Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia

Phase 4

• Conditions: Severe Aplastic Anemia
• Interventions: Drug: ATG

• Registration Number: NCT01997372
• Lead Sponsor: Chinese Academy of Medical Sciences

Brief Summary

Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. However ,there are no clinical trials to illustrate the response and complete remission rate with different doses of ATG.And there are no data reported on children with SAA so far.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-12
• Study First Submitted: 2012-03-01
• Study First Submitted QC: 2013-11-27
• Study First Posted: 2013-11-28
• Status Verified: 2013-12
• Primary Completion: 2013-12
• Last Update Submitted: 2013-12-02
• Last Update Posted: 2013-12-03
• Study Completion: 2015-11

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• clinical diagnosis of childhood acquired severe aplastic anemia(SAA)

Exclusion Criteria

• clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
high dose ATG,low dose ATG	ATG	-

Primary Outcome Measures

Name	Time	Method
the response and complete remission rate with different doses of ATG to treat child severe aplastic anemia	1 years	Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count \>100×109/L, and ANC\>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count \>30×109/L, platelet count \>20×109/L, and ANC \>0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).

Secondary Outcome Measures

Name	Time	Method
the relapse rate with different doses of ATG to treat child severe aplastic anemia	4-10 years	relapse was defined as transfusion dependence again; or progressed to paroxysmal nocturnal hemoglobinuria (PNH) /acute myeloid leukemia/myelodysplasia syndrome (MDS); or CSA dependence

Trial Locations

Locations (1)

Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China