Chronic graft-versus-host disease (cGVHD) is a significant risk factor for patients who have undergone allogeneic hematopoietic stem cell transplantation (alloHSCT). For patients with acute myeloid leukemia who received peripheral blood alloHSCT from human leukocyte antigen (HLA)-identical sibling donors, a human anti-T-lymphocyte globulin (ATLG)-based cGVHD prophylaxis approach may show promising long-term results, according to a recent report in The Lancet Haematology.

In a prior open label study ( Identifier: NCT00678275), patients with acute myeloid leukemia or acute lymphoblastic leukemia in remission were divided according to prophylaxis type. One treatment cohort (83 patients) was assigned to receive cGVHD prophylaxis with human ATLG and ciclosporin with methotrexate; in the initial study, this combination had shown efficacy against cGVHD by multiple metrics. The control cohort (72 patients) was assigned to receive standard prophylaxis with ciclosporin and methotrexate and no ATLG. All patients had received peripheral blood alloHSCT from HLA-identical sibling donors and had been given 1 of a few possible conditioning regimens.

The latest report was a follow-up ( Identifier: NCT03042676) that focused on previously unpublished data from the initial study and long-term outcomes in the patients who received ATLG-based prophylaxis, with a primary endpoint of 2-year cumulative cGVHD incidence and a secondary endpoint of quality of life (QoL). QoL was measured using European Organisation for Research and Treatment of Cancer QLQ-C30 and QLQ-HDC29 questionnaires.

Upon follow-up at a median time of 5.9 years (interquartile range: 1.7-7.9 years), ATLG was associated with a lower incidence of cGVHD (30.0%; 95% confidence interval [CI]: 21.4%-41.9%) compared with non-ATLG prophylaxis (69.1%; 95% CI: 59.1%-80.1%; P <.001). ATLG was also associated with fewer patients remaining in immunosuppression (9.6% with ATLG vs 28.3% without; P =.017), improvement in the composite endpoint of severe cGVHD-free and relapse-free survival (34.3% with ATLG vs 13.9% without; P =.005), and no significant increase in the rate of relapse (35.4% with ATLG vs 22.5% without; P =.09). Overall survival, nonrelapse mortality, and relapse-free survival rates were not significantly different between patients receiving prophylaxis with or without ATLG.

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Patients receiving ATLG prophylaxis showed significant improvements in QoL by several metrics including global health status over time (P =.02), gastrointestinal side effects (P =.008), physical function (P =.014), effect on family (P =.032), and social function (P =.047).

For the patients included in this study, ATLG as a component of cGVHD prophylaxis enabled improvements in long-term outcomes and superior QoL when compared with prophylaxis lacking ATLG.


1. Bonifazi F, Solano C, Wolschke C, et al. Acute GVHD prophylaxis plus ATLG after myeloablative allogeneic haemopoietic peripheral blood stem-cell transplantation from HLA-identical siblings in patients with acute myeloid leukaemia in remission: final results of quality of life and long-term outcome analysis of a phase 3 randomised study published online February 1, 2019]. Lancet Haematol. doi: 10.1016/S2352-3026(18)30214-X