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Alternative treatment modality for severe aplastic anemia in a resource-limited setting: a single-institution
Mervat A M Youssef1, Mohammed H Ghazaly2, Mai A Abdelfattah2
1Children Hospital, Hematology Unit, Faculty of Medicine, Assiut University, Assiut, Egypt. mamuosif2000@aun.edu.eg.
Insights
Eltrombopag (E-PAG) plus Cyclosporine A (CsA) significantly improved hematological response and overall survival in children with severe aplastic anemia (SAA). This combination therapy offers a safe and effective alternative for SAA treatment.
Area of Science:
- Pediatric Hematology
- Hematopoietic Stem Cell Transplantation
- Immunosuppressive Therapy
Background:
- Severe aplastic anemia (SAA) is a rare, life-threatening condition.
- Cyclosporine A (CsA) is a standard immunosuppressive therapy for SAA.
- Limited treatment options exist for SAA, especially in resource-limited settings.
Purpose of the Study:
- To compare the efficacy and safety of CsA monotherapy with eltrombopag (E-PAG) + CsA combined treatment in pediatric SAA patients.
- To evaluate hematological response, overall response (OR), complete response (CR), and overall survival (OS) rates.
- To assess the safety profile of the combined E-PAG + CsA regimen.
Main Methods:
- Retrospective analysis of 10 SAA patients treated with CsA monotherapy.
- Prospective study of 20 SAA patients treated with E-PAG + CsA.
- Evaluation of PR, CR, OR, OS, and safety at 3, 6, and 12 months post-treatment.
Main Results:
- E-PAG + CsA achieved 75% OR and 40% CR by 6 months, significantly higher than CsA alone.
- After 12 months, E-PAG + CsA showed 85% OR and 50% CR versus 20% OR in the CsA group (p=0.0001).
- OS at 12 months was 100% for E-PAG + CsA versus 80% for CsA; at 24 months, OS was 90% for E-PAG + CsA.
Conclusions:
- Eltrombopag (E-PAG) combined with Cyclosporine A (CsA) is a safe and effective treatment for pediatric severe aplastic anemia (SAA).
- This combination therapy demonstrates superior hematological response and survival rates compared to CsA monotherapy.
- E-PAG + CsA represents a valuable therapeutic option for SAA, particularly in resource-limited settings.
Abstract:
This study compared the efficacy and safety of CsA monotherapy with eltrombopag (E-PAG) + CsA combined treatment in children with severe aplastic anemia (SAA). 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. OR for the E-PAG patients was 40% after 3 months of therapy. At 6 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 + CsA 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+ CsA group compared to 80% in the CsA cohort. At 24 months, the OS in the E-PAG + CsA group was 90%. In conclusion, E-PAG+ CsA was found to be a safe and effective alternative treatment for children with SAA particularly in countries with limited resources.
