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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-01-31

Study Completion Date

2021-12-31

Brief Summary

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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.

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Detailed Description

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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.

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Conditions

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Severe Aplastic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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cord blood and IST group

Administration of antithymocyte ( Thymoglobulin ) 3.5mg/kg/d for 5 days, Cyclosporine Oral Product 5mg/kg/d with trough serum concentration of 200-300ng/ml, plus one unit of at least 4/6 HLA loci matched cord blood transfusion 24 hours after last dose of ATG.

Group Type EXPERIMENTAL

Thymoglobulin

Intervention Type DRUG

administration of ATG 3.5mg/kg/d (Thymoglobuline®, Genzyme) intravenously for 5 days

Cord blood

Intervention Type PROCEDURE

transfusion of one unit of at least 4/6 HLA loci matched cord blood.

Cyclosporine Oral Product

Intervention Type DRUG

administration of cyclosporine A 5mg/kg orally, and maintain the trough serum concentration between 200ng/ml to 300ng/ml.

IST group

Antithymocyte ( Thymoglobulin ) 3.5mg/kg/d for 5 days , Cyclosporine Oral Product 5mg/kg/d with trough serum concentration of 200-300ng/ml.

Group Type ACTIVE_COMPARATOR

Thymoglobulin

Intervention Type DRUG

administration of ATG 3.5mg/kg/d (Thymoglobuline®, Genzyme) intravenously for 5 days

Cyclosporine Oral Product

Intervention Type DRUG

administration of cyclosporine A 5mg/kg orally, and maintain the trough serum concentration between 200ng/ml to 300ng/ml.

Interventions

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Thymoglobulin

administration of ATG 3.5mg/kg/d (Thymoglobuline®, Genzyme) intravenously for 5 days

Intervention Type DRUG

Cord blood

transfusion of one unit of at least 4/6 HLA loci matched cord blood.

Intervention Type PROCEDURE

Cyclosporine Oral Product

administration of cyclosporine A 5mg/kg orally, and maintain the trough serum concentration between 200ng/ml to 300ng/ml.

Intervention Type DRUG

Other Intervention Names

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Thymoglobuline cyclosporine A

Eligibility Criteria

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Inclusion Criteria

1．Diagnosis of AA confirmed by bone marrow aspirate and biopsy, myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria were excluded. To confirm severe AA, the patient must fulfill at least two of the criteria: i) ANC\<0.5×109/L,ii)PLT\<20×109/L and iii) Ret\<20×109/L ,in addition, ANC\<0.5×109/L must be included.

2\. Under 60 years old, male or female.

3\. No HLA matched siblings.

4\. No previous ATG treatment history.

5\. Performance status score no more than 2 (ECOG criteria).

6．Adequate organ function as defined by the following criteria:ALT, AST and total serum bilirubin \<2×ULN (upper limit of normal) Serum creatinine and BUN \<1.25×ULN.

7\. Adequate cardiac function without acute myocardial infarction, arrhythmia or atrioventricular block, heart failure, active rheumatic heart disease and cardiac dilatation.

8．Signed and dated informed consent document indicating that the patient (or legally acceptable representative) has been informed of all pertinent aspects of the trial prior to enrollment.

9\. Willingness and ability to comly with scheduled visits, treatment plans, laboratory tests, and other study procedures.

Exclusion Criteria

1. Presence of any condition inappropriate for HSCT.
2. Presence of any fatal disease, including respiratory failure, heart failure, liver or kidney function failure et al.

3．Severely allergic to biologic products.

4．Pregnancy or breastfeeding.

5．Current treatment on another clinical trail.

6．Any other condition the investigator judged the patient inappropriate for entry into this study.

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No