Randomized Clinical Trial of the Use of Eltrombopag in Children with Aplastic Anemia


Jameel Alghaberi*, Eman Fathalla Gad, Mai A Abdelfattah, Mohammed H ghazaly, Khalid A Sanousy, Douaa M Sayed, Shimaa Hosny Hassan and Mervat A M Youssef

Abstract: Severe aplastic anemia is a life - threatening disorder that requires prompt treatment. For children not eligible for Hematopoietic Stem Cell Transplantation (HSCT), the Standard Immunosuppressive Therapy (IST) consisting of Antithymocyte Globulin (ATG) and Cyclosporine (CSA) is the ideal treatment for patients with Severe Aplastic Anemia (SAA). The addition of ELTROMBOPAG (E - PAG) with IST shows good improvement in the response rates, a rise of blood cell counts, and recovery of trilineage hematopoiesis. The purpose of this study was to compare the efficacy and safety of E - PAG combined with CsA and immunosuppressive mono therapy of CsA for treatment in children with SAA.

Method: This was a prospective clinical trial with a patient sample of 20 children diagnosed with SAA. Half of the participants were treated with CsA monotherapy. The other half was treated with combined E - PAG + CsA. All patients were evaluated for hematological response, complete response, and partial response at 3, 6, and 12 months. We also measured treatment safety and tolerability.

Results: The overall response rate for the E - PAG patients was 40 % after three months of therapy. At six months, this had increased to 80 %, and the percentage of patients showing a complete response (40 %) was significantly higher than in the CsA group (p = 0.006). After a year of regular treatment, the complete response rate for the E - PAG regimen had increased to 60 % and the overall response rate to 90 %, compared to a rate of 20 % in the CsA group (p = 0.01). The survival rate at 12 months was 100 % in the E - PAG group compared to 80 % in the CsA cohort. In conclusion, combined treatment with CsA + E - PAG was found to be a safe, well - tolerated and effective alternative treatment for children with SAA.