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. Intra- and Inter-Organ Endothelial Cell Differences Contribute to TA-TMA through HMOX1, Hypoxia and Reactive Oxygen Species Signaling. Transplantation and Cellular Therapy.Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clinical Immunology. 2024; 261:109942.
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. Success and Feasibility of Multiple Breath Washout: Prospective, Multi-Center Results from the Transpire Study. Transplantation and Cellular Therapy.2024; 84:1464.
. Abstract 1464: Smoking carcinogen-induced inflammation promotes lung carcinogenesis via IRAK4 activation. Cancer Research.ATF7IP2/MCAF2 directs H3K9 methylation and meiotic gene regulation in the male germline. Genes and Development. 2024; 38:115-130.
.Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. The Lancet Haematology. 2024; 11:e228-e239.
.Immunophenotypic predictors of influenza vaccine immunogenicity in pediatric hematopoietic cell transplant recipients. Blood Advances. 2024; 8:1880-1892.
.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.
.A toxicology study of Csf2ra complementation and pulmonary macrophage transplantation therapy of hereditary PAP in mice. Molecular Therapy-Methods & Clinical Development. 2024; 32:101213.
.Postinfectious Pulmonary Complications: Establishing Research Priorities to Advance the Field: An Official American Thoracic Society Workshop Report. Annals of the American Thoracic Society. 2024; 21:1219-1237.
.2024; 26:0.
. NFS-24. TUMOR VOLUME IN NEWLY DIAGNOSED OPTIC PATHWAY GLIOMAS ASSOCIATED WITH NF1 (NF1-OPG): PRELIMINARY RESULTS FROM THE INTERNATIONAL MULTICENTER NF1-OPG NATURAL HISTORY STUDY. Neuro-Oncology.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; 84:3092.
. Abstract 3092: MICRO-TAG: A novel fluorescence-based real-time cellular target engagement platform for drug discovery. Cancer Research.Neural responses to decision-making in suicide attempters with youth major depressive disorder. NeuroImage-Clinical. 2024; 43:103667.
.nnU-Net-based Segmentation of Tumor Subcompartments in Pediatric Medulloblastoma Using Multiparametric MRI: A Multi-institutional Study. Radiology: Artificial Intelligence. 2024; 6:e230115.
.Dysregulated innate immune signaling cooperates with RUNX1 mutations to transform an MDS-like disease to AML. iScience. 2024; 27:109809.
.Fanconi anemia neuroinflammatory syndrome: brain lesions and neurologic injury in Fanconi anemia. Blood Advances. 2024; 8:3027-3037.
.Adult microglial TGFβ1 is required for microglia homeostasis via an autocrine mechanism to maintain cognitive function in mice. Nature Communications. 2024; 15:5306.
.Real-world use of tisagenlecleucel in children and young adults with relapsed or refractory B-cell lymphomas. Blood Advances. 2024; 8:4164-4168.
.Cardiac Dysfunction in Children and Young Adults Treated With MEK Inhibitors: A Retrospective, Single-Center Study. JACC: CardioOncology. 2024; 6:794-796.
.Immortalised chronic myeloid leukemia (CML) derived mesenchymal stromal cells (MSCs) line retains the immunomodulatory and chemoprotective properties of CML patient-derived MSCs. Cellular Signalling. 2024; 116:111067.
.Soluble glycoprotein VI predicts abdominal aortic aneurysm growth rate and is a novel therapeutic target. Blood. 2024; 144:1663-1678.
.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.
.Return to school practices after hematopoietic cell transplantation: a survey of transplant centers in the United States. Bone Marrow Transplantation. 2024; 59:653-659.
.Langerhans cell histiocytosis: NACHO update on progress, chaos, and opportunity on the path to rational cures. Cancer. 2024; 130:2416-2439.
.14q22.3 duplication including OTX2 in a girl with medulloblastoma: A case report with literature review. American Journal of Medical Genetics, Part A. 2024; 194:e63604.
.Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation. Blood. 2024; 143:872-881.
.Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study. Pediatric Blood and Cancer. 2024; 71:e30779.
.Chemical genetic screens reveal defective lysosomal trafficking as synthetic lethal with NF1 loss. Journal of Cell Science. 2024; 137:jcs262343.
.In vivo models of subclonal oncogenesis and dependency in hematopoietic malignancy. Cancer Cell. 2024; 42:1955-1969.e7.
.2024; 26:0.
. QOL-06. RECURRENCE PATTERNS AND SURVEILLANCE IMAGING IN PEDIATRIC BRAIN TUMOR SURVIVORS. Neuro-Oncology.Recurrence Patterns and Surveillance Imaging in Pediatric Brain Tumor Survivors. Journal of Pediatric Hematology/Oncology. 2024; 46:e227-e232.
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. MDB-45. THE PTBP2 SPLICING FACTOR IS ESSENTIAL IN GROUP 3/4 MEDULLOBLASTOMA. Neuro-Oncology.Time to therapy and safety of testicular tissue cryopreservation in children undergoing gonadotoxic treatment or hematopoietic stem cell transplant. Journal of Pediatric Urology. 2024; 20:747.e1-747.e7.
.2024; 26:0.
. MDB-09. WNT AND GROUP 4 MEDULLOBLASTOMAS OF EARLY CHILDHOOD TREATED WITH HIGH DOSE CHEMOTHERAPY AND CRANIOSPINAL IRRADIATION SPARING APPROACH. Neuro-Oncology.Springer Nature; Springer Nature; 2024.
. Early Complications of Endothelial Origin. The EBMT Handbook. :Hemoglobin concentration and body mass index are determinants of plasma von Willebrand factor and factor VIII levels. Thrombosis Research. 2024; 240:109061.
.SETDB1 suppresses NK cell-mediated immunosurveillance in acute myeloid leukemia with granulo-monocytic differentiation. Cell Reports. 2024; 43:114536.
.Toxicity profile of high-dose methotrexate in young children with central nervous system tumors. Pediatric Blood and Cancer. 2024; 71:e31213.
.Integrating education for clinical practice change. The Clinical Teacher. 2024; 21:e13753.
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. Abstract We146: CHD8 is Essential for Postnatal Heart Function through Preserving Mitochondrial Integrity. Circulation Research.2024; 26:0.
. TRLS-09. CONNECT1903: A PILOT AND SURGICAL STUDY OF LAROTRECTINIB FOR TREATMENT OF CHILDREN WITH NEWLY DIAGNOSED HGG WITH NTRK FUSION (NCT04655404). Neuro-Oncology.2024; 262:110010.
. 68 LRBA dysfunction: a new diagnostic entity caused by biallelic LRBA missense variants results in reduced CTLA-4 expression and autoimmunity. Clinical Immunology.Efficacy of T-cell assays for the diagnosis of primary defects in cytotoxic lymphocyte exocytosis. Blood. 2024; 144:873-887.
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. Uncommon BK polyomaviruses detected in allogeneic hematopoietic cell transplant recipients. Microbiology Resource Announcements.Uncommon BK polyomaviruses detected in allogeneic hematopoietic cell transplant recipients. Editor, Matthijnssens J. Microbiology Resource Announcements. 2024; 13:e0000524.
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. An in Vitro Patient-Derived Model of Bronchiolitis Obliterans Syndrome Yields New Insight into Steroid Sensitivity and Resistance. Transplantation and Cellular Therapy.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.
.Ruxolitinib Pharmacokinetics and Pharmacodynamics in Children with Acute and Chronic Graft-versus-Host Disease. Transplantation and Cellular Therapy. 2024; 30:528.e1-528.e12.
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. Case report: Childhood erythrocytosis due to hypermanganesemia caused by homozygous SLC30A10 mutation.Evolution of Proton Radiation Therapy Brainstem Constraints on the Pediatric Proton/Photon Consortium Registry. Practical Radiation Oncology. 2024; 14:e507-e514.
.Successes and pitfalls in orphan drug development for sickle cell disease. Blood Advances. 2024; 8:2455-2465.
.Dabrafenib and trametinib in Langerhans cell histiocytosis and other histiocytic disorders. Haematologica: the hematology journal. 2024; 109:1137-1148.
.Zinc-Finger Protein ZFP488 Regulates the Timing of Oligodendrocyte Myelination and Remyelination. The Journal of neuroscience : the official journal of the Society for Neuroscience. 2024; 44:e0141242024.
.Chemotherapy resistance in acute myeloid leukemia is mediated by A20 suppression of spontaneous necroptosis. Nature Communications. 2024; 15:9189.
.Body Image Concerns in Adolescent and Young Adult Oncology Patients within Six Months of Upfront Diagnosis. Journal of Adolescent and Young Adult Oncology. 2024; 13:792-798.
.In Pursuit of Optimal Outcomes: A Framework for Quality Standards in Immune Effector Cell Therapy. Transplantation and Cellular Therapy. 2024; 30:942-954.
.Feeding critically ill patients at the right time of day. Critical Care (UK). 2024; 28:206.
.FLASH radiotherapy for the treatment of symptomatic bone metastases in the thorax (FAST-02): protocol for a prospective study of a novel radiotherapy approach. Radiation Oncology. 2024; 19:34.
.Temporal changes in treatment and late mortality and morbidity in adult survivors of childhood glioma: a report from the Childhood Cancer Survivor Study. Nature Cancer. 2024; 5:590-600.
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. Longitudinal serum chloride as a marker for poor prognosis in severely ill COVID-19 patients: A joint model approach. Brazilian Journal of Biometrics.A Phase 0/I Pharmacokinetic and Pharmacodynamics and Safety and Tolerability Study of Letrozole in Combination with Standard Therapy in Recurrent High-Grade Gliomas. Clinical Cancer Research. 2024; 30:2068-2077.
.2024; 12:100227.
. O114 / #164 DOSE TO ORGANS AT RISK IMPACTS HEALTH-RELATED QUALITY OF LIFE OUTCOMES FOR CHILDREN WITH BRAIN TUMORS. International Journal of Particle Therapy.Macrophage memories of early-life injury drive neonatal nociceptive priming. Cell Reports. 2024; 43:114129.
.The mediating role of family functioning between childhood trauma and depression severity in major depressive disorder and bipolar disorder. Journal of Affective Disorders. 2024; 365:443-450.
.2024; 84:5912.
. Abstract 5912: Rational design of a small-molecule inhibitor targeting the allosteric site of sos1 in oncogenic k-ras pancreatic cancer. Cancer Research.Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. Journal of Allergy and Clinical Immunology. 2024; 153:287-296.
.Prophylactic VSTs: a promising start but still work to do. Blood Advances. 2024; 8:4738-4739.
.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.
.Pediatric spindle cell/sclerosing rhabdomyosarcoma with FUS-TFCP2 fusion: a case report and literature review. Translational Pediatrics. 2024; 13:178-191.
.2024; 26:0.
. LGG-05. REPORT ON PEDIATRIC BRAIN TUMOR CONSORTIUM (PBTC)-029B, A PROSPECTIVE PHASE 2 CLINICAL TRIAL OF SELUMETINIB IN TREATING YOUNG PATIENTS WITH RECURRENT/PROGRESSIVE LOW-GRADE GLIOMA: NEW RESULTS ON STRATA 2, 5, AND 6 WITH UPDATED LONG-TERM SURVIVAL OUTCOMES ON STRATA 1, 3, AND 4. Neuro-Oncology.2024; 35:10.1681/asn.20241j3dwf9j.
. Single-Center Case Series of Congenital Anomalies of the Kidney and Urinary Tract and Hepatoblastoma. Journal of the American Society of Nephrology : JASN.A core outcome domain set to assess cutaneous neurofibromas related to neurofibromatosis type 1 in clinical trials. British Journal of Dermatology. 2024; 190:216-225.
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. 0820 Challenges to Pediatric Patients Sleeping Well in the Hospital: Environmental and Practice Considerations. Sleep.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; 26:0.
. NFS-21. DEMOGRAPHIC, CLINICAL AND IMAGING CHARACTERISTICS OF NEWLY DIAGNOSED OPTIC PATHWAY GLIOMAS ASSOCIATED WITH NF1: RESULTS FROM THE INTERNATIONAL MULTICENTER NF1-OPG NATURAL HISTORY STUDY. Neuro-Oncology.2024; 3:1339870.
. Research and clinical updates on IRAK4 and its roles in inflammation and malignancy: themes and highlights from the 1st symposium on IRAK4 in cancer.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.
.Histogram Analysis of Apparent Diffusion Coefficient Maps Provides Genotypic and Pretreatment Phenotypic Information in Pediatric and Young Adult Rhabdomyosarcoma. Academic Radiology. 2024; 31:2550-2561.
.EZH2 is a key component of hepatoblastoma tumor cell growth. Pediatric Blood and Cancer. 2024; 71:e30774.
.Overlap chronic GVHD is associated with adverse survival outcomes compared to classic chronic GVHD. Bone Marrow Transplantation. 2024; 59:680-687.
.Gestational diabetes in mice induces hematopoietic memory that affects the long-term health of the offspring. The Journal of Clinical Investigation. 2024; 134:e169730.
.Virus-Specific T Cells for the Treatment of Systemic Infections Following Allogeneic Hematopoietic Cell and Solid Organ Transplantation. Journal of the Pediatric Infectious Diseases Society. 2024; 13:S49-S57.
.2024; 30:s89.
. Preliminary Results of a Phase 2a Clinical Trial to Evaluate Safety, Tolerability and Antiviral Activity of Intravenous Brincidofovir (BCV IV) in Immunocompromised Patients with Adenovirus Infection. Transplantation and Cellular Therapy.Part 5: Allogeneic HSCT in refractory SJIA with lung disease; recent cases from centers in North America & Europe. Pediatric Rheumatology Online Journal. 2024; 21:86.
.Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study. Journal of Allergy and Clinical Immunology. 2024; 153:1423-1431.e2.
.Inhibition of Histone Deacetylase Activity Increases Cisplatin Efficacy to Eliminate Metastatic Cells in Pediatric Liver Cancers. Cancers. 2024; 16:2300.
.Cellular origin and molecular mechanisms of lung metastases in patients with aggressive hepatoblastoma. Hepatology Communications. 2024; 8:e0369.
.Cystatin C Outperforms Creatinine in Predicting Cefepime Clearance in Pediatric Stem Cell Transplant Recipients. Transplantation and Cellular Therapy. 2024; 30:614.e1-614.e11.
.Cefepime-Associated Neurotoxicity in a Pediatric Patient With Stage V Chronic Kidney Disease. Journal of Pharmacy Practice. 2024; 37:243-247.
.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.
.Ionized Magnesium Correlates With Total Blood Magnesium in Pediatric Patients Following Kidney Transplant. Annals of Laboratory Medicine. 2024; 44:21-28.
.Inhibition of RHOA activity preserves the survival and hemostasis function of long-term cold-stored platelets. Blood. 2024; 144:1732-1746.
.Diagnostic guidelines for familial hemophagocytic lymphohistiocytosis revisited. Blood. 2024; 144:2308-2318.
.Discovery of IRAK1/4/pan-FLT3 Kinase Inhibitors as Treatments for Acute Myeloid Leukemia. ACS Medicinal Chemistry Letters. 2024; 15:1843-1851.
.DOR activation in mature oligodendrocytes regulates α-ketoglutarate metabolism leading to enhanced remyelination in aged mice. Nature Neuroscience. 2024; 27:2073-2085.
.2024; 30:s344-s345.
. HSCT Patient and Family Education Concept Identification. Transplantation and Cellular Therapy.2024; 30:s43-s44.
. Chronic Graft Versus Host Disease Adversely Affects School Performance and Quality of Life in Children and Young Adults. Transplantation and Cellular Therapy.Topical ruxolitinib is promising as sole or adjunctive therapy in treating maculopapular rash of acute and chronic skin GVHD. Bone Marrow Transplantation. 2024; 59:425-427.
.Shared patterns of glial transcriptional dysregulation link Huntington's disease and schizophrenia. Brain: a journal of neurology. 2024; 147:3099-3112.
.Hereditary hemorrhagic telangiectasia - pediatric review. Current Opinion in Pediatrics. 2024; 36:592-598.
.2024; 26:e20-e21.
. CMV SEROSTATUS IS MOST IMPORTANT TO SELECT DONORS OF MONONUCLEAR CELL PRODUCTS AIMED TO THE GENERATION OF ANTI-CMV VIRUS-SPECIFIC T CELL (VSTS) PRODUCTS. Cytotherapy (Informa).2024; 26:0.
. MDB-42. RADIOMIC TEXTURAL AND MORPHOLOGICAL-BASED FEATURES TO RISK-STRATIFY PEDIATRIC MEDULLOBLASTOMA PATIENTS. Neuro-Oncology.2024; 26:0.
. MDB-40. DEFORMATION-HETEROGENEITY RADIOMIC MEASURES TO RISK-STRATIFY PEDIATRIC MEDULLOBLASTOMA PATIENTS: A PRELIMINARY ANALYSIS. Neuro-Oncology.A Radiomic Approach for Evaluating Intra-Subgroup Heterogeneity in SHH and Group 4 Pediatric Medulloblastoma: A Preliminary Multi-Institutional Study. Cancers. 2024; 16:2248.
.Inhibition of NRF2 signaling overcomes acquired resistance to arsenic trioxide in FLT3-mutated Acute Myeloid Leukemia. Annals of Hematology. 2024; 103:1919-1929.
.A prospective multi-institutional study of eculizumab to treat high-risk stem cell transplantation-associated TMA. Blood. 2024; 143:1112-1123.
.Late effects after allogeneic haematopoietic cell transplantation in children and adolescents with non-malignant disorders: a retrospective cohort study. The Lancet Child and Adolescent Health. 2024; 8:740-750.
.A randomized phase 2 trial of oral vitamin A for graft-versus-host disease in children and young adults. Blood. 2024; 143:1181-1192.
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. Vitamin A to Keep GVHD Away: Single Oral High-Dose Vitamin A Before Bone Marrow Transplant Reduces Graft-Versus-Host Disease in Pediatric and Young Adult Patients.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.
.Third-party virus-specific T cells for the treatment of double-stranded DNA viral reactivation and posttransplant lymphoproliferative disease after solid organ transplant. American Journal of Transplantation. 2024; 24:1634-1643.
.Biomolecular condensates: hubs of Hippo-YAP/TAZ signaling in cancer. Trends in Cell Biology. 2024; 34:566-577.
.Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. Blood Advances. 2024; 8:5529-5538.
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. Longitudinal prospective study of Sturge–Weber syndrome urine angiogenic factors and neurological outcome.Social determinants of health predict health outcomes following pediatric allogeneic hematopoietic stem cell transplant. Pediatric Blood and Cancer. 2024; 71:e30892.
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. Outcomes Following Radiation Therapy (RT) for Embryonal Tumor with Multilayered Rosettes (ETMR): Results from the Pediatric Proton/Photon Consortium Registry (PPCR). International Journal of Radiation: Oncology - Biology - Physics.Red cell abnormalities characterized by ektacytometry in children with cholestasis. Pediatric Research. 2024; 95:1035-1040.
.Patients and mice with deficiency in the SNARE protein SYNTAXIN-11 have a secondary B cell defect. The Journal of Experimental Medicine. 2024; 221:e20221122.
.2024; 30:s291.
. Longitudinal Bone Health in Patients with Fanconi Anemia and Other Major Inherited Bone Marrow Failure Syndromes. Transplantation and Cellular Therapy.2024; 30:s122.
. Invasive Fungal Infections Are Rare in Pediatric and Young Adult Autologous Hematopoietic Stem Cell Transplant Patients. Transplantation and Cellular Therapy.Levofloxacin prophylaxis in pediatric oncology and hematopoietic stem cell transplantation: a literature review. Pediatric Hematology-Oncology (Informa). 2024; 41:432-448.
.Infrequent fractures and resilient bone mineral density: bone health in patients with Fanconi anemia. Haematologica: the hematology journal. 2024; 109:3435-3438.
.Invasive fungal infections are rare in pediatric and young adult autologous hematopoietic stem cell transplant patients. Pediatric Blood and Cancer. 2024; 71:e31336.
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. Ceramides Are Elevated in Transplant-Associated Thrombotic Microangiopathy in Hematopoietic Stem Cell Transplant Recipients and Are a Druggable Target for Prophylaxis. Transplantation and Cellular Therapy.A road map for the treatment of pediatric diffuse midline glioma. Cancer Cell. 2024; 42:1-5.
.Low-grade glioma in children with neurofibromatosis type 1: surveillance, treatment indications, management, and future directions. Child's Nervous System. 2024; 40:3241-3250.
.2024; 26:0.
. NFS-08. CLINICAL AND MOLECULAR FEATURES OF HIGH-GRADE GLIOMA IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1: A PRELIMINARY ANALYSIS. Neuro-Oncology.Absence of PNH-clones in DDX41mutant-GPS aids in their distinction from acquired BM failure syndromes. Leukemia Research: clinical and laboratory studies. 2024; 145:107561.
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. Hypusine Modulation in Hematopoietic Stem Cell Transplant Recipients: A Novel Insight into GI Gvhd Mechanisms. Transplantation and Cellular Therapy.In vivo ablation of NF-κB cascade effectors alleviates disease burden in myeloproliferative neoplasms. Blood. 2024; 143:2414-2424.
.2024; 26:viii180.
. PATH-13. MOLECULAR AND BIOLOGY CORRELATIVE RESULTS FROM THE PHASE 1B TRIAL OF UNESBULIN (PTC596) WITH RADIOTHERAPY IN CHILDREN NEWLY-DIAGNOSED WITH DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG) AND HIGH-GRADE GLIOMA (HGG): A REPORT FROM THE COLLABORATIVE NETWORK FOR NEURO-ONCOLOGY CLINICAL TRIALS (CONNECT). Neuro-Oncology.Clinical outcomes of hypomethylating agents and venetoclax in newly diagnosed unfit and relapsed/refractory paediatric, adolescent and young adult acute myeloid leukaemia patients. British Journal of Haematology. 2024; 205:1055-1066.
.A case of co-occurring acute myeloid leukemia and relapsed diffuse large B-cell lymphoma in a young adult with Shwachman-Diamond syndrome. Pediatric Blood and Cancer. 2024; 71:e31137.
.Eculizumab for Shiga-toxin-induced hemolytic uremic syndrome in adults with neurological involvement. ejHaem. 2024; 5:548-553.
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. A Standardized Approach to Bone Health Screening in Pediatric HSCT and the Study of Bone Health Biomarkers. Transplantation and Cellular Therapy.Pancytopenia in the Emergency Department. The Journal of Pediatrics. 2024; 272:114111.
.2024; 26:0.
. LGG-37. A PHASE III STUDY COMPARING TWO CARBOPLATIN CONTAINING REGIMENS FOR CHILDREN AND YOUNG ADULTS WITH PREVIOUSLY UNTREATED LOW-GRADE GLIOMA. Neuro-Oncology.Infectious Diseases Evaluation of the Child With Suspected Hemophagocytic Lymphohistiocytosis. Journal of the Pediatric Infectious Diseases Society. 2024; 13:220-227.
.Tumor-derived RHOA mutants interact with effectors in the GDP-bound state. Nature Communications. 2024; 15:7176.
.Intrinsic link between PGRN and Gba1 D409V mutation dosage in potentiating Gaucher disease. Human Molecular Genetics. 2024; 33:1771-1788.
.Small-molecule-induced epigenetic rejuvenation promotes SREBP condensation and overcomes barriers to CNS myelin regeneration. Cell. 2024; 187:2465-2484.e22.
.Navigating the Critical Translational Questions for Implementing FLASH in the Clinic. Seminars in Radiation Oncology. 2024; 34:351-364.
.Race and Ethnic Group Enrollment and Outcomes for Wilms Tumor: Analysis of the Current Era Children's Oncology Group Study, AREN03B2. Journal of the American College of Surgeons. 2024; 238:733-749.
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. Single Cell Rnaseq Identifies Chemokine Responses to BK Polyomavirus Infection and Novel Contribution of MAIT Cells. Transplantation and Cellular Therapy.Vorinostat, temozolomide or bevacizumab with irradiation and maintenance BEV/TMZ in pediatric high-grade glioma: A Children's Oncology Group Study. Neuro-Oncology Advances. 2024; 6:vdae035.
.Methodological issues in the evaluation of cold snare endoscopic mucosal resection for colon polyps. Endoscopy. 2024; 56:79.
.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.
.Salvage Therapy and Allogeneic Hematopoietic Cell Transplantation for the Severe Cytokine Storm Syndrome of Hemophagocytic Lymphohistiocytosis. Advances in Experimental Medicine and Biology. 2024; 1448:611-622.
.Impact of Multiple Beams on the FLASH Effect in Soft Tissue and Skin in Mice. International Journal of Radiation: Oncology - Biology - Physics. 2024; 118:253-261.
.Metabolic reprogramming regulated by TRAF6 contributes to the leukemia progression. Leukemia. 2024; 38:1032-1045.
.Characteristics and outcomes of children, adolescent, and young adult patients with myelodysplastic neoplasms: A single-center retrospective analysis. Leukemia Research: clinical and laboratory studies. 2024; 144:107563.
.COVID-19 Vaccination in Patients with Inborn Errors of Immunity Reduces Hospitalization and Critical Care Needs Related to COVID-19: a USIDNET Report. Journal of Clinical Immunology. 2024; 44:86.
.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.
.Feasibility pilot trial of a tailored medication adherence-promotion intervention for adolescents and young adults with cancer: Study design and protocol. Contemporary Clinical Trials. 2024; 139:107483.
.Antiresorptive and anti-angiogenic drug therapy in the pediatric population with reference to medication-related osteonecrosis of the jaw. International Journal of Oral and Maxillofacial Surgery. 2024; 53:496-502.
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. DIPG-17. DYSREGULATED PURINE METABOLISM IN A LETHAL CHILDHOOD BRAINSTEM TUMOR REVEALS NEW TREATMENT STRATEGIES WITH OLD DRUGS. Neuro-Oncology.Neuromuscular and cardiovascular phenotypes in paediatric titinopathies: a multisite retrospective study. Journal of medical genetics. 2024; 61:356-362.
.High symptom burden in female X-linked chronic granulomatous disease carriers. Clinical Immunology. 2024; 268:110364.
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. IMMU-17. A PHASE I STUDY OF INTRATUMORAL/PERITUMORAL HERPES SIMPLEX VIRUS-1 MUTANT HSV1716 IN PATIENTS WITH REFRACTORY OF RECURRENT HIGH-GRADE GLIOMAS: A PEDIATRIC BRAIN TUMOR CONSORTIUM STUDY. Neuro-Oncology.Germline PTCH1: c.361_362insAlu alteration identified by comprehensive exome and RNA sequencing in a patient with Gorlin syndrome. American Journal of Medical Genetics, Part A. 2024; 194:e63788.
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. 5. Human Papillomavirus Vaccination Uptake in Adolescent Survivors of Bone Marrow Transplant. Journal of Pediatric and Adolescent Gynecology.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.
.Consensus framework for conducting phase I/II clinical trials for children, adolescents, and young adults with pediatric low-grade glioma: Guidelines established by the International Pediatric Low-Grade Glioma Coalition Clinical Trial Working Group. Neuro-Oncology. 2024; 26:407-416.
.Thrombotic risk associated with gender-affirming hormone therapy. Journal of Thrombosis and Haemostasis. 2024; 22:2129-2139.
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