NCT02745717

The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic Anemia

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 60 (actual)
• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 60 Years
• Healthy volunteers: Not accepted

Summary

Aim: To evaluate if additional cord blood transfusion could accelerate the hematopoietic reconstitution in severe aplastic anemia(SAA) patients receiving immunosuppressive therapy (IST). Study design: open-labed, prospective, multicenter, randomized control study Number of subjects: 60 each group Treatment: IST group: ATG (Thymoglobuline®, Genzyme) 3.5mg/kg/d×5d plus oral cyclosporine A (CSA) Cord blood transfusion group: In addition to the same dose and course of ATG and CSA , one unit of cord blood having no more than 2 HLA-A, B or DRB1 mismatches is transfused 24h after last dose of ATG administration.

Detailed description

Eligible patients should be under 60 years old with confirmed SAA, without HLA matched siblings and previous ATG treatment history. Patients will be excluded if they present any fatal disease, including respiratory failure, heart failure, liver or kidney function failure et al or severely allergic to biologic products. To evaluate if additional cord blood transfusion could accelerate the hematopoietic reconstitution in severe aplastic anemia(SAA) patients receiving IST therapy, 120 eligible patients will be randomized to two groups, the IST group and the cord blood transfusion group. Patients in the IST group receive standard IST which including ATG (Thymoglobuline®, Genzyme) 3.5mg/kg/d×5d plus oral cyclosporine A（CSA ) started from 5mg/kg/d and adjusted to maintain trough serum concentration of 200-300ng/ml. While patients in the cord blood transfusion group receive the same dose and course of ATG and CSA as the control group and one unit of cord blood having no more than 2 HLA-A, B and DRB1 mismatches is transfused 24h after last dose of ATG administration. The neutrophil recovery day is defined as the first day of 3 consecutive days during which the absolute neutrophil count (ANC) is \>0.5×109/L, without G-CSF adminstration. Platelet recovery day is defined to have occurred on the first of 7 consecutive days with a blood platelet count (BPC) of \>20×109/L, without transfusion support. Response (CR, PR or NR) is evaluated on 3, 4, 6,9, 12, 18 and 24months after treatment. The primary end point is the neutrophil recovery day and second end points are response rate (CR+PR), treatment related mortality, disease free survival and overall survival. .

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Thymoglobulin	administration of ATG 3.5mg/kg/d (Thymoglobuline®, Genzyme) intravenously for 5 days
PROCEDURE	Cord blood	transfusion of one unit of at least 4/6 HLA loci matched cord blood.
DRUG	Cyclosporine Oral Product	administration of cyclosporine A 5mg/kg orally, and maintain the trough serum concentration between 200ng/ml to 300ng/ml.

Timeline

• Start date: 2016-01-01
• Primary completion: 2021-12-01
• Completion: 2021-12-01
• First posted: 2016-04-20
• Last updated: 2023-03-17

Locations

1 site across 1 country: China