Addition of Antithymocyte Globulin to Graft-vs-Host Disease Prophylaxis in Matched Sibling Donor Stem Cell Transplant

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In a Chinese trial reported in the Journal of Clinical Oncology, Chang et al found that the addition of antithymocyte globulin to standard graft-vs-host disease prophylaxis reduced the risk of acute graft-vs-host disease in patients undergoing human leukocyte antigen (HLA)-matched sibling donor hematopoietic stem cell transplantation for hematologic malignancies.

Study Details

The open-label multicenter trial included 263 patients with standard-risk hematologic malignancies undergoing matched sibling donor transplant. Patients were randomly assigned between November 2013 and April 2018 to receive antithymocyte globulin (ATG; 4.5 mg/kg thymoglobulin) plus cyclosporine (CsA), methotrexate (MTX), and mycophenolate mofetil (MMF; n = 132) or CsA/MTX/MMF alone (n =131). The primary endpoint was grade 2 to 4 acute graft-vs-host disease on day 100.

Key Findings

The cumulative incidence of grade 2 to 4 acute graft-vs-host disease at 30 days was 13.7% in the ATG group vs 27.0% in the control group (P = .007). A reduction in grade 3 or 4 acute graft-vs-host disease did not reach statistical significance (8.4% vs 14.2%, P = .151).

The incidence of 2-year overall chronic graft-vs-host disease was 27.9% vs 52.5% (P < .001), with extensive chronic graft-vs-host disease observed in 8.5% vs 23.2% (P = .029). Graft-vs-host disease relapse–free survival at 3 years was 38.7% vs 24.5% (P = .003).

At 3 years, nonrelapse mortality was 9.9% vs 14.3% (P = .552); cumulative incidence of relapse was 20.8% vs 15.4% (P = .362); and overall survival was 69.0% vs 70.4% (P = .937). There were no differences between the ATG and control groups with regard to cytomegalovirus or Epstein-Barr virus reactivation.

The investigators concluded, “Our study is the first prospective randomized controlled trial in our knowledge to demonstrate that ATG can effectively decrease the incidence of acute [graft-vs-host disease] after [HLA-matched sibling donor transplantation] in the CsA era without affecting the cumulative incidence of relapse or nonrelapse mortality.”

Xiao-Jun Huang, MD, of Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, is the corresponding author for the Journal of Clinical Oncology article.

Disclosure: The study was supported by the Foundation for Innovative Research Groups of the National Natural Science Foundation of China and others. For full disclosures of the study authors, visit

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