Characterization of Plasma Factor XIII Antigen and Activity in Very Low Birth Weight Premature Neonates

Michael Tarantino, MD

Sponsoring Agency: Novo Nordisk, Inc.

Hypothesis: Plasma FXIII concentration in the fetus and neonate is dependent upon hepatic maturation and gestational age and low cord blood FXIII activity is associated with an increased risk of intraventricular hemorrhage (IVH).

Specific Aims:

  1. To collect data on the incidence of IVH in premature neonates from a dataset of a large Level 3 Neonatal Intensive Care Unit and use neurodevelopmental outcome data to gauge the impact of IVH in premature neonates.
  2. To measure cord blood antigen concentration and activity of Factor XIII in premature neonates to identify a correlation between low Factor XIII activity and the incidence of IVH.
  3. To measure other morphometric, co-morbid and hemostatic parameters to identify possible contributors and potential confounding variables to role of low Factor XIII activity in IVH in premature neonates.


Investigate efficacy, safety, tolerability and pharmacokinetics of eltrombopag, a thrombopeietin receptor agonist in previously treated patients with chronic ITP

Michael Tarantino, MD

Sponsoring Agency: Glaxo SmithKline

A three part, staggered cohort, open-label and double blind, randomized, placebo controlled study to investigate the efficacy, safety, tolerability and pharmacokinetics of eltrombopag, a thrombopoietin receptor agonist, in previously treated pediatric patients with chronic idiopathic thrombocytopenic purpura (ITP).

Hematologic manifestations in pediatrics patients with Mycoplasma infections.

Karen Fernandez, MD

Mycoplasma pneumoniae is one of the common pathogenic bacteria responsible for a wide variety of infectious syndromes. Infection with M. pneumoniae primarily affects the respiratory tract system with symptoms ranging from mild fever, nonproductive cough to severe pneumonia. Extrapulmonary manifestations are also common and include the central nervous system, cardiovascular, gastrointestinal, and hematological systems (1). Hematological complications that are known to occur with M. pneumoniae infection include hemolytic anemia, thrombocytopenia, thrombotic thrombocytopenic purpura (TTP) and hemophagocytosis.

For the past few years, we observed several pediatrics patients who presented with severe clinical manifestations of M. pneumoniae infection had the changes in peripheral blood esosinophilia. The mechanisms of eosinophilia in M. pneumoniae infection are poorly understood. In fact, it is not yet certain whether peripheral blood eosinophilia was caused directly by M. pneumoniae infection. Interestingly, Medina et al recently reported that M. pneumoniae can produces an ADP-ribosylating and vacuolating toxin termed the CARDS toxin which induced an increased expression of the Th-2 cytokines IL-4 and IL-13 and Th-2 chemokines CCL17 and CCL22 resulting in a mixed cellular inflammatory response comprised of a robust eosinophilia in pulmonary tissues (2). However, the role of M. pneumoniae infection and eosinophilia is just emerging and data are very limited.

Thus, the objective of this study is to evaluate prevalence of peripheral blood eosinophilia among patients with M. pneumoniae infection.

Sickle Cell Disease 

Medical Services for Newborns and Children with Abnormal Hemoglobins

Kay Saving, MD

Sponsoring Agency: Illinois Department of Public Health

The Illinois Department of Public Health (IDPH), Office of Health Promotion, will provide funding to hematology centers to assure the availability of statewide services to families in Illinois who have a newborn or child with a hemoglobin disorder or trait.  Sickle cell centers will provide diagnostic services, treatment and counseling of patients.  In addition, education and information about hemoglobin disorders and treatment will be provided to health care providers and the public.

Sickle Cell Disease Treatment Demonstration Project

Kay Saving, MD

In 2004 Congress enacted legislation authorizing a demonstration program for the prevention and treatment of Sickle Cell Disease (SCD). The legislation was enacted to (1) create an optional medical assistance program for individuals with Sickle Cell Diseases for treatment and education, genetic counseling and other services to prevent mortality and decrease morbidity from Sickle Cell Disease; and (2) create a demonstration program, the Sickle Cell Disease Treatment Demonstration Project (SCDTDP), under Health Resources and Services Administration (HRSA).

Under the authorizing legislation, a National Coordinating Center (NCC) was established to collect, coordinate, monitor, and distribute data, best practices and findings regarding the activities of the demonstration program.