Testing Early Use of Oral Cyclosporine and Eltrombopag in Treating Severe Aplastic Anemia in Patients Aged 3 and Older

What this Trial Is About

Severe aplastic anemia SAA is a rare and serious blood disorder where the immune system attacks bone marrow cells, leading to low blood counts. Researchers are evaluating if starting a lower dose of cyclosporine CsA and eltrombopag EPAG early, before giving horse anti-thymocyte globulin h-ATG, is safe and effective for people with untreated SAA. The study focuses on treatment-naive patients aged 3 years and older. Participants begin oral therapy with low-dose CsA and EPAG, with dosing adjusted by age and ethnicity. CsA is taken twice daily for six months, and EPAG is taken for six months starting day one. Some participants may receive h-ATG intravenously for four days at the Clinical Center, unless they have already achieved a complete response after remote oral therapy. CsA doses increase upon starting h-ATG and are adjusted based on response, with a treatment period that may extend up to 24 months. During the study, participants undergo screening tests including medical history, physical exams, blood tests, bone marrow biopsy, and ECGs, with many tests repeated throughout the study. Follow-up visits occur at 3 months, 6 months, and annually for five years. Researchers monitor safety, feasibility, blood count recovery, relapse, survival, and the development of other blood disorders. Weekly phone calls support participants who start treatment remotely until they can visit the Clinical Center.

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)