Alternative treatment modality for severe aplastic anemia in a resource-limited setting: a single institution prospective cohort study from Upper Egypt

Background: This study compared the efficacy and safety of CsA monotherapy with eltrombopag (E-PAG) + CsA combined treatment in children with severe aplastic anemia (SAA). Method: The study including 30 children had SAA. Ten were a retrospective cohort treated with CsA monotherapy. The other 20 were prospective cohort received E-PAG + CsA . All patients were evaluated for partial (PR) and complete (CR) hematological response at 3, 6, and 12 months. overall response (OR), overall survival rates (OS) and treatment safety. Results: OR for the E-PAG patients was 40% after three months of therapy. At six months, this had increased to 75 % with significantly higher CR rate (40%) than in the CsA group (p = 0.0001). After a year of treatment, the CR for the E-PAG regimen had increased to 50% and the OR to 85%, compared to 20% in the CsA group (p = 0.0001). The OS at 12 months was 100% in the E-PAG group compared to 80% in the CsA cohort. At 24 months, the OS in the E-PAG group was 90%. Conclusion: CsA + E-PAG was found to be a safe and effective alternative treatment for children with SAA particularly in countries with limited resources.