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Research Study for Severe Aplastic Anemia (SAA)

Aplastic Anemia diagram

Doctors at the National Institutes of Health (NIH) are conducting a research study to determine the viability and safety of early initiation of oral therapy with cyclosporine and eltrombopag in patients with severe aplastic anemia (SAA).

Study design:

  • Patients with SAA will be treated with cyclosporine and eltrombopag either before arrival to the NIH or at the NIH while work up is being completed
  • When the treatment is initiated remotely, it will be under the supervision of investigators at the National Heart, Lung, and Blood Institute (NHLBI)
  • All patients will receive standard treatment with cyclosporine, eltrombopag, and
  • Horse Anti-thymocyte-Globulin (h-ATG) at the NIH-Clinical Center, unless there is complete count recovery with oral therapy

Who can participate:

  • Patients ages 3 and older with SAA who:
    • have not received previous ATG-based immunosuppressive treatment and lack a suitable matched sibling marrow donor; OR
    • are not allogeneic transplantation candidates due to patient choice, advanced age, or infeasibility of transplantation

To learn more, call:
NIH Clinical Center Office of Patient Recruitment
800-411-1222
(TTY users dial 7-1-1)
Email: ccopr@nih.gov

Or go online:
http://bit.ly/3mxl7gY
Refer to study # 20-H-0033

Department of Health and Human Services
National Institutes of Health Clinical Center (CC)
National Heart, Lung and Blood Institute (NHLBI)