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

June 15, 2025 updated by: Zhongming Zhang

Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major Treatment: a Multi-center, Open, Randomized, Controlled Clinical Study

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

205

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Guangxi
      • Liuzhou, Guangxi, China
        • Liuzhou Worker's Hospital
      • Nanning, Guangxi, China, 530021
        • The Affiliated Hospital OF Guangxi Medical University
    • Hainan
      • Haikou, Hainan, China
        • Hainan General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

(8) Other circumstances which do not meet the inclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Basiliximab,
Basiliximab was used on 0d (after transplantation) and +4d (10mg for weight under 35kg, 20mg for over 35kg) .
Other Names:
  • chimeric mouse-human antiCD25
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.
Other Names:
  • FK506
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.
Other Names:
  • Ametnopterin
Drug: Mycophenolate mofetil
Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.
Other Names:
  • Mycophenolic acid
Active Comparator: 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.
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.
Other Names:
  • FK506
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.
Other Names:
  • Ametnopterin
Drug: Mycophenolate mofetil
Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.
Other Names:
  • Mycophenolic acid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
grade II-IV acute graft-versus-host disease incidence
Time Frame: 100 days after matched unrelated donor hematopoietic stem cell transplantation.
The cumulative incidence of grade II-IV acute graft-versus-host disease after matched unrelated donor hematopoietic stem cell transplantation +100d.
100 days after matched unrelated donor hematopoietic stem cell transplantation.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: three years
three years
Implantation rate
Time Frame: three years
three years
Transplanted-related mortality
Time Frame: three years
three years
Infection incidence
Time Frame: three years
CMV reactivation, EBV reactivation, bacterial infection, fungal infection, etc.
three years
Chronic graft-versus-host-disease incidence
Time Frame: five years
five years
Disease-free-survival
Time Frame: three years
three years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Zhongming Zhang

Collaborators

Liuzhou Workers Hospital
McMaster University
Hainan General Hospital
Guangxi Key Laboratory of Thalassemia Research
NHC Key Laboratory of Thalassemia Medicine

Investigators

  • Principal Investigator: yongrong lai, PhD, First Affiliated Hospital of Guangxi Medical University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2015

Primary Completion (Actual)

August 20, 2022

Study Completion (Actual)

August 20, 2022

Study Registration Dates

First Submitted

January 14, 2015

First Submitted That Met QC Criteria

January 16, 2015

First Posted (Estimated)

January 19, 2015

Study Record Updates

Last Update Posted (Actual)

June 18, 2025

Last Update Submitted That Met QC Criteria

June 15, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • gxmuh-2014-14

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Graft-versus-host Disease (GVHD)

Clinical Trials on Basiliximab,

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malawi

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe