A5054980108- Hemophilia Treatment and Research
- Acute Myeloid Leukemia Research
- Platelet Disorders and Treatments
- Chronic Myeloid Leukemia Treatments
- Acute Lymphoblastic Leukemia research
- Eosinophilic Disorders and Syndromes
- Blood groups and transfusion
- Hemoglobinopathies and Related Disorders
- Blood properties and coagulation
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Lymphoma Diagnosis and Treatment
- Childhood Cancer Survivors' Quality of Life
- Pharmacological Effects and Toxicity Studies
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Hematopoietic Stem Cell Transplantation
- Cancer Genomics and Diagnostics
- Cancer Treatment and Pharmacology
- Cell death mechanisms and regulation
- Medical Imaging and Pathology Studies
- Sarcoma Diagnosis and Treatment
- Neutropenia and Cancer Infections
- Pediatric Pain Management Techniques
- Prenatal Screening and Diagnostics
- Congenital Diaphragmatic Hernia Studies
- Cardiac tumors and thrombi
Children's Hospital of Richmond at VCU
2012-2025
Virginia Commonwealth University
2012-2023
Virginia Commonwealth University Medical Center
1995-2019
May Institute
2013
Children's Hospital of Michigan
1999-2008
Arkansas Children's Hospital
2008
St. Jude Children's Research Hospital
2008
Lucile Packard Children's Hospital
2008
Children's Oncology Group
2008
University Health System
2007
Emicizumab is a recombinant humanized bispecific antibody that bridges factor IXa and X to mimic the cofactor function of VIII. It approved prevent bleeding in patients with haemophilia A (HA). Outside clinical trials, there limited data on outcomes treated emicizumab, particularly children without inhibitors.To report our experience treating including (a) rates pre postemicizumab, (b) peri-procedural management (c) serious drug-related adverse events.Multicentre observational study HA who...
Transient leukemia (TL or transient myeloproliferative disorder) occurs in approximately 10% of newborn infants with Down syndrome. The disorder is characterized by the presence megakaryoblasts peripheral blood; most cases resolve spontaneously within first 3 months life, and child well thereafter. However, there are which a severe, potentially lethal form disease develops, manifesting as hepatic fibrosis cardiopulmonary failure. Hitherto, incidence these severe forms has not been reported....
To determine the outcomes, with particular attention to toxicity, of children Down syndrome (DS) and acute myeloid leukemia (AML) treated on Pediatric Oncology Group (POG) protocol 9421.Children DS newly diagnosed AML (n = 57) were prospectively enrolled onto standard-therapy arm POG 9421 administered five cycles chemotherapy, which included daunorubicin 135 mg/m(2) mitoxantrone 80 mg/m(2). Outcomes toxicity evaluated compared non-DS-AML cohort 565). A retrospective chart review was...
ABSTRACT Patients with vascular anomalies (VAs) require expert multidisciplinary care. No prior studies have characterized the sociodemographic characteristics across multiple VA centers. We collected data on 5783 patients receiving care at 20 centers between July 1, 2020 through December 31, 2022. Male were underrepresented compared to state‐level census 17/20 Black and Asian 14/20 12/20 centers, respectively. The median distance hospital was 48 miles, age 12.0 years. These suggest that...
Summary Inhibitors are a rare but serious complication of treatment patients with haemophilia. Phase III clinical trials enrol too few to adequately assess new product inhibitor risk. This project explores the feasibility using public health surveillance system conduct national for inhibitors. Staff at 17 U.S. haemophilia centres ( HTC ) enrolled A and B into this prospective study. staff provided detailed historic data on use inhibitors baseline, postenrolment monthly infusion logs. central...
Despite aggressive pain management with opiates, debilitating still occurs in a subset of children terminal cancer. A 5-year-old girl metastatic retinoblastoma, profound opiate tolerance, and refractory was treated. Continuous lidocaine infusion initiated at dose 35 μg/kg per minute increased over 4 days to 50 minute, which point the patient discharged for continued end-of-life comfort care. The had excellent relief without associated lethargy high-dose opiates. No complicating...
A limited number of small studies have examined the vitamin D status pediatric oncology patients, and results indicate an increased prevalence hypovitaminosis. We conducted a cross-sectional study with primary aim describing our cancer patients any associations demographic characteristics. Our secondary was to compare this that healthy population. collected data on children seen in clinic determined overall then compared populations described literature. The hypovitaminosis population 72%....
Abstract Introduction Emicizumab is the first approved non‐factor therapy for haemophilia A. It provides superior prophylactic bleeding control compared to other products in both patients with and without inhibitors. However, there no real‐world data about monetary consequences of starting emicizumab. Aim To examine estimated costs emicizumab a cohort A Methods The cost haemostatic 6 months before after initiating participants multicentre observational study was calculated based on type...
Little is known about the risk of post-COVID-19 multisystem inflammatory syndrome in children (MIS-C) setting childhood cancer.
Angiofollicular lymph node hyperplasia is a heterogeneous disorder of unclear etiology and has wide spectrum systemic symptoms. This report describes case this in 15-year-old girl examines the response primary mass, symptoms, alterations selected immune parameters at diagnosis, as result steroid therapy radiation (RT). The patient had 1-year history growth failure, delayed puberty, refractory iron deficiency anemia. Computed tomography scan showed posterior mediastinal mass. Biopsy revealed...
Abstract Pathologic variants in TP53 are known risk factors for the development of cancer. We report a 17‐year‐old male who presented with two primary sarcomas. Germline sequencing revealed novel c.672 G>A mutation. Sequencing wild‐type parents, and there was no history cancer first‐degree relatives. This de novo synonymous germline mutation results 5′ cryptic splice site that is bound by U1, resulting shift 5 base pairs. The frame truncated protein at residue 246, which disrupts...
Valproic acid is an effective anti-epileptic medication often used for long-term control of seizure disorders that has been implicated in hematological toxicities, including rare reports myelodysplasia and acute leukemia. Here, we report a case valproic acid-related leukemia-like syndrome with t(8;16) chromosomal translocation. After discontinuing acid, the findings completely resolved.
A temporary elevation of serum alkaline phosphatase has been described in young children who have no evidence liver or bone disease. This phenomenon termed benign hyperphosphatasemia infancy. Its occurrence is three undergoing chemotherapy for acute lymphoblastic leukemia and lymphoma. All were remission the consolidation maintenance phase their therapy when occurred. also receiving methotrexate (IM IV), oral 6-mercaptopurine, sulfamethoxazole/trimethoprim. Although these agents are...