Active Studies
Pediatric ITP Consortium Open Research Studies
PINES Study: A phase 3 study of Eltrombopag vs. Standard First-Line Management for newly diagnosed immune thrombocytopenia (ITP) in children
Study contact: Kristin Shimano, MD (UCSF)
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
Biobank contacts: Amanda Grimes, MD and Bogdan Dinu
This is a multi-center study 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 are being used to investigate biological markers and genetic variants that may affect likelihood of development of ITP, progression to chronic ITP, disease severity, and response to treatment.
ITP-APPS study
Study contact: Larry Frelinger, PhD (Boston Children's)
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.
ITP Pathway for Newly Diagnosed Pediatric ITP
Study contacts: Rachael Grace, MD and Kathryn Carrier
Evidence-based guidelines from the American Society of Hematology (ASH) guide the management of children with newly diagnosed ITP. Despite the availability of guidelines, there is significant practice variation in the approach to these patients. In this quality improvement project, an algorithm is made available to guide the initial management of newly diagnosed children across a number of ICON centers. The algorithm is based directly on the ASH recommendations. Clinicians then complete brief surveys regarding their management to understand decision-making. Iterative changes will be made over time based on the ongoing results.
Autoimmune Hemolytic Anemia Cohort Study
Study Contact: Rachael Grace, MD (Boston Children’s)
Evidence-based guidelines for the diagnosis, evaluation, and treatment of autoimmune hemolytic anemia in children are lacking. This is a multicenter observational cohort study of the diagnostic testing and management of pediatric autoimmune hemolytic anemia over a 10-year period.
Immature Platelet Fraction Study
Study contacts: Emily Harris, MD and Rachael Grace, MD (Boston Children’s)
This is a multi-center observation study evaluating the relationship between the immature platelet fraction and clinical features of ITP including treatment response. The study is funded by the James B Bussel ITP Young Investigator Award from the Platelet Disorder Support Association.