2025 Studies Synopsis

Current Projects

  • PI: Marshall Mazepa
    Population: iTTP

    The P200 study is a prospective biobank and clinical registry for patients with iTTP, collecting longitudinal clinical data and biological samples. The study includes 21 participating sites and 277 enrolled patients, with data cleaning currently underway, and maintains stored aliquots of sodium citrate plasma, EDTA plasma, and buffy coat. Investigators may request access to data and specimens for approved research projects, with limited funding available to support collaborative studies.

  • PI: Meera Sridharan
    Population: CM-TMA

    The R400 is a planned retrospective protocol focused on characterizing clinical features, management strategies, and outcomes in patients with complement-mediated thrombotic microangiopathy. The study is currently in development and seeking funding, with Charlotte Story serving as a co-collaborator. This effort will leverage existing clinical data to inform future prospective studies and therapeutic strategies.

  • PI: Anuja Java and Satheesh Chonat
    Population: Children and adults with TMA

    This study will recruit children and adults with TMA to gather standardized longitudinal clinical data and biospecimens across participating institutes. The objective is to enable mechanism-based classification and guide future research for TMA studies.

  • PI: Shruti Chaurvedi
    Population: iTTP

    The NeST (Neurologic Sequelae of iTTP) study is a multicenter cohort study evaluating the impact of acute iTTP therapies on long-term neurologic and cognitive outcomes in adult iTTP survivors. Conducted across six sites (JHU, OSU, University of Minnesota, UNC, Cleveland Clinic, and UAB), the study compares patients treated with early caplacizumab (within 3 days of diagnosis) to matched controls treated without caplacizumab. Outcomes include silent cerebral infarction on brain MRI, long-term cognitive impairment measured by the NIH ToolBox Cognition Battery, and patient-reported outcomes related to cognition, quality of life, and depression.

  • PI: TBD
    Population: TBD

    Description:  The Pilot Core supports the development of innovative, early-stage research projects in thrombotic microangiopathies by providing seed funding and infrastructure support. Through the U54 mechanism, investigators may apply for pilot grants to generate preliminary data, foster new collaborations, and advance projects toward larger external funding. The Pilot Core is designed to encourage novel ideas and accelerate translational and clinical research across the consortium.

  • PI: Satheesh Chonat
    Population: Pediatric iTTP

    Description: The R300 study is an IRB-exempt, retrospective registry focused on pediatric iTTP. The registry collects de-identified clinical data only (no PHI) and is open to any investigators interested in contributing data on pediatric iTTP cases. This study aims to expand understanding of disease presentation, management, and outcomes in children with iTTP.

  • PI: Dee Terrell and Senthil Sukumar
    Population: TMA

    Description: The International Decentralized Registry is a newly launched study designed to support the collection of real-world data across international sites using a decentralized approach. The registry aims to facilitate broad participation while reducing site burden, with some funding available to support study activities. This effort will enable international collaboration and expand the global understanding of thrombotic microangiopathies.

  • PI: Satheesh Chonat (US)
    Population: cTTP

    Description: The UK Congenital TTP Project is an emerging collaborative effort focused on patients with cTTP in the United Kingdom. The project is currently in development and aims to establish a framework for studying clinical characteristics, management strategies, and outcomes in this rare population. This initiative will strengthen international collaboration and inform future prospective research in cTTP.