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. Longitudinal analysis of electrolyte prolife in intensive care COVID-19 patients. Egyptian Journal of Anaesthesia.Fractionated plasma N-glycan profiling of novel cohort of ATP6AP1-CDG subjects identifies phenotypic association. Journal of Inherited Metabolic Disease. 2023; 46:300-312.
.Hydroxyurea with dose escalation for primary stroke risk reduction in children with sickle cell anaemia in Tanzania (SPHERE): an open-label, phase 2 trial. The Lancet Haematology. 2023; 10:e261-e271.
.Viral specific T cell therapy in kidney transplant recipients - A single-center experience. Transplant Infectious Disease. 2023; 25:e14179.
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. Clinical Characteristics, Management, and Allogeneic Hematopoietic Cell Transplantation of Patients with Toll-like Receptor 8 Gain-of-Function Mutations. Blood.Proceedings of the 2023 Second Annual ASTCT-NMDP ACCESS Initiative Workshop. Transplantation and Cellular Therapy. 2023; 29:739-746.
.Assessing Medicaid Coverage for Hematopoietic Cell Transplantation and Chimeric Antigen Receptor T Cell Therapy: A Project from the American Society for Transplantation and Cellular Therapy and the National Marrow Donor Program ACCESS Initiative. Transplantation and Cellular Therapy. 2023; 29:713-720.
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. Immunogenicity and Safety of Alternative Influenza Vaccination Strategies in Repeated Seasons in Pediatric Hematopoietic-Cell Transplant Recipients. Journal of the Pediatric Infectious Diseases Society.Tisagenlecleucel utilisation and outcomes across refractory, first relapse and multiply relapsed B-cell acute lymphoblastic leukemia: a retrospective analysis of real-world patterns. EClinicalMedicine. 2023; 65:102268.
.Optimized vitamin D repletion with oral thin film cholecalciferol in patients undergoing stem cell transplant. Blood Advances. 2023; 7:4555-4562.
.Reduced-Intensity Conditioning Mitigates Risk for Primary Ovarian Insufficiency but Does Not Decrease Risk for Infertility in Pediatric and Young Adult Survivors of Hematopoietic Stem Cell Transplantation. Transplantation and Cellular Therapy. 2023; 29:130.e1-130.e8.
.Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. Journal of Allergy and Clinical Immunology. 2023; 152:1619-1633.e11.
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. Human Hematopoietic Stem Cells Have Altered Mitochondrial Activity after Stem Cell Transplantation. Blood.Systematic Review of Signs and Symptoms Associated with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy. Transplantation and Cellular Therapy. 2023; 29:282.e1-282.e9.
.Incidence of Bloodstream Infections after Hematopoietic Stem Cell Transplantation for Hurler Syndrome. Transplantation and Cellular Therapy. 2023; 29:707.e1-707.e4.
.The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. Journal of Allergy and Clinical Immunology. 2023; 151:547-555.e5.
.Lorlatinib use in an infant with thalamic ALK-positive histiocytosis. Pediatric Blood and Cancer. 2023; e30378.
.Urinary Biomarkers for the Assessment of Acute Kidney Injury of Pediatric Sickle Cell Anemia Patients Admitted for Severe Vaso-occlusive Crises. Journal of Pediatric Hematology/Oncology. 2023; 45:309-314.
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. 356 Children Under 2 Years Old Treated with Ruxolitinib for Acute and Chronic Graft-Versus-Host-Disease Demonstrate Variable Pharmacokinetics. Transplantation and Cellular Therapy.Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults. Transplantation and Cellular Therapy. 2023; 29:305-310.
.Durable remissions achieved with reinfusion of CD19-directed CAR-T despite failure to induce or maintain B-cell aplasia and single-center experience with reinfusion of tisagenlecleucel. Pediatric Blood and Cancer. 2023; 70:e30271.
.Alemtuzumab and CXCL9 levels predict likelihood of sustained engraftment after reduced-intensity conditioning HCT. Blood Advances. 2023; 7:3725-3734.
.Use of extracorporeal immunomodulation in a toddler with hemophagocytic lymphohistiocytosis and multisystem organ failure. Pediatric Nephrology. 2023; 38:927-931.
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. 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. Blood.Center volume effect on acute cellular rejection and outcomes in pediatric lung transplant recipients. The Journal of Heart and Lung Transplantation. 2023; 42:1030-1039.
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. Sex and Sex Hormones Matter in Lung Transplant Outcomes. The Journal of Heart and Lung Transplantation.Changing racial and ethnic differences for lung transplantation in cystic fibrosis. Pediatric Transplantation. 2023; 27:e14404.
.Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome. Transplantation and Cellular Therapy. 2023; 29:438.e1-438.e16.
.Posttransplant complications in patients with marrow failure syndromes: are we improving long-term outcomes?. Hematology-American Society of Hematology Education Program. 2023; 2023:141-148.
.Alpha-1 antitrypsin limits neutrophil extracellular trap disruption of airway epithelial barrier function. Frontiers in Immunology. 2023; 13:1023553.
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. Reduced-intensity conditioning mitigates risk for primary ovarian insufficiency but does not decrease risk for infertility in pediatric and young adult survivors of hematopoietic stem cell transplantation.Using participatory research to identify actionable facilitators and barriers to effective inpatient interdisciplinary communication. Journal of hospital medicine (Online). 2023; 18:130-138.
.Adopting Model-Informed Precision-Dosing for Eculizumab in Transplant Associated-Thrombotic Microangiopathy to Gene Therapies. Clinical Pharmacology and Therapeutics. 2023; 114:511-514.
.Reeling in complement in transplant-associated thrombotic microangiopathy: You're going to need a bigger boat. American Journal of Hematology. 2023; 98 Suppl 4:S57-S73.
.How do we improve the translation of new evidence into the practice of hematopoietic cell transplantation and cellular therapy?. Blood Reviews. 2023; 60:101079.
.Transcriptome analysis in acute gastrointestinal graft-versus host disease reveals a unique signature in children and shared biology with pediatric inflammatory bowel disease. Haematologica: the hematology journal. 2023; 108:1803-1816.
.No impact of CD34+ cell dose on outcome among children undergoing autologous hematopoietic stem cell transplant for high-risk neuroblastoma. Bone Marrow Transplantation. 2023; 58:1390-1393.
.Effect of Autograft CD34+ Dose on Outcome in Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors. Transplantation and Cellular Therapy. 2023; 29:380.e1-380.e9.
.Extracorporeal membrane oxygenation bridge to pediatric lung transplantation: Modern era analysis. Pediatric Transplantation. 2023; 27:e14570.
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. 166 Reduced Sphingosine-1-Phosphate Leads to Hypoxemia and Lung Injury after Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy.Gonadal function in pediatric Fanconi anemia patients treated with hematopoietic stem cell transplant. Haematologica: the hematology journal. 2023; 108:2358-2368.
.Increased Body Mass Index Augments Endothelial Injury and Clinical Outcomes after Hematopoietic Stem Cell Transplantation. Transplantation and Cellular Therapy. 2023; 29:704.e1-704.e8.
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. Efficiency of observed information adaptive designs in linear models. Journal of Statistical Planning and Inference.Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD. Blood. 2023; 142:2105-2118.
.Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome. Journal of Allergy and Clinical Immunology. 2023; 151:1081-1095.
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. 426 Impact of Trimethoprim/Sulfamethoxazole Prophylaxis on Engraftment in Pediatric Hematopoietic Cell Transplant Recipients. Transplantation and Cellular Therapy.Phenotypic Alteration of an Established Human Airway Cell Line by Media Selection. International Journal of Molecular Sciences. 2023; 24.
.Ocular survival after intra-arterial chemotherapy for retinoblastoma improves with accrual of experience and programmatic evolution. Pediatric Blood and Cancer. 2023; 70:e30071.
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. 262 High Disease Burden and Severe Neutropenia Predict HLH Toxicity in Patients with B-Acute Lymphoblastic Leukemia (B-ALL) Treated with Tisagenlecleucel in the PRWCC. Transplantation and Cellular Therapy.HLH-like toxicities predict poor survival after the use of tisagenlecleucel in children and young adults with B-ALL. Blood Advances. 2023; 7:2758-2771.
.Lineage switching of the cellular distribution of BRAFV600E in multisystem Langerhans cell histiocytosis. Blood Advances. 2023; 7:2171-2176.
.Dual phenotype: co-occurring Leber congenital amaurosis and familial exudative vitreoretinopathy: a case report. Ophthalmic Genetics (Informa). 2023; 44:89-92.
.Viral-specific T cells for Cytomegalovirus retinitis following hematopoietic stem cell transplantation: A success story. Pediatric Blood and Cancer. 2023; 70:e30429.
.Humoral Response in Hemodialysis Patients Post-SARS-CoV-2 mRNA Vaccination: A Systematic Review of Literature. Vaccines. 2023; 11.
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. 429 BK Polyomavirus after Pediatric Hematopoietic Stem Cell Transplantation. Transplantation and Cellular Therapy.BK Polyomavirus Diversity After Hematopoietic Stem Cell Transplantation. The Journal of Infectious Diseases. 2023; 228:1208-1218.
.Hydroxyurea treatment is associated with lower malaria incidence in children with sickle cell anemia in sub-Saharan Africa. Blood. 2023; 141:1402-1410.
.Outcomes among racial and ethnic minority groups with X-linked agammaglobulinemia from the USIDNET registry. The Journal of Allergy and Clinical Immunology: in Practice. 2023; 11:1945-1946.
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. 194 Adherence Barriers in Pediatric Hematopoietic Stem Cell Transplant in an Outpatient Setting. Transplantation and Cellular Therapy.Autoimmune Lymphoproliferative Syndrome (ALPS) Disease and ALPS Phenotype: Are They Two Distinct Entities?. HemaSphere. 2023; 7:e845.
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. 409 Delayed CD4+ T Cell Recovery after Allogeneic Hematopoietic Cell Transplantation Is Associated with Decreased Overall Survival in Adult but Not Pediatric Recipients. Transplantation and Cellular Therapy.2023; 2:1151744.
. Post CAR T-cell therapy outcomes and management in HSCT-naive patients: a single-center experience.Evaluating Focal Areas of Signal Intensity (FASI) in Children with Neurofibromatosis Type-1 (NF1) Treated with Selumetinib on Pediatric Brain Tumor Consortium (PBTC)-029B. Cancers. 2023; 15.
.Novel FOXF1-Stabilizing Compound TanFe Stimulates Lung Angiogenesis in Alveolar Capillary Dysplasia. American Journal of Respiratory and Critical Care Medicine. 2023; 207:1042-1054.
.The incidence and impact of clostridioides difficile infection on transplant outcomes in acute leukemia and MDS after allogeneic hematopoietic cell transplant-a CIBMTR study. Bone Marrow Transplantation. 2023; 58:360-366.
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. Lymphoid Malignancies in Shwachman-Diamond Syndrome: Incidence, Clinical Features, and Outcomes from the North American SDS Registry. Blood.Allowing Relationships to Unfold: Consult Reason and Topics Discussed in Initial and Subsequent Palliative Care Visits Among Children Who Died From Relapsed/Refractory Cancer. Journal of Pediatric Hematology/Oncology Nursing. 2023; 40:170-177.
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. Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Outcome and Prognostic Factors for Survival and Subsequent Neoplasms. Blood.The Choice of Either Conventional Chemotherapy or Inotuzumab Ozogamicin as Bridging Regimen Does Not Appear To Impact Clinical Response to CD19-Directed CAR-T Therapy in Pediatric B-ALL. Transplantation and Cellular Therapy. 2023; 29:311.e1-311.e7.
.Mechanisms of endothelial injury and transplant-associated thrombotic microangiopathy in tandem autologous hematopoietic stem cell transplant for neuroblastoma. Haematologica: the hematology journal. 2023; 108:3449-3453.
.Peering into the darkness of drug-induced thrombotic microangiopathy: complement, are you in there?. Journal of Gastrointestinal Oncology. 2023; 14:468-471.
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. 406 All Eyes on Interferon in Graft Failure after Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy.2023; 29:s20-s21.
. 24 Longitudinal Risk of Bone Disease in a Diverse Pediatric Population Undergoing Allogeneic Hematopoietic Stem Cell Transplant. Transplantation and Cellular Therapy.2023; 29:s64-s65.
. 77 Proteomic Analyses Reveal Molecular Connections That Support the Clinical Association of Cystitis with Thrombotic Microangiopathy in Pediatric Transplant Recipients. Transplantation and Cellular Therapy.Harmonizing Definitions for Diagnostic Criteria and Prognostic Assessment of Transplantation-Associated Thrombotic Microangiopathy: A Report on Behalf of the European Society for Blood and Marrow Transplantation, American Society for Transplantation and Cellular Therapy, Asia-Pacific Blood and Marrow Transplantation Group, and Center for International Blood and Marrow Transplant Research. Transplantation and Cellular Therapy. 2023; 29:151-163.
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. 162 Harmonizing Definitions for Diagnostic Criteria and Prognostic Assessment of Transplant Associated Thrombotic Microangiopathy: A Report on Behalf of the European Society for Blood and Marrow Transplantation (EBMT), American Society for Transplantation and Cellular Therapy (ASTCT), Asia-Pacific Blood and Marrow Transplantation Group (APBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR). Transplantation and Cellular Therapy.Diffuse alveolar hemorrhage after hematopoietic cell transplantation- response to treatments and risk factors for mortality. Frontiers in Oncology. 2023; 13:1232621.
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. 161 Healthcare Utilization in Children with Transplant Associated Thrombotic Microangiopathy and the Impact of Eculizumab Therapy. Transplantation and Cellular Therapy.Outcomes After Nonresponse and Relapse Post-Tisagenlecleucel in Children, Adolescents, and Young Adults With B-Cell Acute Lymphoblastic Leukemia. Journal of Clinical Oncology. 2023; 41:354-363.
.Influenza Vaccine in Pediatric Recipients of Hematopoietic-Cell Transplants. The New England Journal of Medicine. 2023; 388:374-376.
.The 2022 EULAR/ACR Points to Consider at the Early Stages of Diagnosis and Management of Suspected Haemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS). Arthritis and Rheumatology. 2023; 75:1714-1732.
.The 2022 EULAR/ACR points to consider at the early stages of diagnosis and management of suspected haemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS). Annals of the Rheumatic Diseases. 2023; 82:1271-1285.
.Stroke Prevention with Hydroxyurea Enabled through Research and Education: A Phase 2 Primary Stroke Prevention Trial in Sub-Saharan Africa. Acta Haematologica. 2023; 146:95-105.
. .Higher doses of tisagenlecleucel are associated with improved outcomes: a report from the pediatric real-world CAR consortium. Blood Advances. 2023; 7:541-548.
.Higher abatacept exposure after transplant decreases acute GVHD risk without increasing adverse events. Blood. 2023; 142:700-710.
.Tocilizumab for Treatment of Children and Young Adults With Severe Acute COVID-19: Experience at a Quaternary-care Children's Hospital. The Pediatric Infectious Disease Journal. 2023; 42:119-121.
.Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium. The Lancet. 2023; 402:129-140.
.PROTECTIVE EFFECTS OF HUMANIN-G IN HEMORRHAGIC SHOCK IN FEMALE MICE VIA AMPKα1-INDEPENDENT MECHANISMS. Shock. 2023; 60:64-74.
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. GATA2-TGF-b1 axis regulates human NK cell development. Journal of immunology (Baltimore, Md. : 1950).Intestinal permeability in patients undergoing stem cell transplantation correlates with systemic acute phase responses and dysbiosis. Blood Advances. 2023; 7:5137-5151.
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. Go to bed!: Sleep as a risk factor for adolescent hypertension. Progress in Pediatric Cardiology.2023; 29:s135.
. 167 Earlier Oral Intake in Pediatric Stem Cell Transplant Patients Receiving Time-Restricted Feeding: A Prospective, Randomized Trial. Transplantation and Cellular Therapy.Phenotypes of adults with Fanconi anaemia. British Journal of Haematology. 2023; 201:133-139.
.Daytime-restricted parenteral feeding is associated with earlier oral intake in children following stem cell transplant. The Journal of Clinical Investigation. 2023; 133.
.Sustained and Boosted Antibody Responses in Breast Milk After Maternal SARS-CoV-2 Vaccination. Breastfeeding Medicine. 2023; 18:612-620.
.Preventable harm because of outpatient medication errors among children with leukemia and lymphoma: A multisite longitudinal assessment. Cancer. 2023; 129:1064-1074.
.Pediatric myelofibrosis due to compound heterozygous MPIG6B mutations in a patient of European ancestry. Pediatric Blood and Cancer. 2023; 70:e30023.
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. 441 Evolution of BK Polyomavirus in Immunocompromised Pediatric Patients. Transplantation and Cellular Therapy.