A5075871834- Platelet Disorders and Treatments
- Hemophilia Treatment and Research
- Acute Myeloid Leukemia Research
- Histiocytic Disorders and Treatments
- Neuroblastoma Research and Treatments
- Blood groups and transfusion
- Chemotherapy-induced cardiotoxicity and mitigation
- Virus-based gene therapy research
- Autoimmune and Inflammatory Disorders Research
- Cancer-related Molecular Pathways
- Neutropenia and Cancer Infections
- Chronic Lymphocytic Leukemia Research
- CAR-T cell therapy research
- Genital Health and Disease
- Hemostasis and retained surgical items
- Venous Thromboembolism Diagnosis and Management
- Viral-associated cancers and disorders
- Cancer therapeutics and mechanisms
- Acute Lymphoblastic Leukemia research
- Systemic Sclerosis and Related Diseases
- Hematopoietic Stem Cell Transplantation
- Sarcoma Diagnosis and Treatment
- Immunodeficiency and Autoimmune Disorders
- Viral Infectious Diseases and Gene Expression in Insects
- Bone Metabolism and Diseases
Arkansas Children's Hospital
2014-2025
University of Arkansas for Medical Sciences
2005-2021
Lucile Packard Children's Hospital
2008
Children's Oncology Group
2008
St. Jude Children's Research Hospital
2008
Children's Hospital of Michigan
2008
National Institute on Aging
2005
Dana-Farber Cancer Institute
2005
SUNY Upstate Medical University
2005
Memorial Sloan Kettering Cancer Center
2005
PURPOSE: To determine the response rate of combination cyclophosphamide and topotecan in pediatric patients with recurrent or refractory malignant solid tumors. PATIENTS AND METHODS: A total 91 patients, 83 whom were fully assessable for toxicity, received (250 mg/m 2 /dose) followed by (0.75 /dose), each given as a 30-minute infusion daily 5 days. All filgrastim (5 mcg/kg) until absolute neutrophil count (ANC) was ≥ 1,500 μL after time expected ANC nadir. RESULTS: 307 treatment courses to...
Prognosis is poor for Ewing sarcoma patients with metastasis at diagnosis. We intensified a five-drug therapy (ifosfamide, etoposide alternated vincristine, doxorubicin, and cyclophosphamide) using filgrastim but not stem-cell support. studied topotecan alone combined cyclophosphamide in therapeutic windows before the therapy. A randomly assigned proportion of received amifostine as cytoprotective agent.Eligible were < or = 30 years old had histologically proven primitive neuroectodermal...
Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option children who require an increased platelet count.We performed multicenter retrospective study of ITP followed at Consortium North America (ICON) sites to characterize TPO-RA use.Seventy-nine had total 87 treatments (28 eltrombopag, 43 romiplostim, and eight trialed both). The majority primary (82%) most...
Objective. To seek insights into the heterogeneity of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA) through analysis a large patient sample collected in multinational survey. Methods. International pediatric rheumatologists and hemato-oncologists entered their data, retrospectively, Web-based database. The demographic, clinical, laboratory, histopathologic, therapeutic, outcome data were analyzed relation to (1) geographic location caring...
HIV-1-specific cell-mediated cytotoxicity (CMC) is a form of antibody-dependent cellular (ADCC) in which antibodies arm NK cells directly to become cytotoxic for targets bearing HIV-1 antigenic determinants. This non-MHC-restricted activity present early stages disease and declines markedly with progression. To understand the humoral factors contributing reduction this activity, conditions under maximal arming occurs was examined vitro. With use large patient cohort, strong positive...
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...
Purpose To determine the efficacy and safety of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia (AML). Patients Methods A phase II, open-label, multicenter study was conducted single-agent AML. Clofarabine administered intravenously over 2 hours at maximum-tolerated dose (MTD) 52 mg/m daily for 5 consecutive days. Cycles were repeated every to 6 weeks. Responses determined by an independent response review panel. Results The 42 treated on had a median age...
Treatment of chronic severe pediatric ITP is not well studied. In a phase 1/2 12-16-week study, 15/17 romiplostim-treated patients achieved platelet counts ≥50 × 109 /L, and romiplostim treatment was tolerated. subsequent open-label extension (≤109 weeks), 20/22 received romiplostim; all >50 /L. Twelve continued in second (≤127 weeks). Longitudinal data from start through the two extensions were evaluated to investigate safety efficacy long-term ITP.Patients weekly subcutaneous romiplostim,...
Abstract Patients with hemophilia A require exogenous factor VIII (FVIII) or nonfactor hemostatic agents to prevent spontaneous bleeding events. Adeno-associated virus (AAV) vector–based gene therapy is under clinical investigation enable endogenous FVIII production. Giroctocogene fitelparvovec a recombinant AAV serotype 6 vector containing the coding sequence for B-domain–deleted human F8 gene. In ongoing phase 1/2, dose-ranging Alta study, 4 sequential cohorts of male participants severe...
To determine the maximum-tolerated dose (MTD) and dose-limiting toxicity of topotecan when combined with cyclophosphamide in pediatric patients recurrent or refractory malignant solid tumors.A total 33 received (250 mg/m2/dose) followed by escalating doses (0.6 to 0.75 mg/m2/dose), each given as a 30-minute infusion daily for 5 days. A 154 fully assessable treatment courses were these patients.Neutropenia was therapy at both levels. The addition filgrastim allowed escalation 0.75-mg/m2 level...
To present two patients as illustrations of the risk developing secondary acute myelogenous leukemia (sAML) when theoretically safe doses etoposide (VP-16) are used.Patient no. 1 was a 15-year-old white girl diagnosed with stage IIa Hodgkin's disease. She treated combination vincristine, doxorubicin, bleomycin, and VP-16 (2 g/m2 total) over 4 months, followed by 25.5 Gy involved-field radiotherapy. Patient 2 an 11-year-old boy virus-associated hemophagocytic syndrome (VAHS). He intravenously...
Human genetic variation contributes to differences in susceptibility HIV-1 infection. To search for novel host resistance factors, we performed a genome-wide association study (GWAS) hemophilia patients highly exposed potentially contaminated factor VIII infusions. Individuals with A and documented history of infusions before the introduction viral inactivation procedures (1979–1984) were recruited from 36 treatment centers (HTCs), their variants compared those matched HIV-infected...
Background Pediatric patients with Langerhans cell histiocytosis (LCH) may become refractory to conventional therapy or present repeated recurrences over several years. Current therapeutic options such as prednisone, vinblastine, etoposide, and cyclosporine are associated significant acute toxicities late effects. Recent reports suggested that 2-chlorodeoxyadenosine (2-CDA) be an effective agent in adults LCH. The purpose of this study was determine the safety efficacy 2-CDA children Methods...
Osteosarcoma (OS) is the most common malignant bone tumor affecting children and adolescents. Many patients are treated with a combination of chemotherapy, resection, limb salvage protocols. Surgical reconstructions after resection include structural allografts, non-cemented endoprostheses, distraction osteogenesis (DO), which require direct formation. Although cisplatin (CDP) extensively used for OS effects on regeneration not well studied. The CDP formation in DO were compared using two...
Purpose Patients with Ehlers-Danlos syndrome (EDS), especially types IV, VI, and VIII, are at increased risk of bleeding, most do not have specific hemostatic deficiencies that would be amenable to replacement therapy. We investigated the ability DDAVP (desmopressin acetate) control bleeding in EDS. Methods Two children EDS, VIII presented hemorrhagic symptoms scheduled surgical procedures. Ivy times (BTs) were measured before after intravenous (i.v.) challenge, i.v. was used...
Abstract Background Treatment choice in pediatric immune thrombocytopenia (ITP) is arbitrary, because few studies are powered to identify predictors of therapy response. Increasingly, rituximab becoming a treatment those refractory other therapies. Methods The objective this study was evaluate univariate and multivariable platelet count response rituximab. After local IRB approval, 565 patients with chronic ITP enrolled met criteria for the longitudinal, North American Chronic Registry...
Acquired von Willebrand factor (vWF) disease is associated with a decrease in the amount of circulating high molecular weight (HMW) vWF multimers. has not been previously investigated children on extracorporeal membrane oxygenation (ECMO) support. We hypothesized that HMW multimers and activity over course ECMO support these patients. This prospective, single center, observational, cohort pilot study was carried out between December 2010 April 2011 included patients 0 to 18 years old...
Abstract Patients with Langerhans cell histiocytosis (LCH) may behave differently depending on what sites are involved and the response or lack of to earlier therapies. Therapy for high‐risk patients those multiple reactivations continues be challenging because variable rates frequent toxicities. The goals this study were determine long‐term disease free survival in children multiply reactivated LCH treated 2‐CDA, toxicity low dose continuous infusion (CI). Ten as defined by Histiocyte...