Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major

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

205 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-04-30

Study Completion Date

2022-08-20

Brief Summary

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The purpose of this study is to evaluate the basiliximab for prevention of graft-versus-host disease in unrelated allo-genetic hematopoietic stem cell transplantation for thalassemia major. The objective was to evaluate the effect and safety of basiliximab for acute graft-versus-host disease.

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Detailed Description

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Allo-geneic stem cell transplantation(allo-HSCT) cure thalassemia major by destroying the original hematopoietic and immune systems with a large dose of chemotherapy, rebuilding a new system to correct the abnormal hematopoietic globin chain synthesis which leads to hemolysis. Currently, it is the only curative means. According to donors, allo-HSCT could be sibling allogeneic hematopoietic stem cell transplantation and unrelated allogeneic hematopoietic stem cell transplantation(URD-HSCT). URD-HSCT could expand the range of treatment among β-thalassemia major patients. As recently reported , 68 cases of thalassemia patients at the median age of 15 (2 to 37 ) received unrelated donor BMT. According to Pesaro rating classification, 14 patients were attributed to type Ⅰ, 16 cases Ⅱ type , 38 cases type III, overall survival and thalassemia free survival rates were 79.3% and 65.8%. A survey among 59 evaluable patients indicated that grade Ⅱ \~ Ⅳ aGVHD occurred in 24 cases (40%) , in which 10 cases (17%) were grade Ⅲ \~ Ⅳ aGVHD. Similar results were seen in other reports, 21 patients received unrelated donor BMT, with a 2-year thalassemia free survival rate of 71%. GVHD happened in 3 cases, and 3 patients died. Our institution has conducted a total of 10 cases of URD-HSCT to treat severe thalassemia, using methotrexate + cyclosporine A+ mycophenolate mofetil to prevent graft-versus-host disease, 9 cases of disease-free survival, 1 case with graft rejection. Incidence of Ⅲ-Ⅳ severe acute graft-versus-host disease (aGVHD) was 20%. Severe aGVHD incidence was 20%. Our research group has found there is a high risk to develop aGVHD, especially severe aGVHD for heavy thalassemia patients who receive URD-HSCT, which seriously affects the prognosis and survival, while increasing medical costs and the financial burden on the patients' families.

The key factor affecting URD-HSCT's success is GVHD. Thus effective prevention and treatment of GVHD is a prerequisite to ensure a successful transplant. CD25 is a humanized monoclonal IgG1,with murine anti-human IL-2RA chain complement determining region retained. IL-2RA chain expressed only on the surface of activated cytotoxic T cells, which could convert the IL-2R complexes into a higher affinity. The feature that IL-2RA distributes only on the surface of activated lymphocytes indicates it's a ideal target when designing the policy to scavenge antigen-specific allogeneic reactive T cells. In vitro experiments, CD25 monoclonal antibody binds specifically with IL-2RA+ cells by inhibiting IL-2 binding to its receptor competitively. Basiliximab has now been used as first-line medication for aGVHD treatment, as well as the combined prevention of hematologic malignancies URD-HSCT treatment . However as for thalassemia major URD-HSCT, few cases have been reported.

This study was aimed at the high incidence of aGVHD, especially severe aGVHD in thalassemia major URD-HSCT. Basiliximab was added to the original prevention program. The aGVHD incidence, implantation rate, transplant-related mortality, infection incidence would be observed. It is hopeful to reduce the aGVHD incidence after URD-HSCT and promote curative effect.

Conditions

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Beta-Thalassemia Major Graft-versus-host Disease (GVHD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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group A

The patients were used FK506 (0.03mg/kg/day), Methotrexate (15mg/m2 on +1d, 10mg/m2 on +3d, +6d, +11d), Mycophenolate Mofetil (0.25g/d, days-1 to 90) and Basiliximab (use 10mg for weight under 35kg, 20mg for over 35kg, 0d and +4d) for prevention of acute graft-versus-host-disease.

Group Type EXPERIMENTAL

Basiliximab,

Intervention Type DRUG

Basiliximab was used on 0d (after transplantation) and +4d (10mg for weight under 35kg, 20mg for over 35kg) .

Tacrolimus

Intervention Type DRUG

Specifically Tacrolimus was used by intravenous drip infusion on 0.03mg/kg dosage from -1d and change to 0.1mg/kg oral when gastrointestinal function recovers. The blood concentrations of cyclosporine A was maintained 5-10 ng/ml.

Methotrexate

Intervention Type DRUG

Methotrexate was used 15mg/m2 on +1d and 10mg/m2 on +3d,+6d,+11d by intravenous for prevention of graft-versus-host-disease.

Mycophenolate mofetil

Intervention Type DRUG

Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.

group B

The patients were used FK506 (0.03mg/kg/day), Methotrexate (15mg/m2 on +1d, 10mg/m2 on +3d, +6d, +11d), Mycophenolate Mofetil (0.25g/d, days-1 to 90) for prevention of acute graft-versus-host-disease.

Group Type ACTIVE_COMPARATOR

Tacrolimus

Intervention Type DRUG

Specifically Tacrolimus was used by intravenous drip infusion on 0.03mg/kg dosage from -1d and change to 0.1mg/kg oral when gastrointestinal function recovers. The blood concentrations of cyclosporine A was maintained 5-10 ng/ml.

Methotrexate

Intervention Type DRUG

Methotrexate was used 15mg/m2 on +1d and 10mg/m2 on +3d,+6d,+11d by intravenous for prevention of graft-versus-host-disease.

Mycophenolate mofetil

Intervention Type DRUG

Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.

Interventions

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Basiliximab,

Basiliximab was used on 0d (after transplantation) and +4d (10mg for weight under 35kg, 20mg for over 35kg) .

Intervention Type DRUG

Tacrolimus

Specifically Tacrolimus was used by intravenous drip infusion on 0.03mg/kg dosage from -1d and change to 0.1mg/kg oral when gastrointestinal function recovers. The blood concentrations of cyclosporine A was maintained 5-10 ng/ml.

Intervention Type DRUG

Methotrexate

Methotrexate was used 15mg/m2 on +1d and 10mg/m2 on +3d,+6d,+11d by intravenous for prevention of graft-versus-host-disease.

Intervention Type DRUG

Mycophenolate mofetil

Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.

Intervention Type DRUG

Other Intervention Names

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chimeric mouse-human antiCD25 FK506 Ametnopterin Mycophenolic acid

Eligibility Criteria

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Inclusion Criteria

1. a diagnosis of TM with hemoglobin electrophoresis, a genetic diagnosis of -thalassemia by DNA analysis, and blood transfusion dependence;
2. a cardiac ejection fraction \>50%;
3. normal pulmonary function tests and pulmonary examination results;
4. normal kidney function.

Exclusion Criteria

1. aspartate aminotransferase level \>4- fold the upper limit of normal range in our institution's laboratory criteria;
2. uncontrolled bacterial, viral, or fungal infection;
3. positive serology for HIV;
4. cytomegalovirus (CMV) or Epstein-Barr virus (EBV) copy number \>200 copies/mL in blood by quantitative PCR. Patients positive for hepatitis C or hepatitis B virus were also excluded.

Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No