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

  • STATUS
    Recruiting
  • days left to enroll
    59
  • participants needed
    120
  • sponsor
    Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Updated on 17 October 2021
immunosuppressant
hla-a
cyclosporine
thymoglobulin
immunosuppression
aplastic anemia
rheumatic heart disease
paroxysmal nocturnal hemoglobinuria
cyclosporine oral product

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/d5d 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.

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/d5d plus oral cyclosporine ACSA ) 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 >0.5109/L, without G-CSF adminstration. Platelet recovery day is dened to have occurred on the rst of 7 consecutive days with a blood platelet count (BPC) of >20109/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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Details
Condition Severe Aplastic Anemia
Treatment Thymoglobulin, IST, cord blood transfusion, Thymoglobuline® and oral CSA, cord blood, Cyclosporine Oral Product
Clinical Study IdentifierNCT02745717
SponsorShanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Last Modified on17 October 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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<0.5109/L,ii)PLT<20109/L and iii) Ret<20109/L ,in addition, ANC<0.5109/L
must be included
\. Under 60 years old, male or female
\. No HLA matched siblings
\. No previous ATG treatment history
\. Performance status score no more than 2 (ECOG criteria)
Adequate organ function as defined by the following criteria:ALT, AST and
total serum bilirubin <2ULN (upper limit of normal) Serum creatinine and BUN
<1.25ULN
\. Adequate cardiac function without acute myocardial infarction, arrhythmia
or atrioventricular block, heart failure, active rheumatic heart disease and
cardiac dilatation
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
\. Willingness and ability to comly with scheduled visits, treatment plans
laboratory tests, and other study procedures

Exclusion Criteria

Presence of any condition inappropriate for HSCT
Presence of any fatal disease, including respiratory failure, heart failure, liver or kidney function failure et al
Severely allergic to biologic products
Pregnancy or breastfeeding
Current treatment on another clinical trail
Any other condition the investigator judged the patient inappropriate for
entry into this study
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