Research

Open ICON studies

PINES Study

A phase 3 study of Eltrombopag vs. Standard First-Line Management for.newly diagnosed immune thrombocytopenia (ITP) in children: Eltrombopag is an oral thrombopoietin receptor agonist that is FDA approved for the treatment of chronic ITP in children. Due to the lack of immune suppression and overall favorable safety profile, off-label use of this agent for adults and children with newly diagnosed ITP is occurring with greater frequency, although this approach has not been systematically studied. ICON3 is a prospective, open label, randomized, multi-center phase 3 clinical trial comparing eltrombopag with the enrolling physician’s choice of front-line therapy.

ICON Research Sample Bank

This is multi-center study is initiated, funded, and run through Baylor College of Medicine and includes select sites that are affiliated with ICON. The purpose of this study is to establish a repository of samples from patients with ITP to facilitate genetic and biologic research with the goal of improved understanding of disease susceptibility, pathophysiology, and treatment response. The samples will be used in future research studies investigating biological markers and genetic variants that may affect likelihood of development of ITP, progression to chronic ITP, disease severity, and response to treatment.

ITP Pathways Study

The purpose of this study is to examine the role of health-related quality of life on the decision making of physicians and choice of treatment for pediatric patients with ITP. The survey data will identify information such as geographic location, time of day, inpatient versus outpatient care, and day of week on the patient and family’s quality of life at diagnosis. The study will further identify the role of physician-patient and physician-caregiver interactions on the quality of life and emotional well-being of patients and their families.

ITP-APPS study

ITP-APPS (Immune Thrombocytopenia – Association of Platelet Parameters Study) is a multi-center, cross-sectional study of platelet biomarkers in children with ITP. The goal of the study is to determine the association, independent of platelet count, of immature platelet fraction and other platelet tests with concurrent, subsequent and worst-ever bleeding in children with ITP.