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy. 2025; 31(2):s148-s149.
Male Gender and Underlying Diagnosis Predisposes to Increased HLA Antibody Burden in Children before Allogeneic Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy. 2025; 31(2):s147.
Frequent Pulmonary Complications after Allogeneic Hematopoietic Stem Cell Transplant: A Brief Transpire Report. Transplantation and Cellular Therapy. 2025; 31(2):s149-s150.
005 Baseline clinical characteristics of newly diagnosed optic pathway gliomas associated with NF1: results from the international multicenter NF1-OPG Natural History Study. Journal of AAPOS. 2025; 29(4):104287.
Long-term Ocular Safety in Children Treated with Mitogen-Activated Protein Kinase Pathway Inhibitor Therapy. Ophthalmology (Rochester, Minn.). 2026; 133(2):291-294.
Erythroid-specific, inducible CDAN1∆/∆ mouse model phenocopies congenital dyserythropoietic anemia type-la. Blood. 2025; 146(Supplement 1):186-186.
MICRO-TAG enzyme complementation enables quantification of cellular drug-target engagement in temperature series. SLAS Discovery. 2026; 38:100291.
Abstract 5480: Live cell real-time quantification of drug-target engagement for rapid drug discovery. Cancer Research. 2025; 85(8_Supplement_1):5480-5480.
Abstract LB266: A multicenter phase 1-2a clinical study of Orotecan® (oral irinotecan HCl, VAL-413) in patients with recurrent pediatric solid tumors. Cancer Research. 2025; 85(8_Supplement_2):lb266-lb266.
Decoding DDX41: Clinical impact of germline and somatic mutations in 77 high-risk myeloid neoplasm patients. Blood. 2025; 146(Supplement 1):1442.
Immunogenicity and Reactogenicity of High- or Standard-Dose Influenza Vaccine in a Second Consecutive Influenza Season. Journal of Infectious Diseases. 2025; 231(1):e123-e131.
LINE-1 Retroelement Activation and Neuroinflammation in Persons With Fanconi Anemia. Pediatric Blood and Cancer. 2025; 72(12):e32081.
Impact of Graft Versus Host Disease Following Allogeneic Hematopoietic Cell Transplantation on Leukemia Free Survival in Hematologic Malignancies: A CIBMTR Analysis. Transplantation and Cellular Therapy. 2025; 31(2):s60-s61.
A Single-Cell Transcriptome Atlas of Epithelial Subpopulations in HPV-Positive and HPV-Negative Head and Neck Cancers. Viruses. 2025; 17(4).
The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care (COMETS) Trial: Protocol for a Randomized Controlled Trial. JMIR Research Protocols. 2025; 14:e69239.
Beyond Survival: Assessing Renal and Cardiovascular Health in Fanconi Anemia Adolescents and Young Adults Post-Stem Cell Transplant. Transplantation and Cellular Therapy. 2025; 31(2):s375-s376.
Updated favourable-histology Wilms tumour risk stratification: rationale for future Children's Oncology Group clinical trials. Nature Reviews Urology. 2025; 22(11):775-788.
Multicenter Study on Caregiver Experiences in Pediatric Hematopoietic Stem Cell Transplantation Part I: Integrative Analysis of Mental Health, Psychosocial Stressors, and Support Mechanisms. Transplantation and Cellular Therapy. 2025; 31(7):456.e1-456.e16.
Real-World Assessment of Patient Adherence to the Late Effects Screening Recommendations Among Hematopoietic Cell Transplantation Survivors. Transplantation and Cellular Therapy. 2025; 31(2):s364-s365.
Expression of mutant TIE2 p.L914F during mouse development causes embryonic lethality and defects in vascular remodeling. Developmental Dynamics. 2025.
The Fas-mediated apoptosis assay: From concept to clinical application. Journal of Immunological Methods. 2025; 537:113812.
633. Incidence and Outcomes of Pediatric Allogeneic Hematopoietic Cell Transplant (allo-HCT) Recipients Under Surveillance and Not Under Surveillance for Human Adenovirus (HAdV) in the Post-HCT Period. Open Forum Infectious Diseases. 2025; 12(Supplement_1):ofae631.198.
Differential Filtration of Macromolecules: A Novel Indicator of Mortality Risk in Pediatric HSCT Patients. Transplantation and Cellular Therapy. 2025; 31(2):s145-s146.
Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement. Blood. 2025; 145(13):1369-1381.
Multiomics dissection of human RAG deficiency reveals distinctive patterns of immune dysregulation but a common inflammatory signature. Science immunology. 2025; 10(103):eadq1697.
ASH's Commitment to SCD: Building on 10 Years of Progress. Blood advances. 2025.
Three-generation female cohort with macrocytic anemia and iron overload. American Journal of Hematology. 2025; 100(1):133-138.
High Household Transmission Among Asymptomatic Contacts Across Pandemic Waves in Cincinnati, Ohio. Epidemiologia. 2025; 6(4).
GATA Lot to Learn: Decoding the Drivers of Down Syndrome-Associated Acute Leukemias. The Hematologist. 2025; 22(4).
1187: ECMO IN PEDIATRIC ONCOLOGIC AND HEMATOPOIETIC STEM CELL TRANSPLANT PATIENTS: A COMPARATIVE ANALYSIS. Critical Care Medicine. 2025; 53(1).
Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study. Journal of Human Immunity. 2026; 2(1):e20250163.
Etiologies and clinical predictors of pediatric eosinophilia: A retrospective analysis from a tertiary care center. Blood. 2025; 146(Supplement 1):6554-6554.
Abstract 1596: Tumor-derived RAC1A159V gain-of-function mutation promotes an immunosuppressive tumor microenvironment and represses response to immune checkpoint inhibitors. Cancer Research. 2025; 85(8_Supplement_1):1596-1596.
Tumor-derived RAC1A159V mutation promotes an immunosuppressive microenvironment that represses response to immune checkpoint inhibitor. Science Advances. 2025; 11(44):eaea1212.
Feasibility of Atezolizumab in Combination With Chemotherapy for Children With Relapsed or Refractory Solid Tumors. Pediatric Blood and Cancer. 2025; 72(12):e32046.
118: Randomized, Double-Blind, Phase 2 Study of Alpelisib in Pediatric and Adult Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Journal of Investigative Dermatology. 2025; 145(3):e29.
Prothrombin prevents fatal T cell-dependent anemia during chronic virus infection of mice. JCI Insight. 2025; 10(4).
Outcomes of Patients with Intracranial Germinoma Enrolled on the Pediatric Proton Photon Consortium Registry. International Journal of Radiation Oncology Biology Physics. 2025; 123(1):e144-e145.
Perioperative Management with Efanesoctocog Alfa in Adults, Adolescents, and Children with Severe Hemophilia A in the Phase 3 XTEND Clinical Program. Hamostaseologie. 2025; 45:s57-s58.
Novel CSF Biomarkers for facilitating diagnosis of CNS-HLH from other Neuroinflammatory Disorders. Blood advances. 2025.
Emapalumab Treatment in Patients With Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study. Arthritis & Rheumatology. 2025; 77(2):226-238.
ZMYND11 functions in bimodal regulation of latent genes and brain-like splicing to safeguard corticogenesis. Nature Communications. 2025; 16(1):9010.
Grandchildren of GRNDaD: Shifts in disease-modifying therapy at the adolescent transition in sickle cell disease. British Journal of Haematology. 2025; 207(3):1070-1075.
WU Hoo! The Promise of Allogeneic WU-CART-007 in Relapsed/Refractory T-Cell Malignancies. The Hematologist. 2025; 22(5).
Neurons derived from NeuroD1-expressing astrocytes transition through transit-amplifying intermediates but lack functional maturity. Science Advances. 2025; 11(30):eadw9296.
YTHDF2 promotes ATP synthesis and immune evasion in B cell malignancies. Cell. 2025; 188(2):331-351.e30.
An engineered β-globin homology donor reveals insights into β-globin expression and betters HDR. Molecular Therapy. 2025; 33(4):1308-1309.
Validation of an Interferon Type I Score Test on the Nanostring nCounter Platform. Journal of Human Immunity. 2025; 1(CIS2025).
Clinical and Analytical Validation of a NOD2 Functional Test for XIAP/XLP2 Diagnosis. Journal of Human Immunity. 2025; 1(CIS2025).
Case Report: A patient with a novel heterozygous IRF8 variant with repeated infection and immune-mediated organ disease, but without disseminated mycobacterial disease despite BCG immunization. Frontiers in Immunology. 2025; 16:1654617.
Lipopolysaccharide-responsive and beige-like anchor protein (LRBA) functional deficiency caused by biallelic LRBA missense variants characterized by Evans syndrome or colitis. Journal of Allergy and Clinical Immunology. 2025; 156(2):270-278.
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Atypical Teratoid Rhabdoid Tumor. Journal of Molecular Pathology. 2025; 6(2):13.
Role in of primary VTE prophylaxis in pediatric patients in hospitalized and critically ill settings: A systematic review and meta-analysis. Blood. 2025; 146(Supplement 1):3137-3137.
Real-world clinical outcomes with novel agent combination therapies for the frontline treatment of pediatric and adult advanced-stage Hodgkin lymphoma. Blood. 2025; 146(Supplement 1):1852-1852.
Yellow Fluorescent Protein Quenching Assay for Analyzing Odorant Receptor Activity. Methods in Molecular Biology. 2025; 2915:169-177.
Protein-Protein Interactions of Odorant and G Protein-Coupled Receptors: A BRET Analysis Approach. Methods in Molecular Biology. 2025; 2915:161-168.
A strongly degenerate fully nonlinear mean field game with nonlocal diffusion. Journal of Differential Equations. 2025; 440:113436.
Characterization of E1 enzyme dependencies in mutant-UBA1 human cells reveals UBA6 as a novel therapeutic target in VEXAS syndrome. Leukemia. 2025; 39(8):1997-2009.
166-OR: Mean RBC Age (MRBC) Variation Accounts for the Predominance of Mismatch (MM) between Measured HbA1c (mA1c) and CGM-Derived Estimated HbA1c (eA1c) Diabetes. 2025; 74(Supplement_1).
Associations Between Pain Scores and Opioid Doses With Emergency Department Disposition and Return Visit Rates in Children With Sickle Cell Disease. Pediatric Blood and Cancer. 2025; 72(7):e31750.
EBV-Associated T/NK-LPD Manifesting As HLH Cured By Nivolumab and Emapalumab, Avoiding the Need for Allogeneic HCT. Transplantation and Cellular Therapy. 2025; 31(2):s43.
Experience with T Cell–Depleted Allogeneic HSCT for Refractory sJIA Associated with Lung Disease. Journal of Human Immunity. 2025; 1(CIS2025).
Central Venous Catheter Removal After Hematopoietic Stem Cell Transplant: A Review and Perspective. Pediatric Blood and Cancer. 2025; e70001.
Oxidative Stress Early After Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy. 2025; 31(3):135.e1-135.e10.
Thirty Years of Hydroxyurea for Sickle Cell Anemia - Scientific Progress, Global Health Gaps. New England Journal of Medicine. 2025; 393(16):1556-1559.
Globalization in clinical drug development for sickle cell disease. American Journal of Hematology. 2025; 100(1):4-9.
ReInspire: A phase 2 study of mutant-selective PI3Kα inhibitor, RLY-2608, in adults and children with PIK3CA-related overgrowth spectrum and malformations driven by PIK3CA mutation. Blood. 2025; 146(Supplement 1):3092.
Social support and barriers to medication adherence among adolescents and young adults with leukemia and lymphoma. Blood. 2025; 146(Supplement 1):2706.
Wide Ranging Neurobehavioral Phenotype in Individuals With Costello Syndrome. American Journal of Medical Genetics, Part A. 2026.
A Mixed Methods Study of Medication Adherence in Adults with Neurofibromatosis Type 1 (NF1) on a Clinical Trial of Selumetinib. Cancers. 2025; 17(2).
"From Drowning to Treading Water": Adolescents and Young Adults Living with Incurable and Indolent Metastatic Soft Tissue Sarcoma for More than Two Years. Cancers. 2025; 17(3).
Engraft: A Collaborative Learning Health Network for Enhanced Transplant and Cellular Therapy Outcomes. Transplantation and Cellular Therapy. 2025; 31(3):123-134.
Gene Therapy with Reduced-Intensity Conditioning for Sickle Cell Disease. Transplantation and Cellular Therapy. 2025; 31(2):s2-s3.
Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part II. Organ Dysfunction and Immune Reconstitution Considerations for Children with Chronic Graft-versus-Host Disease after Hematopoietic Cell Transplantation. Transplantation and Cellular Therapy. 2025; 31(6):347.e1-347.e17.
667: SEVERE METHOTREXATE-INDUCED NEUROTOXICITY IN A HISPANIC ADOLESCENT GIRL WITH B-ALL. Critical Care Medicine. 2025; 53(1).
Hydroxyurea for Children and Adults with Hemoglobin SC Disease. NEJM Evid. 2025; 4(2):EVIDoa2400402.
A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric and Young Adult Solid Tumors. Pediatric Blood and Cancer. 2025; 72(7):e31692.
Upstream integration of implementation planning in clinical trials through the blood and marrow transplant clinical trials network. Blood. 2025; 146(Supplement 1):7913-7913.
Redox Control in Platelet Activity and Therapy. Antioxidants. 2025; 14(11).
Outcome Analysis Post Virus-Specific T Cell Therapy for the Treatment of Adenoviremia in Immunocompromised Pediatric Patients. Transplantation and Cellular Therapy. 2025; 31(2):s221-s222.
Challenges in Assessing Response in Clinical Trials for Neurofibromatosis Type 1 Plexiform Neurofibromas. Journal of Clinical Oncology. 2025; 43(15):1843-1844.
Relevance of Recent Thymic Emigrants Following Allogeneic Hematopoietic Cell Transplantation for Pediatric Patients with Inborn Errors of Immunity. Transplantation and Cellular Therapy. 2025; 31(4):265.e1-265.e12.
When Treatment Becomes Personal: Cytarabine Pharmacogenomics Score Elucidates Outcome Disparities in Pediatric and Adolescent and Young Adult Acute Myeloid Leukemia. The Hematologist. 2025; 22(6).
Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy. Molecular Therapy. 2025; 33(10):4848-4857.
Reconstitution of Norovirus-Specific T-Cell Responses Following Hematopoietic Stem Cell Transplantation in Patients With Inborn Errors of Immunity and Chronic Norovirus Infection. Journal of Infectious Diseases. 2025; 231(3):773-783.
Inpatient and emergency healthcare utilization and payments for care of SCD: an analysis of all payers in Florida 2010-2019. Journal of Sickle Cell Disease. 2025; 2(1).
Spatial Profiling Identifies Regionally Distinct Microenvironments and Targetable Immunosuppressive Mechanisms in Pediatric Osteosarcoma Pulmonary Metastases. Cancer Research. 2025; 85(12):2320-2337.
A proposed path to explaining the unexplained anemia of aging. The journals of gerontology. Series A, Biological sciences and medical sciences. 2026; 81(1).
Molecular surprises in evaluations of red cell disorders. Hematology. American Society of Hematology. Education Program. 2025; 2025(1):370-376.
Characterization of the human erythromyeloblastic islands (EMBIs) Blood. 2025; 146(Supplement 1):182-182.
Biomarkers of ineffective erythropoiesis in patients with sickle cell anemia, at baseline and while treated with hydroxyurea, in comparison to patients with other iron-loading red cell disorders. Blood. 2025; 146(Supplement 1):4686.
Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome. Orphanet Journal of Rare Diseases. 2025; 20(1):375.
Comment on: "Comment on: Bivalirudin During Thrombolysis With Catheter-Directed tPA in a Heparin-Refractory Patient: A Case Report": The Promise of Dilute Thrombin Time and Drug-Calibrated Assays for Improved Bivalirudin Monitoring. Pediatric Blood and Cancer. 2025; 72(1):e31418.
A phase 2 PBTC study of selumetinib for recurrent/progressive pediatric low-grade glioma: Strata 2, 5, and 6 with long-term outcomes on strata 1, 3, and 4. Neuro-Oncology. 2025; 27(9):2415-2428.
Clinically relevant bleeding and heavy menstrual bleeding among patients <21 years old with provoked VTE: Findings from the multinational kids-DOTT trial. Blood. 2025; 146(Supplement 1):316-316.
BraTS-PEDs: Results of the Multi-Consortium International Pediatric Brain Tumor Segmentation Challenge 2023. MELBA journal. 2025; 3(June 2025):72-87.
Age-based pegaspargase dosing is safe and achieves therapeutic levels in infants with ALL: report from COG AALL15P1. Blood advances. 2026; 10(1):70-74.
Roadmap to a Global Template for Implementation of Ototoxicity Management for Cancer Treatment. Ear and Hearing. 2025; 46(2):286-297.
How to Improve the Sleep of Hospitalized Pediatric Patients: Family and Staff Focus Groups. Transplantation and Cellular Therapy. 2025; 31(8):586.e1-586.e11.
The somatic hotspot mutation of DDX41 (p.R525H) elicits dominant-negative effects upon non-truncating germline variants, explaining similar disease risk as truncating variants. Blood. 2025; 146(Supplement 1):3221-3221.
Dopaminergic encoding of future defensive actions in the mouse nucleus accumbens. PNAS Nexus. 2025; 4(5):pgaf128.
NTRK1-rearranged histiocytosis: clinicopathologic and molecular features. Blood advances. 2025; 9(14):3617-3628.
Feasibility and Safety of Intratympanic Administration of Sustained-Exposure Dexamethasone Thermosensitive Gel (OTO-104) for Prevention of Cisplatin-Induced Hearing Loss in Children: A Multisite Phase 2 Randomized Clinical Trial. Pediatric Blood and Cancer. 2025; 72(6):e31680.
Itacitinib for the prevention of IEC therapy-associated CRS: results from the 2-part phase 2 INCB 39110-211 study. Blood. 2025; 146(4):422-436.
Elevated Bromide Levels Due to Tazemetostat Therapy Treated With Salt and Water Supplementation in Two Children With Atypical Teratoid Rhabdoid Tumor. Pediatric Blood and Cancer. 2025; 72(8):e31803.
CPI203, a BET inhibitor, down-regulates a consistent set of DNA synthesis genes across a wide array of glioblastoma lines. PLoS ONE. 2025; 20(5):e0306846.
Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321. Pediatric Blood and Cancer. 2025; 72(3):e31490.
Physician Advocacy for Hematopoietic Cell Transplantation and Cell Therapy: Overcoming Challenges to Reduce Disparities and Improve Outcomes for Patients. Transplantation and Cellular Therapy. 2026; 32(1):12.e1-12.e4.
T2-weighted MRI radiomics for the prediction of pediatric and young adult rhabdomyosarcoma alveolar subtype and distant metastasis: a pilot study. Pediatric Radiology. 2025; 55(6):1149-1161.
Dramatic multifocal osteosarcoma treatment response in the setting of POT1 tumor predisposition syndrome. Cancer Genetics. 2025; 298-299:39-43.
The Threat of Medical Misinformation: A Case of Ivermectin Toxicity in a Pediatric Oncology Patient. Pediatric Blood and Cancer. 2025; 72(9):e31876.
A novel POT1-TPD presentation: A germline pathogenic POT1 variant discovered in a patient with newly diagnosed posterior fossa ependymoma. Cancer Genetics. 2025; 292-293:38-43.
Reprioritizing and embracing change: Implications of the ACGME revised requirements on pediatric medical education and hospital medicine. Journal of Hospital Medicine. 2025; 20(9):1009-1011.
Understanding Diversity: An ASTCT-NMDP Access Initiative Survey of the Hematopoietic Cell Transplant and Cellular Therapy Nonphysician Workforce. Transplantation and Cellular Therapy. 2025; 31(2):s343-s344.
Physician Workforce Diversity in the Hematopoietic Cell Transplant and Cellular Therapy Ecosystem: An ASTCT-NMDP Access Initiative Survey. Transplantation and Cellular Therapy. 2025; 31(2):s361.
Prolonged, Unintentional Overdose of Hydroxyurea in a Child With Sickle Cell Anemia. Pediatric Blood and Cancer. 2025; e70076.
Cellular taxonomy of the preleukemic bone marrow niche of acute myeloid leukemia. Leukemia. 2025; 39(1):51-63.
Duration of anticoagulation for venous thromboembolism in pediatric patients: Evaluation of the Duration of Therapy for Thrombosis in Children (Kids-DOTT) trial outcomes at 2 years. Journal of Thrombosis and Haemostasis. 2025; 23(2):651-656.
Examining the role of the photopigment melanopsin in the striatal dopamine response to light. Frontiers in Systems Neuroscience. 2025; 19:1568878.
Shining a TORCH on a Case of Blueberry Muffin Rash in a Newborn Infant. NeoReviews. 2025; 26(3):e191-e196.
Pulmonary function testing in pediatric allogeneic stem cell transplant recipients to monitor for Bronchiolitis obliterans syndrome: a systematic review. BMC Pediatrics. 2025; 25(1):250.
An inflammatory state defines a high-risk T-lineage acute lymphoblastic leukemia subgroup. Science Translational Medicine. 2025; 17(779):eadr2012.
DARA to Believe: Anti-CD38 Antibody Improves Outcomes in Relapsed/Refractory Pediatric T-Cell Lymphoblastic Leukemia and Lymphoma. The Hematologist. 2025; 22(3).
Managing Diffuse Alveolar Hemorrhage in Pediatric HSCT with Inhaled and Intrabronchial Therapy: A Case Series of Targeted Hemostatic Intervention. Transplantation and Cellular Therapy. 2025; 31(2):s153.
Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial. Nature Medicine. 2025; 31(7):2204-2212.
Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial. JAMA dermatology. 2025; 161(5):533-537.
Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial. Nature Medicine. 2025; 31(1):105-115.
Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease. Blood. 2025; 146(Supplement 1):2542-2542.
Opioid Timeliness in the Emergency Department and Hospitalizations for Acute Sickle Cell Pain. JAMA pediatrics. 2025; 179(11):1194-1202.
Anemia in Young Children and the Association With Socioeconomic Deprivation Indices. Pediatric Blood and Cancer. 2025; 72(7):e31663.
Reply to: Comment on Anemia in Young Children and the Association With Socioeconomic Deprivation Indices. Pediatric Blood and Cancer. 2025; 72(7):e31743.
Incidence of Transplant-Associated Thrombotic Microangiopathy (TA-TMA) Is Not Lowered By Reduction of Acute GvHD (aGvHD) in Patients Receiving Post-Transplant Cyclophosphamide (PTCy) GvHD Prophylaxis: A Report from the MIDAS (Microangiopathy, Endothelial Damage in Adults undergoing Stem cell transplantation) Consortium. Transplantation and Cellular Therapy. 2025; 31(2):s282.
Impact of Hepatoblastoma on Infectious Complications Following Pediatric Liver Transplantation. Pediatric Transplantation. 2025; 29(1):e70035.
Title: Role of primary VTE prophylaxis in pediatric leukemia, lymphoma, and solid tumors: A systematic review and meta-analysis. Blood. 2025; 146(Supplement 1):3139-3139.
Congenital dyserythropoietic anemia type I: Updated report from the congenital dyserythropoietic anemia registry of North America. Blood. 2025; 146(Supplement 1):4685-4685.
Biomarkers of ineffective erythropoiesis in patients with transfusion- dependent thalassemia. Blood. 2025; 146(Supplement 1):4684-4684.
Reclassification of ATM Missense Variants of Uncertain Significance by Integrating Results from Systematic Functional Assays into an ACMG Points-Based Framework. Clinical Cancer Research. 2025; 31(12):2426-2440.
Posttransplant EBV-Positive Smooth Muscle Tumors in Children, Adolescents, and Young Adults: A Multi-Institution Experience. Pediatric Blood and Cancer. 2025; 72(6):e31678.
Community respiratory viruses are generally welltolerated in hematopoietic stem cell transplant recipients: a brief report from the TRANSPIRE study. Haematologica. 2025; 110(8):1882-1886.
467 Hereditary predisposition syndrome for myelodysplastic syndrome with SF3B1 mutation; a case report. American Journal of Clinical Pathology. 2025; 164(Supplement_1):aqaf121.272.
Reducing Harm in Cultural Transition: Repurposing the 5 Stages of Grief Model for Global Health Experiences. Journal of Graduate Medical Education. 2025; 17(6):689-693.
Causes and Consequences of Persistent Anemia after 6 Months of Antiretroviral Therapy in Tanzania: An Observational Comparative Cohort Study. American Journal of Tropical Medicine and Hygiene. 2025; 112(1):234-241.
Engaging Parents of Children With Sickle Cell Disease in Shared Decision-Making for Hydroxyurea: The ENGAGE-HU Study. Pediatric Blood and Cancer. 2025; 72(5):e31639.
Discrepancies between primary and secondary interpretations of pediatric nuclear medicine imaging examinations. Pediatric Radiology. 2025.
IRAK1/4/pan-FLT3 Kinase Inhibitors with Reduced hERG Block as Treatments for Acute Myeloid Leukemia. ACS Medicinal Chemistry Letters. 2025; 16(5):887-895.
Neutrophil-derived vesicles control complement activation to facilitate inflammation resolution. Cell. 2025; 188(6):1623-1641.e26.
Establishing the Engraft Multi-Center Tri-Use Registry. Transplantation and Cellular Therapy. 2025; 31(2):s529-s530.
Acute Toxicities and Early Outcomes of Tandem Autologous Stem Cell Transplantation in Pediatric High-Risk Neuroblastoma: A Multicenter Study. Transplantation and Cellular Therapy. 2025.
Acute Toxicities and Early Outcomes of Tandem Autologous Stem Cell Transplantation in Pediatric High-Risk Neuroblastoma: A Multicenter Study. Transplantation and Cellular Therapy. 2025; 31(2):s93-s94.
Abatacept Prevents Severe Acute Graft-Versus-Host Disease Without Increasing Graft Failure Risk in Pediatric Bone Marrow Failure Syndromes. Pediatric Blood and Cancer. 2025; 72(12):e32085.
Chronic Graft-Versus-Host Disease Adversely Impacts School Performance in Children and Young Adults. Transplantation and Cellular Therapy. 2025; 31(2):99.e1-99.e11.
Longitudinal Outcomes of Fertility Potential in Female Pediatric and Young Adult Allogeneic Hematopoietic Stem Cell Transplant Survivors. Transplantation and Cellular Therapy. 2025; 31(2):s98.
A Novel Dominant Negative Variant in IL2RG Gene that Failed to Be Corrected by Lentiviral Gene Therapy. Journal of Human Immunity. 2025; 1(CIS2025).
Stretchable Heat Transfer Eco-Materials: Mesogen Grafted NR-Based Nanocomposites with High Thermal Conductivity and Low Dielectric Constant. Small. 2025; 21(2):e2406066.
Real world experience using belumosudil for treatment of chronic graft versus host disease in children and young adults. Bone Marrow Transplantation. 2026.
Abatacept for acute graft versus host disease prophylaxis in children with sickle cell disease patients undergoing HLA identical sibling hematopoietic stem cell transplantation. Bone Marrow Transplantation. 2025; 60(8):1102-1107.
Kinetics of DNases after hematopoietic stem cell transplant. Blood Vessels, Thrombosis & Hemostasis. 2025; 2(2):100055.
Single Center Experience of Belumosudil in Children < 12 Years, Adolescents and Young Adults. Transplantation and Cellular Therapy. 2025; 31(2):s305-s306.
Ubiquitin-conjugating enzyme UBE2N modulates proteostasis in immunoproteasome-positive acute myeloid leukemia. Journal of Clinical Investigation. 2025; 135(10).
IMG-71. Stability of radiomics in pediatric medulloblastoma: a multi-institutional preliminary analysis. Neuro-Oncology. 2025; 27(Supplement_5):v289-v290.
Radiomic-based prognostic score for survival risk-stratification in pediatric medulloblastoma tumors: A multi-institutional study. Neuro-Oncology Advances. 2025; 7(1):vdaf107.
Context-dependent inhibitory roles of RhoA in 3D invasive cell migration within the extracellular matrix. Cell reports. 2025; 44(12):116649.
Letter to the editor. European Archives of Oto-Rhino-Laryngology. 2025.
Community Health Worker and Mobile Health Interventions for Quality of Life Among Young Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA Network Open. 2025; 8(11):e2543571.
Targeting the lectin pathway in TA-TMA. Blood advances. 2026; 10(1):181-182.
Translating biomarker insights into practice: a path forward in TA-TMA management. Frontiers in Medicine. 2025; 12:1550365.
Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry. Blood advances. 2025; 9(20):5249-5262.
DEK promotes mammary hyperplasia and is associated with H3K27me3 epigenetic modifications. Life Science Alliance. 2025; 8(9).
FLT3-F691L confers kinase-independent resistance via Grb2-dependent MAPK signaling in AML. Blood. 2025; 146(Supplement 1):5049-5049.
Intrabreath Oscillometry (IBO) vs. Spectral Oscillometry (SO) in Detecting Respiratory Disease in Patients Undergoing Hematopoietic Stem Cell Transplantation (HSCT) American Journal of Respiratory and Critical Care Medicine. 2025; 211(Supplement_1):a5118-a5118.
The diagnosis and treatment of red cell membrane disorders: algorithm for the general hematologist. Clinical Advances in Hematology and Oncology. 2025; 23(9):534-538.
Efficacy and safety of thrombolysis for pediatric venous thromboembolism: a systematic review and meta-analysis. Blood advances. 2025; 9(21):5512-5527.
Patient-Level Barriers and Facilitators to Inpatient Physical Therapy in Adolescents and Young Adults With a Hematological Malignancy: A Qualitative Study. Pediatric Blood and Cancer. 2026; e70129.
Development of Mouse-Tumor Model Using Prostate Cancer (PC3) Cell Line for High-Intensity Focused Ultrasound (HIFU) Ablation. Prostate Cancer. 2025; 2025:5678314.
Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part III. Long-Term Impact of Chronic Graft-versus-Host Disease on Endocrinologic, Cardiovascular, and Metabolic Outcomes in Survivors of Pediatric Hematopoietic Cell Transplantation. Transplantation and Cellular Therapy. 2025; 31(5):297.e1-297.e15.
Abatacept improves posttransplant survival and reduces endothelial injury syndromes in β-thalassemia major. Blood advances. 2025; 9(24):6370-6379.
miR-219a-Robo1 axis acts as a conserved switch for slit-mediated commissural axon guidance. iScience. 2025; 28(12):114242.
Addition of Thiotepa to Alemtuzumab, Fludarabine, and Melphalan Reduced-Intensity Conditioning Reduces Secondary Graft Failure in Allogeneic HSCT for Inborn Errors of Immunity. Journal of Human Immunity. 2025; 1(CIS2025).
Developmental Origins and Oncogenesis in Medulloblastoma. Annual Review of Neuroscience. 2025; 48(1):85-102.
The somatic DDX41 hot spot mutation (p.R525H) causes skewed differentiation into plasmacytoid dendritic cells in human iPSC and leukemia models. Blood. 2025; 146(Supplement 1):678-678.
Germline DDX41 mutations in myeloid neoplasms: the current clinical and molecular understanding. Current Opinion in Hematology. 2025; 32(2):67-76.
SARC031: A Phase 2 Trial of Selumetinib and Sirolimus for Patients with Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors (MPNST). Clinical Cancer Research. 2026.
In vitro determination of patient-specific variation challenges the universal RBE gold standard for proton radiation therapy. Clinical and Translational Radiation Oncology. 2026; 56:101046.
Pain, Internalizing Symptoms, Posttraumatic Stress, and Alcohol Use Among Young Adult Survivors of Cancer. Pediatric Blood and Cancer. 2026; e70154.
396 Pain Experiences Among Children Receiving Treatment for Acute Lymphoblastic Leukemia. Journal of Pain. 2025; 29:105194.
Systematic Review of Pain Assessment Measures Used in Pediatric Acute Lymphoblastic Leukemia. Psycho-Oncology. 2025; 34(1):e70063.
Requirements and Study Design for the Next Proton FLASH Clinical Trials: an International Multidisciplinary Delphi Consensus. International Journal of Radiation Oncology Biology Physics. 2025; 123(1):296-305.
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Pediatric Medulloblastoma. Journal of Molecular Pathology. 2025; 6(2):11.
BIOM-88. LEPTOMENINGEAL METASTASIS IN THE INTERNAL AUDITORY CANAL AS A PREDICTOR OF OUTCOMES FOR CHILDREN WITH CENTRAL NERVOUS SYSTEM TUMORS. Neuro-Oncology. 2025; 27(Supplement_5):v45-v45.
Wolcott-Rallison syndrome - crosstalk between PERK- EIF2A and type II interferon signaling. European Journal of Medical Genetics. 2025; 76:105022.
Proteomic analysis of pericardial effusions in hematopoietic stem cell transplant recipients. Haematologica. 2025.
The Tell-Tale Heart: A Cytokine Storm Story of Pericardial Effusions after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s137.
High Prevalence of Abnormal Baseline Lung Function in Pediatric and Young Adult Hematopoietic Stem Cell Transplant Recipients: A Report from the TRANSPIRE Study. Pediatric Blood and Cancer. 2025; 72(10):e31916.
Hepatocyte senescence and persistent liver injury in Fanconi anemia. Haematologica. 2025; 110(8):1870-1874.
Transcriptomic Alterations and Clonal Evolution Identifies a Pre-Emptive Indication for Hematopoietic Stem Cell Transplant in Fanconi Anemia. Transplantation and Cellular Therapy. 2025; 31(2):s308-s309.
Pravastatin Prophylaxis Is Safe and Feasible in Children and Young Adults Undergoing Allogeneic HSCT: A Prospective, Single Arm Cohort Study. Transplantation and Cellular Therapy. 2025; 31(2):s153-s154.
Beyond Graft-Vs-Host Disease: The Role of Cellular Senescence in Persistent Liver Injury in Fanconi Anemia after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s311-s312.
Lipid dysregulation after hematopoietic stem cell transplant. Haematologica. 2025.
Liver disease in shwachman-diamond syndrome: A report from the shwachman-diamond syndrome registry. Blood. 2025; 146(Supplement 1):1436.
Bone matters: Defining the burden of skeletal disease in telomere biology disorders. Blood. 2025; 146(Supplement 1):6747.
Immunodeficiency in TBDs: making the lymphocyte count. Blood. 2025; 146(10):1148-1150.
The spectrum of pathogenic NF1 variants in participants enrolling on clinical trials of MEK inhibitors for plexiform neurofibroma. Human Genetics. 2025; 144(11-12):1199-1206.
The Prostate is a Common Site of Disease in Patients with MEN2B and Medullary Thyroid Carcinoma. Endocrine-Related Cancer. 2025.
Outcomes following radiation therapy for embryonal tumor with multilayered rosettes (ETMR): results from the Pediatric Proton/Photon Consortium Registry (PPCR). Journal of Neuro-Oncology. 2025; 174(2):369-380.
Cell-extrinsic factors contribute toward myeloid-biased expansion in Tet2-mutant clonal hematopoiesis. Experimental Hematology. 2025; 148:104816.
Identifying barriers to clinical trial enrollment for children with relapsed or refractory hematologic malignancies. Blood. 2025; 146(Supplement 1):4404-4404.
Prevalence of cytopenia(s) and somatic variants in patients with DDX41 mutant germline predisposition syndrome. British Journal of Haematology. 2025; 206(4):1109-1120.
Utilization of Intravenous Posaconazole in Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy. 2025; 31(12):1033.e1-1033.e11.
Higher Dose Intravenous Posaconazole Is Safe and Necessary for Achievement of Therapeutic Targets and Prevention of Infections in Pediatric Hematopoietic Stem Cell Transplant Patients. Transplantation and Cellular Therapy. 2025; 31(2):s546.
Medulloblastoma of early childhood. Salvage and outcome after initial CSI sparing approaches. OncoDaily Medical Journal. 2025.
Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions. Clinical Cancer Research. 2025; 31(3):561-572.
Optimal relevant subset designs in nonlinear models. Canadian Journal of Statistics, The. 2025; 53(3).
Co-designing educational materials about SARS-CoV-2 (COVID-19) vaccines with individuals with sickle cell disease (SCD) and their families. Journal of Sickle Cell Disease. 2025; 2(1).
Late Morbidity and Mortality in Survivors of Childhood Ependymoma: A Report from the Childhood Cancer Survivor Study (CCSS). Cancers. 2025; 17(22).
Urine Kidney Injury Biomarkers Predict TMA Risk in Pediatric HSCT Recipients. Transplantation and Cellular Therapy. 2025; 31(2):s314.
Supar Is a Novel and Powerful Biomarker of TMA and Kidney Injury after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s313-s314.
DDDR-42. Modeling and Translating Precision Therapies for NF1-Associated Tumors Across the Disease Spectrum. Neuro-Oncology. 2025; 27(Supplement_5):v172-v172.
Building capacity for sickle cell stroke prevention in low-resource settings. Discover Public Health. 2025; 22(1):881.
Health status of Ugandan children born with sickle cell anemia and congenital HIV identified through newborn screening. Blood. 2025; 146(Supplement 1):2668.
Assessing educational needs of sickle cell anemia healthcare providers in sub-Saharan Africa and the Caribbean. Frontiers in Public Health. 2025; 13:1693285.
Primary Stroke Screening and Hydroxyurea Treatment for Sickle Cell Anemia in Pediatric Healthcare Settings in East and Central Africa: A Narrative Review of Capacity Gaps and Opportunities. Public Health Reviews. 2025; 46:1608359.
IMGP-04. Targetable somatostatin receptor expression assessed by DOTATATE PET imaging in children and young adults with recurrent/progressive high-risk central nervous system (CNS) tumors: Initial screening results from the CONNECT2007 Lutathera Phase I/II trial. Neuro-Oncology. 2025; 27(Supplement_5):v311-v311.
ZIC1 is a context-dependent medulloblastoma driver in the rhombic lip. Nature Genetics. 2025; 57(1):88-102.
Anthropometric measurements of children with neurofibromatosis type I: impact of plexiform neurofibroma volume and treatment. Pediatric Research. 2025; 97(6):1918-1928.
Recurrent thrombosis and major bleeding in children treated for VTE. Blood advances. 2025; 9(15):3824-3831.
Integrated clinical and molecular landscape of disseminated pediatric low-grade glioma. Neuro-Oncology. 2025.
Maternal-Fetal Transmission of Diffuse Large B-Cell Lymphoma. Pediatric Blood and Cancer. 2025; e32174.
Loss of Cpt1a results in elevated glucose-fueled mitochondrial oxidative phosphorylation and defective hematopoietic stem cells. Journal of Clinical Investigation. 2025; 135(5).
Current Advances and Future Directions for Sensitizing Gastric Cancer to Immune Checkpoint Inhibitors. Cancer Medicine. 2025; 14(14):e71065.
SUV39H1 maintains cancer stem cell chromatin state and properties in glioblastoma. JCI Insight. 2025; 10(5).
KLF5 Shapes Developing Respiratory Tubules by Inhibiting Actin Asymmetry in Epithelial Cells. American Journal of Respiratory Cell and Molecular Biology. 2025; 72(6):663-677.
Effect of Clinically Relevant Immunosuppressants on Viral Specific T-Cell (VST) in Vitro Function. Transplantation and Cellular Therapy. 2025; 31(2):s509-s510.
Use of Ovarian Tumor Markers in Prediction of Malignancy Risk in Pediatric and Adolescent Patients. Journal of Surgical Research. 2025; 315:839-846.
Fusion-specific chromatin profiles mediate transcriptional heterogeneity in MLL-rearranged acute lymphoblastic leukemia. Blood advances. 2025; 9(4):856-861.
Structural Dynamics of Rho GTPases. Journal of Molecular Biology. 2025; 437(3):168919.
Glutaredoxin 2 is essential for AML survival through mitochondrial permeability transition pore regulation. Blood. 2025.
A Pilot Acceptance and Commitment Intervention for Parents of Children With RASopathies. Clinical Practice in Pediatric Psychology. 2025; 13(1):37-48.
Inhibitors and Obesity: The Prevalence and Strength of Association in People With Haemophilia. Haemophilia. 2025; 31(6):1226-1235.
Improving Time to Bleeding Disorder Treatment in the Emergency Department. Pediatric Blood and Cancer. 2025; 72(4):e31570.
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery. Molecular Therapy. 2025; 33(3):1154-1165.
The role of oligodendrocyte progenitor cells in the spatiotemporal vascularization of the human and mouse neocortex. Glia. 2025; 73(1):140-158.
Consensus recommendations for an integrated diagnostic approach to peripheral nerve sheath tumors arising in the setting of Neurofibromatosis Type 1. Neuro-Oncology. 2025; 27(3):616-624.
CNSC-41. IDENTIFICATION AND VALIDATION OF TRANSITIONAL CEREBELLAR PROGENITORS (TCP) IN THE RHOMBIC LIP OF THE HUMAN FETAL CEREBELLUM. Neuro-Oncology. 2025; 27(Supplement_5):v63-v63.
Real-world outcomes for young adult patients receiving CD19 CAR T-cell therapy. Blood advances. 2025; 9(11):2763-2772.
Disseminated pediatric low-grade glioma and hydrocephalus: a multinational consortium analysis of incidence and mortality. Journal of Neurosurgery: Pediatrics. 2025; 1-14.
Clinical, Immunologic, and Genetic Characteristics in Patients With Syndrome of Undifferentiated Recurrent Fevers. Arthritis & Rheumatology. 2025; 77(5):596-605.
Benefits of Hydroxyurea in Mitigating Early Onset Lung Disease in Children With Sickle Cell Anemia. American Journal of Respiratory and Critical Care Medicine. 2025; 211(Abstracts):a1272-a1272.
Establishing a Quality Improvement Education Platform in the Engraft Learning Network. Transplantation and Cellular Therapy. 2025; 31(2):s501.
Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe hemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. Journal of Thrombosis and Haemostasis. 2025; 23(11):3481-3491.
HTRS2025.P1.6 Clinical Outcomes Over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children From North America With Severe Hemophilia A in the Phase 3 XTEND-ed Long-Term Extension Study. Research and Practice in Thrombosis and Haemostasis. 2025; 9:103026.
SAT-218 Longitudinal outcomes of fertility potential in female pediatric hematopoietic stem cell transplant survivors. Journal of the Endocrine Society. 2025; 9(Supplement_1):bvaf149.1785.
The HCT Protein Shake: Moderate and Severe Proteinuria Are Specific to TA-TMA. Transplantation and Cellular Therapy. 2025; 31(2):s145.
P-103. Adenoviremia Clearance Following Treatment with Intravenous (IV) Brincidofovir (BCV) in Immunocompromised Patients is Associated with Positive Clinical Disease Response: Preliminary Outcomes from the ATHENA Phase IIa Study. Open Forum Infectious Diseases. 2025; 12(Supplement_1):ofae631.310.
Precision Alemtuzumab Dosing Results in Day 0 Target Range Achievement in 80% of IEI Patients and Minimizes Risks of GVHD, Clinically Significant Mixed Chimerism, and Secondary Graft Failure. Journal of Human Immunity. 2025; 1(CIS2025).
Diagnostic testing for hemophagocytic lymphohistiocytosis. Journal of Immunological Methods. 2025; 537:113816.
Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study. The Lancet Rheumatology. 2025; 7(4):e243-e251.
Reduced chronic graft versus host disease after allogeneic HSCT with cryopreserved peripheral blood stem cell grafts: A CIBMTR analysis. Blood. 2025; 146(Supplement 1):933-933.
Defining and Assessing Poor Graft Function after Allogeneic HSCT in Children and Young Adults. Transplantation and Cellular Therapy. 2025; 31(2):s317-s318.
The First Prospective Screening Study of BK Polyomavirus (BKPyV) Hemorrhagic Cystitis after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s513-s514.
Plasma LILRA6 Levels Influence BK Polyomavirus Cystitis after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s514-s515.
Feasibility Trial of a Tailored Adherence-Promotion Intervention for Adolescents and Young Adults With Cancer. Pediatric Blood and Cancer. 2026; 73(1):e32127.
Developing Behavior Change Model-Informed and Participant-Engaged Retention Strategies for Adolescents and Young Adults With Cancer Enrolled on Behavioral and/or Psychosocial Trials. Pediatric Blood and Cancer. 2025; 72(5):e31583.
Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia. Blood advances. 2025; 9(8):1927-1939.
Targeting RhoA nuclear mechanoactivity rejuvenates aged hematopoietic stem cells. Nature Aging. 2026; 6(1):68-87.
Use of angiopoietin-2 as a biomarker in vascular anomalies beyond complex lymphatic anomalies. Blood. 2025; 146(Supplement 1):4877.
Emapalumab for the Treatment of Immune-Mediated Graft Failure after HSCT. Bone Marrow Transplantation. 2025; 60(3):406-411.
Integrated cytogenetic and genomic profiling of the MDS-L cell line. Molecular Cytogenetics. 2025; 18(1):11.
Nurse Driven Central Line Rounding Utilized to Improve Bone Marrow Transplant Patient Outcomes: An Update. Transplantation and Cellular Therapy. 2025; 31(2):s451.
Tunneling nanotube-like structures regulate distant cellular interactions during heart formation. Science. 2025; 387(6739):eadd3417.
Runx2 takes center stage in regulating HSC expansion. Blood. 2025; 146(26):3135-3136.
Unraveling the Challenges of Non-Responders to the Eculizumab Treatment with High-Risk Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (TA-TMA) Transplantation and Cellular Therapy. 2025; 31(2):s542-s543.
American Society of Hematology/International Society on Thrombosis and Haemostasis 2024 updated guidelines for treatment of venous thromboembolism in pediatric patients. Blood advances. 2025; 9(10):2587-2636.
Physical activity among adolescents and young adults living with chronic pain and sickle cell disease: a qualitative examination. Journal of Pediatric Psychology. 2025; 50(12):1079-1087.
Randomized controlled trial-in-progress of an interdisciplinary treatment program for youth with chronic SCD pain: Integrative strong body and mind training for sickle cell disease (I-STRONG) Blood. 2025; 146(Supplement 1):4753.
Quality of Life for Rural and Appalachian Pediatric Brain Tumor Survivors in Ohio and Kentucky. Clinical Practice in Pediatric Psychology. 2025; 13(1):11-16.
Post-transplant cyclophosphamide based GVHD prophylaxis in HLA-matched related and unrelated donor hematopoietic cell transplantation in adults with hematologic malignancies undergoing myeloablative conditioning: A CIBMTR analysis of 8,272 participants. Blood. 2025; 146(Supplement 1):931.
Receipt of Fluoroquinolone Prophylaxis is not Associated With Development of Vancomycin-Resistant Enterococcus Colonization During Pediatric Hematopoietic Cell Transplantation. Journal of the Pediatric Infectious Diseases Society. 2025; 14(3).
Erythrocyte alloimmunization in children with sickle cell anemia living in Kilifi, Kenya. Blood Global Hematology. 2025; 1(2):100016.
SUPERIOR SURVIVAL FOLLOWING HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR HIGH-RISK MARROW FEATURES IN PATIENTS WITH SHWACHMAN-DIAMOND SYNDROME. EJC Paediatric Oncology. 2025; 6:100336.
Low hematologic event-free survival with age in shwachman-diamond syndrome. Blood. 2025; 146(Supplement 1):748-748.
Clinical Use of ZSCAN4 for Telomere Elongation in Hematopoietic Stem Cells. NEJM Evid. 2025; 4(3):EVIDoa2400252.
Blood proteomics of paediatric bronchiolitis obliterans syndrome after hematopoietic cell transplant. ERJ Open Research. 2025; 00745-02025.
Characterization and prediction of prolonged severe neutropenia in pediatric patients receiving tisagenlecleucel. Blood advances. 2025; 9(19):5070-5084.
Microbiota from young mice restore the function of aged ISCs. Stem Cell Reports. 2026; 102788.
Circulating Immune Complexes and Glucose-6-Phosphate Dehydrogenase Deficiency Predict Recurrent Blackwater Fever in Ugandan Children With Severe Malaria. Journal of Infectious Diseases. 2025; 232(2):285-297.
Transplant outcomes using older matched sibling donors compared with young alternative donors: a CIBMTR analysis. Blood advances. 2025; 9(14):3469-3478.
Perspectives on the current diagnostic and treatment paradigms in secondary hemophagocytic lymphohistiocytosis (HLH). Orphanet Journal of Rare Diseases. 2025; 20(1):200.
Soluble Fms-like Tyrosine Kinase-1 (sFlt-1) As a Clinical Biomarker for Individual Susceptibility to Post-Transplant Complications: A Report from the MIDAS Consortium. Transplantation and Cellular Therapy. 2025; 31(2):s26-s27.
Acute Toxicities of Proton Craniospinal Irradiation in Pediatric Medulloblastoma: A Pediatric Proton/Photon Consortium Registry (PPCR) Study. International Journal of Particle Therapy. 2025; 16:100747.
Systemic T-cell activation and IFN-γ activity in indeterminate severe hepatitis are reminiscent of hemophagocytic lymphohistiocytosis: Implications for T-cell- and IFN-γ-directed therapies. Journal of Allergy and Clinical Immunology. 2025; 155(1):199-212.
Stroke prevention in Hispanic children with sickle cell anemia: the SACRED trial. Blood advances. 2025; 9(8):1791-1800.
Longitudinal changes and predictors of cardiac extracellular volume fraction in sickle cell anemia. Blood Red Cells Iron. 2025; 1(3).
A New First-Line Option for Pediatric ITP-Incremental Progress or Paradigm Shift? Journal of the American Medical Association (JAMA). 2025; 334(20):1802-1803.
Reassessing voxelotor safety and efficacy: Real-world outcomes from the ASH research collaborative data hub. Blood. 2025; 146(Supplement 1):6226-6226.
Impact of disease genotype and severity of anemia on myocardial extracellular volume fraction in sickle cell disease (SCD) and non-SCD hemolytic anemias. Blood. 2025; 146(Supplement 1):2957.
The ASH consa consortium on newborn screening in Africa for sickle cell disease: Interim updates of progress in screening, follow-up and supporting initiatives. Blood. 2025; 146(Supplement 1):944-944.
Hereditary spherocytic pyropoikilocytosis: Expanding the clinical and genetic spectrum of red blood cell membrane disorders. Blood. 2025; 146(Supplement 1):4667.
Plasma lipid levels predict chemotherapy response and survival in acute myeloid leukemia. Blood. 2025; 146(21):2589-2596.
Unraveling lipid metabolism for acute myeloid leukemia therapy. Current Opinion in Hematology. 2025; 32(2):77-86.
Haematopoietic stem cell transplant for inborn errors of immunity-Who and when? British Journal of Haematology. 2025; 207(3):750-753.
Prevalence of Antiplatelet and Anticoagulation Therapy in Children with Sickle Cell Anemia and Stroke. Pediatr Stroke. 2025; 11:111-140.
Neurocognitive outcomes and functional independence in adult survivors of childhood medulloblastoma diagnosed over 3 decades. Neuro-Oncology. 2025; 27(1):254-266.
Renin-angiotensin-aldosterone system inhibitors exacerbate anemia in sickle cell disease. Blood. 2025; 146(Supplement 1):297-297.
61430 Dermatologic adverse events in neurofibromatosis patients on MEK inhibitors. Journal of American Academy of Dermatology. 2025; 93(3):ab33.
Delayed T-cell recovery after hematopoietic cell transplantation is associated with decreased overall survival in adults. Blood advances. 2025; 9(14):3502-3517.
Understanding Diversity: An ASTCT-NMDP ACCESS Initiative Survey of the Hematopoietic Cell Transplant and Cellular Therapy Workforce in the United States. Transplantation and Cellular Therapy. 2025.
Hallmark discoveries in the biology of non-Wilms tumour childhood kidney cancers. Nature Reviews Urology. 2025; 22(8):526-543.
Transition of care of youth with bone marrow failure conditions: Current practices, barriers, and burden- a survey of hematology-oncology providers in the US. Blood. 2025; 146(Supplement 1):4394.
TMOD-42. TREM2 modulates glioma-associated microglia and macrophage function in a genetic driver dependent manner. Neuro-Oncology. 2025; 27(Supplement_5):v483-v483.
Multicenter Study on Caregiver Experiences in Pediatric Hematopoietic Stem Cell Transplantation: Part II. Treatment Challenges, Communication Barriers, and Caregiver-Driven Approaches to Mitigation. Transplantation and Cellular Therapy. 2025; 31(8):590.e1-590.e16.
‘Blin’-ing Is Winning for Children With B-Cell Acute Lymphoblastic Leukemia. The Hematologist. 2025; 22(1).
Persisting Inflammatory and Fibrotic Gene Signatures Linking Acute Cellular Rejection to Chronic Lung Allograft Dysfunction. American Journal of Transplantation. 2025; 25(8):s823-s824.
(750) Single Cell Technology Shines LIGHT on Acute Lung Allograft Dysfunction. The Journal of Heart and Lung Transplantation. 2025; 44(4):s355.
(276) Gene Expression Alterations in Lung Allografts with ACR and After Treatment Compared to Never ACR. The Journal of Heart and Lung Transplantation. 2025; 44(4):s140.
Liquid Hydroxyurea (Xromi) for Children With Sickle Cell Anemia: A New Solution Compounding Existing Problems. Pediatric Blood and Cancer. 2026; e70143.
The feasibility of pharmacokinetic-based dosing of hydroxyurea for children with sickle cell anaemia in Uganda: Baseline results of the alternative dosing and prevention of transfusions trial. British Journal of Clinical Pharmacology. 2025; 91(6):1865-1872.
The feasibility of pharmacokinetic-based dosing of hydroxyurea for children with sickle cell anaemia in Uganda: Baseline results of the alternative dosing and prevention of transfusions trial. British Journal of Clinical Pharmacology. 2025; 91(6):1865-1872.
Pharmacokinetic-Guided Hydroxyurea to Reduce Transfusions in Ugandan Children with Sickle Cell Anemia: Study Design of the Alternative Dosing And Prevention of Transfusions Trial. Acta Haematologica. 2025; 148(2):208-219.
Hydroxyurea pharmacokinetics in children with sickle cell anemia across different global populations. Blood advances. 2026; 10(2):418-427.
Full assessment of clinical transfusion support (FACTS): A prospective Study to determine the burden of sickle cell anemia on pediatric blood transfusion use in a malaria-endemic region in Africa. Blood. 2025; 146(Supplement 1):300-300.
Measles vaccine-induced sero-protection among children with sickle cell anemia. Blood. 2025; 146(Supplement 1):1168.
23P Pre-diagnostic imaging reveals rapid emergence and progression of small cell carcinoma of the ovary hypercalcemic type (SCCOHT): Implications for monitoring carriers of SMARCA4 germline pathogenic variants (GPVs) ESMO Open. 2025; 10:104332.
Rehabilitation of the Pediatric Cancer Patient. In: Cancer Rehabilitation. Springer Publishing Company; 2025.
Granulocyte-Macrophage Colony Stimulating Factor Receptor Contributes to Plexiform Neurofibroma Initiation. Cancers. 2025; 17(5).
Feasibility, reliability, and accuracy of point-of-care measurements of fetal hemoglobin. Blood. 2025; 146(Supplement 1):4739-4739.
The modern use of hydroxyurea for children with sickle cell anemia. Haematologica. 2025; 110(5):1061-1073.
The Voxelotor Effect: Decreased Affinity for New Drugs for Sickle Cell Disease? Pediatric Blood and Cancer. 2025; 72(3):e31475.
Perception and Reality of Racial and Ethnic Workforce Diversity in the U.S HCT/CT Workforce: Results from the ASTCT-NMDP Access Initiative Survey. Transplantation and Cellular Therapy. 2025; 31(2):s342.
Pre-Transplant Complement Activation and Transplant-Associated Thrombotic Microangiopathy (TA-TMA) across Diagnostic Groups. Transplantation and Cellular Therapy. 2025; 31(2):s25-s26.
Real-world outcomes of infections following tisagenlecleucel in patients with B-cell ALL: a CIBMTR analysis. Blood advances. 2025; 9(21):5489-5500.
Impact of Extramedullary Disease at Diagnosis on Outcomes Post Allogeneic Hematopoietic Cell Transplant in Children and Young Adults With Acute Myeloid Leukemia: A CIBMTR Report. Transplantation and Cellular Therapy. 2025.
IMMU-79. An Updated Analysis of PD1 and Immune Pathway Activity in Malignant Pediatric Central Nervous System (CNS) Tumors: A Pediatric Brain Tumor Consortium (PBTC) Study, PBTC-N13. Neuro-Oncology. 2025; 27(Supplement_5):v221-v221.
Isotretinoin has no effect on event-free survival across high-risk medulloblastoma molecular groups when added to maintenance: A secondary analysis of the Children's Oncology Group ACNS0332 data. Neuro-Oncology Advances. 2025; 7(1):vdaf054.
137 Schwann Cell Glial Cell Line-Derived Neurotrophic Factor Signaling Influences Pain in Neurofibromatosis 1. Journal of Pain. 2025; 29:104935.
Outcomes Following Matched Sibling Donor Transplantation for Severe Combined Immunodeficiency: A Report from the PIDTC. Blood advances. 2025.
Novel anticoagulant-preservative solution maintained the hemostatic function of cold stored whole blood for 56 days. Transfusion. 2025; 65 Suppl 1(Suppl 1):S185-S192.
GENETIC RE-EVALUATION OF SDS-LIKE AND NEUTROPENIA CONDITIONS. EJC Paediatric Oncology. 2025; 6:100400.
Lymphoid malignancies in patients with Shwachman-Diamond syndrome. Blood. 2025; 145(21):2528-2532.
Long-term kidney outcomes in survivors of Wilms tumor: a single-center retrospective cohort study. Pediatric Nephrology. 2025; 40(5):1603-1611.
44: Capillary Malformation-Arteriovenous Malformation Syndrome: Proposed Consensus Guidelines. Journal of Investigative Dermatology. 2025; 145(3):e12.
Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, and Spectrum of Thrombotic Microangiopathy. In: Pediatric Nephrology. Springer Nature; 2025:1-41.
Validation of Pediatric Self-Report Pain Scales in Sub-Saharan Africa: A Systematic Review. Journal of Pain and Symptom Management. 2025; 69(2):e103-e112.
Imaging flow cytometry for detection of red blood cell inclusions: A quantitative approach to assess splenic filtration function in sickle cell disease. Blood. 2025; 146(Supplement 1):1188.
Advances on the genetic basis of red cell membrane disorders. Current Opinion in Hematology. 2025; 32(5):279-286.
Ontogeny of the spinal cord dorsal horn. Science. 2026; 391(6781):eadx5781.
Kymriah Real-World Effectiveness and Functional Persistence in Acute Lymphoblastic Leukemia (ALL) (KareALL): A Retrospective Analysis of the Effect of Current Tisagenlecleucel (tisa-cel) Manufacturing in Pediatric and Young Adult Patients (pts) with ALL. Transplantation and Cellular Therapy. 2025; 31(2):s219.
Outcomes with third-party virus-specific T cells after the use of single-antigen cell lines to predict HLA restriction. Blood advances. 2025; 9(24):6305-6313.
The Fanconi Anemia Pathway Inhibits mTOR Signaling and Prevents Accelerated Translation in Head and Neck Cancer Cells. Cancers. 2025; 17(15).
TA-TMA Evolution and Individual Susceptibility at the Proteome Level. Transplantation and Cellular Therapy. 2025; 31(2):s133.
Malignant peripheral nerve sheath tumors in neurofibromatosis type 1 arise from distinct nodular lesions: A retrospective imaging analysis. Neuro-Oncology Advances. 2025; 7(1):vdaf201.
Variation in Central Venous Catheter Practices and Complications after Stem Cell Transplant: A Multi-Center Survey of the Engraft Learning Network. Transplantation and Cellular Therapy. 2025; 31(2):s535-s536.
STAT6 gain-of-function disease: p.D519N is a new disease-causing variant that responds well to dupilumab treatment. Journal of Allergy and Clinical Immunology: Global. 2025; 4(3):100494.
Abstract 3155: High-throughput drug repositioning identifies dinaciclib as a potent osteosarcoma inhibitor. Cancer Research. 2025; 85(8_Supplement_1):3155-3155.
Clinicopathologic Features, Management, and Outcomes Of Pediatric CRTC1::TRIM11 and MED15::ATF1 Tumors With Spitzoid Morphology: A Case Series. Pediatric Blood and Cancer. 2025; 72(8):e31814.
Pharmacokinetic Monitoring of JAK Inhibitor and Tacrolimus for Safe and Effective Management of Graft-Versus-Host Disease After Pediatric Liver Transplantation. Pediatric Transplantation. 2025; 29(7):e70187.
Global consensus from the PHITT consortium surrounding an interim chemotherapy guidance for the treatment of children with hepatoblastoma, hepatocellular neoplasm – not otherwise specified (HCN-NOS), hepatocellular carcinoma and fibrolamellar carcinoma after trial closure. EJC Paediatric Oncology. 2025; 5:100235.
An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy. Transplantation and Cellular Therapy. 2025; 31(9):610-623.
Semaphorin 3A and 3F Promote Lumen Expansion in TIE2-Mutated Venous Malformation. Arteriosclerosis, Thrombosis, and Vascular Biology. 2025; 45(12):2243-2260.
The potential and risks of FLASH radiotherapy in pediatric patients. Neuro-Oncology Pediatrics. 2025.
Graduating Residents' Readiness for Unsupervised Practice. Pediatrics. 2025; 155(5).
TTLL4 glutamyltransferase is a therapeutic target for NPM1-mutated Acute Myeloid Leukemia. Blood. 2025; 146(Supplement 1):869-869.
Growth Patterns in Shwachman-Diamond Syndrome: Findings from the North American Shwachman-Diamond Syndrome Registry. Journal of Pediatrics. 2025; 287:114780.
Parallels in Canonical Developmental Signaling Pathways between Normal Development and the Tumor Microenvironment. Cold Spring Harbor Perspectives in Medicine. 2025.
Congenital Lymphatic Malformations. NeoReviews. 2025; 26(8):e578-e586.
Oxidative stress and hemoglobin-c denaturation drive hemoglobin SC pathophysiology and can be ameliorated by antioxidants and hydroxyurea. Blood. 2025; 146(Supplement 1):7.
A novel mouse model of hemoglobin SC disease reveals mechanisms underlying beneficial effects of hydroxyurea. Blood. 2025; 146(1):13-28.
Impact of Abatacept Inclusive Graft-Versus-Host Disease Prophylaxis in Pediatric Stem Cell Transplantation for Hemoglobinopathy. Transplantation and Cellular Therapy. 2025; 31(10):826.e1-826.e10.
Sinusoidal obstruction syndrome and other outcomes in pediatric patients with acute lymphoblastic leukemia who received inotuzumab ozogamicin before hematopoietic cell transplantation. Journal of Clinical Oncology. 2025; 43(16_suppl).
Challenges of Engaging Primary Care Providers in Specialized Telementoring Education About Sickle Cell Disease for Sickle Cell Specialists: Results from the Sickle Cell Disease Training and Mentoring Program for Primary Care Providers (STAMP) Project ECHO. AJPM Focus. 2025; 4(1):100304.
Clinical Utility of the Addition of Molecular Genetic Testing to Newborn Screening for Hemoglobinopathies for Confirmation of Alpha-Thalassemia Trait. International Journal of Neonatal Screening. 2025; 11(1).
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models. Molecular Therapy: Methods & Clinical Development. 2025; 33(2):101439.
Microsensor Array for the Electrochemical Analysis of Hydroxyurea in Blood Samples of Children Affected by Sickle Cell Anemia. Analytical Chemistry. 2025; 97(44):24399-24414.
PA2G4 as a Candidate Therapeutic Target in Pediatric Diffuse Gliomas. International Journal of Radiation Oncology Biology Physics. 2025; 123(1):e510.
Critical bleeding in adults and children with immune thrombocytopenia: a multicenter cohort study. Blood advances. 2025; 9(13):3238-3248.
Sociodemographic Characteristics of Patients Seen at Vascular Anomaly Centers in the United States. Pediatric Blood and Cancer. 2025; 72(6):e31661.
Benefits of hydroxyurea in hemoglobin SC: Results of the open-label phase of the prospective identification of variables as outcomes for treatment (PIVOT) trial. Blood. 2025; 146(Supplement 1):409.
Pharmacokinetic (PK)-guided vs weight-based dosing of hydroxyurea for tanzanian children with sickle cell anemia. Blood. 2025; 146(Supplement 1):2959-2959.
Pre-Transplant Endothelial Activation May Increase Individual Susceptibility Transplant-Associated Thrombotic Microangiopathy in Adults: A Report from the MIDAS Consortium. Transplantation and Cellular Therapy. 2025; 31(2):s133-s134.
Living on the edge: Uncommitted OPC-like cells drive glioblastoma invasiveness. Developmental Cell. 2025; 60(18):2383-2385.
Scrubbing Olympics: Next Steps. Transplantation and Cellular Therapy. 2025; 31(2):s489.
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Ependymoma. Journal of Molecular Pathology. 2025; 6(3):23.
The Novel Role of Pilra in BK Polyomavirus Cystitis after HSCT. Transplantation and Cellular Therapy. 2025; 31(2):s167.
Local application of sodium thiosulfate as an otoprotectant for cisplatin-exposed patients – A narrative literature review to explore the potential benefit for children with cancer. EJC Paediatric Oncology. 2025; 5:100211.
Optimization of IRAK1/4/pan-FLT3 kinase inhibitors as treatments for acute myeloid leukemia. Bioorganic and Medicinal Chemistry Letters. 2025; 129:130355.
Remaining Challenges in the Treatment of Relapsed Wilms Tumor: Children's Oncology Group and International Society of Paediatric Oncology Perspectives. Pediatric Blood and Cancer. 2025; 72(8):e31790.
Inbody Assessments Reveal Important Changes in Body Mass Composition during HCT. Transplantation and Cellular Therapy. 2025; 31(2):s146.
The Adverse Event Landscape of Stem Cell Transplant: Evidence for AGVHD Driving Early Transplant Associated Toxicities. Transplantation and Cellular Therapy. 2025; 31(2):109.e1-109.e13.
The Role of Second Hematopoietic Cell Transplantation (HCT2) in Patients with Severe Combined Immunodeficiency (SCID): A Report from the Primary Immune Deficiency Treatment Consortium (PIDTC) Transplantation and Cellular Therapy. 2025; 31(2):s92-s93.
Inhibitor development and clinical characteristics in children with severe hemophilia A in the ATHN 8 US cohort study. Blood Vessels, Thrombosis & Hemostasis. 2025; 2(3):100082.
Understanding monocyte-driven neuroinflammation in Alzheimer's disease using human cortical organoid microphysiological systems. Science Advances. 2025; 11(34):eadu2708.
Hemostatic derangements associated with cardiopulmonary bypass predict outcomes in pediatric patients undergoing corrective heart surgery. Journal of Thrombosis and Haemostasis. 2025; 23(2):492-503.
Development of disease-specific growth curves from Kenyan children with sickle cell anemia. Blood Global Hematology. 2025; 1(3):100031.
Comprehensive flow cytometry-based diagnosis of XIAP deficiency. Clinical and Experimental Immunology. 2025; 219(1).
1013-P: Estimates of Mean Red Blood Cell Age (MRBC) Obtained with Continuous Glucose Monitoring (CGM)-Based Computational Methods Are Comparable to Those Obtained with Stable Isotope (SI) In Vivo Measurements. Diabetes. 2025; 74(Supplement_1).
Essential Knowledge and Skills for the Practice of Pediatric Surgical Oncology: A Modified Delphi Consensus Study by the APSA Cancer Committee. Annals of Surgical Oncology. 2025; 32(8):5752-5771.
Characteristics of patients with liver tumors deemed ineligible for enrollment on Children's Oncology Group trial AHEP1531: An opportunity to expand inclusion criteria and improve outcome. Pediatric Blood and Cancer. 2025; 72(1):e31389.
Splenomegaly patterns and clinical impact in African children with sickle cell anemia on hydroxyurea treatment. Blood. 2025; 146(Supplement 1):2971-2971.
Modelling a pathological GSX2 variant that selectively alters DNA binding reveals hypomorphic mouse brain defects. Disease Models & Mechanisms. 2025; 18(2).
Scaffolding-dependent CASP1 constrains excessive cell-intrinsic inflammatory signaling in leukemia. Cell Chemical Biology. 2026; 33(1):59-73.e10.
Home Spirometry for Early Identification of Pulmonary Dysfunction After Hematopoietic Stem Cell Transplant in the Transpire Research Study. American Journal of Respiratory and Critical Care Medicine. 2025; 211(Abstracts):a7235-a7235.
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Pediatric High-Grade Glioma. Journal of Molecular Pathology. 2025; 6(3):17.
High incidence of severe TA-TMA increases mortality in adult allogeneic transplant recipients: a prospective MIDAS Consortium study. Blood. 2025; 146(5):638-646.
Pre-HCT Creatinine and Early Post-HCT Changes in Creatinine and Blood Pressure Associate with Severe Transplant Associated Thrombotic Microangiopathy (TA-TMA): A Report from the MIDAS (Microangiopathy, Endothelial Damage in Adults undergoing Stem cell transplantation) Consortium. Transplantation and Cellular Therapy. 2025; 31(2):s149.
Splicing regulatory dynamics for precision analysis and treatment of heterogeneous leukemias. Science Translational Medicine. 2025; 17(797):eadr1471.
Influence of the Incorporation of a Muslim Chaplain on Medical Decision-Making in a Pediatric Hematopoietic Cell Transplant Program. Transplantation and Cellular Therapy. 2025; 31(2):s355.
Characterizing the Gut Microbiome in Pediatric Patients Undergoing Hematopoietic Cell Transplant for a Non-Malignant Diagnosis. Transplantation and Cellular Therapy. 2025; 31(2):s316-s317.
Metabolic reprogramming in Fanconi anemia: Evidence of compromised glucose oxidation, enhanced ketogenesis, and metabolic inflexibility. Science Advances. 2025; 11(48):eady6117.
IRAK signaling in cancers: mechanisms, targeting, and clinical implications. Expert Opinion on Investigational Drugs. 2025; 34(10):775-792.
Targeting of IRAK4 and GSPT1 enhances therapeutic efficacy in AML via c-Myc destabilization. Leukemia. 2025; 39(9):2163-2173.
The IRAK4 long isoform as widely upregulated in non-splicesome mutated acute myeloid leukemia and as altered by hypomethylating agent therapy. Journal of Clinical Oncology. 2025; 43(16_suppl):6524-6524.
Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia. Cell Stem Cell. 2025; 32(1):38-52.e6.
Management, Trends, and Recommendations for Intra-abdominal Lymphatic Malformations: A Single-Center Retrospective Review. Journal of Pediatric Surgery. 2025; 60(4):162223.
Using cancer phenotype sex-specificity to enable unbiased penetrance estimation of SMARCA4 pathogenic variants for small cell carcinoma of the ovary, hypercalcemic type (SCCOHT). Genetics in Medicine. 2025; 27(1):101287.
BK polyomavirus genotype IV isolates from virus-specific T-cell therapy recipients. Microbiology Resource Announcements. 2025; 14(10):e0062525.
Single-nucleotide polymorphisms within the BK polyomavirus non-coding control region are genotype-associated. Microbiology Spectrum. 2025; 13(8):e0049425.
Insight Into Current BK Polyomavirus Research Efforts: BK Day Meeting 2024. Transplantation. 2025.
Factors associated with and kinetics of anti-IFN-α autoantibodies in RAG1/2 deficiency. Journal of Allergy and Clinical Immunology: Global. 2025; 4(3):100521.
TIFAB modulates metabolic pathways in KMT2A::MLLT3-induced AML through HNF4A. Blood advances. 2025; 9(4):844-855.
Abstract 4355964: Probiotic Extracellular Vesicles Stimulate Macrophage Efferocytosis to Limit Ischemia/Reperfusion-Induced Damage by Delivering 5s-rRNA-Derived Short RNA. Circulation. 2025; 152(Suppl_3):a4355964-a4355964.
Safety and efficacy of ten years of hydroxyurea treatment for young children with sickle cell anemia in Uganda. Blood. 2025; 146(Supplement 1):412.
Longitudinal changes in haemoglobin, iron stores, and inflammatory markers following surgery and in critical illness: an analysis from the Practical Anaemia Bundle for Sustained Blood Recovery randomised clinical trial. British Journal of Anaesthesia. 2025.
Single Cell Rnaseq Identifies Novel Graft Rejection Mechanisms and Resurgence of Recipient NK and T-Cells. Transplantation and Cellular Therapy. 2025; 31(2):s307-s308.
Interferon-induced cell senescence as a novel mechanism of eculizumab resistance in transplant associated thrombotic microangiopathy. Blood. 2025; 146(Supplement 1):509.
Beyond genotype: challenges in predicting disease risk for carriers of biallelic perforin variants. Blood. 2025; 145(25):2992-3006.
Differential regulation of fetal bone marrow and liver hematopoiesis by yolk-sac-derived myeloid cells. Nature Communications. 2025; 16(1):4427.
Lung Disease in Systemic Juvenile Idiopathic Arthritis: An Atypical Path to Diagnosis. American Journal of Respiratory and Critical Care Medicine. 2025; 211(Abstracts):a7865-a7865.
Effects of whole brain proton irradiation at conventional or ultra-high dose rate (FLASH), in adult male Sprague Dawley rats. Scientific Reports. 2025; 15(1):10602.
Pharmacogenomic synthetic lethal screens reveal hidden vulnerabilities and new therapeutic approaches for treatment of NF1-associated tumors. Molecular Cancer Therapeutics. 2025.
Malaria prophylaxis in sickle cell anaemia: some answers, more questions. The Lancet Infectious Diseases. 2025; 25(6):601-602.
Secondary Anticoagulation Use in Patients < 21 Years Old following Primary Anticoagulant Treatment for Provoked Venous Thromboembolism: Findings from the Kids-DOTT Trial. Seminars in Thrombosis and Hemostasis. 2025; 51(3):343-347.
Infection after CD19 chimeric antigen receptor T-cell therapy for large B-cell lymphoma: real-world analysis from CIBMTR. Blood advances. 2025; 9(21):5460-5472.
Single-Cell Multi-omics Analyses of Medulloblastoma. In: Translational Research Methods for Pediatric Brain Tumors. Springer Nature; 2025:1-21.
Intelligent sensing devices and systems for personalized mental health. Med X. 2025; 3(1).
Genetic and epigenetic regulation of Treg cells by autism-related CHD8 2064. Journal of Immunology. 2025; 214(Supplement_1).
In vivo expression of CD3/CD19 bispecific T-cell engager and α-PD-L1-Fc enables effective and durable immunotherapy for CD19+/PD-L1+ leukemia. Leukemia. 2025.
Genetic and epigenetic regulation of Treg cell fitness by autism-related chromatin remodeler CHD8. Cellular & Molecular Biology Letters. 2025; 30(1):36.
B-252 Functional Validation of Novel FAS Intronic and Splice Region VUS Through Minigene Assays. Clinical Chemistry. 2025; 71(Supplement_1):hvaf086.640.
Transcriptomic analysis uncovers hidden intronic and synonymous pathogenic variants in red cell membrane disorders. Blood. 2025; 146(Supplement 1):282-282.
National Trends in Dexrazoxane and Cardiovascular Health Care Utilization for Children With Acute Myeloid Leukemia. JAMA Oncology. 2025; 11(12):1545-1548.
TMOD-15. Native Histone H3.3K27M Sustains Progenitor Identity and Impairs Differentiation to Rewire Neural Development. Neuro-Oncology. 2025; 27(Supplement_5):v476-v477.
The Functional Characterization of Venous Thromboembolic Disease (FUVID) study: rationale, design, and methods of a prospective, observational, multicenter study to evaluate mechanisms of exercise intolerance and dyspnea following pediatric pulmonary embolism. Research and Practice in Thrombosis and Haemostasis. 2025; 9(1):102669.
Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: The REAL-HLH study. Blood advances. 2025.
First Relapse, Second Chance: Determinants of Survival After First Relapse in Pediatric Acute Lymphoblastic Leukemia. The Hematologist. 2025; 22(2).