Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA

Launched by FEDERAL RESEARCH INSTITUTE OF PEDIATRIC HEMATOLOGY, ONCOLOGY AND IMMUNOLOGY · Jan 26, 2018

Keywords

ClinConnect Summary

This trial will evaluate safety and efficacy of combination eltrombopag with standard hATG/CSA as first line therapy in patients with SAA. The primary endpoint is going to be estimating the rate of complete hematologic response at the point in four months after the end of the treatment. Secondary endpoints are probability of relapse, hematologic blood count recovery in 6 and 12 months after the treatment, survival, clonal evolution into myelodysplasia and leukemia

Aplastic anemia can be treated effectively with allogeneic bone marrow transplantation and immunosuppressive therapy with horse...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Clinical diagnosis of severe and very severe Aplastic anemia
• • 2. 2 - 18 years old
• • 3. Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.
• • 4. Absence of HLA-identical family member
• Exclusion Criteria:
• • 1. myelodysplastic syndrome 4. Prior immunosuppressive therapy 5. Patients with hepatic, renal or cardiac failure, or any other life- threatening concurrent disease 6. hypersensitivity to any of the component medications 7. Creatinine \>2.5 mg/dL× the upper limit of normal, 8. Total bilirubin \>1.5 × the upper limit of normal mg/dL , 9. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3-5 × the upper limit of normal