A5004008487- Blood Coagulation and Thrombosis Mechanisms
- Venous Thromboembolism Diagnosis and Management
- Childhood Cancer Survivors' Quality of Life
- Cancer therapeutics and mechanisms
- Acute Lymphoblastic Leukemia research
- Cardiac Arrhythmias and Treatments
- Pharmaceutical studies and practices
- Pharmacogenetics and Drug Metabolism
- Lung Cancer Research Studies
- Atrial Fibrillation Management and Outcomes
- Heart Failure Treatment and Management
- Sarcoma Diagnosis and Treatment
- Mechanical Circulatory Support Devices
- Platelet Disorders and Treatments
- Neuroblastoma Research and Treatments
- Cardiac Structural Anomalies and Repair
- Blood disorders and treatments
- Cancer Treatment and Pharmacology
- Multiple Myeloma Research and Treatments
- Herpesvirus Infections and Treatments
- Congenital Heart Disease Studies
- Acute Myeloid Leukemia Research
- Cytomegalovirus and herpesvirus research
- Diabetes Management and Research
- Testicular diseases and treatments
Texas Children's Hospital
2004-2024
Baylor College of Medicine
2013-2024
Children's Cancer Center
2009-2022
Dan L Duncan Comprehensive Cancer Center
2021
Mayo Clinic in Florida
2018
Seattle Children's Hospital
2016-2018
Boehringer Ingelheim (India)
2018
Janssen (Belgium)
2018
Boehringer Ingelheim (Australia)
2018
Pfizer (United States)
2018
PURPOSE: To assess thiopurine S-methyltransferase (TPMT) phenotype and genotype in patients who were intolerant to treatment with mercaptopurine (MP) or azathioprine (AZA), evaluate their clinical management. PATIENTS AND METHODS: TPMT metabolism assessed all referred between 1994 1999 for evaluation of excessive toxicity while receiving MP AZA. activity was measured by radiochemical analysis, determined mutation-specific polymerase chain reaction restriction fragment length polymorphism...
The use of radiographic response as the primary end point in phase II osteosarcoma trials may limit optimal detection treatment because calcified tumor matrix. We performed this study to determine if time progression could be used an for subsequent studies.We a retrospective analysis outcome patients with recurrent/refractory enrolled one seven conducted by Children's Oncology Group and predecessor groups from 1997 2007. All RECIST or WHO criteria rate. following potential prognostic...
To determine the efficacy and safety of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia (AML).
The exponential increase in the number of drugs used to treat infant and childhood illnesses necessitates an understanding ontogeny drug biotransformation for development safe effective therapies. Healthy infants received oral dose (0.3 mg/kg) dextromethorphan (DM) at 0.5, 1, 2, 4, 6, 12 months age. DM its major metabolites were measured urine. CYP2D6 genotype was determined by polymerase chain reaction-restriction fragment length polymorphism. Genotyping data indicated a strong correlation...
Purpose Intermediate-risk rhabdomyosarcoma (RMS) includes patients with either nonmetastatic, unresected embryonal RMS (ERMS) an unfavorable primary site or nonmetastatic alveolar (ARMS). The aim of this study was to improve the outcome intermediate-risk by substituting vincristine and irinotecan (VI) for half vincristine, dactinomycin, cyclophosphamide (VAC) courses. All received a lower dose earlier radiation therapy than in previous trials. Patients Methods were randomly assigned at entry...
Abstract Purpose: A phase 1 study to determine the maximum-tolerated dose, dose-limiting toxicity, pharmacokinetics, and biological effects of bortezomib in children with recurrent/refractory leukemia. Experimental Design: Bortezomib was administered twice weekly for 2 consecutive weeks at either 1.3 or 1.7 mg/m2 dose followed by a 1-week rest. pharmacokinetics nuclear factor κB (NF-κB) binding activity were evaluated during first treatment cycle. Results: Twelve patients (nine acute...
Purpose A phase II study was performed to determine the efficacy of irinotecan (IRN) in children with refractory solid tumors. Secondary objectives were evaluate toxicity, pharmacokinetics, pharmacodynamics, and UGT1A1 genotype. Patients Methods total 181 patients enrolled, whom 171 eligible. received IRN 50 mg/m 2 /d for 5 days repeated every 3 weeks. Pharmacokinetic studies genotyping performed. Results Of 161 assessable response, one patient hepatoblastoma had a complete partial responses...
We performed a phase I trial of intrathecal (IT) liposomal cytarabine (DepoCyt; Enzon Pharmaceuticals, Piscataway, NJ and SkyePharma Inc, San Diego, CA) to determine the maximum-tolerated dose, dose-limiting toxicities, plasma CSF pharmacokinetics IT lipsomal in children >/= 3 years age with advanced meningeal malignancies.Eighteen assessable patients received through either an indwelling ventricular access device or via lumbar puncture. Liposomal was given once every 2 weeks during...
OBJECTIVE Exenatide improves postprandial glycemic excursions in type 2 diabetes. could benefit 1 diabetes as well. We aimed to determine an effective and safe glucose-lowering adjuvant exenatide dose adolescents with RESEARCH DESIGN AND METHODS Eight subjects completed a three-part double-blinded randomized controlled study of premeal exenatide. Two doses (1.25 2.5 μg) were compared insulin monotherapy. Prandial was reduced by 20%. Gastric emptying hormones analyzed for 300 min postmeal....
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...
Efficacious ventricular assist device (VAD) support in pediatric patients depends on successful antithrombotic management. The experience with management for the EXCOR Pediatric VAD Investigational Device Exemption (IDE) study is described. All 68 children North America enrolled IDE from May 9, 2007 to December 10, 2010 are included. Edmonton Anticoagulation and Platelet Inhibition Protocol was provided guidance. Monitoring parameters, drug dosing, targeted serious adverse events, pump...
Postprandial hyperglycemia and preprandial hypoglycemia contribute to poor glycemic control in type 1 diabetes. We hypothesized that postprandial excursions could be normalized diabetes by suppressing glucagon with pramlintide acetate the immediate period supplementing late period. A total of 11 subjects were compared 8 diabetic on insulin pump therapy, using usual bolus–to–carbohydrate ratio during a standard liquid meal. Type then randomized two open-labeled studies. On one occasion,...
Childhood ITP can have a negative impact on the child and his/her family even though it is typically benign disorder with low risk of serious bleeding. In adults now children, romiplostim increases platelet count without significant adverse effects. this study, treatment HRQoL children chronic was assessed using Kid's Tools (KIT).Subjects 1-18 years old, (>6 months), were enrolled in multi-center, randomized, double-blind, placebo-controlled phase 1/2 study (reported elsewhere). Subjects...
Objective: Coagulation system activation in extracorporeal membrane oxygenation results hemostatic derangements. Thrombin generation markers like prothrombin fragment 1+2 and thrombin-antithrombin complex are sensitive of hypercoagulability. Plasmin-antiplasmin is a marker for fibrinolysis. D-dimers reflect thrombin The aim was to identify the extent hemostasis during by measuring complex, 1+2, plasmin-antiplasmin D-dimer. Design: Prospective cohort study. Setting: Tertiary care academic...
Background Congenital heart disease (CHD) is common in children and associated with greater risk of thrombotic complications. Management these complications standard‐of‐care treatment suboptimal for children. Methods Results The effectiveness safety dabigatran were demonstrated pivotal pediatric studies the acute venous thromboembolism (VTE; NCT01895777) secondary VTE prevention (NCT02197416). We report efficacy outcomes from subgroup analyses CHD (diagnosed according to local practice)...
Essentials Dabigatran etexilate may provide a new treatment option for pediatric venous thromboembolism. Children aged 1 to < 12 years were given dabigatran in an open-label, single-arm study. The pharmacokinetic-pharmacodynamic relationship was similar that seen adult patients. There no serious adverse events, bleeding events or recurrent thromboembolism.Background current standard-of-care treatments thromboembolism (VTE) have limitations. (DE), direct thrombin inhibitor, offer alternative...
Summary Severe congenital protein C (PC) deficiency (SCPCD) is associated with disseminated intravascular coagulation (DIC), purpura fulminans (PF), and vascular thromboembolic events (TE), often leading to organ failure death. PC replacement therapy offers a safe, effective treatment for complications of SCPCD secondary prophylaxis recurrent DIC, PF, TEs. A prospective, multi-centre, open-label, phase 2/3 study was conducted demonstrate the safety efficacy concentrate PF acute Fifteen...