Beta-Thalassemia Major Clinical Trial
Official title:
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.
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.
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Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention
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