A5081987561- Platelet Disorders and Treatments
- Blood groups and transfusion
- Immunodeficiency and Autoimmune Disorders
- Hemophilia Treatment and Research
- Chronic Lymphocytic Leukemia Research
- Blood disorders and treatments
- Blood properties and coagulation
- Autoimmune Bullous Skin Diseases
- Heparin-Induced Thrombocytopenia and Thrombosis
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Hematopoietic Stem Cell Transplantation
- Blood Coagulation and Thrombosis Mechanisms
- Traumatic Brain Injury and Neurovascular Disturbances
- Monoclonal and Polyclonal Antibodies Research
- Renal Diseases and Glomerulopathies
- Cell Adhesion Molecules Research
- COVID-19 Clinical Research Studies
- Polyomavirus and related diseases
- S100 Proteins and Annexins
- Cancer-related gene regulation
- Antiplatelet Therapy and Cardiovascular Diseases
- Neutropenia and Cancer Infections
- Intracerebral and Subarachnoid Hemorrhage Research
- COVID-19 and healthcare impacts
- Acute Ischemic Stroke Management
University of Chicago
1990-2025
Emory University
2016-2025
Children's Healthcare of Atlanta
2016-2025
Neurological Surgery
2024
NeuroDevelopment Center
2023
University of Virginia
2023
Spark Therapeutics (United States)
2023
Aflac (United States)
2013-2022
Versiti Blood Center of Wisconsin
2022
Michigan State University
2015
Abstract The Joint Outcome Study (JOS), a randomized controlled trial, demonstrated that children with severe hemophilia A (HA) initiating prophylactic factor VIII (FVIII) prior to age 2.5 years had reduced joint damage at 6 compared those treated episodic FVIII for bleeding. Continuation (JOS-C) evaluated early vs delayed prophylaxis effects on long-term health, following JOS participants 18 in an observational, partially retrospective study. Index magnetic resonance imaging (MRI) scores of...
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...
Immune thrombocytopenia (ITP) during childhood spontaneously remits in up to 80% of children. Predictors remission are not well understood.We analyzed data from Intercontinental Cooperative ITP Study Group (ICIS) Registry II, a large prospective cohort children with ITP, investigate factors that might predict remission.In ICIS 705 patients had collected through 12 months following diagnosis, 383 having available at 24 as well. Younger age and pharmacologic treatment diagnosis were...
Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence inform treatment relapsed or refractory patients is also limited. The clinical features 314 children treated from 2002 2014 with acquired were analyzed retrospectively 25 institutions in the North American Pediatric Aplastic Anemia Consortium. majority subjects (n=264) received horse anti-thymocyte globulin (hATG) plus...
The novel severe acute respiratory syndrome coronavirus 2 is a worldwide pandemic. morbidity and mortality associated with disease 2019 has mostly affected the elderly or those underlying medical conditions. We present case of 12-year-old girl no past history who presented fever, cough, vomiting. Laboratory evaluation revealed thrombocytopenia elevated markers inflammation. patient progressed to failure, testing results for returned positive. Because severity her thrombocytopenia, she was...
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with isolated and hemorrhagic risk. While many children ITP can be safely observed, treatments are often needed for various reasons, including to decrease bleeding, or improve health related quality of life (HRQoL). There a number available second-line treatments, rituximab, thrombopoietin-receptor agonists, oral immunosuppressive agents, splenectomy, but data comparing treatment outcomes lacking. ICON1 prospective,...
Summary The development of antibodies against infused factor VIII (FVIII) in patients with haemophilia A is a serious complication leading to poorly controlled bleeding and increased morbidity. No treatment has been proven reduce high titre who fail immune tolerance induction or are not candidates for it. Rituximab the Treatment Inhibitors Congenital Hemophilia (RICH) study was phase II trial assess whether rituximab can anamnestic FVIII antibody (inhibitor) titres. Male subjects severe...
Abstract Introduction Recombinant factor IX Fc fusion protein (rFIXFc) has demonstrated efficacy for treatment of haemophilia B in the Phase 3 B‐LONG and Kids studies. However, long‐term rFIXFc safety data have not yet been reported. Aim To report subjects with B. Methods B‐YOND (NCT01425723) was an open‐label extension eligibl previously treated who completed or B‐LONG. Subjects received ≥1 regimen: weekly prophylaxis (WP), individualized interval (IP), modified episodic treatment. could...
Abstract Background We previously showed in a prospective study that rituximab appears to be effective some children and adolescents with severe chronic immune thrombocytopenia. Eleven of 36 patients achieved maintained platelet counts over 50,000/mm 3 within the first 12 weeks. These were followed for next year. Methods Platelet monitored monthly all subsequent bleeding manifestations need further treatment was noted. Results Eight 11 initial responders count 150,000/mm without...
Background Randomized clinical trials in pediatric aplastic anemia (AA) are rare and data to guide standards of care scarce. Procedure Eighteen institutions formed the North American Pediatric Aplastic Anemia Consortium foster collaborative studies AA. The initial goal NAPAAC was survey diagnostic therapies utilized Results Our indicates considerable variability among diagnosis treatment There were areas general consensus, including need for a bone marrow evaluation, cytogenetic specific...
Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated and risk of hemorrhage. While most children ITP promptly recover or without drug therapy, persistent chronic in others. When needed, how to select second-line therapies not clear. ICON1, conducted within the Pediatric Consortium North America (ICON), a prospective, observational, longitudinal cohort study 120 from 21 centers starting treatments for examined treatment decisions. Treating...
Summary The safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) Kids (children <12 studies subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety efficacy data from B-YOND, rFIXFc extension study. Eligible who completed or could enrol B-YOND. There four treatment groups: weekly prophylaxis (20–100 IU/kg every 7 days), individualised (100 8–16 modified...
Introduction: Familial cerebral cavernous malformations (FCCM), Sturge-Weber Syndrome (SWS), and hereditary hemorrhagic telangiectasia with brain arteriovenous (HHT) are neurovascular disorders driven by genetic mutations while microbleeds (CMBs) primarily associated the aging process. All different vascular dysmorphisms and/or propensity to bleed. Hypothesis: We hypothesized that common distinct circulating microribonucleic acids (miRNAs), reflecting shared pathobiology, can serve as...
Neutralizing alloantibodies (inhibitors) to factor VIII or IX develop in approximately 25% of patients with haemophilia A and <3% B treated concentrate. Patients high titre inhibitors, whom immune tolerance therapy fails, have few treatment options. Targeted anti-B-cell rituximab (chimeric anti-CD20) has been useful several antibody-mediated autoimmune states. Case reports small numbers inhibitors inconclusive. We describe three adolescent severe four weekly doses rituximab, 375 mg m(-2)....
We describe monozygotic female twins discordant for hemophilia A, born to a carrier mother and normal father. Affected twin A presented at age 1 year with excessive bruising factor VIII procoagulant activity (FVIII:C) of less than 1% normal. Twin B is an asymptomatic FVIII:C level 42%. Peripheral blood DNA was tested X-chromosome inactivation (methylation) patterns the X-linked human androgen receptor gene, comparing twins' parental. showed nonrandom skewed toward paternal X, whereas random...
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...
Multiple daily injection insulin regimens (MDI) and continuous subcutaneous infusion (CSII) allow adolescents with type 1 diabetes mellitus (DM) meal flexibility, may improve metabolic control. The dosage calculations, however, involve ratios of to carbohydrate corrections for high blood glucose values, are labor-intensive prone error. We evaluated the impact an calculation device (IDC) on control, treatment satisfaction, regimen adherence quality life in using MDI or CSII.We conducted a...